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FDA approves clinical trial of human embryonic stem cell therapy

Green light for US stem cell work

US regulators have cleared the way for the world’s first study on human embryonic stem cell therapy.

The US Food and Drug Administration have been considering the 21,000 page application for months.

The decision by the FDA to give the go-ahead comes at a symbolic moment, just days after the inauguration of President Barack Obama. Since 2001 there have been limits on federal funding for embryonic stem cell research.The decision of the FDA is independent of White House control, but the new president is widely expected to adopt a more pragmatic and science-oriented approach to stem cell research.

Under President Bush, federal funding had been limited to around 60 stem cell lines created from embryos destroyed prior to August 2001.

Scientists had warned that only 20 eligible cell lines remained useful for research and many of these were problematic. Researchers had told the BBC that the restrictions had slowed down their work.

Controversy

Geron Corp, the company behind the research, plans to initiate a clinical trial in a handful of patients paralysed due to spinal cord injury. Interest in use of embryonic stem cells is due to their ability to turn into any of the body’s 200 cell types.

Using embryos donated through IVF treatment scientists have coaxed the stem cells inside into many types of tissue. One embryo can provide a limitless supply because the cell lines can be grown indefinitely.

But the use of human embryonic stem cells in research is controversial with some campaigners saying it is unethical.

Geron, a biotech company based in “silicon valley” south of San Francisco, has spent $170m on developing a stem cell treatment for spinal cord injury. The research will use cells coaxed to become nerve cells which are injected into the spinal cord.

In animal trials of the treatment, paralysed rats regained some movement.

Company chief Dr Tom Okarma said: “What stem cells promise for a heart attack or spinal cord injury or diabetes is that you go to the hospital, you receive these cells and you go home with a repaired organ, that has been repaired by new heart cells or new new nerve cells or new islet cells that have been made from embryonic stem cells.”

The cells being used in the trial are derived from the H1 human embryonic stem cell line, which was created before 9 August 2001 and thus eligible for federal research funding. However, Geron received no federal funding for the studies leading up to the clinical trial or to support the trial itself.

‘Pivotal decision’

Professor Chris Mason, an expert in regenerative medicine at University College London, described the FSA decision as “historic” and a “pivotal milestone in the development of embryonic stem cell therapies.

He said: “The knowledge that will be gained in this first clinical trial deploying embryonic stem cell derived material will accelerate the development of all future stem cell therapies.”

Professor Pete Coffey, director of the London Project to cure blindness, said: “It’s great news for the field.

“This strengthens our recent call for regulators in the UK to help provide a clear process for researchers to take this forward.

“It’s also exciting for me because it brings our own moves towards clinical trials with embryonic stem cells for age-related macular degeneration a step forward.”

Professor Robin Lovell-Badge, of the MRC National Institute for Medical Research, said it often took 15 to 20 years to develop a therapy.

“It takes a long time and much work to derive processes that will efficiently and reproducibly give an appropriate cell type for grafting and many animal experiments to test efficacy and safety.

“An appropriate set of patients have to be identified for the first tests and clinicians willing to participate in trials.

“And the regulatory hurdle is, understandably, a huge one - in this case it required 21,000 pages of documents.”

He added that for those patients desperate for treatment, and for their families, the news showed the research is moving in the right direction. But Josephine Quintavalle, director of Comment on Reproductive Ethics (Core), which opposes embryonic stem cell therapies, dismissed the research as “highly speculative”.

“The work is at a highly experimental stage and there’s still a question mark over the capacity of these cells to form tumours,” she said.

“What worries me is that patients will really believe this is going to cure their spinal injury.”

She pointed out that other research teams in Australia and Portugal were developing spinal therapies using adult stem cells.

“We’ve never changed our point of view, which is that embryonic stem cell treatments cannot ever be justified,” she said.

BBC NEWS
http://news.bbc.co.uk/go/pr/fr/-/2/hi/health/7847450.stm

January 26th 2009 Posted to Stem Cells, US, Biomedical Ethics, Spring 2009

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White House stem cell shift expected (BBC)

By Fergus Walsh - BBC News medical correspondent

A major shift in White House policy on stem cell research is expected when Barack Obama takes office.

The president-elect has been a strong supporter of embryonic stem cell research. He is widely expected to lift restrictions imposed by President Bush in August 2001 who was opposed to the destruction of embryos for scientific research.

This limited federal funding to stem cell lines created before that date.It would simply take an executive order - not legislation - for President Obama to reverse the Bush restrictions.

Although the Obama transition team has not made specific promises on stem cells, the change is widely anticipated.

Restrictions

The current restrictions limit federal funding to around sixty stem cell lines created from embryos destroyed prior to August 2001.But scientists say only twenty eligible cell lines remain useful for research and many of these are problematic.

