Medical Device Daily Washington Writer

WASHINGTON – The Obama administration will "vigorously support scientists" pursuing human embryonic stem cell (hESC) research, the president said Monday just before signing an executive order lifting a federal funding restriction imposed by his predecessor in 2001.

Overturning the ban, Barack Obama said, will "bring the change that so many scientists and researchers, doctors and innovators, patients and loved ones have hoped for and fought for" over the past eight years.

While his order is an important step in advancing the cause of science in America, the president stressed that promoting science is not just about providing resources. "It is also about protecting free and open inquiry."

Obama said his order also is about letting scientists do their jobs "free from manipulation or coercion, and listening to what they tell us, even when it's inconvenient, especially when it's inconvenient."

"It is about ensuring that scientific data is never distorted or concealed to serve a political agenda, and that we make scientific decisions based on facts, not ideology," he declared.

Obama also signed a presidential memorandum directing his head of the White House Office of Science and Technology Policy, John Holdren, to develop a strategy for restoring scientific integrity to government decision making, which the president said would ensure the administration bases its public policies on the soundest science and appoints scientific advisers "based on their credentials and experience, not their politics or ideology."

The memorandum also is intended to ensure that "we are open and honest with the American people about the science behind our decisions," Obama pledged.

The president noted that while the full promise of stem cell research remains unknown, the potential of such research "will not reveal itself on its own" or "happen simply by accident."

When government fails to make investments in medical research, Obama said, "opportunities are missed, promising avenues go unexplored, some of our best scientists leave for other countries that will sponsor their work, and those countries may surge ahead of ours in the advances that transform our lives."

Obama said his administration plans to actively, responsibly, and with the urgency required "make up for lost ground. Not just by opening up this new frontier of research today, but by supporting promising research of all kinds, including groundbreaking work to convert ordinary human cells into ones that resemble embryonic stem cells."

He vowed that hESC research conducted with U.S. funding would never be undertaken lightly.

"We will support it only when it is both scientifically worthy and responsibly conducted," he said. "We will develop strict guidelines, which we will rigorously enforce, because we cannot ever tolerate misuse or abuse," Obama maintained, adding that the government would never open the door to the use of cloning for human reproduction.

"It is dangerous, profoundly wrong, and has no place in our society, or any society," he said.

"Today is an important day for science, and for biotechnology in particular, and for patients," said Jim Greenwood, CEO of the Biotechnology Industry Organization (Washington). "The president's action will now free up federal funding to be spent in academic settings and elsewhere on research that is extraordinarily promising in enabling us to create new therapies and medicines and to have that decision made by scientists as opposed to being made on an ideological basis by politicians."

However, he noted, the order does not fully "get us to the place we want to be, which is companies actually being able to produce medicines that will treat spinal cord injuries, cancer and Parkinson's."

Congress and the president, Greenwood said, "need to be equally supportive of the policies that are necessary to accomplish that."

Evan Snyder, program director of the Burnham Institute for Medical Research's Stem Cell Research Center, said Obama's executive order is evidence of the president's pledge that science would play an important role in his administration and policymaking and "it's not just lip service to science but actually data-driven."

The National Institutes of Health (NIH), Snyder added, can now go "full bore" with its research and awarding grants.

NIH officials Monday said they have 120 days to get guidelines crafted and in place. During a briefing with reporters, the officials also said that some of the $10 billion allocated under the stimulus package also may be used for hESC research.

The order will make hESC research less wasteful because research organizations no longer will be forced to have partitions between federally acceptable research and non-federally acceptable, Snyder told Medical Device Daily's sister publication, BioWorld Today.

For example, Snyder explained, researchers have to ensure that no materials in projects that were funded with California dollars are mixed with materials funded by federal money.

"If I had a grant from NIH to study Parkinson's disease, I had to make sure that the reagents and equipment that I was using on that project were not used for any of my California projects," Snyder said. "It was totally wasteful."

But, he said, under the Obama order, "funds will be used more economically."

Research centers engaged in hESC using federal dollars can now "design experiments and generate grants that are also dictated by science, logic and data rather than by politics," he declared.

While Obama's order reversing the Bush administration's ban on federal funding is an important step forward, Snyder said he approached it with somewhat "dampened enthusiasm," because Congress now must pass legislation that ensures hESC is protected.

"I'm very enthusiastic, but I'm a little concerned that it is not exactly yet what we need, and that Obama started this but Congress has to pick up the ball and bring it all the way home for a score," he said. "What we need to do is to generate new stem lines, particularly new stem cell lines from embryos that bear human diseases for which there are no animals because that's what we need for drug testing, drug discovery, identifying drug targets, diagnostics, prognostics. And that is new cell lines, whether from embryos or from nuclear transfer."

Even if scientists get to a time where they can start using reprogrammed cells, "we still need to even test those against the gold standard to know how representative they are," Snyder added.

Snyder noted that stem cell biology is not solely transplanting cells into the body.

"It's also trying to model diseases and development in a dish and understand those mechanisms and that gives rise to perhaps some drugs," he said. "That may even be lower hanging fruit than the transplantation stuff and that really does feed directly into the interests of many of the biotech industries much more quickly than transplant therapies might."

While Obama's order is clearly a positive correct first step, said Thomas Okarma, CEO of Geron (Menlo Park, California) which in January received the first FDA clearance to conduct clinical trials with hESCs in humans the impact "remains to be seen."

"The next steps are really where the rubber meets the road, specifically, how much money is going to be appropriated, and after that, how will it be allocated, how will the advances made by industry, like Geron, and academia that are funded with private sources be leveraged by this moving forward," Okarma told BioWorld Today.

If the order only opens just more money to the academic circles, "they are still going to be pretty far behind where we are," he said. But, he added, "I don't think that's the intention of this."

Okarma noted that his firm has been struggling with how it can be influential in teaching the academic centers "what we learned how to do."

"There is no one in line behind us for another IND approval for embryonic stem cells," he said. "We have done all of that heavy lifting without a dime" from California or the NIH, Okarma said.

He said he was awaiting the details on whether companies, like Geron, will receive federal dollars under the Obama administration's plan moving forward.

In addition, he said, it remains to be seen whether his firm will be able to work directly with the NIH to disseminate its technologies.

"So there's a lot of unanswered questions," Okarma said. "It's clear that the intent is there. The speech by the president was brilliant and forceful. We are all pretty happy to hear all of this. But the next steps are really as important if not more important than this first one."

Most importantly, he said. Congress needs to pass legislation to ensure hESC research does not "get ping-ponged around again" if the next president disagrees with Obama's policies.

Okarma said he plans to "keep an eye on the language that the legislators come up to be sure there aren't little things in there that could cause problems."

While some researchers have complained that the so-called "Bush-approved lines" were useless," Okarma noted that it was those lines his firm has been using, and it was the H1 that is the manufacturing source "for the world's first clinical trial in spinal cord injury."

"So the notion bandied about for years by the academics that the Bush lines were useless is not true," he said. "You have to know what you are doing with them and how to keep them clean and to produce them appropriately so they are appropriate for human use. The FDA and the state of California completely bought off on that."

Michael West, CEO of BioTime (Emeryville, California) and its subsidiary, Embryome Sciences, and a co-founder of Geron, said the president's order has helped to "clear the ethical clouds of controversy" that have "lingered much too long" over hESC research.

Academics and industry, he said, can now have a much clearer path forward.

No Comments