Stem cell bill sails through House; Bush vows to veto

By Rick Weiss
THE WASHINGTON POST
The San Francisco Chronicle and New York Times News Service contributed to this report.

January 12, 2007

WASHINGTON – The House overwhelmingly approved a bill yesterday that would loosen the restrictions on human embryonic stem cell research imposed by President Bush in 2001, inaugurating the second such assault on the administration's stem cell policy in as many years.

Thirty-seven Republicans joined 216 Democrats to pass the Stem Cell Research Enhancement Act, which would allow federal funding for research on stem cells from embryos slated for destruction at fertility clinics.

The 253-to-174 vote fell 37 votes short of what it would take to override a presidential veto, which Bush promised yesterday would be forthcoming, assuming the Senate passes the same bill, as expected. Bush vetoed the same legislation last year.

But buoyant research proponents promised to persevere until the legislation becomes law.

“While it's not enough to override a veto, it's enough to show we have tremendous momentum,” said Rep. Diana DeGette, D-Colo., who co-sponsored the measure with Rep. Michael Castle, R-Del.

“We can do this the easy way or the hard way,” DeGette said. “By January 2009, we will have a president who favors embryonic stem cell research,” she added, noting that at the moment the leading presidential candidates in both parties support it.

She suggested that it is time for the president to begin negotiating with Congress over compromise language.

“The vote today shows that productive discussions might be a very, very good idea for all concerned,” DeGette said.

The legislation was backed by a large coalition of patient advocacy groups, universities and medical associations. But unless it becomes law, embryonic stem cell research in the United States will continue to be limited mainly to privately funded lab work and such state-funded programs as California's voter-approved $3 billion initiative.

Under the existing policy, federal funds may be used to study only those stem cells taken from embryos destroyed by Aug. 9, 2001 – or about 21 of the nearly 400 stem cell colonies created since 1998.

Many scientists say the 21 lines are contaminated with mouse cells and aren't useful for research into a roster of diseases that advocates say includes cancer, diabetes, Parkinson's and Lou Gehrig's diseases, spinal cord injuries and heart disease.

The House bill would expand that pool of available cells to include those from any of the thousands of embryos that are discarded by fertility clinics each year, as long as those cells were freely donated for research by the parents. It would also impose some of the country's first ethics rules on embryo research.

Opponents of the bill said they favor all forms of stem cell research except for the work on embryonic cells, which they say is immoral because it involves the destruction of human embryos.

Echoing arguments by the White House, House Republicans opposed to the measure noted that some studies suggest that stem cells taken either from adults or from amniotic fluid may provide alternatives to the use of those from human embryos.

But the bill's advocates produced statements from researchers saying that work on amniotic cells and adult stem cells in no way precludes the need for embryonic stem cell research.

Reps. Bob Filner, D-San Diego; Susan Davis, D-San Diego; Brian Bilbray, R-Carlsbad; and Darrell Issa, R-Vista, backed the stem cell measure. Rep. Duncan Hunter, R-Alpine, opposed.

The House vote came after about three hours of impassioned speeches by members on both sides of the issue.

“I've got 11 brothers and sisters,” noted Minority Leader John Boehner, R-Ohio. “The sanctity of life is the issue. Taxpayer funds should not be used to destroy human life, plain and simple.”

Rep. Roger Wicker, R-Miss., warned that passage would lead to a “slippery slope.” Before long, he said, scientists would routinely be creating human embryos “for the express purpose of killing that embryo” for research.

Rep. Roscoe Bartlett, R-Md., touting his credentials as a former research scientist who studied human embryology, spoke up in favor of alternative methods of getting stem cells – methods that virtually all leading stem cells scientists have discounted as having uncertain value.

On the other side, Rep. Edward Markey, D-Mass., warned that if Congress does not override a Bush veto this time, “this will be remembered as a Luddite Moment in American history, where fear triumphed over hope and ideology triumphed over science.”

Rep. Rahm Emanuel, D-Ill., wondered aloud – not seriously, he assured – whether those voting against the legislation would be willing to waive their right to access the cures that could come from the research.

With the bill's approval, the House hit the halfway mark in passing Speaker Nancy Pelosi's six-bill agenda, which she vows will be completed in the new House's first 100 legislative hours.

Senate leaders have said they plan to take up the stem cell measure next month.

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me@rescam.org

Holding the line on stem cell research

CHARLES KRAUTHAMMER
THE WASHINGTON POST

January 12, 2007

When President Bush announced in August 2001 his restrictive funding decision for federal embryonic stem cell research, he was widely attacked for an unwarranted intrusion of religion into scientific research.

His solicitousness for a 200-cell organism – the early embryo that Bush declared should not be destroyed to produce a harvest of stem cells – was roundly denounced as reactionary and anti-scientific. And cruel to boot. It was preventing the cure for thousands of people with hopeless and terrible diseases, from diabetes to spinal cord injury. As John Edwards put it most starkly and egregiously in 2004: If John Kerry becomes president, Christopher Reeve will walk again.

This kind of stem cell advocacy did not just shamefully inflate its promise. It tended to misrepresent the basis for putting restrictions on embryonic research, insisting that it was nothing more than political enforcement of the religious fundamentalist belief that life begins at conception.

This has always been a tendentious characterization of the argument for restricting stem cell research that relies on the destruction of embryos. I have long supported legal abortion. And I don't believe that life – meaning the attributes and protections of personhood – begins at conception. Yet many secularly inclined people like me have great trepidation about the inherent dangers of wanton and unrestricted manipulation – to the point of dismemberment – of human embryos.

You don't need religion to tremble at the thought of unrestricted embryo research. You simply have to have a healthy respect for the human capacity for doing evil in pursuit of the good. Once we have taken the position of many stem cell advocates that embryos are discardable tissue with no more intrinsic value than a hangnail or an appendix, then all barriers are down. What is to prevent us from producing not just tissues and organs, but human-like organisms for preservation as a source of future body parts on demand?

South Korea enthusiastically embraced unrestricted stem cell research. The subsequent greatly heralded breakthroughs – accompanied by lamentations that America was falling behind – were eventually exposed as a swamp of deception, fraud and coercion.

The slope is very slippery. Which is why, even though I disagreed with where the president drew the line – I would have permitted the use of fertility clinic embryos that are discarded and going to die anyway – I applauded his insistence that some line must be drawn, that human embryos are not nothing, and that societal values, not just the scientific imperative, should determine how they are treated.