The Bush restrictions led to some bizarre complications for scientists according to Professor David Schaffer, of the University of California, Berkeley. “To work with non-federal stem cell lines means we have to separate labs, separate lab books, separate personnel, payroll even building facilities and infrastructure to be able to be able to conduct work on the better non-registry lines that have derived since that time.”

Professor Schaffer, the co-director of the Berkeley Stem Cell Center, says the restrictions have slowed down their work. His lab is working on basic research into Parkinson’s and Alzheimer’s, trying to create the cells which are lost in these diseases.

He cannot put a time-frame on when that might translate into treatments. A policy change in the White House would receive broad support in California. It has become the epicentre of stem cell research in the US. In 2004 the state passed Proposition 71 which promised $3bn to be spent over 10 years. To date the California Institute of Regenerative Medicine (CIRM) has given out more than $600m in state grants to universities.

CIRM has helped funded a separate building at Berkeley for research on newer stem cells lines, which is nearing completion, but in the future such separation of science may not be necessary.

The change in policy at the White House would also be welcomed by industry.

Industry

Geron, a biotech company based in “silicon valley” south of San Francisco, is set to take embryonic stem cells into the clinic. It has spent $170m on developing a stem cell treatment for spinal cord injury. Company chief Dr Tom Okarma would like to see a presidential commission on stem cell policy.

“The impact of eight years under the previous administration for stem cell research has really been devastating. “We want the new administration to do more than the merely political - of reversing the prior administration’s ban.”

Geron has submitted a 22,000 page submission to the Food and Drug Administration - the body which approves clinical trials in the US - the largest application on record. Geron wants to inject embryonic stem cells direct into the spine of newly paralysed patients. In trials on rats, the animals regained some function.

For the moment the stem cells remain cryo-preserved in liquid nitrogen but they are ready to be shipped to the clinic if and when approval is given.

“This goes beyond what a pill or a scalpel can do”, says Dr Okarma. “Twenty or thirty years from now, hopefully I’ll be full of embryonic stem cell products that will keep me healthy into my 90s and beyond.”

Geron’s next application will be for a stem cell treatment for heart failure. It’s also working on treatments for diabetes, liver disease, bone and cartilage repair.

That may seem like science fiction to many, and private companies don’t usually talk down the promise of their products.But it is far from being an isolated view in the industry. Dr Michael West is another leading figure in the field of stem cell research.

He used to be in charge at Geron and helped fund the research which led to first human embryonic stem cell line being created in 1998 and now heads another company, BioTime.
“For the first time we can see a way to rebuilding the human body and to adding new heart muscles and saving many millions of lives. “These cells not only do that for the heart, for the kidney, but for everything in the human body.”

Lifespan

He believes embryonic stem cells could profoundly extend human lifespan as scientists work out how to reset the normal ageing process of DNA.

All this of course is just theory at the moment. Dr West’s experiments are in mice not men. Successful trials in a dish or a rat are one thing; success in humans is quite another.

What we know is that embryonic stem cells are special. Of the bundle of cells found in a three day old embryo, each has the ability to turn into any of the body’s two hundred cell types.
Using embryos donated through IVF treatment scientists have coaxed the stem cells inside into many types of tissue. One embryo can provide a limitless supply because the cell lines can be grown indefinitely.

Geron says it has enough undifferentiated stem cells to potentially treat every spinal cord injured patient in the United States for the next two decades.

But embryonic stem cell research is controversial. President Bush said the destruction of embryos in scientific experiments as immoral. Many evangelical Christians are worried about where stem cell policy may be heading.

Dr Robert Evans is the founder of a new church, so new it holds its services in a school hall in Pleasanton California .

As pastor at Christ Church and a bio-ethicist, he is disturbed by the new freedoms scientists may be given. “What it signals is that the human embryo has been denied moral standing and a corresponding right to life.

“As an evangelical who believes all life is created equal in the image of God, and that we have moral standing from the moment of conception, I would view that as a very chilling decision.”
Dr Evans points out that there are adult sources of stem cells such as bone marrow which avoid the moral issues.

“All therapeutics to date have been done with adult sources, so we can still help those in need without having to violate the standing of the human embryo.”
A seismic shift in White House policy on stem cells seems likely, but the state of the economy could still leave scientists frustrated that President Obama won’t back words with cash.

January 16th 2009 Posted to Stem Cells, US, Spring 2009

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She may never get breast cancer - but girl’s birth raises new doubts over designer babies

Ben Quinn, The Guardian, Saturday 10 January 2009

The birth of the first British baby genetically screened before conception to be free of a breast cancer gene was hailed yesterday as a breakthrough by doctors but raised fresh questions about the ethics of creating so-called designer babies.

The baby girl grew from an embryo screened to ensure that it did not contain the faulty BRCA1 gene, which would have meant she had a 50%-85% of developing breast cancer.

While mother and daughter were said by a spokesman at University College hospital, London, to be doing “very well” following the birth at this week, medical experts and those involved in cancer research were considering the implications.

