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Stemedica Files with FDA to Become a Licensed Manufacturer of Adult Stem Cells for U.S. Based Clinical Trials
Stemedica Cell Technologies, Inc. announced that it has
filed with the Food and Drug Branch of the State of California's Department of Public Health for
certification as a licensed manufacturer of adult stem cells for U.S.-based
Phase I and Phase II Clinical Trials. Readiness for manufacture includes
obtaining a California Drug Manufacturing License for the Stemedica facility in
San Diego, California.
"Completion of this certification will allow us to
manufacture stem cells under contract for clinical trials for Stemedica as well
as for other companies and research centers in California
and across the United States.
This marks a significant milestone along the journey toward the future
availability of stem cell treatment for patients who so urgently need it,"
said Dr. Maynard Howe, Vice Chairman and CEO at Stemedica.
To prepare for inspection, Stemedica fully reconstructed its
facility according to FDA requirements, which had previously focused on
research and development. Stemedica's manufacturing facility now includes a
fully compliant manufacturing wing with entry-level gowning rooms, two ISO
class 8 sterile gowning suites, an ISO Class 7 Manufacturing Clean Room, an ISO
Class 7 Processing Clean Room, a Good Manufacturing Process compliant materials
warehouse and a controlled-access Quality Control testing laboratory.
"As soon as Stemedica secures its license from the
State of California's Department of Public
Health, we plan on filing for one or more IND's (Investigative New Drug) for specific
indications with the FDA using our proprietary technology. The final major step
by the Food and Drug Branch within the Department of Public Health is an
in-depth, in-person inspection by their personnel. We're particularly proud of
the fact that we are ready for, and capable of, seeking licensing in California, which has
exceptionally stringent standards," said Dr. Alex Kharazi, Vice President,
Research & Manufacturing at Stemedica.
"Becoming a Licensed Manufacturer of clinical grade
adult stem cells is a significant scientific and medical step forward for the
Company," said Dr. Nikolai Tankovich, Stemedica's President and Chief
Medical Officer. "Securing this status will allow us to become a primary
CMO (Contract Manufacturing Organization) for other for-profit companies and
the many research organizations interested in advancing stem cell research
through clinical application. Additionally, achieving this regulatory status
within the manufacturing world will provide Stemedica with the foundation to
advance its own Phase I and Phase II Clinical Studies once approved by the
FDA."
Source: Stemedica Cell Technologies, Inc.
Geron Receives FDA Clearance to Begin World's First Human Clinical Trial of Embryonic Stem Cell-Based Therapy
Geron to Study GRNOPC1 in Patients with Acute Spinal Cord
Injury
MENLO PARK, Calif., January 23, 2009 - Geron Corporation announced that the U.S. Food and Drug Administration (FDA)
has granted clearance of the company's Investigational New Drug (IND)
application for the clinical trial of GRNOPC1 in patients with acute spinal
cord injury.
The clearance enables Geron to move forward with the world's
first study of a human embryonic stem cell (hESC)-based therapy in man. Geron
plans to initiate a Phase I multi-center trial that is designed to establish
the safety of GRNOPC1 in patients with "complete" American Spinal Injury
Association (ASIA) grade A subacute thoracic spinal cord injuries.
"The FDA's clearance of our GRNOPC1 IND is one of
Geron's most significant accomplishments to date," said Thomas Okarma,
Ph.D., M.D., Geron's president and CEO. "This marks the beginning of what
is potentially a new chapter in medical therapeutics - one that reaches beyond
pills to a new level of healing: the restoration of organ and tissue function
achieved by the injection of healthy replacement cells. The ultimate goal for
the use of GRNOPC1 is to achieve restoration of spinal cord function by the
injection of hESC-derived oligodendrocyte progenitor cells directly into the
lesion site of the patient's injured spinal cord."
GRNOPC1, Geron's lead hESC-based therapeutic candidate,
contains hESC-derived oligodendrocyte progenitor cells that have demonstrated
remyelinating and nerve growth stimulating properties leading to restoration of
function in animal models of acute spinal cord injury (Journal of Neuroscience,
Vol. 25, 2005).
"The neurosurgical community is very excited by this
new approach to treating devastating spinal cord injury," said Richard
Fessler, M.D., Ph.D., professor of neurological surgery at the Feinberg School
of Medicine at NorthwesternUniversity.
"Demyelination is central to the pathology of the injury, and its reversal
by means of injecting oligodendrocyte progenitor cells would be revolutionary
for the field. If safe and effective, the therapy would provide a viable
treatment option for thousands of patients who suffer severe spinal cord
injuries each year."
The GRNOPC1 Clinical
Program
Patients eligible for the Phase I trial must have documented
evidence of functionally complete spinal cord injury with a neurological level
of T3 to T10 spinal segments and agree to have GRNOPC1 injected into the lesion
sites between seven and 14 days after injury. Geron has selected up to seven U.S. medical
centers as candidates to participate in this study and in planned protocol
extensions. The sites will be identified as they come online and are ready to
enroll subjects into the study.
Although the primary endpoint of the trial is safety, the
protocol includes secondary endpoints to assess efficacy, such as improved
neuromuscular control or sensation in the trunk or lower extremities. Once
safety in this patient population has been established and the FDA reviews
clinical data in conjunction with additional data from ongoing animal studies,
Geron plans to seek FDA approval to extend the study to increase the dose of
GRNOPC1, enroll subjects with complete cervical injuries and expand the trial
to include patients with severe incomplete (ASIA grade B or C) injuries to
enable access to the therapy for as broad a population of severe spinal
cord-injured patients as is medically appropriate.
Preclinical Evidence
of Safety, Tolerability and Efficacy
Geron submitted evidence of the safety, tolerability and
efficacy of GRNOPC1 to the FDA in a 21,000-page IND application that described 24 separate
animal studies requiring the production of more than five billion GRNOPC1
cells. Included in the safety package were studies that showed no evidence of
teratoma formation 12 months after injection of clinical grade GRNOPC1 into the
injured spinal cord of rats and mice. Other studies documented the absence of
significant migration of the injected cells outside the spinal cord, allodynia
induction (increased neuropathic pain due to the injected cells), systemic
toxicity or increased mortality in animals receiving GRNOPC1.
In vitro studies have shown that GRNOPC1 is minimally
recognized by the human immune system. GRNOPC1 is not recognized in vitro by
allogeneic sera, NK cells or T cells (Journal of Neuroimmunology, Vol. 192,
2007). These immune-privileged characteristics of the hESC-derived cells allow
a clinical trial design that incorporates a limited course of low-dose
immunosuppression and provide the rationale for an off-the-shelf, allogeneic
cell therapy.
Also included in the IND
application were published studies supporting the utility of GRNOPC1 for the
treatment of spinal cord injury. Those studies showed that administration of
GRNOPC1 significantly improved locomotor activity and kinematic scores of
animals with spinal cord injuries when injected seven days after the injury
(Journal of Neuroscience, Vol. 25, 2005). Histological examination of the
injured spinal cords treated with GRNOPC1 showed improved axon survival and
extensive remyelination surrounding the rat axons. These effects of GRNOPC1
were present nine months after a single injection of cells. In these nine-month
studies, the cells were shown to migrate and fill the lesion cavity, with
bundles of myelinated axons crossing the injury site.
Production and
Qualification of GRNOPC1
GRNOPC1 is produced using current Good Manufacturing
Practices (cGMP) in Geron's manufacturing facilities. Geron's GRNOPC1
production process and clean-room suites have been inspected and licensed by
the state of California.
The cells are derived from the H1 human embryonic stem cell line, which was
created before August 9, 2001. Studies using this line qualify for U.S. federal
research funding, although no federal funding was received for the development
of the product or to support the clinical trial.
Geron's H1 hESC master cell bank is fully qualified for
human use and was shown to be karyotypically normal and free of measurable
contaminants of human or animal origin. Production of GRNOPC1 from
undifferentiated hESCs in the master cell bank uses qualified reagents and a
standardized protocol developed at Geron over the past three years. Each
manufacturing run of GRNOPC1 is subjected to standardized quality control
testing to ensure viability, sterility and appropriate cellular composition
before release for clinical use. GRNOPC1 product that has passed all such specifications
and has been released is available for the approved clinical trial. The current
production scale can supply product needs through pivotal clinical trials. The
existing master cell bank could potentially supply sufficient starting material
for GRNOPC1 to commercially supply the U.S. acute spinal cord injury
market for more than 20 years.
Intellectual Property
The production and commercialization of GRNOPC1 is protected
by a portfolio of patent rights owned by or exclusively licensed to Geron.
Patent rights owned by Geron protect key technologies developed at Geron for
the scalable manufacturing of hESCs, as well as the production of neural cells
by differentiation of hESCs. The fundamental patents covering hESCs are
exclusively licensed to Geron from the Wisconsin Alumni Research Foundation
(WARF) for the production of neural cells, cardiomyocytes and pancreatic islets
for therapeutic applications. The validity of these patents was recently
confirmed by the U.S. Patent and Trademark Office in a re-examination
proceeding. Geron funded the original research at the University of
Wisconsin-Madison that led to the first isolation of hESCs. The production of
oligodendrocytes from hESCs is covered by patent rights exclusively licensed to
Geron from the University
of California. These
patent rights cover technology developed in a research collaboration between
Geron and University
of California scientists.
About Geron
Geron is developing first-in-class biopharmaceuticals for
the treatment of cancer and chronic degenerative diseases, including spinal
cord injury, heart failure and diabetes. The company is advancing an
anti-cancer drug and a cancer vaccine that target the enzyme telomerase through
multiple clinical trials. Geron is also the world leader in the development of
human embryonic stem (hESC) cell-based therapeutics. The company has received
FDA clearance to begin the world's first human clinical trial of a hESC-based
therapy: GRNOPC1 for acute spinal cord injury. For more information, visit
www.geron.com.
Source: Geron Press Release
CALIFORNIA STEM CELL AGENCY SENDS OPEN LETTER TO PRESIDENTIAL CANDIDATES REGARDING FUNDING FOR STEM CELL RESEARCH
SAN DIEGO, Calif., September 25, 2008 - Governor Arnold Schwarzenegger has declared Sept. 25 to be Stem Cell Awareness Day. In the proclamation he said, "The discoveries being made today in our GoldenState will have a great impact on many around the world for generations to come." The full text of the proclamation can be seen here: http://www.gov.ca.gov/proclamation/10589/
Dr. Alan Trounson, president of the California Institute for Regenerative Medicine (CIRM), the state stem cell agency, said that Stem Cell Awareness Day will help draw attention to science that will one day bring dramatic advances in human health. "The events on Stem Cell Awareness Day are designed to inform a broad audience of patients, clinicians, students and the general public about some of the many advances in the field that California is helping to accelerate through CIRM funding," he said.