Congress voted yesterday to erase Bush's line. But future generations may nonetheless thank Bush for standing athwart history, if only for a few years. It gave technology enough time to catch up and rescue us from the moral dilemmas of embryonic destruction. It has just been demonstrated that stem cells with enormous potential can be harvested from amniotic fluid.

This is a revolutionary finding. Amniotic fluid surrounds the baby in the womb during pregnancy. It is routinely drawn out by needle in amniocentesis. The procedure carries little risk and is done for legitimate medical purposes that have nothing to do with stem cells. If it nonetheless yields a harvest of stem cells, we have just stumbled upon an endless supply.

And not just endless, but uncontroversial. No embryos are destroyed. The cells are just floating there, as if waiting for science to discover them.

Even better, amniotic fluid might prove to yield an ideal stem cell – not as primitive as embryonic stem cells and therefore less likely to grow uncontrollably into tumors, but also not as developed as adult stem cells and therefore more “pluripotential” in the kinds of tissues it can produce.

If it is proved that these are the Goldilocks of stem cells, history will record the amniotic breakthrough as the turning point in the evolution of stem cell research from a narrow, difficult, delicate and morally dubious enterprise into an uncontroversial one with raw material produced unproblematically every day.

It will have turned out that Bush's unpopular policy held the line, however arbitrary and temporary, against the wanton trampling of the human embryo just long enough for a morally neutral alternative to emerge. And it did force the country to at least ponder the moral cost of turning one potential human being into replacement parts for another. Who will be holding the line next time, when another Faustus promises medical nirvana if he is permitted to transgress just one moral boundary?

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me@rescam.org

Stem debate

New cell findings may moot issue. Or not.

SAN DIEGO UNION-TRIBUNE EDITORIAL

January 12, 2007

One promising item on the Democrats' to-do list in their first 100 hours at the helm of Congress faces an almost certain veto: legislation to allow federal funding for embryonic stem cell research.

The House passed that legislation yesterday – but without enough votes to override the expected veto by President Bush. So, however the Senate votes, if it votes on this at all, federal dollars would still be limited to the few embryonic stem cell lines that existed in 2001, when Bush vetoed similar legislation. Congress couldn't override his veto then, either.

Fortunately, the impact on beneficial stem cell research if the veto stands may not be dire. This week researchers at Wake Forest University School of Medicine created body cells from stem cells found in amniotic fluid donated by pregnant women. Like adult stem cell research, amniotic research destroys no embryos. If amniotic stem cells turn out to have all the healing properties of embryonic cells – they may not – the emotional debate over destroying embryos for research could end.

But not yet. For opponents, stopping federal funding is a means to end embryonic stem cell research that destroys embryos. For most Americans, there is hope, not harm, in research that uses embryos destined to be discarded, anyway, because they're no longer needed for fertility treatments. Such research could reveal ways to control or reverse catastrophic or chronic diseases that disable millions. Those ways elude us now, and may forever if embryonic stem cell research must forever depend on private and state funding.

If opponents prevail, however, Bush and Congress can allot the funds nixed for embryonic stem cell research to amniotic stem cell research. The sooner we know the extent of its healing powers, the sooner we can help the ill, and drop or prolong the debate.

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me@rescam.org

More good news on amniotic stem cells...


THERE IS AN ALTERNATIVE
Stem cells could come from womb
Alok JhaLondon,
Hindustan Times
January 8, 2007


SCIENTISTS HAVE found a new source of stem cells that does not involve destroying embryos. The cells can be harvested easily from the fluid surrounding developing babies in the womb and could help overcome ethical concerns.

It has been known for decades that the placenta and the amniotic fluid in the womb contain important cells. “We asked the question: is there a possibility that within this cell population we can capture true stem cells? The answer is yes,” said Anthony Atala, director of the Institute for Regenerative Medicine at Wake Forest, North Carolina, who led the research.

Stem cells can grow into any type of body tissue and are used to research cures for conditions such as diabetes and brain disorders, including Alzheimer’s and Parkinson’s disease. It is hoped that one day they may be used to grow replacement tissue that is a perfect genetic match for patients with damaged organs.

Stem cells from embryos are highly prized because they are the most adaptable. They are hard to obtain, however, because they are normally harvested from embryos left over from fertility treatments. Anti-abortion campaigners argue this leads to destruction of human life. Adults also have stem cells, but these can turn into fewer types of body tissue.

Researchers said the newly discovered amniotic fluid-derived stem (AFS) cells represent an intermediate stage between embryonic and adult stem cells. They grew AFS cells into muscle, bone, fat, blood vessel, nerve and liver cells. "Our hope is that these cells will provide a valuable resource for tissue repair and for engineered organs as well," said Professor Atala. His results are published in Monday's issue of the journal Nature Biotechnology.

"These cells are capable of extensive self-renewal, a defining property of stem cells," he said. "The could be used to produce a broad range of cells for therapy."

Aside from ethical concerns, it is difficult to extract cells from leftover embryos, which are often of poor quality. AFS cells are readily available from samples taken for amniocentesis - which involves testing the amniotic fluid for signs of genetic siorders - and from the placenta. Around 1% of cells in these samples have been found to be stem cells. They grow quickly without turning into tujours, a problem for other types of stem cell.

So far we've been successful with every cell type we've attempted to produce from these stem cells," said Professor Atala. "The AFS cells can also produce mature cells that meet tests of function, which suggests their therapeutic value."

In one test researchers grew brain cells from AFS cells and implanted them into mice suffering from a degenerative brain disease. The cells successfully repopulated the damaged parts of the brain. Viable bone cells were made the same way.

[Also reported in The Guardian, UK]

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Hm, we had the option to extract and store the blood in the placenta (think it includes stem cells as well) when my son was born. I agreed to do so even though it might be mostly money making right now nobody knows how technology will develop further.

Stem cell case arguments set for Feb. 14
Critics say initiative is unconstitutional

By Terri Somers
Union Tribune

January 19, 2007

A state appeals court yesterday set Feb. 14 as the date for oral arguments in a case challenging the constitutionality of California's $3 billion stem cell initiative.

Several advocacy groups are appealing an April 2006 Superior Court decision that found the stem cell initiative to be constitutional. The court in Alameda also found that the California Institute for Regenerative Medicine, which was created to oversee the $300 million annually to be spent on research, was in full compliance of the law and accountable to the public.

Leaders of California's stem cell initiative have repeatedly pointed to the ties the legal challengers have to anti-abortion groups, which oppose embryonic stem cell research because it requires the destruction of human embryos.