Paul Serhal, medical director of the assisted conception unit at the hospital, said: “This little girl will not face the spectre of developing this genetic form of breast cancer or ovarian cancer in her adult life.

“The parents will have been spared the risk of inflicting this disease on their daughter. The lasting legacy is the eradication of the transmission of this form of cancer that has blighted these families for generations.”

In June the mother, then 27, told how she decided to undergo the screening process after seeing all her husband’s female relatives suffer the disease. The woman, who wanted to remain anonymous, said at the time: “We felt that, if there was a possibility of eliminating this for our children, then that was a route we had to go down.”

The technique, pre-implantation genetic diagnosis (PGD), has already been used in the UK to free babies of inherited disorders such as cystic fibrosis and Huntington’s disease. But breast cancer is different because it does not inevitably affect a child from birth and may or may not develop later in life. There is also a chance it can be cured, if caught early enough.

Permission to carry out PGD for breast cancer had to be obtained from the Human Fertilisation and Embryology Authority by the London clinic which performed the procedure.

Dr Sarah Cant, policy manager at Breakthrough Breast Cancer, said the decision to screen embryos to see whether they have a faulty breast cancer gene was a complex and very personal issue. “Women with a family history of breast cancer tell us that what might be right for one person may not be right for another. It’s important for anyone affected to have appropriate information and support so they can make the right choice for them.”

Kath McLachlan, a clinical nurse specialist at the charity Breast Cancer Care, said it would give those carrying the faulty BRCA1 gene “another option” to consider when starting a family.

She said: “However, there are many complex issues to take into account and the decision will finally come down to an individual’s personal ethics. While the selection of an embryo through PGD can reduce a person’s risk of developing breast cancer, the procedure cannot prevent a non-genetic form of the disease in later life. It is essential that anyone considering using the technique is offered comprehensive information, high-quality support and advice.”

Doctors at the private clinic at University College hospital conducted tests on 11 embryos by removing just one cell from each when they were three days old. Six embryos were found to carry the defective BRCA1 gene. Two embryos which were free of the gene were implanted, resulting in a single pregnancy.

Faulty genes are responsible for between 5% and 10% of the 44,000 cases of breast cancer that occur in the UK each year. BRCA1 and its sister gene BRCA2 are the two most commonly involved. Women with a defective BRCA1 or BRCA2 gene are up to seven times more likely to develop breast cancer than those without the mutations.

As the debate about the ethics involved in the procedure was renewed, the main objection from critics remains the charge that it opens the door to the creation of babies for parents who may want their off spring to be top of the class, excel in sport, and have hair, eyes and other physical characteristics that into a particular family’s wish list.

Alternatively, deaf or blind couples might want their disabilities passed on to their child. Some members of the deaf community who claim they belong to a “linguistic minority” are campaigning for the right to have hearing-impaired children.

January 11th 2009 Posted to Ethics and Science, News, Europe, Biomedical Ethics, Spring 2009

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NIST Calls Personalized Medicine a ‘Critical National Need,’ Seeks Advice for New Funding Programs

January 2, 2009
By a GenomeWeb staff reporter

NEW YORK (GenomeWeb News) — The National Institute of Standards and Technology wants genomics, proteomics, and other biomedical researchers to submit ideas about needed advances in personalized medicine, and has asked for white papers detailing these pitches.

The NIST call is part of a new program asking for input on a number of subjects it has deemed “areas of critical national need,” including personalized medicine, and the advice will be used to develop new competitions for funding under its Technology Innovation Program.

In the area of personalized medicine, NIST said in a listing in the Federal Register, researchers could describe needs for advances in genomics and proteomics that could be used to help doctors develop personalized drug treatments and dosages.

NIST is not seeking proposals; it is asking for descriptions of the need and associated societal challenge, why government support is needed, the consequences of inaction, and potential technical solutions.

In its call, NIST said personalized medicine, based on genetic, environmental, and metabolic influences on disease, could be a key to addressing the “trial and error” nature of treatment in the current health care system.

White papers covering personalized medicine could include descriptions of the challenges of cost-effective tools and techniques for genomics and proteomics research, technologies used in identifying biomarkers, drug and vaccine delivery systems, and better methods of integrating and analyzing biological data when it is combined with environmental and patient history information.

Alongside personalized medicine, NIST also sees several other research areas as critical, including civil infrastructure, energy, water supply, manufacturing, nanomaterials and nanotechnology, and sustainable chemistry. It has asked for white papers in those areas as well.

More information about the NIST call for white papers addressing areas of critical national need may be found here.

January 05th 2009 Posted to DNA Test & Personalized Medicine, News, US, Spring 2009

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FDA approves clinical trial of human embryonic stem cell therapy

White House stem cell shift expected (BBC)

She may never get breast cancer - but girl’s birth raises new doubts over designer babies

NIST Calls Personalized Medicine a ‘Critical National Need,’ Seeks Advice for New Funding Programs

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