To mark the day, Trounson and Robert Klein, Chairman of the Independent Citizen's Oversight Committee, co-signed an open letter to the presidential candidates regarding funding for stem cell research and biomedical research in general.
The letter reads:
"Today, California is marking Stem Cell Awareness Day. For the millions who suffer from incurable diseases and injury, today is a day to celebrate the scientific advances made to-date and be hopeful of what is yet to come.
The research being accelerated by the California Institute for Regenerative Medicine (CIRM) could one day offer stem cell derived therapies and cures for more than 70 currently incurable diseases and injuries. But no single state can provide the level of funding or the political will that is needed to ensure the long term success of this vital medical research. Leadership and funding at the Federal level is desperately needed to ensure that we realize the benefits from what is one of the most promising fields of science in this century.
That is why we implore both Republican Sen. John McCain, and Democrat Sen. Barack Obama to commit their administration to sustained levels of funding for all biomedical research and in particular, to all types of stem cell research. We believe that if they answer this call to action, we will soon realize significant medical benefits for patients around the world and increase our ability to control health care costs here at home.
Earlier this month, a panel of experts convened by the U.S. National Academy of Sciences confirmed what stem cell researchers throughout the world know - that the use of embryonic stem cells is still necessary. They remain the gold standard. As the expert panel, chaired by Richard Hynes of the Massachusetts Institute of Technology stated "It is far from clear at this point which types cell types will prove to be the most useful for regenerative medicine, and it is likely that each will have some utility."
By fully capitalizing on this area of scientific research, we have a chance to develop therapies and cures for a long list of serious diseases, injuries and
genetic conditions, from cancer to spinal cord injury to Alzheimer's, and an opportunity to restore quality of life for these patients and their families. We also have an opportunity to save billions of dollars we now spend on lost productivity and medical care for these patients. Stem cell research also holds promise for developing powerful environmental toxicity tests, drug screening, and patient diagnostic tests that can help save lives and eliminate serious potential roadblocks on the road to faster, more effective clinical trials for all kinds of intractable conditions.
California is making a bold investment in the promise of stem cell research, but it is up to the new leadership in Washington to ensure we capitalize on the full potential this science holds for us all."
Sincerely,
Alan Trounsan
President
CIRM
Robert Klein
Chairman
Independent Citizen's Oversight Committee
The President of the Karolinska Institute, Dr. Harriet Wallberg-Henriksson Joins the Scientific and Medical Advisory Board of Cellastra, a California Stem Cell and Regenerative Medicine Company
August 12, 2008-San Francisco, California- Cellastra Inc., a pioneering stem cell engineering and therapeutics company, today announced the appointment of Harriet Wallberg-Henriksson, MD, PhD, President of the Karolinska Institutet, Stockholm, Sweden to its Scientific and Medical Advisory Board.
Dr. Henriksson’s inclusion in Cellastra’s advisory board is part of Cellastra’s strategy to work with scientific and medical leaders around the world to help expand clinical applications of pluripotent, especially embryonic stem cells.
Professor Harriet Wallberg-Henriksson has been President of Karolinska Institutet since 2004. She is a member of Karolinska Institutet´s Nobel Assembly, which selects the winner of the Nobel Prize in Physiology or Medicine every year. Harriet Wallberg-Henriksson was appointed President after over 25 years at Karolinska Institutet, one of Europe’s largest and one of the highest ranking medical universities in the world.
CellastraInc.is an innovative stem cell company focused on developing the highest quality, xeno-free (i.e. containing no foreign contaminants), clinical grade and Good Manufacturing Practice (GMP) quality pluripotent stem cell lines and stem cell derived therapeutics. Cellastra’s technology is developed in collaboration with its co-founder Dr. Outi Hovatta, MD, PhD, Professor at the Karolinska Institutet and the KarolinskaUniversityHospital, Huddinge, Sweden. Dr. Hovatta is an internationally renowned stem cell expert with pioneering research on stem cell cultivation, stem cell engineering and therapeutics development.
The alliance with Dr. Wallberg-Henriksson further strengthens Cellastra’s plans for technological and scientific exchange and collaboration between Karolinska Institutet in Sweden and Cellastra in California. The California Institute of Regenerative Medicine (CIRM)has recently initiated a 10 year grant and loan programwith more than USD 3 billion made available to support pluripotent/embryonic stem cell research in institutions and companies located in California . “We are extremely honored and excited to strengthen the bridges between Sweden and California at this unique time by appointing Dr. Wallberg-Henriksson to Cellastra’s Advisory board,” said Dr. Neerja Sethi, CEO and co-founder of Cellastra Inc.
Dr Sethi added that “Cellastra takes pride in developing the highest quality pluripotent and embryonic stem cell lines and related technologies to help catapult the field of stem cell research into the era of biomedical and clinical applications.”
About Cellastra
CELLASTRA Inc. is a Stem Cell Therapeutics and Pharmacogenomics company that aims to develop, patent and commercialize innovative, cutting-edge technologies for developing human pluripotent/embryonic stem cells based therapeutics and research tools.CELLASTRA’s technology platform has clinical and market potential for multiple diseases with unmet needs. These include diabetes, spinal cord injuries, Parkinson’s, Multiple Sclerosis, Alzheimer’s, cardiovascular ailments, liver ailments, organ replacements, cancer, blood disorders and other applications of tissue regeneration. CELLASTRA is currently in a unique leadership position in stem cell engineering field in having access to the highest clinical quality, xeno-free (free of animal additions and contaminations), pharmaceutical level, Good Manufacturing Practice (GMP) grade, human embryonic/pluripotent stem cell lines and the related technologies. Cellastra is headquartered in San Francisco, California. Visit www.cellastra.com
GlaxoSmithKline and the Harvard Stem Cell Institute Announce a Unique Collaboration in Stem Cell Science to Enable the Discovery of New Medicines
-- Industry and academia link up in pioneering agreement--
GlaxoSmithKline (GSK) and the Harvard Stem Cell Institute (HSCI) announced that they have entered into a five-year, $25 million-plus collaborative agreement to build a unique alliance in stem cell science, leading to the development of new medicines.
GSK’s investment, one of the largest by a pharmaceutical company in stem cell science, will support innovative research at HarvardUniversity and in at least four Harvard-affiliated hospitals in the areas of neuroscience, heart disease, cancer, diabetes, musculoskeletal diseases and obesity. In addition, GSK will fund an annual grant, which supports early stage research in stem cell biology, as part of HSCI’s seed grant program
This agreement marks the beginning of an exciting collaboration with multiple researchers at multiple Harvard institutions, making it possible for academic and industrial scientists to work side-by-side to develop treatments in areas of unmet medical need.
“GSK believes stem cell science has great potential to aid the discovery of new medicines by improving the screening, identification and development of new compounds. We have carefully chosen the Boston biomedical community to collaborate with on this important venture. It has the highest concentration of leading stem cell scientists, and the Harvard Stem Cell Institute is the epicentre of that community,” said Patrick Vallance, Head of Drug Discovery at GSK.
The collaboration aims to integrate HSCI’s world-class stem cell expertise with GSK’s pharmaceutical capabilities to drive advances in drug discovery research. This will include, for example, a staff exchange programme where HSCI and GSK researchers will spend up to several months in each other’s laboratories. The collaboration will be overseen by a joint steering committee made up of HSCI and GSK scientists and managers.
“This is an exciting, extremely significant moment in the history of HSCI,” said Brock Reeve, the Stem Cell Institute’s Executive Director. “This kind of collaborative alliance with GlaxoSmithKline, one of the world's leading research-based pharmaceutical and healthcare companies, will allow the Harvard Stem Cell Institute to ultimately fulfil its promise of advancing stem cell science to benefit patients.”
GlaxoSmithKline – one of the world’s leading research-based pharmaceutical and healthcare companies – is committed to improving the quality of human life by enabling people to do more, feel better and live longer.For more information, visit www.gsk.com.
Harvard Stem Cell Institute – The Harvard Stem Cell Institute is a scientific collaborative within Harvard University and its affiliated hospitals, established to fulfill the promise of stem cell biology as a basis for cures and treatments for a wide range of chronic and medical conditions. HSCI takes a broad approach, focusing not only on stem cell science, but also on teacher education and programs in bioethics and in the public policy issues generated by stem cell research. For more information, visit www.hsci.harvard.edu.
Source: GSK Press Release
$24 Million in New Stem Cell Research Funding Awarded
to 25 California Institutions
Grants to Help Create New Stem Cell Lines and Drive Research on Specific Diseases
SAN FRANCISCO, Calif., June 27, 2008—The governing board of the California Institute for Regenerative Medicine (CIRM), the state’s stem cell agency, today awarded $24 million under two separate grant programs; one that will fund research for the development of new lines of pluripotent human stem cells, and the other that will fund the planning stages of an innovative model for research teams that will collaborate on therapies for a specific disease or injury.
New Cell Lines Awards
The CIRM New Cell Lines Awards support the derivation and propagation of new lines of pluripotent human stem cells with important research and clinical application for understanding, diagnosing and treating serious injury and disease. $23 million in funding for a total of 16 grants were approved at today’s meeting.
The New Cell Lines Awards support two categories of research and give particular consideration to research that cannot be currently funded by federal programs:
- Derivation of new human embryonic stem cell lines using excess or rejected early-stage human embryos generated by in vitro fertilization.
- Derivation of pluripotent human stem cell lines from other sources using alternative methods such as, but not limited to, somatic cell nuclear transfer (SCNT) or reprogramming of neonatal or adult cells (iPS cells).
To ensure that research moves forward in all of the areas that have potential to deliver medical advances to patients, the grants support research across the spectrum of approaches used to derive pluripotent stem cell lines, including the well-established means of human embryonic stem cells, which remain the gold standard for research into pluripotent cells, as well as new technologies such as iPS.
“Ultimately, our goal is to apply the knowledge gained in basic research towards treatments and cures for patients” stated Dr. Alan Trounson, president of CIRM. “Pluripotent stem cells play a key role in developing stem-cell based therapies because of their unique ability to renew themselves and their potential to form almost all of the cell types of the body. As such, derivation of new human embryonic stem cell lines is a priority for both basic and translational research that could be the foundation for advancing new therapies.”