But the plaintiffs, who refer to themselves as taxpayer advocates, have avoided raising the heated moral debate about embryonic stem cell research. Their primary argument is that the initiative is unconstitutional because state elected officials do not have control over the $3 billion in taxpayer funding that would be spent.

The plaintiffs include the People's Advocate, the California Family Bioethics council and the National Tax Limitation Foundation, which is represented by the Life Legal Defense Foundation.

The lawsuits were originally filed in April 2005, just four months after California voters approved the initiative by a 17 percent margin.

The state has been unable to sell the bonds to support the initiative pending the resolution of the lawsuits. To survive, the stem cell institute has received monetary gifts and loans from the state and numerous philanthropists, including Padres owner John Moores and Qualcomm founder Irwin Jacobs.

The appeals court has 90 days after the Feb. 14 hearing to issue a decision, absent any further briefings the judges may request.

The challengers have said that if they lose in appeals court, it is likely they will seek to bring their case to the state Supreme Court. The appeal will be heard in the First District Court of Appeals in San Francisco.

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me@rescam.org

NIH official sees flaws in stem cell policy

The Washington Post

January 20, 2007

WASHINGTON – The National Institutes of Health official overseeing the implementation of President Bush's embryonic stem cell policy suggested yesterday that the controversial program is delaying cures.

Under questioning by Sen. Edward Kennedy, D-Mass., Story Landis, director of NIH's National Institute of Neurological Disorders and Stroke and interim chair of the agency's stem cell task force, said: “We are missing out on possible breakthroughs.”

The ability to work on newly derived stem cell colonies – precluded from federal funding under the Bush plan – “would be incredibly important,” she added.

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me@rescam.org

Stem cell scientists shout out hallelujah
Rule changes expected to improve research

By Terri Somers
Union-Tribune

January 23, 2007

Scientists expect access to human embryonic stem cells for research to improve thanks to new policies announced yesterday by a University of Wisconsin agency that controls the patents on the cells.

The policy changes and “clarifications” include one that is specifically directed at California: The taxpayer-funded California Institute for Regenerative Medicine will not have to buy a license to distribute funding for embryonic stem cell research.

“Hallelujah,” said Larry Goldstein, a University of California San Diego stem cell researcher who lobbied for the state stem cell initiative, when he heard about the policy changes.

“I've been writing letters all over the place complaining about their policies hampering research,” Goldstein said.

For several years researchers around the nation have accused the Wisconsin Alumni Research Foundation, or WARF, of stifling human embryonic stem cell research with its licensing policies on the cells.

WARF controls all human embryonic stem cells used in research in the United States because the process to derive them was patented by University of Wisconsin scientist James Thomson.

“These latest changes reflect an ongoing dialogue with researchers and university administrators across the country,” WARF managing director Carl Gulbrandsen said yesterday.

The new policies allow companies to support research at universities and nonprofit institutes without having to buy a license to use the cells. However, once the company wants to bring that research back in-house and use it toward a commercial product, it would have to buy a license.

“Our belief is that this will increase the number of companies involved in research and increase the funding for universities and nonprofit research institutes, which will lead to more treatments reaching patients,” said Andy Cohn, a WARF spokesman.

As a result, it could also increase the number of companies that ultimately seek a WARF license.

Researchers are also now free to provide their colleagues with copies of embryonic stem cells that they received from WARF.

“I couldn't even share cells with colleagues before, so this is good,” said Jeanne Loring, an embryonic stem cell researcher at the Burnham Institute in La Jolla.

Loring and two taxpayer advocate groups have filed a challenge of the WARF patents with the U.S. Patent and Trademark Office, citing exorbitant licensing fees and arguing that Thomson's work was not unique enough to qualify for a patent.

She suspects that the patent challenge and publicity about it contributed to the new policies.

But despite the policy changes, the patent challenge will not be dropped, said Loring and John Simpson, of the Foundation for Taxpayer and Consumer rights, one of the groups challenging the patents.

“A change in licensing policy of the human ES cell patents doesn't solve the fundamental problem that the patents should not have been issued in the first place,” Simpson said. “The right thing for WARF to do is admit that it doesn't deserve the patents and abandon them in their entirety.”

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me@rescam.org

Guidelines on stem cell research offered
Group promotes worldwide practices

By Terri Somers
Union Tribune

February 2, 2007

An international organization of stem cell scientists released guidelines yesterday that aim to dictate rigorous ethical standards for research on human embryonic stem cells.

The guidelines were compiled by a task force of renowned scientists, as well as ethicists and lawyers from 14 countries, to promote responsible, transparent and uniform practices worldwide, according to the group, known as the International Society for Stem Cell Research.

“Realizing that stem cell research is an international community, we have to be able to share cells and our scientific methods across borders with some confidence that we have been doing our work to some agreed-upon ethical standards,” said Larry Goldstein, an embryonic stem cell researcher at the University of California San Diego.

Goldstein is a member of the task force that worked for more than a year on the guidelines.

The Foundation For Taxpayer and Consumer Rights in Santa Monica praised provisions in guidelines that emphasize the need for researchers “to promote public benefit as their primary objective” when turning over discoveries to commercial firms.

The guidelines also say “scientists and clinicians must be transparent and truthful about issues relating to human stem cell research and its potential to advance medicine.” And they urge scientists “to guard against the creation of unrealistic expectations of success.”

“Too often stem cell advocates have hyped the immediate benefit of stem cell research,” said John Simpson, of the foundation. “I'm delighted to see the call for realism.”

But the guidelines had their critics, especially from contingencies that oppose human embryonic stem cell research because it destroys embryos.

“This is worthless as an ethical guide because it is issued by scientists and entrepreneurs who have dedicated their careers to destructive human cloning and human embryo research and who will profit from the expansion of these abuses,” said Richard Doerflinger, an official with the U.S. Conference of Catholic Bishops.

In the United States, the National Academy of Science has issued guidelines for controversial human embryonic stem cell research. Those guidelines are very U.S. centric because they reflect restrictions on federal funding used for human embryonic stem cell research.

The international standards reflect the same spirit as those issued in the United States, Goldstein said. But the international rules do not get as specific, allowing room for different government policies, legal systems and cultures to dictate how the guidelines will be met, he said.

For instance, U.S. standards require some kind of independent committee composed of ethicists and scientists to review all proposals for research involving human embryonic stem cells.

The international standards call for independent review but do not dictate specifically how that should be done because many countries already have established their protocols, Goldstein said.

“The good thing about international agreements and standards is that they set the bar for people to perform reasonably and to prevent people from hiding behind an excuse like 'I live in a different country, with different standards,' ” he said.