Disease Team Planning Grants
Separately, the ICOC awarded 22 grants totaling $1.1 million to support multi-disciplinary teams of scientists in pursuit of therapies for specific diseases. The Disease Team Planning grants provide relatively modest grants to scientists who will use the funds to assemble multi-disciplinary teams that will help prepare proposals that can respond to an upcoming request for proposals for major grants for translational research that could lead to clinical trials. Ultimately, the goal is to fund the work of disease teams that would result in a therapy or diagnostic for a particular disease or serious injury.
The CIRM Disease Team Award Request for Application (RFA) will be posted in March and considered by the ICOC in June 2009. The goal of these awards is to facilitate the integration and organization of the highest quality basic, translational, and clinical research in a team setting. This innovative disease team approach has the potential to advance therapies into the clinic more rapidly. Receipt of a Disease Team Planning Grants is not a requirement for applying for a Disease Team Award.
“Since CIRM began making grants in 2006, we have been steadily advancing a scientific strategic plan, through a combination of research, training and facility grants, that establishes California as one of the most comprehensive and robust stem cell research environments in the world” stated Robert N. Klein, chairman of the governing board of CIRM. “The grants awarded today play a tremendously important role in advancing our ultimate goal of delivering new therapies and cures to Californians afflicted with a range of devastating diseases and injuries.”
Other ICOC Business
In other business, Chairman Klein read into the record an official thanks to the State Controller’s Office for the partnership they have extended in working with the staff at CIRM to distribute $195 million in funding of the CIRM Major Facilities grants. Earlier in the week, these funds were distributed to the eight institutions that opted to receive their funding this summer with a nine percent reduction, rather than spreading their funding over the two years it will take to build the facilities. Chairman Klein stated, “John Chiang, the state Controller, and his staff demonstrated how government can perform at its best. In recognizing how critical these research facilities are to advancing medical therapies for heart disease, blindness, diabetes, cancer and 70 other areas of chronic disease and injury, the Controller’s office clearly cut substantial time out of the normal payment process to accelerate the building of these vital medical facilities.”
Additionally, the ICOC voted to approve a new concept plan for a Translational 1 RFA for research that enables translation of basic stem cell research to potential clinical application. This award will support two types of early translational research including research that results in a therapy development candidate that meets an unmet medical need, and research that addresses a significant bottleneck that, if overcome, would advance effective translation of discoveries towards testing in patients. The Translational 1 RFA will be released in August 2008, with applications due in November 2008 and awarded in the Winter/Spring of 2009.
The tables below detail the grants approved at today’s ICOC meeting:
New Cell Lines Awards
Application Number
Institution
PI Name
First Year Total
Total Budget
RL1-00630-1
StanfordUniversity
Dr. Julie C. Baker
$474,804
$1,424,412
RL1-00634-1
StanfordUniversity
Professor Michele P. Calos
$464,418
$1,406,875
RL1-00636-1
University of California, Los Angeles
Dr. Amander T. Clark
$390,048
$1,177,648
RL1-00639-1
The J. David Gladstone Institutes
Dr. Bruce R. Conklin
$569,520
$1,708,560
RL1-00644-1
University of California, San Diego
Dr. Steven F. Dowdy
$462,600
$1,387,800
RL1-00648-1
University of California, San Francisco
Dr. Susan J. Fisher
$462,372
$1,383,419
RL1-00650-1
The J. David Gladstone Institutes
Dr. Fen-Biao Gao
$569,520
$1,708,560
RL1-00660-1
University of California, San Francisco
Dr. Long-Cheng Li
$453,288
$1,375,144
RL1-00662-1
StanfordUniversity
Professor Michael T. Longaker
$474,804
$1,424,412
RL1-00667-1
University of Southern California
Professor Martin Frederick Pera
$490,258
$1,387,508
RL1-00669-1
University of California, San Francisco
Dr. Miguel Ramalho-Santos
$417,695
$1,307,201
RL1-00670-1
StanfordUniversity
Professor Renee Reijo Pera
$470,245
$1,410,042
RL1-00681-1
University of California, Los Angeles
Dr. Jerome Zack
$460,800
$1,382,400
RL1-00682-1
Burnham Institute for Medical Research
Dr. Zhuohua Zhang
$529,920
$1,589,760
RL1-00649-1
The Salk Institute for Biological Studies
Professor Fred H. Gage III
$579,240
$1,737,720
RL1-00678-1
University of California, Irvine
Professor Leslie Michels Thompson
$455,400
$1,369,800
Total
$23,181,261
Disease Disease Team Planning Awards
Application Number
Institution
PI Name
Total Budget
DT1-00652-1
University of California, San Francisco
Dr. Jeffrey Allen Bluestone
$55,000
DT1-00653-1
University of Southern California
Dr. David Timothy Woodley
$42,574
DT1-00656-1
University of California, San Francisco
Dr. Jeffrey Charles Lotz
$55,000
DT1-00657-1
University of Southern California
Dr. Mark S Humayun
$50,001
DT1-00659-1
The Salk Institute for Biological Studies
Samuel L. Pfaff
$54,798
DT1-00669-1
University of California, Los Angeles
Dr. Stanley Thomas Carmichael
$44,792
DT1-00671-1
The J. David Gladstone Institutes
Dr. Deepak Srivastava
$53,972
DT1-00672-1
Novocell, Inc.
Dr. Emmanuel Edward Baetge
$48,950
DT1-00674-1
StanfordUniversity
Dr. Thomas A. Rando
$52,650
DT1-00675-1
University of California, San Diego
Dr. Lawrence S. B. Goldstein
$55,000
DT1-00683-1
University of California, Los Angeles
Professor Irvin S.Y. Chen
$52,500
DT1-00688-1
Buck Institute for Age Research
Dr. Xianmin Zeng
$55,000
DT1-00690-1
University of California, Irvine
Dr. Henry John Klassen
$37,367
DT1-00696-1
Ludwig Institute for Cancer Research
Dr. Webster K. Cavenee
$55,000
DT1-00697-1
Children's Hospital Oakland
Mark Walters
$55,000
DT1-00698-1
Cedars-SinaiMedicalCenter
Dr. Eduardo Marban
$46,886
DT1-00700-1
University of California, Irvine
Leslie Michels Thompson
$54,618
DT1-00701-1
Children's Hospital of Los Angeles
Donald Kohn
$33,110
DT1-00704-1
Burnham Institute for Medical Research
Professor Mark Mercola
$53,150
DT1-00708-1
Beckman Research Institute of the City of Hope
Professor Michael Edward Barish
$55,000
DT1-00709-1
University of California, San Diego
Dr. Dennis Carson
$55,000
DT1-00710-1
StanfordUniversity
Dr. Robert C. Robbins
$55,000
Total
$1,120,368
About CIRM CIRM was established in early 2005 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure, which provided $3 billion in funding for stem cell research at California universities and research institutions, was overwhelmingly approved by voters, and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities, and other vital research opportunities. To date, the CIRM governing board has approved 206 research and facility grants totaling more than $554 million, making CIRM the largest source of funding for human embryonic stem cell research in the world. For more information, please visit www.cirm.ca.gov.
Source: CIRM Press Release
UCLA RESEARCHERS IDENTIFY LEUKEMIA STEM CELLS AND THE ALTERATIONS THAT CAUSE NORMAL CELLS TO BECOME CANCEROUS
Stem cell researchers at UCLA have identified a type of leukemia stem cell and uncovered the molecular and genetic mechanisms that cause normal blood cells to become cancerous.
The discovery may lead to the development of new therapies that target these leukemia stem cells, attacking the disease at its very root and killing the early cells that give rise to mature cancer cells. The study appears in the May 22, 2008 issue of the journal Nature.
Recent studies suggest that cancer stem cells may contribute to development of several types of cancer, cancer relapse and drug resistance. New anti-cancer therapies will need to kill or stop the cancer stem cell from proliferating to battle the cancer most effectively. Therapies targeting the derivatives of the cancer stem cell, the mature cancer cells, are not as effective and leave the “seeds” of the disease untouched.
If scientists could understand the biology of cancer stem cells and find a way to kill them, new and much more effective therapies could be developed to seek out and eliminate the cancer stem cells, robbing the cancer of its ability to re-grow and crippling, if not completely eliminating it, from the body.
Led by Dr. Hong Wu, a professor of medical and molecular pharmacology and a scientist with the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, the UCLA team has for the first time identified and isolated the cancer initiating cells responsible for a type of leukemia known as acute T-lymphoblastic leukemia, an aggressive and deadly cancer that occurs both in children and adults. Using a genetically engineered model, the team also discovered the key steps that turn normal blood cells into malignant leukemia stem cells, providing potential targets for news therapies.
“One of the main challenges in cancer biology is to identify cancer stem cells and define the molecular and genetic events required for transforming normal cells into cancer stem cells,” said Wu, senior author of the Nature study and also a researcher at UCLA’s JonssonComprehensiveCancerCenter.
The origin of cancer stem cells is not entirely clear. Normal tissue stem cells – in this case blood cells - are likely targets for insults that can initiate cancer due to their long
life span, necessary for the accumulation of the multiple genetic or epigenetic changes required for malignant transformation. However, mutated stem cells do not necessarily become cancer stem cells. Recent experimental evidence suggests that cancer can originate from either a self-renewable stem cell population or their more differentiated progenies that have acquired self-renewal capacity due to accumulated mutations. In a leukemia model generated for this study, the leukemia stem cells carry T lymphocytic marker, suggesting a T-cell origin.
The UCLA team wanted to know how blood stem cells become cancerous and studied the cells at the molecular and genetic level to uncover those mechanisms. “We know that cancer formation requires multiple genetic or molecular alterations,” said Wei Guo, the first author of the study and a postdoctoral associate in Wu’s lab who received support from the California Institute of Regenerative Medicine. “Our study suggests that such alterations may happen at the level of cancer stem cells.”
The alterations found that collaboratively contribute to leukemia stem cell formation were the deletion of the PTEN tumor suppressor gene, a chromosomal translocation involving c-myc, an oncogene, and the activation of a signaling molecule, called beta-catenin, that is known to be involved in stem cell self-renewal.
“Interestingly, the events that we have identified in our model system have also been found in human leukemia patients,” Wu said. Wu and her team currently are testing therapies that target the alterations they discovered, hoping to interrupt the process that causes the normal blood cells to become leukemia stem cells, thereby stopping the cancer. They’re also looking for other alterations that might be at play in resistance to these targeted therapies. Dr. Luisa Iruela-Arispe, a professor of molecular, cell and developmental biology and a Broad Stem Cell Center researcher, also is a co-author of the Nature paper.
The stem cell center was launched in 2005 with a UCLA commitment of $20 million over five years. A $20 million gift from the Eli and Edythe Broad Foundation in 2007 resulted in the renaming of the center. With more than 150 members, the Eli and EdytheBroadCenter for Regenerative Medicine and Stem Cell Research is committed to a multi-disciplinary, integrated collaboration of scientific, academic and medical disciplines for the purpose of understanding adult and human embryonic stem cells. The institute supports innovation, excellence and the highest ethical standards focused on stem cell research with the intent of facilitating basic scientific inquiry directed towards future clinical applications to treat disease. The center is a collaboration of the David Geffen School of Medicine, UCLA’s JonssonCancerCenter, the Henry Samueli School of Engineering and Applied Science and the UCLA College of Letters and Science. To learn more about the center, visit our web site at http://www.stemcell.ucla.edu/.
Source: CIRM Press Release
Cellartis Enters into a Research Collaboration with Pfizer to Develop a Screening System for Detection of Human Toxicity using Human Embryonic Stem Cell Lines
Gothenburg, Sweden-- Cellartis announced that it has entered into a collaborative research agreement with Pfizer for the development of a novel model system for the detection of human toxicity in vitro. The collaboration aims to generate a predictive developmental screening model for new chemical entities using the unique patented Cellartis human Embryonic Stem (ES) cell platform.
It is important to identify toxic substances as early as possible in the drug development process. Through this collaboration, Pfizer and Cellartis are embarking on potentially pioneering work on human relevant developmental toxicity models, which may yield a solution long sought by the industry.
While presumed human teratogens are presently identified using data from mouse, rat, or rabbit, the novel human ES cell-based system would enable the identification of genuine human developmental toxicity data at the pre-clinical in vitro stage. Cellartis will take the lead in the development of the human ES cell model system and will conduct validation compound testing. Pfizer will provide expertise and capabilities in the design and optimization of the developmental toxicity prediction model. The collaboration provides Pfizer with access to specific human ES technology, based on Cellartis’ patented human ES platform, for developing the toxicity screening system.
Cellartis receives an upfront fee as well as research funding from Pfizer. In addition, Cellartis retains the right to sublicense, to make, use, and sell the developmental toxicity screening model.
Announcing the collaboration, Mats Lundwall, the Chief Executive Officer of Cellartis, commented: ”We are delighted that Pfizer has chosen to collaborate with Cellartis in the human ES field. We are confident that the unique characteristics of our human ES cell technology have the potential to produce breakthroughs in the prediction of human developmental toxicity. I can think of no stronger partner for Cellartis in developmental toxicity research than Pfizer.”
About Cellartis
CellartisAB is a Swedish/British biotechnology company focused on human embryonic stem (hES) cells for drug discovery, toxicity testing and regenerative medicine with the main objective to develop hepatocytes and cardiomyocytes from these cells. The company is the world’s largest single source of defined hES cell lines, and has developed more than 30 well documented cell lines. Two cell lines are listed on the NIH Stem Cell Registry and 22 are approved by the UK Stem Cell Bank. In addition, Cellartis has built the world’s first large-volume production facility for human ES cells. The company’s strategy is to accelerate product development by working in partnership with academia and industry. The company was founded in 2001, has 49 employees and is located in Gothenburg, Sweden and Dundee, UK.
For more information, please visit www.cellartis.com .
Source: Cellartis Press Release
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CALIFORNIA STEM CELL INSTITUTE DISAGREES WITH PRESIDENT BUSH’S MISLEADING POSITION ON STEM CELL RESEARCH
SAN FRANCISCO, Calif., January 28, 2008 – The California Institute of Regenerative Medicine (CIRM)issued the following statement in response to President Bush’s State of the Union address: Tonight, in his State of the Union address, President Bush distorted the scientific facts on stem cell research and did a disservice to the millions of patients suffering from chronic disease and injury for whom stem cell research holds great promise for future therapies and cures.
The stem cell research community is united in the position that human embryonic stem cells clearly remain the gold standard for research into pluripotent cells – cells that have the capacity to form all tissues of the body. Human embryonic stem cells are also the model against which all other potentially pluripotent cells need to be compared. The President’s proposals to further limit medical research in this area fail to take into account the intricate realities of the state of stem cell research. Indeed, the recent advances in which skin cells were induced to become pluripotent would not have been possible without research involving human embryonic stem cells. Furthermore, induced pluripotency is a technology still in its infancy. Though this technology offers great hope and promise, it will not, for the foreseeable future, be suitable for clinical studies in humans because of safety concerns.
Therefore it is critical that all avenues of stem cell research be aggressively advanced. To do otherwise would increase the already devastating restrictions that have burdened Federal support of stem cell research and patients who are depending upon it. This Administration’s position on stem cell research has already cost years in lost research productivity. Further restrictions would result in more lost time in developing stem cell based therapies and cures that hold great promise to alleviate suffering for the most destructive and costly diseases such as spinal injury, loss of sight, heart muscle injury, Parkinson’s Disease, ALS and diabetes.
CIRM supports and applauds any programs the White House advances that accelerate NIH funding for research on induced pluripotency. There is much work to be done on all cell types, including this highly promising but early stage technology. CIRM looks forward to engaging with NIH and other state and federal organizations in accelerating the progress of stem cell therapies to the clinic.
CIRM also strongly opposes reproductive cloning.
About CIRM
CIRM was established in 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure, which provided $3 billion in
funding for stem cell research at California universities and research institutions, was overwhelmingly approved by voters, and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities, and other vital research opportunities. To date, the CIRM governing board has approved 156 research grants totaling almost $260 million, making CIRM the largest source of funding for human embryonic stem cell research in the world. For more information, please visit www.cirm.ca.gov.
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e-published on MedicineandBiotech.com December 1st, 2007
Stem Cells are once again in the forefront of news with two major announcements last month- one involving the “production of cloned primate embryonic stem cells” at Oregon Health & Science University and the other on “reprogramming of adult human cells” by a US team and a Japanese team. Both represent key developments in innovative methods for creating 'pluripotent' cells, which can develop into almost any of the body's cell types. However, for true clinical applications for these technologies, the road is a long one. This hype should not derail the research and clinical developments in progress using “true” human embryonic stem cells, which will at any time, any day be the most optimum cells for developing treatments for complex diseases like Diabetes, Parkinson’s and Spinal Cord Injuries. Successful development of any treatment modality will involve pursuing every possible research and clinical source, whether human embryonic stem cells or those derived from adult cells.
The US and Japanese studies on “reprogramming of adult human cells” feature in the journals Science and Cell. The Japanese team treated human skin cells with a cocktail of chemicals so they reverted to a pliable embryonic state. Some of the cells were developed to beat like those found in the muscle of the heart. The US team, from the University of Wisconsin-Madison, achieved the same effect by using a slightly different combination of chemicals.
Researchers at OregonHealth & ScienceUniversity’s Oregon National PrimateResearchCenter have also made a significant breakthrough. For the first time, scientists have successfully derived embryonic stem cells by reprogramming of genetic material from skin cells while studying rhesus macaque monkeys.
This breakthrough follows several previously unsuccessful attempts by the OHSU-based team and other scientific teams worldwide. The results of the work were released online by the scientific journal Nature.
Prior to the OHSU team’s recent success in a species closely related to humans, scientists worldwide have isolated stem cells only in mice using a technique called “somatic cell nuclear transfer” (SCNT). The method involves transplanting the nucleus of the cell, containing an individual’s DNA, to an egg cell which has had its genetic material removed. For various reasons and despite numerous attempts, previous efforts to use the SCNT technology to clone stem cells in primates have failed repeatedly.
“Many scientists believe that embryonic stem cells hold great promise for treating a variety of diseases including Parkinson’s disease, multiple sclerosis, cardiac disease and spinal cord injuries,” explained Shoukhrat Mitalipov, Ph.D., director of the OHSU-based research team and an assistant scientist in the Division of Reproductive Sciences at ONPRC, the Oregon Stem Cell Center and the department of Obstetrics and Gynecology of Oregon Health & Science University. Don Wolf, Ph.D. of the primate center also played a significant role in the research.
“Using our advanced methods, it is conceivable that years from now, patients could receive therapeutic embryonic stem cells developed from their very own cells meaning that there would be no concerns about transplant rejection. Another noteworthy aspect of this research is that it does not involve the use of fertilized embryos, a topic which has been the source of a significant ethical debate in this country. ”
The Munroe-Meyer Institute and the Whitehead Institute for Biomedical Research collaborated with OHSU to conduct this research. The studies were funded by the Oregon National PrimateResearchCenter, the NationalCenter for Research Resources, and the National Institute of Neurological Disorders and Stroke, both components of the National Institutes of Health.
“This advance at the Oregon National PrimateResearchCenter builds on studies supported over several years by the NCRR aimed at understanding the basic biology of stem cells and at developing methods to investigate non human primate models of disease,” said John D. Harding, Ph.D., NCRR's Director of Primate Resources. “These studies have great potential to accelerate progress in the field of regenerative medicine.”
The reason why Mitalipov’s team was successful when so many other previous attempts were not, lies in the method for identifying and extracting the nuclei of the eggs being used. Prior attempts resulted in damaged eggs due to the difficulties involved in removing the nucleus. This means that the eggs were not fully functional and failed to divide and develop.
To conduct the research, researchers obtained skin cells from a nine-year-old male rhesus macaque monkey at the Oregon National PrimateResearchCenter. The researchers then used specialized imaging software called, Oosight Spindle Imaging System, to spot and remove the nuclear material attached to the egg’s spindle fibers. The nuclei of skin cells were then inserted into nucleus-free eggs. Using this technique, two embryonic stem cell lines - groups of cells that can grow indefinitely and differentiate into any cells of the body- were successfully developed. The genetic material (DNA) of cell lines was then matched to DNA from the male donor male monkey to ensure that they were a direct clone.
Successful development of the cell lines required numerous attempts. Overall, 304 monkey eggs (oocytes) from 14 female rhesus monkeys were used to generate the two embryonic stem cell lines, a .7 percent success rate
“While development of the stem cell lines required hundreds of attempts, this research proves it can be done and will likely lead to refinements which will make the process more efficient and lead to a higher success rate,” explained Mitalipov. “This is the next step for our research team as other scientists continue to investigate the promise of stem cell therapies.”
LITIGATION AGAINST CALIFORNIA STEM CELL PROJECT ENDS
---State Supreme Court Refuses Appeal of Constitutional Challenge----
SAN FRANCISCO, May 16, 2007 – The California Supreme Court today declined to hear an appeal in the litigation challenging the constitutionality of Proposition 71, the California Stem Cell Research and Cures Act. The Court’s decision effectively ends the lawsuits that have held up bond funding for the California Institute for Regenerative Medicine (CIRM), the state agency created to manage the stem cell project.