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me@rescam.org

Appeals court hears from stem cell opponents
Voter-approved law would fund research

By Terri Somers
San Diego Union-Tribune

February 15, 2007

SAN FRANCISCO – An opponent of California's stem cell initiative told a state appeals court yesterday that voters were hoodwinked into approving the $3 billion plan based on the popularity of stem cell research and didn't realize they were voting for a law that allows their money to be spent on all kinds of research.

Another critic argued that the initiative, known as Proposition 71, also violates the state constitution's requirement that its governing body be accountable to the public.

The three-judge panel heard arguments yesterday in the appeal of an Alameda Superior Court judge's April 2006 ruling that the stem cell initiative was constitutional.

But based on the questions and comments from the panel during an hour of arguments, Proposition 71 opponents had a tough sell in trying to convince the appellate judges to throw out the initiative, which aims to direct $300 million annually to stem cell research.

David Llewellyn, a lawyer for the California Family Bioethics Council, argued that the overly broad nature of the proposition violates the constitution's single-subject rule, which requires laws to be about one thing.

But Justice Stuart Pollak asked, for example, whether the proposition weren't written broadly to eliminate the need for scientists to worry that they had crossed a legal line if their research took them from stem cells to another area of science.

And Justice Peter Siggins asked whether the many aspects of the initiative – building labs, training and educating the public – weren't focused on one mission: advancing the quest for new therapies.

Deputy Attorney General Tamar Pachter told the court that the intent of the initiative was clear.

“In November 2004, when voters approved Proposition 71, they knew what they were voting for: a public institute that would raise money through the issuance of bonds to fund research that is not funded by the federal government,” Pachter said. “There is no single-subject defect in the law.”

The legality of Proposition 71 has been a question for the courts for the past two years, preventing the state from issuing bonds to fund the taxpayer initiative.

Lawsuits challenging the initiative were filed in February 2005 by the Bioethics Council and the People's Advocate, a legal organization representing the National Tax Limitation Foundation. The groups include individuals who oppose abortion and embryonic stem cell research, which requires the destruction of human embryos.

In February 2006, the groups unsuccessfully argued their case in Alameda Superior Court. But they have said they will bring their challenge all the way to the state Supreme Court if necessary.

The People's Advocate argues that the constitution requires the initiative's $3 billion in taxpayer money to be spent by people under exclusive management of the state.

The stem cell institute's 29-member oversight board is an independent body and not wholly accountable to the public, People's Advocate lawyer Robert Taylor argued. Its policies also lack the teeth to remove members who are not performing their job, Taylor said.

For instance, Taylor said, University of California chancellors, who are not accountable to the public, appoint four board members.

“The chancellors are appointed by the trustees, are they not?” Justice Joanne Parrilli asked Taylor. “Are the trustees not accountable to the public?”

But Taylor persisted.

“In all situations, the (board) members are independent,” Taylor said. “They have no accountability to the people who appointed them or anyone else other than the institutes they serve, their employer. So they have at least a divided interest. As long as that is present, the state doesn't have control.”

Pachter argued that the stem cell institute is subject to an annual auditthat must be reviewed and accepted by the state controller. The board members also take an oath of office for public officials, file financial disclosure forms and are bound by a code of ethics.

“If they decide to take the money and build a baseball stadium, there are procedures in place in government to preclude them from doing so, are there not?” asked Justice Pollak.

Pachter conceded in her arguments that Proposition 71 was written to insulate its governing board from the changing whims of politics, so that a new governor who opposed stem cell research could not redirect the funding.

The court has 90 days to issue its ruling but could extend that deadline by asking lawyers to file further written arguments.

Robert Klein, chairman of the stem cell institute's governing board and an author of Proposition 71, said the opponents were ignoring a clear mandate from the state's voters.

“(The opponents) are seeking to delay our funding, to force our scientists to leave this great state to pursue their research elsewhere, and to dampen the hopes of tens of millions of patients everywhere,” Klein said in a statement issued after the hearing.

Outside the courtroom, Llewellyn said he could not predict the outcome based on the judges' questions. But he said he did expect them to try to uphold Proposition 71.

“If I was in their position, I'd want to save the statute, too. But when it goes out of its way to be disjunctive . . . it's not a strong argument.”

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me@rescam.org

Board to hand out stem cell research grants
Legal battle plays out as loans fund program

By Terri Somers
San Diego Union-Tribune

February 16, 2007

BURLINGAME – The board of California's Institute for Regenerative Medicine today is expected to award the first round of research grants funded by the state's groundbreaking $3 billion stem cell initiative.

People around the world will be watching.

California's initiative, voted into law in November 2004, is the nation's first state program that seeks to circumvent federal funding restrictions on human embryonic stem cell research. Its mission, and success with voters, prompted other states to follow suit with their own taxpayer-supported funding programs, fueled in part by a desire to stem a brain drain of talent to California.

“This is very exciting. It's what we've been working toward for the past two years,” institute president Zach Hall said yesterday, as the institute's governing board prepared to review the grant applications.

Lawsuits challenging the initiative kept the institute from making its first grants in May 2005 as originally planned. State lawyers were in state appeals court Wednesday, arguing against the latest legal challenges. It could be 90 days before the appellate court rules.

Meanwhile, $180 million in loans from philanthropists and the state are allowing the grants to get rolling while the legal battle plays out.

The board had planned a two-day meeting to review the 232 applications it received for its “Seed Grants,” which are designated to support innovative research by young investigators or work in new areas by more seasoned scientists.

The 27-member board of scientists, patient advocates and biotechnology executives isn't making its decision in a vacuum. The grant applications have been reviewed by a working group made up of patient advocates who serve on the board and by scientists from outside California. That working group scored the applications based on the quality of the proposed experiments, the researchers' background ,and access to the proper lab facilities and support.

But the board could only make “provisional” votes on the recommendations last night because only 16 members attended the meeting. Eighteen were required for a quorum. Several board members who had said they would be present for the historic vote could not make it at the last minute, said Dale Carlson, an institute spokesman.

“It's outrageous that they could not get a quorum for what is their most high-profile event next to their first board meeting,” said John Simpson, of the Foundation for Consumer and Taxpayer Rights, who has been following the board's actions closely.

Robert Klein, board chairman, defended those absent.

The initiative made the board large so that it would encompass a wide range of experience and expertise, including executives, medical professionals and patient advocates, Klein said. And professional obligations or personal emergencies can prevent people from attending meetings, Klein said.