“More than two years ago, the people of California voted overwhelmingly to support stem cell research with $3 billion in bond funds,” said Robert N. Klein, chairman of the Independent Citizens Oversight Committee, the CIRM governing board. “The groups opposed to stem cell
research immediately filed a constitutional lawsuit to block the funding of this critical frontier for medical research.”
Faced with litigation that was intended to shut down all funding, Klein stated at the time,“Against all odds, we shall succeed. We have a moral obligation -- to every patient, to every family, to every Californian, to every American -- to succeed; and so we shall.”
“It had to be demonstrated that litigation could not destroy the progress of every democratic mandate in this country; the funding had to proceed during the course of this litigation,” Klein said today. “The agency’s board, with the extraordinary support of the agency’s staff, proceeded to fund $158 million in stem cell research, representing 103 research grants at 23 non-profit research institutions and 169 research fellows at 16 universities, medical schools and research institutions. CIRM is now the largest source of funding for human embryonic stem cell research in the world.”
Today’s decision permits the state agency to immediately move into the next round of funding which will include up to $48.5 million in shared laboratories grants and loans and $222 million for major facilities at California’s universities, research hospitals, medical schools, and research
institutes.
“The voters’ mandate will be fulfilled and the promise of stem cell research will be lifted to new heights with California’s billions in funding focused on helping millions of children, husbands and wives, grandparents and friends in their struggle against chronic disease and injury,”
continued Klein. “California’s fight against human suffering has achieved lift off. Today is a day of victory for all of those men and women who have dedicated their lives to medical research and the fight against chronic disease and injury, and it is a day of celebration for everyone with a
loved one suffering from chronic disease or injury. We can only pray that the rest of the country will now join California in dedicating the entire nation’s medical resources to advance the stem cell frontier to therapies and cures for every patient.’
After losing at the Superior Court and Court of Appeals, plaintiffs in the litigation – the California Family Bioethics Council, along with the People’s Advocate and National Tax Limitation Foundation, represented by the Life Legal Defense Foundation – filed petitions for
review with the Supreme Court in April. The Court today denied those petitions. Under California law, no additional litigation can further delay the issuance of general obligation bonds authorized by Proposition 71. “We will work closely with the State Treasurer in the coming weeks to prepare for the initial stem cell bond offering,” said Klein. “We need to first repay the state and the private philanthropists who advanced funds to CIRM for research grants, then move forward aggressively with additional training, research and facility grants.”
Governor Arnold Schwarzenegger authorized a $150 million loan to the CIRM this past summer from the state’s general fund. Another $45 million came from 14 individuals and institutions through the purchase of bond anticipation notes (BANs).
About CIRM
Governed by the ICOC, the CIRM was established in 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Initiative. The statewide ballot measure, which provided $3 billion in funding for stem cell research at California universities and research institutions, was approved by California voters, and called for the establishment of an entity to make grants and provide loans for
stem cell research, research facilities, and other vital research opportunities. To date, the ICOC has approved 119 research grants totaling more than $158 million. For more information, please visit www.cirm.ca.gov.
###
CALIFORNIA STEM CELL PROJECT PREVAILS
--Appellate Court Affirms Constitutionality of Proposition 71--
SAN FRANCISCO, CA, Feb 27, 2007 – For the second time in less than a year, the California judiciary has issued a commanding decision affirming the Constitutionality of the state’s innovative stem cell research project.
Proposition 71, the California Stem Cell Research and Cures Act, was approved by 7 million voters (59 percent) in November, 2004, and authorized a $3 billion bond program to fund stem cell research in the state. Since then, litigation challenging the Constitutionality of the Act and the California Institute for Regenerative Medicine (CIRM), the agency created to manage the project, has precluded the issuance of bonds.
The State Court of Appeal ruled unanimously yesterday that:
…the objective of the proposition is to find, "as speedily as possible," therapies for the treatment and cure of major diseases and injuries, an aim the legitimacy of which no one disputes. The very pendency of this litigation, however, has interfered with implementation for more than two years. After careful consideration of all of appellants’ legal objections, we have no hesitation in concluding, in the exercise of "our solemn duty to jealously guard the precious initiative power…," that Proposition 71 suffers from no constitutional or other legal infirmity. Accordingly, we shall affirm the well-reasoned decision of the trial court upholding the validity of the initiative.
Robert N. Klein, chairman of the Independent Citizens Oversight Committee (ICOC), the CIRM’s 29-member governing board, said, "Once again, the judiciary has upheld the Constitutionality of California’s innovative stem cell research project – in its entirety, without equivocation, and with absolutely no room for further argument. We are grateful that the Court rendered this decision so quickly, as it speeds the day when the will of 7 million voters can be fully realized."
The Appellate Court heard oral arguments in the case on February 14, 2007. Plaintiffs – the California Family Bioethics Council, along with the People’s Advocate and National Tax Limitation Foundation, represented by the Life Legal Defense Foundation – had appealed an Alameda Superior Court decision which also upheld Prop 71. That decision had been rendered last April.
The CIRM and the ICOC were defended by Attorney General Jerry Brown, who said, "This is a bright day for the people of California who want to see stem cell research accelerated. The Court of Appeal gave us a rock solid opinion. This is the end of the road for the obstructionists."
Despite the litigation, the stem cell research project is moving forward. Last summer, Governor Arnold Schwarzengger authorized a $150 million loan from the state’s general fund to the CIRM, and private philanthropists provided an additional $45 million in loans. That funding allowed the ICOC to approve 72 grants totaling nearly $45 million for embryonic stem cell research at 20 California institutions earlier this month. Another $80 million in grants will be considered by the ICOC on March 15th and 16th, when it meets in Los Angeles. At that point, California will become the world’s leading funder of stem cell research, searching for therapies and cures for Parkinson’s, cancer, heart disease, diabetes, Alzheimer’s, and more than 70 other chronic diseases and injuries.
Yesterday’s decision is expected to be appealed to the California Supreme Court within the 40-day statutory limit. The Supreme Court must then decide within 90 days whether it will accept the case.
"We are extremely confident in the strength of our position," said Klein. "Our arguments are grounded in more than 100 years of legal precedent. We are convinced that the mandate of the voters will be upheld and that this litigation will conclude by yearend. Patients and families should take heart. We are growing closer to our goal of turning stem cells into therapies and cures."
"The first years will yield knowledge in how to mitigate the tragic consequences of disease," Klein continued. "The major breakthroughs will take years of dedicated research and medical trials."
"Throughout this litigation, we have been ably represented by former Attorney General Bill Lockyer, Attorney General Jerry Brown, and Deputy Attorney General Tamar Pachter," Klein said. "The efforts of our outside counsel, James Harrison of Remcho, Johansen & Purcell, and our interim counsel, Scott Tocher, have also been invaluable. And, of course, we’ve enjoyed the strong support of the many scientists, research institutions, and patient advocate organizations who filed amicus briefs in both the Superior Court and Court of Appeal cases."
About CIRM
Governed by the ICOC, CIRM was established in 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure, which provided $3 billion in funding for stem cell research at California universities and research institutions, was approved by California voters, and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities, and other vital research opportunities. For more information, please visit www.cirm.ca.gov.
Source: CIRM Press Release
Taking Stem Cells to the Clinic…a discussion with Dr. Outi Hovatta
By Neerja Sethi, Managing Editor
Professor Outi Leena Hovatta, MD, PhD is Professor of Obstetrics and Gynecology at the Karolinska Institute, Karolinska University Hospital Huddinge, Stockholm, Sweden (www.ki.se) and also serves as the current Director of Stem Cells Research at REGEA, Institute for Regenerative Medicine in Tampere, Finland (www.regea.fi). Dr. Hovatta is a world-renowned leading expert in developing technologies for cultivating human embryonic stem cell lines. She has extensive experience in developing organ cultures since 1970's. She has been active in developing clinical in vitro fertilization (IVF) treatment since 1986, and ovarian tissue and oocyte cryopreservation and culture of ovarian follicles and oocytes since 1995. In the laboratory of the IVF Unit of the hospital, her team has derived more than 20 embryonic Stem cell lines since March 2002, paying particular attention to the quality aspects. Dr. Hovatta is the lead member of the inter-disciplinary program on characterization, growth and differentiation of human embryonic stem cells at the KarolinskaUniversityHospital. Her team has wide collaborations within Sweden and internationally. She has published extensively on stem cells research in majority of leading scientific journals.
Advances in stem cell research are taking us closer to therapeutic applications in the clinic for a wide variety of chronic human diseases including Parkinson’s, Spinal Cord injuries, Diabetes, Cardiovascular ailments and others. However, there are a number of skeptics both in the scientific community and the business community who are still not convinced about the potential of this technology. In this New Year 2007 issue of MedicineandBiotech.com, we have a candid talk with Dr. Hovatta, one of the most trusted names in the field of Stem Cell biology and Medicine on the facts about the current status of the field. Dr. Hovatta’s opinions are very frequently sought by the leading scientific journals and news media.
What is the current status of embryonic stem cells research in the European Union and in the USA?
Currently in the US, due to a number of independent initiatives by various states, the funding situation for embryonic stem cell research is improving. However, there is a limited group of researchers who can qualify for these funds. The NIH grants only support research using the older stem cell lines and do not fund any derivation of newer stem cell lines. The old lines are clinically, quantitatively and qualitatively not sufficient. The hopes are now focused on funding availability in California.
In the EU, there is limited amount of funding available for embryonic stem cell research. Currently, 12 million euros have been assigned for the next four years for stem cell research for the whole EU. Today there are very few good, clinical quality embryonic stem cells lines available in the EU. Therefore, 12 million euros are not sufficient to promote the science adequately. The European Stem Cell Integrated Project (www.estools.eu) is developing an initiative to produce standardized, quality embryonic stem cell lines.
What is the current status of human embryonic stem cell lines in the EU?
There are number of groups all over the EU who are actively deriving embryonic stem cell lines -for example, about 7 groups in the UK, 3 in Sweden, 2 in Finland, 2 in France, 1 group in Switzerland and several groups in Spain. In addition, number of European biotech companies are also specializing in stem cell engineering. Therefore, the science and technology is progressing very actively.
How many human embryonic stem cell lines derived therapies are heading towards the clinic?
In the laboratory at the tissue culture level, stem cell technology is showing remarkable results as therapeutic agents, especially for neurodegenerative diseases and cardiovascular ailments, like myocardial infarction. There are numerous studies proceeding in animal models, including studies in primates being conducted in France that show very promising results. Soon many of these studies will make a transition into the clinical trials in humans.
According to the new EU directive, human embryonic stem cells for transplantation or clinical applications have to be cultured under Good Manufacturing Practice (GMP) standards to guarantee the safety of stem cells. Therefore, now as we are moving closer to the clinic, identifying all culture constituents meeting GMP standards is an important requirement for those who derive human-embryonic stem-cell lines of clinical quality. Other aspects for clinical quality stem cell lines that are critical are- how to avoid immune reactions in transplantation, and how to prevent genetic changes during long-term culture.
What are the current limitations for the progress of embryonic stem cell technology?
Currently, majority of stem cell research is being conducted in University laboratories which have very limited funding. There are few small private biotech companies specializing in stem cell technologies. However, the technology has not been able to attract substantial private investments. Well-established Pharmaceutical companies and venture capitalists are not investing large amounts in the technology and are cautiously watching the progress. However, the promising aspect is that a large pool of brilliant scientists is lining up to work in this exciting field of science.
Can Adult stem cells fill this void?
Today we know that adult stem cells have limited therapeutic applications. For example, mesenchymal bone marrow cells are effective in some revascularization in the heart. However, detailed studies have shown that adult stem cells cannot integrate correctly into the heart and can cause arrhythmia. Slowly adult stem cell researchers are becoming unanimous in their decision that adult stem cells are not enough.
As a clinician. scientist and as a woman, what keeps you motivated in pursuing stem cell research despite so many hurdles?
I have been in the field of reproductive biology for over 30 years. My interests developed in reproductive biology as a medical student and then I specialized in the techniques of in vitro fertilization. My interests focused on improving in vitro cultures and subsequently, developing and improving stem cell culture methods was a logical progression. My motivation is to solve and cure a number of diseases in the mankind from a reproductive biology based technology.
*Dr. Outi Hovatta and Dr. Neerja Sethi collaborate on various scientific and educational projects.
SAN FRANCISCO, CA-October 2006 – The California Institute for Regenerative Medicine (CIRM) announced that it has received 232 applications for Scientific Excellence through Exploration and Development (SEED) Grants, the first stem cell research grants it will award since passage of Proposition 71 in November 2004.The applications are from individual researchers at 36 non-profit institutions in California.
SEED Grants are intended to bring new ideas and new investigators into the field of human embryonic stem cell research, and offer an opportunity for investigators to carry out studies that may yield preliminary data or proof-of-principle results that could then be extended to full scale investigations.
"The response demonstrates the keen interest in the field and the pent up demand for funding for human embryonic stem cell research in California,” said Dr. Arlene Y. Chiu, CIRM’s Director of Scientific Programs.“We’re delighted that there is such strong competition for our inaugural research initiative, and that the applicants are considering such a broad range of approaches.It bodes well for the future of our program."
CIRM's governing board, the Independent Citizens Oversight Committee (ICOC), authorized up to $24 million to support as many as 30 SEED Grants over two years.The applications will be reviewed by a committee of scientific experts from outside California and patient advocates from the ICOC.The committee will evaluate the scientific merit of each proposal and make funding recommendations to the full ICOC, which has final authority to award CIRM grants.Grant recipients will be named at the ICOC’s January meeting.
About CIRM
Governed by the ICOC, CIRM was established in 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Initiative. The statewide ballot measure, which provided $3 billion in funding for stem cell research at California universities and research institutions, was approved by California voters, and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities, and other vital research opportunities. For more information, please visit www.cirm.ca.gov.
STEM CELL AGENCY BOARD APPROVES PROPOSAL FOR HUMAN EMBRYONIC STEM CELL RESEARCH PROGRAM
Governor Schwarzenegger-Authorized Loan Allows Science to Move Forward
August 2, 2006,
San Francisco, CA – Twelve days after Governor Arnold Schwarzenegger’s announcement of a $150 million loan to the California Institute for Regenerative Medicine (CIRM), its oversight board responded with a new proposal funding human embryonic stem cell research at California’s non-profit research institutions.
The Independent Citizens’ Oversight Committee (ICOC) approved a proposal for three Requests for Application (RFAs):
Comprehensive Research Grants—four-year grants to investigators with a record of accomplishment in human embryonic stem cell research or closely-related field that relate to a long-term therapeutic goal;
Seed Grants—two-year grants to fund innovative ideas by scientific investigators who are new to the field;
CIRM Shared Research Laboratory Grants—grants for dedicated laboratories for culturing human embryonic stem cells including core equipment and trained personnel. Additional grants in this category will be made for a course to teach culturing methods.
All grants will be made on a competitive basis to ensure that the best scientific proposals are funded. The ICOC will approve all funding decisions following scientific peer review and recommendations by the Scientific and Medical Research Funding Working Group and the Facilities Working Group, as appropriate.
“The Governor’s commitment to this science has electrified the field and given hope to millions that the promise of stem cell research will move forward in California,” said ICOC Chairman Robert Klein. “The board’s decision to focus on human embryonic stem cell research will address the critical funding gap created by the paralysis of federal policy.”
Topics to be considered in the new RFA will include but are not limited to:
Self-renewal and differentiation of human embryonic stem cells;
Derivation of new human embryonic stem cell lines, including disease-specific lines;
Assessment of tumorigenicity of human embryonic stem cells and derived cells;
Reprogramming of adult human somatic nuclei;
Studies related to identification, storage, maintenance, stability and storage of human embryonic stem cells.
“The board’s decision will allow CIRM to jump-start human embryonic stem cell research in California,” said CIRM President Zach Hall. “We have the infrastructure and policies in place to manage this exciting program in a responsible way for the people of California. We can now advance the goal set by California voters—funding the best science that leads toward therapies.”
Human Embryonic Stem Cell Research Program
Preliminary Budget
Year 1 ---
$69.5 million
Year 2 ---
$37 million
Year 3 ---
$25 million
Year 4 ---
$20 million
Total ---
$151.5 million
The first two years of the Human Embryonic Stem Cell Research Program is intended to be funded from the loan from the State of California initiated by the Governor, with the anticipated general obligation bonds funding years three and four. An additional RFA is expected later this year which can address adult, cord blood and other critical stem cell research. The State loan will be available to fund this RFA as well.
The ICOC wrote a mission statement, guiding values and strategic principles for the scientific strategic plan, which is scheduled to be presented for formal approval at the next board meeting in October. The board created a slogan for the program: “Turning stem cells into cures.”
The board also approved final regulations for Medical and Ethical Standards to guide CIRM-funded research and conflict of interest regulations for advisory working group members. They will have the force and effect of California law pending final review and publication by the California Office of Administrative Law.
About CIRM Governed by the ICOC, CIRM was established in 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Initiative. The statewide ballot measure, which provided $3 billion in funding for stem cell research at California universities and research institutions, was approved by California voters, and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities, and other vital research opportunities. For more information, please visit www.cirm.ca.gov.
Source: CIRM Press Release
STEM CELL INSTITUTE RECEIVES $14 MILLION IN PRIVATE FUNDING
Innovative Financing Program Wins Backing from State Officials
SACRAMENTO, CA – The California Stem Cell Research and Cures Finance Committee today reported that six California philanthropic entities have agreed to purchase $14 million of Bond Anticipation Notes (BANs) to fund scientific and research grants administered by the California Institute for Regenerative Medicine (CIRM).
The initial BAN investment commitments are:
Beneficus Foundation -------- $2 million
Blum Capital Partners LP -------- $1 million
William K. Bowes Foundation ------- $2 million
The Broad Foundation --------- $2 million
Jacobs Family Trust -------- $5 million
The Moores Foundation --------- $2 million
The BANs will mature in two years at a variable interest rate payable at maturity, but no more than 5%. They will be repaid when the General Obligation bonds authorized by the California Stem Cell Research and Cures Act are issued.
“This is an important step forward,” said Robert N. Klein, chairman of the Independent Citizens’ Oversight Committee, the CIRM governing board. “Stem cell research may lead to cures and therapies for a host of chronic conditions. Without the support of private investors, our progress would slow. We cannot afford to lose more time. Thanks to the leadership of these foundations, we won’t.”
“Stem cell research is one of the greatest opportunities to improve the human condition,” said Eli Broad, founder of The Broad Foundation and a BANs investor. “California will be America’s leader in stem cell research, which will not only benefit the tremendous need of people suffering from debilitating diseases and conditions but will also help the California economy immeasurably.”
Proposition 71 was approved by voters overwhelmingly in November 2004. Litigation challenging the constitutionality of the measure has impeded the State’s ability to sell approved General Obligation bonds. [That litigation is expected to conclude next spring.]
“With the State Treasurer’s Office, we are in the process of closing the transactions to reach our BANs goal of $50 million,” said Klein. “We will close these transactions incrementally in the near future.”
By Gail Schechter, Ph.D.
President, BioIntelligence
Gail Schechter, Ph.D., is President of BioIntelligence (www.BioIntelligence.com), a consulting company specializing in CNS technology assessment, product development, and grant writing. She started her career at the NIH doing clinical research and subsequently worked in the pharmaceutical industry setting up research collaborations. For the past 10 years, she has helped many companies obtain multi-million dollar grant awards. She can be reached at Brains@BioIntelligence.com.
An unlimited source of cells suitable for transplantation into the central nervous system (CNS) can be derived from embryonic stem cells. Of particular importance, embryonic stem cells can differentiate into diverse neuronal and glial cell types. These cells show a remarkable ability to proliferate, differentiate, migrate and integrate in the brain, as well as ameliorate functional deficits when transplanted into animal models of neurological disorders. In spite of recent progress in neural cell transplantation, extensive preclinical investigation is necessary to determine the optimal factors to control the differentiation process, insure safety, and demonstrate long-term effectiveness. Before clinical trials are initiated, there is much to learn about how to direct stem cell proliferation and differentiation into specific phenotypes, induce integration into existing neural and synaptic circuits, and optimize functional recovery in animal models closely resembling the human disease. In addition to embryonic stem cells, there are (slightly older) fetal stem cells and (much older) adult stem cells, each with its own set of benefits and limitations.
In honor of Christopher Reeve, this report will highlight research advances in spinal cord injury, stroke and Parkinsons disease. Other promising therapeutic targets for neural stem cells include amyotrophic lateral sclerosis, multiple sclerosis, and Alzheimers disease. Taken together, evidence is mounting that neural stem cells are capable of replacing damaged neurons, are suitable for gene therapy, and can serve as a vehicle for drug delivery to the CNS.