The potential embarrassment of not making a quorum rises today, with Gov. Arnold Schwarzenegger planning to attend the meeting to speak to the board. Schwarzenegger has supported the initiative, and made a $150 million loan available to the institute so it could continue to operate during its legal struggles.

There is a second issue the board could vote on today: whether to raise the Seed Grant funding from $24 million to $45 million.

Originally the board estimated that it would fund 30 Seed Grants, for a total of about $24 million. When reviewing the grants, the scientific working group determined that 38 projects could be funded for $24.2 million.

Moreover, the working group was so impressed with the caliber of the proposed research, and with the investigators seeking funding, that it proposed funding a second tier of 34 grants for an additional $21 million, said Jeff Sheehy, a patient advocate and board member who serves on the working group.

If the board does not want to allocate $45 million, the working group suggested funding nine of the 34 projects in the second tier for $5 million, Sheehy said. These are not necessarily the highest-scoring proposals in the tier, but they would expand the portfolio of research funded by the CIRM.

For example, after some discussion, the board agreed it wanted to fund a second-tier proposal for work that would try to coax human embryonic stem cells into cells that comprise the hairs in the inner ear. Loss of these hairs contributes to hearing loss.

A brief synopsis of each grant and comments from the working group reviewers has been posted on the institute's Web site for public review.

All the grants recommended for funding involve work with human embryonic stem cells that would not be funded by the National Institutes of Health, because President Bush ordered that no federal money be spent on stem cell lines created after August 2001.

“That makes sense, and it's how the initiative can make a unique impact on the science,” said University of California San Diego stem cell researcher Larry Goldstein.

Around the state, scientists have been reviewing the list and the projects favored for funding. And many people have been able to identify some grant applicants, although the list does not contain the name of researchers or their institutes.

It's a small scientific community, and its members are familiar with who has a particular expertise, said Jeanne Loring, an embryonic stem cell researcher at the Burnham Institute in La Jolla. But that means many scientists are also uncomfortable with their scores being posted on the Internet for the world to see.

It appears that numerous grant applications filed by researchers in the San Diego region are highly recommended for funding, scientists said. Four of those grants, including one of the most highly rated, appear to be from researchers at the University of California San Diego. Three appear to be from the Burnham Institute.

The grant application that ranked highest with the scientific review committee is from UCSD. The scientist is requesting $612,000 over two years to fund experiments that would coax stem cells to become forebrain neurons, which may one day be used to replace neurons damaged by Alzheimer's and other disease.

The grant receiving the second-highest score asks for $638,000 over two years to perform research using stem cells pulled from human embryos that have been donated to a stem cell bank in La Jolla by a local in-vitro fertilization clinic.

One task this team of researchers – apparently Loring's lab at the Burnham Institute – would try to perform is to standardize embryonic stem cell cloning. This is the technique that a North Korean scientist claimed to have mastered, but his work was revealed to be fraudulent in late 2005.

The Burnham application specifically seeks to clone embryonic stem cell lines predisposed to a childhood disease, so that the cells could be used to track the disease progression and to test possible therapeutics in a Petri dish.

Another grant application recommended for funding would use human embryonic stem cells to track the earliest stages of human development, with special attention given to what leads to abnormalities in implantation of the embryo that could result in the death of the fetus or mother.

All of the work is basic embryonic stem cell research, which would lay the foundation for the still nascent field, said Mahendra Rao, a former embryonic stem cell researcher at the NIH who is now at Invitrogen in Carlsbad.

The projects recommended for funding encompass an array of disease groups, and the basic work of trying to standardize the reproduction of embryonic stem cell lines and characterizing the different changes caused by a change in their environment, Rao said.

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me@rescam.org

State awards stem cell grants

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me@rescam.org

Pediatricians, private banks tussle over cord blood

By Lauran Neergaard
ASSOCIATED PRESS

February 20, 2007

WASHINGTON – Fliers in upscale doctors' offices portray it as the hot new baby-shower gift: a registry where friends and family chip in almost $2,000 to start privately banking a newborn's umbilical cord blood, just in case of future illness.

That idea of biological insurance is a long shot that most mothers-to-be can safely ignore, say new guidelines from the nation's pediatricians that urge more parents to donate their babies' cord blood – so that it might save someone's life today.

The guidelines come as the government begins setting up the first national cord-blood banking system, aiming to prevent some 12,000 deaths a year – if public banks can compete with marketing-savvy private companies that now house the bulk of the world's preserved cord blood.

Cord blood is rich in stem cells, the building blocks that produce blood – and the same stem cells that make up the bone-marrow transplants that help many people survive certain cancers and other diseases. But cord blood has some advantages: These younger stem cells are more easily transplanted into unrelated people than bone marrow is, and they can be thawed at a moment's notice, much easier than searching out a bone-marrow donor.

There should be plenty for both private and public banking, says an optimistic Dr. Elizabeth Shpall of the public M.D. Anderson Cord Blood Bank. After all, cord blood from most of the nation's 4 million annual births is thrown away.

Chief hurdles: Improving consumer awareness – and the small number of hospitals that allow donations.

Shpall's own work illustrates the industry's stark socio-economic contrasts: At Houston's Ben Taub General Hospital, Shpall finds the mostly Hispanic mothers-to-be unable to afford private banking – few have even heard that cord blood has a medical use.

Today, about 50,000 cord blood donations are stored in more than 20 public banks around the country. The new National Cord Blood Inventory aims to triple that number, enough that virtually anyone who needs stem cell treatment could find a match – especially minority patients who today seldom can as most bone marrow donors are white.

Private banks have an estimated 400,000 units stored.

What's the controversy? Deciding who really needs to store a child's own cord blood for later use. Private storage costs $1,500 to $1,900 up front, and about $125 a year thereafter, although some offer special programs for lower-income families.

Guidelines published last month by the American Academy of Pediatrics say:

Parents should consider private storage only if an older sibling has cancer or certain genetic diseases that cord blood is proven to treat.

Everyone else should consider donating their child's cord blood. The odds that a child would need an infusion of his or her own cord blood later in life are slim, between one in 1,000 and one in 200,000.

Private banks vehemently disagree, arguing that as scientists learn more about stem cells, the blood could create personalized treatments for heart disease or other more common killers.

“That's still considered very experimental,” counters Dr. Mitchell Cairo of Columbia University Medical Center, who co-authored the new guidelines.

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me@rescam.org

Pediatricians, private banks tussle over cord blood

By Lauran Neergaard
ASSOCIATED PRESS

February 20, 2007
...
Everyone else should consider donating their child's cord blood. The odds that a child would need an infusion of his or her own cord blood later in life are slim, between one in 1,000 and one in 200,000.
...