Stem Cell Transplantation and Other Novel Approaches to Spinal Cord Injury
Although Christopher Reeve did not live to walk again, he managed to turn the worlds attention to the need for more research in spinal cord injury and the potential of stem cell treatments. To date, experimental results suggest that stem cell transplantation and other new approaches can be beneficial in promoting recovery from spinal cord injury.
Research from the group at Washington University School of Medicine, including John McDonald (now at Johns Hopkins) shows that embryonic stem cells can give rise to neural precursor cells and that transplantation of these cells into the injured spinal cord leads to partial recovery of function. In particular, the focus on optimizing remyelination through transplantation of myelin-producing cells may offer a practical approach to restoring meaningful neurological function. When transplanted into the injured spinal cord of adult rodents, the neural precursor cells are capable of differentiating into oligodendrocytes and myelinating host axons.
Using an alternative approach to embryonic stem cells, Mark Tuszynski and his colleagues at UCSD have investigated a combination of therapeutic modalities to treat spinal cord injury. This group focuses on neurotrophic factors, including BDNF, GDNF and others, and reports that these growth factors, administered alone or in combination with cellular therapy, show a positive effect.
In one study using an ex vivo gene delivery approach to provide both trophic support and a cellular substrate for axonal growth, they implanted primary fibroblasts genetically modified to secrete GDNF into animal models of spinal cord injury. The GDNF-expressing grafts promoted significant regeneration of several spinal systems compared to the control condition, and also induced Schwann cell migration to the lesion site, leading to remyelination of regenerating axons.
In another study, they tested whether a combinatorial approach of stimulating the neuronal cell body with cAMP and the injured axon with the neurotrophic factor NT-3 would extend axonal growth into and beyond sites of spinal cord injury that had been grafted with autologous bone marrow stromal cells. The results demonstrated that significant axonal regeneration could be achieved by these combinatorial approaches.
Fetal Stem Cells to Treat Stroke
Recently, neurosurgeon Gary Steinberg and his colleagues at Stanford University School of Medicine reported the first success using fetal stem cells to re-populate the damaged region in a rat stroke model. The investigators demonstrated that fetal stem cells injected into the brains of rats could migrate to the correct location and turn into the appropriate types of neurons.
Steinberg believes signals from the damaged cells act as a distress call beckoning the transplanted cells. Other signals direct the newly arrived cells to transform into neurons and astrocytes. Steinbergs is the first paper to show that the fetal stem cells can transform into the appropriate cell types in an animal. The next step is to test whether the implanted neurons can also replace the function of the lost cells in animal models before advancing to human clinical trials.
Fetal stem cells are more advantageous than adult human stem cells because they are still able to form many types of brain cells, but they circumvent the federal restrictions and controversy associated with using embryonic stem cells in humans. Stem Cells, Inc., a company founded by study co-author and pathology professor Irving Weissman, initially isolated these cells from human fetal tissue. The company now grows the cells in bulk and distributes them to researchers studying spinal cord injuries as well as Parkinsons, Alzheimers and other brain disorders.
Dopaminergic Neurons from Embryonic Stem Cells to Treat Parkinson's Disease
Much of the initial promise of stem cell transplantation in the CNS started with Parkinsons disease. Progress with human fetal tissue transplants in hundreds of patients (although results were mixed) provided a foundation for subsequent testing of embryonic stem cells in animal models. Recent studies in a number of laboratories have now demonstrated the ability of embryonic stem cells to differentiate into dopaminergic neurons. Following transplantation, these cells have been shown to survive, release dopamine, and reverse motor deficits Parkinsons disease animal models.
The ability to induce efficient differentiation of dopaminergic neurons requires sensitive control over growth factors and culture conditions and/or genetic manipulation. Thus, the combination of genetic engineering and appropriate culture conditions are necessary to generate a robust dopaminergic cell source derived from embryonic stem cells for cell replacement therapy of degenerative diseases such as PD.
In several studies, scientists have transplanted stem cells into the brains of rats with a Parkinson-like syndrome. The cells differentiated into fully mature neurons that produced dopamine, the missing chemical messenger of Parkinson's disease. The treated animals showed functional improvement as well. While these results are encouraging, more work needs to be done before this technique becomes accepted procedure for humans with Parkinson's disease.
Conclusion
The unique features of embryonic stem cells are of extraordinary scientific, clinical, and commercial interest. Hopefully, their promise and potential will translate into discoveries in neural repair to treat a wide range of intractable neurological disorders.
* Please reference or credit any material, article or figure cited from http://www.medicineandbiotech.com as:
Copyright 2004. MedicineandBiotech.com, http://www.medicineandbiotech.com/
Disclaimer: MedicineandBiotech.com is managed by AstraGen LLC. Please re-distribute this e-magazine. AstraGen LLC and MedicineandBiotech.com do not assume, and hereby disclaim, any liability for any loss or damage caused by errors or omissions in any material published at the web-site http://www.medicineandbiotech.com, whether such errors or omissions resulted from negligence, misstatements or other oversights.
JOINT SWEDISH/CALIFORNIA SYMPOSIUM ON STEM CELLS RESEARCH: April 26th-27th, 2005, San Francisco
Bridging Forefront Scientific Achievements with Biotechnology Business Opportunities
Date: April 26 - 27, 2005, CA
Location: The Hyatt Regency San Francisco Airport Hotel,San Francisco, California
For more information, please email: symposium@medicineandbiotech.org
Website Registration: www.medicineandbiotech.org *Early Registration Discount Ends March 27th, 2005
San Francisco, CA - This April, the worlds leading stem cells researchers from
Sweden and California will join pioneers from the fields of life sciences, finance and
the legal community to map out the future of stem cells research and development in
California. The 2005 Stem Cells Symposium, on April 26-27 th , is organized by the
Consulate General of Sweden in San Francisco, the Swedish-American Chamber of
Commerce in San Francisco/Silicon Valley, the Swedish Office of Science and
Technology in Los Angeles and Astragen LLC and will bring together the scientific
and business expertise required to build bilateral world-class research
programs that foster entrepreneurship.
The Symposium will feature six keynote addresses from world-renowned researchers
and leading experts in the stem cells field, and over 12 other researchers and business
representatives will address cutting-edge issues in a series of panel discussions.
The Symposium will also arrange meetings of researchers, companies and potential
financiers to spark international research and business ventures, both in the context
of California's "Proposition 71" funding and beyond.
"Sweden has one of the most advanced Stem Cells research programs in the world
today," says Dr. Kent Persson, member of the Board of Directors at Astragen LLC and
the Chair of the Symposiums Advisory Committee. "The Symposium will attract top
level stem cells researchers and bring together both Swedish and Californian
businesses with Stem Cells Research for world class entrepreneurial ventures," says
Dr. Neerja Sethi, member of the Board of Directors at Astragen LLC and the
Symposiums Advisory Committee.
"This is a unique opportunity for Swedish and American stem cells researchers and
entrepreneurs to exchange knowledge for continued expansive stem cells research,"
says Mr. Nils Welin, Executive Director Swedish American Chamber of Commerce.
"We look forward to bringing together researchers, companies, policy makers and
others from Sweden and the US in this very important area. Due to Californias
Proposition 71 stem cells funding initiative, there is a great potential to form joint
ventures between Swedish and US concerns. Progress in the stem cells area will most
likely lead to major health benefits and economic growth in our countries," says
Helena Jonsson-Franchi, Swedish Attaché of Science and Technology, at the Swedish
Office of Science and Technology in Los Angeles.
This will be a "continuing symposium," with follow-up meetings in future years to
map progress and continue to steer the course for future international cooperation in
this important area of scientific, business and governmental cooperation
The two day symposium will be held in the San Francisco Bay Area and is scheduled to take place in mid-late April 2005. To learn more about the Stem Cell Symposium, please visit the following link: www.medicineandbiotech.org
Consulate General of Sweden in San Francisco The Consulate General of Sweden in San Francisco provides services to Swedish citizens and conducts extensive information and culture activities, arranging and supporting Swedish events in the San Francisco Bay Area.
Swedish-American Chamber of Commerce San Francisco/Silicon Valley
The main objective of the Swedish-American Chamber of Commerce in San Francisco/Silicon Valley is to foster trade between Sweden and the US and to enhance the business interests of its members. By providing a broad spectrum of services and new business opportunities through networking, the Chamber contributes to the development and improvement of goodwill between the business communities in the San Francisco Bay Area and Sweden.
Swedish Office of Science and Technology, Los Angeles The Swedish Office of Science and Technology, a Swedish Governmental Agency, is represented in strategic regions with significant development within science, technology and economic growth. The Swedish Office of Science and Technology monitors and conducts analysis in areas important to economic growth. It also initiates and supports collaboration between the Swedish and US government, academia and industry.
Astragen LLC
ASTRAGEN provides the scientific training and technological expertise to early- and mid-stage Biotech companies to efficiently and effectively transition from Preclinical development platforms to Clinical Trials/Clinical Research platforms. Our expertise is in the fields of ONCOLOGY, INFECTIOUS DISEASES, STEM CELL RESEARCH and GENOMICS. ASTRAGEN facilitates efficient integration of Preclinical Research with Clinical Research by organizing well-designed Clinical Trials Study Design, GCP training and streamlining the process of managing complex Clinical Trials during Phases I, II, III and IV.
Source: Swedish-American Chamber of Commerce, San Francisco/Silicon Valley Press Release
Swiss voters back stem cell research under strict conditions
By Dr. Krishan Maggon, Pharma Biotech R&D Advisor, email: kmaggon@yahoo.com
ICC- 20 route de Pre Bois,
1215 Geneva 15,
Geneva, Switzerland
Volume 5, December 2004-January 2005. In November 2004, voters in Switzerland by 66% majority, have strongly backed stem cell research under strict conditions. The Swiss parliament had passed the original law in 2003 but its application was challenged by a coalition of right wing religious conservatives (Catholic Church and Catholic Doctors, Evangelic, Medical Ethics, Pro-Life) and left wing greens, ecologists and socialists opposed to genetic engineering, globalization and big pharma. In French-speaking Switzerland the percentage was even higher. Geneva recorded the highest level of support at 84.5 per cent. Voter turnout was low at 35% (2 million votes cast) due to the complexity of the subject and 20% of the citizens stated in a poll that the subject was difficult to understand. The Swiss Government, parliament, research institutes and universities and pharmaceutical and biotechnology industry had urged support for stem cell research. The debate in Switzerland closely matched the heated exchanges in USA, California, Germany, Austria, UK and Sweden.
Switzerland is a small landlocked country in the heart of Europe with a population of 7.2 million and has a system of direct democracy and frequent votation. The country is a leader in high technology, science and medical research and is home to major companies like Novartis, Roche, Serono, Nestle and international organizations like World Health Organization, International Red Cross, Center European Nuclear Research and International Olympic Committee.
Swiss voters have backed research in science and medicine and have rejected outright bans in previous national votes. In the year 2000, 72% voters rejected a proposal banning in vitro fertilization and although the first voting on the first "delay for abortion" was rejected by 52% of voters in 1977, the law passed in 2002 where strict conditions were imposed for on demand abortions. Swiss voters have rejected outright ban on animal experimentation, first in 1985 by 70%, second in 1992 by 56% and once again in 1993 by 72%. The shift in voting percentage is reflective of masked language of the proposal. In 1998, a proposal to ban genetic engineering and manipulation was refused by 66% of voters. The present law dealing with stem cells was initially voted in 1992 by 74% of voters. Next year, a new law dealing with clinical research, tissue banks for donors and a proposal to ban all genetic modifications of living organisms for five years will be voted in a national poll. About 170,000 Swiss patients participate in global clinical trials of new medicines each year.
The new legislation will permit research on stem cells from surplus human embryos under strict conditions. The production of stem cells will be limited to embryos not older than seven days. Therapeutic cloning and the trade in embryos will remain banned along with research on the embryos themselves. Informed consent of the couple using in vitro fertilization will be required for initiating any project; otherwise such embryos will be destroyed. Commercialization of the stem cells is not allowed and donors do not have any right to receive free products coming out of research. All projects using stem cells must provide scientific, ethical and medical rationale for research, advance our understanding of a serious medical condition/disease, exhaust all alternatives like using animal or adult cells, obtain the approval of the Institutional Ethical Review Committee, National Fund for Research and Federal Office for Public Health. The investigator will use the minimum number of stem cells and provide regular updates and progress report and any termination or final study reports to authorities and publish the results. For all exports or import of stem cells, the other country legal framework for stem cells should be similar to Switzerland. The Swiss stem-cell law falls between the liberal regulations in Britain, Sweden and Austrias restrictive legislation.
Three years ago Switzerlands National Science Foundation gave its approval for research on stem cells imported from abroad. The present vote will allow about ten research groups to initiate stem cell research for therapeutic use.
The interest in human stem cells is based on the fact that these cells offer the potential into development into any critical tissue or organ thereby providing novel therapy options. Stem cells offer a precious source of undifferentiated material for the study of cell maturation and specialization and are a potential source of material for the cure and replacement of damaged or declining organs and tissues. Stem cell lines with built in genetic diseases like diabetes, Parkinsons and Alzheimers and with selective RNA interference for switching off genes can help us understand how genes trigger diseases. Before any medical or therapeutic use, knowledge about their growth and differentiation into tissues and organs is required. Application areas in cell replacement therapy and bone and tissue engineering for incurable and degenerative diseases (Alzheimer), osteoporosis, for cardiovascular diseases, for diseases of the nervous system (e.g. Parkinson, multiple sclerosis) and cancer (e.g. leukemia) get a major share of research efforts. Only one research group in University of Geneva is active with imported human embryonic stem cells for study of aging process since 2001. Swiss research groups at the Universities of Geneva, Basel and Zurich have published papers and were successful in growing and transforming animal stem cells into myocardium and neurons. It will take about a year for any research group to grow a stable stem cell line and the project cost estimate is about $100,000. The current worldwide market for stem cells is estimated to reach $12 billion in 2005 and $52 billion in 2010.
* Please reference or credit any material, article or figure cited from http://www.medicineandbiotech.com as:
Copyright 2004. MedicineandBiotech.com, http://www.medicineandbiotech.com/
Disclaimer: MedicineandBiotech.com is managed by AstraGen LLC. Please re-distribute this e-magazine. AstraGen LLC and MedicineandBiotech.com do not assume, and hereby disclaim, any liability for any loss or damage caused by errors or omissions in any material published at the web-site http://www.medicineandbiotech.com, whether such errors or omissions resulted from negligence, misstatements or other oversights.
Stem Cell Research in UK
August 11, 2004. For the first time, British scientists have been given permission to perform therapeutic cloning using human embryos.
The Human Fertilisation and Embryology Authority granted the license to experts at the University of Newcastle. They are investigating new treatments for diabetes, Parkinson's and Alzheimer's disease. This decision could open a new era of research by scientists looking for remedies for diseases.
The research will involve experts from the Institute of Human Genetics at Newcastle University, and the Newcastle Fertility Center. This is possibly the first time such a license has been granted in Europe, as well as in the UK. Therapeutic cloning has been legal in Britain since 2001.
This license will be for at least five years - if not many more - before patients could receive stem cell treatments based on their work.
Stem cell treatments are carried out for medical reasons. Even though the science is similar, this technique is different to reproductive cloning, which aims to create a human being.
Cell nuclear replacement (CNR) or the cloning technique involves removing the nucleus of a human egg cell and replacing it with the nucleus from a human body cell, such as a skin cell. The egg is then artificially stimulated. This causes the egg to divide and behave in a similar way to a standard embryo fertilized by sperm. The eggs used in such studies are left over from IVF treatment. They are donated by couples, and would otherwise have been destroyed.
Scientists from the Newcastle NHS Fertility Center say that since they submitted their application, they have had overwhelming support from senior scientists and clinicians from all over the world and many letters from patients who may benefit from the research.
But Professor Murdoch at Newcastle NHS Fertility Center said: "Realistically, we have at least five years of further laboratory-based work to do before we move to clinical trials but this could be reduced if we receive additional funding which would allow us to increase the size of our team."
"This research should give valuable insight into the development of many diseases."
Suzi Leather, chair of the Human Fertilisation and Embryology Authority, said an initial one-year research license had been granted after "careful consideration of all the scientific, ethical, legal and medical aspects of the project".
She said: "This is an important area of research and a responsible use of technology.
"The HFEA is there to make sure any research involving human embryos is scrutinized and properly regulated."
A spokeswoman for the British Medical Association said: "We support strong regulation so that therapeutic cloning to extract embryonic stem cells for life-saving treatment, which most of the public supports, can go ahead while human reproductive cloning, which most of the public opposes, cannot."
But Julia Millington of the ProLife Party, said it planned to take advice over whether it could mount a legal challenge to the HFEA decision.
Cloning human embryos for therapeutic purposes was made legal by an amendment to the Human Embryology Act in January 2001 in UK.
But cloning humans for reproductive purposes remains illegal and is punishable by a 10-year prison sentence and unlimited fines in UK.
Cancer Stem Cells
e-published on MedicineandBiotech.com Jan 1st, 2008
-Cancer Stem Cells May Be at the Root of Brain Tumors-
Stem cells—popularly known as a source of biological rejuvenation—may also play harmful roles in the body, specifically in the growth and spread of cancer. Amongst the wildly dividing cells of a tumor, scientists have located cancer stem cells. Physician-scientists from WeillCornellMedicalCollege are studying these cells with hopes of combating malignant cancers in the brain.
"Some patients' brain tumors respond to chemotherapy and some don't," says Dr. John A. Boockvar, the Alvina and Willis Murphy Assistant Professor of Neurological Surgery and head of the Brain Tumor Research Group at NewYork-Presbyterian Hospital/Weill Cornell Medical Center. "We believe cancer stem cells may be the cause."
Dr. Boockvar is capturing and classifying these cancer stem cells in order to determine how they react to certain available drug therapies. Doing so will lead to more accurate and specific cancer diagnosis, allowing for tailored drug treatments. Results explaining the techniques used to harvest normal neural and brain-tumor-derived stem cells will be described in the January 2008 edition of the journal Neurosurgery.
"Cataloging brain tumor stem cells will be an enormous tool for patient diagnosis," explains Dr. Boockvar, "but it will also help to create a library of knowledge for the scientific community to understand how brain tumors form and to test and develop new drugs."
To stave off cancer stem cell growth in the brain, Dr. Boockvar is studying the use of two drugs already available for cancer treatment. Tarceva—approved for the treatment of lung and pancreatic cancer—works by stopping the growth and spread of cancer cells. Avastin—approved for the treatment of colorectal cancers—is also being studied for inhibiting cancer cell growth and works by stopping the growth of blood vessels (angiogenesis) that feed the tumor.
Preliminary results from these trials have shown that some patients' cancers are wiped out, whereas others remain resistant. Dr. Boockvar believes that these patients' drug resistance might be due to a class of stem cells resilient to available treatments. Finding biomarkers that distinguish these stem cells from those that are destroyed by known drugs might help researchers formulate new drugs.
"Determining a patient's cancer stem cell profile will take a lot of the guessing out of choosing a course of treatment," says Dr. Boockvar. "It will save money, medical resources and precious time for the patient."
NewYork-Presbyterian Hospital/Weill Cornell Medical Center NewYork-Presbyterian Hospital/Weill Cornell Medical Center, located in New York City, is one of the leading academic medical centers in the world, comprising the teaching hospital NewYork-Presbyterian and Weill Cornell Medical College, the medical school of Cornell University. NewYork-Presbyterian/Weill Cornell provides state-of-the-art inpatient, ambulatory and preventive care in all areas of medicine, and is committed to excellence in patient care, education, research and community service. Weill Cornell physician-scientists have been responsible for many medical advances—from the development of the Pap test for cervical cancer to the synthesis of penicillin, the first successful embryo-biopsy pregnancy and birth in the U.S., the first clinical trial for gene therapy for Parkinson's disease, the first indication of bone marrow's critical role in tumor growth, and, most recently, the world's first successful use of deep brain stimulation to treat a minimally-conscious brain-injured patient. NewYork-Presbyterian, which is ranked sixth on the U.S.News & World Report list of top hospitals, also comprises NewYork-Presbyterian Hospital/Columbia University Medical Center, Morgan Stanley Children's Hospital of NewYork-Presbyterian, NewYork-Presbyterian Hospital/Westchester Division and NewYork-Presbyterian Hospital/The Allen Pavilion. WeillCornellMedicalCollege is the first U.S. medical college to offer a medical degree oversees and maintains a strong global presence in Austria, Brazil, Haiti, Tanzania, Turkey and Qatar. For more information, visit www.nyp.org and www.med.cornell.edu.
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Stemedica Files with FDA to Become a Licensed Manufacturer of Adult Stem Cells for U.S. Based Clinical Trials
Neural Stem Cells for Brain Repair
Cancer Stem Cells