That's quite a spread of odds there! Anyway, on a 1%% chance, if I had the money to spare I would go to a private bank to store my child's cord blood. At the 1:200,000 odds I would consider donating, but it really would depend on the institution.

State seeking return on stem cell research investment
Senate panel eyes conditional grants

By Terri Somers
San Diego Union-Tribune

February 23, 2007

The state Senate Health Committee wants to be sure California receives a share of profits that any company may make off stem cell therapies developed with taxpayer-funded grants.

Some committee members believe that policies being developed by the state stem cell institute do not go far enough to ensure a return to the taxpayers through royalties or access to drugs at the lowest possible price.

Today the committee is expected to introduce legislation that would modify or strengthen the stem cell institute's proposed policies.

“Californians are putting billions of dollars into this research. They ought to be guaranteed to get a little bit back, because everyone else is going to be on the take,” said Sen. Sheila Kuehl, D-Santa Monica, a sponsor of the bill.

During the campaign for the $3 billion taxpayer-funded stem cell research initiative Proposition 71, the public was told California could realize as much as $500 million to $1 billion in revenue from the patenting and licensing of discoveries that contribute to new therapies.

However, a report issued in 2005 by a group of academics and biotechnology industry executives said the most likely return to the state will come from jobs that are fueled by stem cell research and savings that therapies contribute to the health care system.

The group, commissioned by the stem cell institute and state Legislature, recommended the institute use federal procedures under which the federal government and other institutions generally get little or no monetary return on the grants they make.

Members of the Legislature and public watchdog groups were troubled by the report. For more than a year, they have sought to use proposed legislation and public discourse to keep the stem cell institute from softening to what they feel is drug industry pressure. Their goal is a guarantee that the state will collect direct revenue from the science it is funding.

The California Institute for Regenerative Medicine, which is implementing the stem cell initiative, has been working on two policies that would govern ownership and financial return on scientific discoveries made from its grants.

One policy deals with discoveries, called intellectual property, coming out of nonprofit research institutions. After a year, the administrative process for that policy still is not complete.

The second policy, which the institute started working on later, would govern how companies give a cut of their profits back to the state if they take institute grants.

Kuehl pointed out that both policies have softened after being criticized by executives from biotechnology companies, which hope to use the state-funded science to make therapies.

For instance, the institute originally planned to require companies using Proposition 71 grants to make the drugs available in California at the lowest available price, or the federal Medicaid price.

But the institute struggled with that definition. Medicaid law refers to drugs, and there were questions as to whether the institute could use that same definition to create regulations governing more complex therapies and diagnostic tools that are likely to be a result of stem cell research.

Now its policy requires companies to make the drugs accessible at the level of the California Prescription Drug Discount Program.

That's a significantly less favorable price, according to the Health Committee's analysis. And it does not specifically provide drugs to the underinsured or poor.

Kuehl's bill would require grant recipients to enter into exclusive licensing with a company developing therapies only if that company agrees to provide therapies, drugs and diagnostics to publicly funded health care programs in California at the “best available prices,” such as the Medicaid price.

The measure would also require grant recipients to give the state 25 percent of the licensing revenue they receive on inventions that they develop with Proposition 71 aid.

The bill also would require grant recipients to share 2 percent to 5 percent of the revenue they receive over the life of the product. The percentage would be negotiated and based on the level of state funds that were used to make the product.

However, it is unclear whether that would remain the percentage range even if the Proposition 71-funded discovery is just one of many discoveries that are combined to create therapy.

The exact working of Kuehl's bill was not available yesterday to the staff at the stem cell institute.

“I prefer not to comment on the specific provision until we actually see them,” said institute spokesman Dale Carlson.

“The issues are enormously complex, as the senator knows. And we are still in the process of finalizing our intellectual property regulations. We look forward to working with her staff toward the successful resolution of the open issues,” Carlson said.

One taxpayer advocacy group that has been a constant critic of the stem cell institute's policies on intellectual property said that Kuehl's bill does not go far enough to protect the uninsured and poor.

“In case of unreasonable pricing by drug companies the state needs to have march-in rights that allow it bring the prices down to a reasonable level,” said John Simpson, of the Foundation for Taxpayer & Consumer Rights in Santa Monica.

Kuehl's is not the first on the Health Committee to try to dictate stem cell institute policy.

Former Sen. Deborah Ortiz, D-Sacramento, introduced legislation regarding intellectual policy. Ortiz, who played a key role in the formation of Proposition 71, later became a critic of the institute and said it did not do enough to see that taxpayers were reimbursed for their investment.

However, the Ortiz legislation lacked support, and she was forced out of office by term limits.

Kuehl's bill would require 70 percent approval by the Senate and Assembly to become law. Proposition 71, which created the stem cell institute, dictates that the Legislature cannot pass any laws to change the initiative for three years.

If Kuehl's measure passes, it would not take effect until 2008, which is three years after the formation of the stem cell institute.

Kuehl said she saw no reason to hold any legislation and work with the stem cell institute staff as it continues forming its intellectual property policy.

“There's no reason for us to wait for them,” Kuehl said. “We represent the people who voted for Proposition 71.”

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me@rescam.org

Britain OKs plan to pay women to donate eggs

ASSOCIATED PRESS

February 23, 2007

LONDON – The government has approved a plan to allow women to donate eggs for stem cell and cloning research and to be compensated for it, an action that scientists hope will improve the supply of eggs.

Women getting costly fertility treatments will receive a discount if they donate eggs for research. Others will receive up to $500 for each fertilization cycle to cover costs such as travel or lost work time.

The United States and some other countries already allow human egg donations for research. The United States has no national policy on payment, but some states limit it to reimbursing women for costs.

Human eggs for research are in short supply, and some researchers believe payment would improve that situation.

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me@rescam.org

Human eggs for research are in short supply, and some researchers believe payment would improve that situation.

It sure worked for human blood, years ago when I was in the US Navy, I used to donate my blood regularly. They would give us $20.00 for a pint, we would then immediately go to the local bar and spend alot less than that on getting drunk. Less blood means you can get drunk quicker. Nice place San Diego, back then!
Used to see signs "Sailors and dogs keep off the grass!" Navy went an paid the Sailors in two dollar bills a few times. Merchants saw all those two dollar bills in their registers and the attitudes changed.

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Stem cell agency is upheld by court
Justices firmly reject challengers' claims

By Terri Somers
San Diego Union-Tribune

February 27, 2007

A state appeals court in San Francisco upheld the constitutionality yesterday of California's $3 billion stem cell initiative, striking down dozens of issues raised two years ago by advocacy groups.

The ruling – issued 12 days after justices heard arguments in the case – leaves the initiative's challengers only one more avenue to block the groundbreaking initiative: an appeal to the state Supreme Court.

“It is a fabulous day for stem cell research,” said Robert Klein, an author of the initiative and chairman of the board overseeing the stem cell institute it created to disburse $3 billion in stem cell research grants.

One of the challengers' lawyers said yesterday that an appeal is likely.

The groups had argued that the initiative is unlawful because elected officials do not have oversight of the taxpayer money to be used on the research. They also argued that voters were hoodwinked into approving the $3 billion plan based on the popularity of stem cell research and didn't realize they were voting for a law that allows their money to be spent on all kinds of research.

The three-judge panel for the 1st Appellate District ruled that there are ample ways elected officials have oversight of the taxpayer money, including annual audits and selection of members of the stem cell institute's board.

To say that issues surrounding Proposition 71 generally were well-aired before the election would be an understatement, the court said.

The initiative, and campaign literature supporting it, clearly explained it would fund “research for which the federal government is not providing adequate funding necessary for the urgent research and facilities needed to develop stem cell therapies to treat and cure disease and serious injury,” the court wrote.

The appeals court decision upholds an April 2006 decision by Alameda County Superior Court Judge Bonnie Sabraw.

The legal challenges, filed two years ago, have prevented the state from issuing bonds to fund the initiative, which voters approved in November 2004. Philanthropists and the state issued loans to the stem cell institute, allowing it to cover operating costs and issue its first round of research grants 10 days ago.

“Today's ruling is a victory. I always believed the courts would uphold the will of California voters,” said Gov. Arnold Schwarzenegger.

The Supreme Court could end the battle quickly by refusing to hear the case. It did just that two years ago, when the challengers tried to take their arguments directly to the state high court.

The challengers were not surprised by the ruling because the appeals court justices seemed skeptical during oral arguments, said Dana Cody, a lawyer with the Life Legal Defense Foundation, which is representing two challengers. Cody had not had a chance to read the decision before talking to a reporter last night.

The three-justice appellate panel noted the two-year delay in implementing the initiative in its ruling. It mentioned that it is the courts' job to try to uphold California's electoral process, which allows taxpayers to decide major issues through initiatives.

“The objective of the proposition is to find, 'as speedily as possible,' therapies for the treatment and cure of major disease and injuries, an aim the legitimacy of which no one disputes,” the appellate court wrote.

“The very pendency of this litigation, however, has interfered with implementation for more than two years,” the court wrote. “After careful consideration of all 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.”

Klein read the paragraph out loud and said: “It doesn't get better than that, does it?”

Local members of the California Institute for Regenerative Medicine's oversight board and scientists who helped lobby for passage of the initiative were equally jubilant last night.

“It's a big win today, and the court did not mince words as they found nothing the plaintiffs argued as having justification in law,” said board member Duane Roth, who runs Connect, an advocacy organization for start-up technology companies in the San Diego region. “They will likely appeal to the California Supreme Court, and I would predict without success.”

Proposition 71 was initiated by a collection of patient advocates, legislators and scientists who sought to circumvent federal funding restrictions on human embryonic stem cell research by getting state taxpayers to pay for it.

Many Americans, including President Bush, oppose human embryonic stem cell research because it requires the destruction of human embryos, just days after fertilization, when they are masses of 100 to 200 cells. Opponents of the research say that the early embryos are life that should be protected.

When Proposition 71 passed, it became the first state taxpayer initiative to fund stem cell research. Several others states since have started their own taxpayer funding programs.

“While certainly this is a great legal victory, it is actually very sad when you realize it took 2½ years for the legal system to get to the conclusion that voters came to in November 2004: that this is where medicine and science should be moving,” said Evan Snyder, who runs the human embryonic stem cell program at the Burnham Institute in La Jolla.

“Hopefully now we will be ale to make up for lost time.”

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me@rescam.org

Stem cell audit finds need for changes
Institute will comply on all but one request

By Terri Somers
San Diego Union-Tribune

February 28, 2007

A performance review of California's stem cell institute says the fledgling agency needs to improve several policies, better document how it reaches its policy decisions and further justify some salaries.

But the report released yesterday by the state Bureau of Audits said the institute already has addressed some issues auditors raised during their four-month review, such as how it solicits bids for services.

And yesterday, the agency said it would follow all of the bureau's recommendations except one: the suggestion that it require public conflict of interest disclosures by a committee of scientists that recommends which research grants should be funded.

“The auditors' careful examination of the policies and procedures of the institute has been very valuable in helping us to assess our performance, as have their suggestions for specific areas for improvement,” said Zach Hall, president of the California Institute for Regenerative Medicine.

The institute is “committed to earning the trust of the public as responsible stewards of the state's funds,” Hall said. “In this regard, the audit report has been helpful and has made us a stronger agency.”

The purpose of the audit was to look at whether the agency is implementing good policies and following transparent and legal procedures. A separate annual financial audit is required under Proposition 71, the 2004 voter initiative that created the stem cell institute to disburse $3 billion in taxpayer money for stem cell research.

The performance audit was originally suggested last year by former Sen. Deborah Ortiz, D-Sacramento, and supported by the Joint Legislative Audit Committee.

The review by four state auditors began when the institute was only 18 months into its existence, had a staff of 20 and was operating only on philanthropic donations and loans because legal challenges have delayed state funding.

In the 97-page report on their work, the auditors take into account that the institute and its governing body are a unique new organization still in the process of drafting and refining many of the key policies that were under review.

One of those still-under-development policies dictates what kind of financial return the state should receive from the sale of therapies developed using institute money.

Auditors recognized the numerous public meetings held by the institute to solicit input into the formation of this policy. But they criticized the institute for failing to provide them with documentation showing how they processed the input into policy.

“It's hard for me to determine whether this is the auditors being overly demanding or the institute continuing to do what it has done in the past – oppose all attempts to make it conduct its business in the open sunshine of the public,” said Jerry Flanagan, of the Foundation for Taxpayer and Consumer Rights, which has been keeping tabs on the institute.

The auditors also said institute policy needs to ensure that California's poor receive access to any new therapies that are a result of state-funded research. The institute needs to identify the benchmarks that will be used as a standard to establish the discount prices, the audit states.

The auditors are not alone in this concern.

Last week, Senators Sheila Kuehl, D-Santa Monica, and George Runner, R-Lancaster, introduced legislation that would require grant recipients to enter into exclusive licensing with a company developing therapies only if that company agrees to provide therapies, drugs and diagnostics to publicly funded health care programs in California at the “best available prices,” such as the Medicaid price.

The legislators worry that the institute has weakened its policies after receiving input and pressure from the biotechnology industry, which would be developing and selling these eventual therapies.

“I think the audit really supports the need for that legislation,” Kuehl said yesterday.

The audit also revealed that the state's Fair Political Practices Committee has concerns with the institute's conflict of interest policy for out-of-state scientists who are members of a working group that reviews grant applications and makes recommendations on which should get funding. The institute's board reviews this groups' recommendations, then votes on who should receive funding.

The board contends that the working group members serve only in an advisory capacity and are therefore not required to publicly disclose their potential conflicts of interest, as required of the institute's staff and governing body.

Instead, the working group members file disclosures to the institute, which keeps them under wraps and available for private review by state auditors.

The Fair Political Practices Committee believes that the working group members are decision makers and therefore should be required to publicly file financial disclosure statements.

The legal stance is a threatening one for the institute.

Groups challenging the institute's legality have also said this working group is a decision-making body, and therefore, the challengers argued, the institute is unconstitutional because the people deciding how to spend the state's money are not accountable to the taxpayers.

In April, a Superior Court judge in Alameda County accepted the institute's contention that these working group members are only advisers, when she threw out the legal challenges. But that was before the institute had issued its first two rounds of grants, which required it to discuss the working group's recommendations.

On Monday, an appeals court upheld the Superior Court judge's ruling.

Acknowledging the legal issue in this matter, the auditors recommended the institute get a formal opinion on its conflict of interest policy from the state attorney general.

It's an issue that has been editorialized in the media and a point of contention for taxpayer advocacy groups that repeatedely attend board meetings and criticize the policy.

“If they are going to be a strong and model agency, make them accountable to the Fair Political Practices Act,” said Jesse Reynolds of the Center for Genetics and Society.

The institute is adamant in sticking to its policy, said Dale Carlson, a spokesman.

The institute is asking scientists from outside the state to take time away from their own research to review grant applications,” Carlson said. “Although they're paid a small fee, they basically do it as a favor . . . to advance the science. They are not eligible to receive any of this grant money.”

Meanwhile, other agencies outside the state are offering to pay them more to review far fewer grants, he said.

“If you are given the opportunity to be paid 10 times as much as we are paying for a small portion of the workload we are going to lay on you, and you don't have to open your kimono, were do you think you're going to go?” Carlson asked.

“We have to strike the balance to be able to recruit the best possible people to be sure we are funding only the best possible science.”

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me@rescam.org

Burnham team is successful in stem cell study

By Terri Somers
San Diego Union-Tribune

March 12, 2007

LA JOLLA – Scientists at the Burnham Institute for Medical Research said they have used human embryonic stem cells to treat a genetically linked degenerative disease by significantly preserving function and prolonging life in mice.

Their findings offer insights into a biological pathway that affects a broad array of diseases, which combined afflict one in 5,000 people, said Evan Snyder and Jean-Pyo Lee, the Burnham scientists.

In a peer-reviewed article in the journal Nature Medicine, published online yesterday, the La Jolla-based scientists write that they implanted two types of stem cells – human neural stem cells taken from a developing brain and human embryonic stem cells coaxed to become neural stem cells – into mice with Sandhoff disease, a deadly degenerative childhood affliction.

Children with Sandhoff, a form of Tay-Sachs disease, are deficient in an enzyme that helps the body metabolize lipids, a fatty material. A buildup of lipids destroys brain cells that control and coordinate body movement and leads to the deterioration of the brain and spinal cord. Children with the disease often don't see their sixth birthday.

When stem cells were implanted in mice, the onset of symptoms was delayed, well-being and motor function were preserved, and life span was extended 70 percent, according to the article.

The study showed the stem cells migrated throughout the brain, with some replacing damaged nerve cells and transmitting nerve impulses.

This is the first evidence that stem-cell-derived nerve cells may integrate electrically and functionally into a diseased brain, Snyder said.

The transplanted cells also boosted the brain's supply of the enzyme Hex, the housecleaning substance that reduces lipid accumulations.

The treatment also dampened the inflammation that typically occurs in the brain as a result of most degenerative diseases. Inflammation is also thought to play a role in disease progression.

“Our study offers the first evidence that (human embryonic stem cells) employ multiple mechanisms – not just cell replacement – to benefit disease,” said Snyder, who runs Burnham's stem cell program.

When the stem cells were transplanted into mice who were also given a drug that permitted the enzymes in the stem cells to work more efficiently, the life span of the mice doubled.

This part of the study highlights how future therapeutic use of stem cells against degenerative disease will likely be multidimensional, using the cells as the glue to hold everything together, Snyder said.

“Our studies suggest that functional neuronal replacement can be complemented and, under some conditions, eclipsed by a range of other stem cell actions that nevertheless exert a number of critical stabilizing forces,” Snyder said.

Neither of the human embryonic stem cells created tumors, the article says. And the scientists also noted that they saw no negative reactions by the mouse immune system, which are often prompted by transplanting foreign cells into a body.

The article is the culmination of a long-standing collaboration between the Burnham scientists and Frances Platt and Mylvaganam Jeyakumar of Oxford University in England.

The scientists noted that the stem cells they used were coaxed to evolve and survive in a solution that does not contain animal cells and would therefore likely be acceptable to the Food and Drug Administration.All new drugs and therapies in the United States require FDA approval.

While Sandhoff is a rare disease, it is one of a broad type of diseases in which the body cannot metabolize lipids. Since its genetic link is known, scientists use it in researching an array of other diseases in which the genetic cause still has not been identified.

The Burnham scientists hope their work with Sandhoff disease, for which there is no cure, lays the groundwork for clinical trials.

“Dr. Snyder's team has extended the promise of stem cell therapies to children with special needs, including those with Sandhoff disease,” said Fia Richmond, the Santa Barbara founder of Children's Neurobiological Solutions, whose son has an undiagnosed neurological disorder.

“The CNS Foundation is proud to have contributed major funding for this project on behalf of the 14 million special-needs children in the United States alone.”

Other funding for the four-year study came from the National Institutes of Health, Oxford University, the Wellcome Trust and private philanthropies including National Tay-Sachs and Allied Diseases Foundation, the Late-Onset Tay-Sachs Foundation, the A-T Children's Project, the Barbara Anderson Foundation for Brain Repair, Project A.L.S., March of Dimes and Hunter's Hope.

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