Live on the Internet: Today's FDA Hearing on Stem Cell Regulation, Preview of California's Comments

The FDA's two-day hearing into stem cell treatments is underway this morning and can be seen live on the Internet, including a presentation this afternoon by the California stem cell agency, which is seeking to ease FDA regulation of stem cell clinical trials.

A likely preview of the comments from the agency popped up this morning on California Healthline in a piece by Emily Bazar. She carried the brief text of an interview with Randy Mills, president of the $3 billion California agency.

He said,

"The problem with (the FDA's current) strategy is twofold. It doesn’t address the patients, or the need side of the equation. And I don’t think it has a chance of actually working because the FDA will acknowledge that they do not have the resources to enforce these types of regulations at the clinic level.

"They would have to be essentially regulating the practice of physicians, which is well beyond their capabilities. Even if they were able to enforce it, it would just drive these patients somewhere else.

"We’re advocating for the creation of some middle pathway that would bring essentially unregulated therapies into the regulatory fold, but in a manner which could be complied with.

"I would rather know these clinics are being regulated and collecting data than have them operating under the radar screen of the FDA. I would like there to be a formal pre-market review of these therapies before they’re put on the market. I would like there to be safety and efficacy data.

"I’m going to try hard to get the FDA to see that just plugging this hole won’t make the problem go away."

Bazar's piece also contained the text of comments from Jeanne Loring, head of the stem cell research program at the Scripps Institute in La Jolla. She said,

"There’s no scientific evidence that the fat cells (unregulated) clinics are using are going to do the patient any good. And there’s no evidence that shows they are safe.

"I’m a stem cell scientist. I need scientific evidence before I will believe anything. Regulation will help determine the efficacy and safety of those fat cells.

"There are several lawsuits or potential lawsuits brewing over these stem cell treatments. People were promised they would get improvements and didn’t. And there are cases where people were actually harmed by stem cells.

"Some people truly believe they have been helped by the stem cell injections. I’m not going to argue with them. It’s a very personal and emotional response. It’s not something that can be scientifically validated. People are really desperate, especially really sick people and their families.

"That means patients are at risk. That’s what bothers me a lot. There’s nothing we can do to talk somebody out of going to a clinic if they feel that’s the only option they have. We just want to make sure nobody gets hurt. We also don’t want people to go broke. These treatments are not covered by insurance and they cost tens of thousands of dollars."

Here is a link to the actual broadcast, which is being watched by about 300 persons at this moment. An overview of the audience seems to indicate that it is being lightly attended. Mills is scheduled to speak at 4:23 p.m. EDT.

Here is a link to the agenda including a list of speakers and schedule. The broadcast also has closed caption capability.

More Than a Minor Headache: The Stem Cell Snake Oil Problem vs. Legitimate Research

Just two weeks ago, the headlines from Bloomberg News offered a glowing view of the prospects for stem cell therapies and the industry. The story in the online publication, which counts nearly 9 million readers monthly, said,

"Stem cell crusader sparks new hope....Regenerative medicine could be a $120 billion industry by 2030"

But only three days later, that article was overwhelmed by a wave of stories with a much different flavor. They carried reports of unproven therapies, possibilities of fraud, lack of regulation and fearful, expensive consequences for desperate patients.

That theme continued even as recently as this past weekend as The Economist carried a piece headlined,

"A dish called hope: The flourishing, unregulated industry in expensive, experimental treatments"

So which is it? Hope for legitimate cures and oodles of cash for stem cell companies? Or "hope" for treatments that do not work and have sometimes damaged both the bodies and wallets of the patients?

The situation is more than a minor public relations headache for the supporters of stem cell research, be they patients, stem cell companies or the $3 billion California stem cell agency, which is on a campaign to ease federal regulation of stem cell clinical trials.

For all practical purposes, the public generally does not make much of distinction between the legitimate research conducted by institutions such as Stanford and the so-called "miracles" reported about the late hockey great Gordie Howe and others. It all goes into the same cognitive bag. The stories about mysterious and fearsome tumors found in little-known patients attract little widespread attention.

But UC Davis scientist Paul Knoepfler and bioethicist Leigh Turner of the University of Minnesota ripped open that bag with their study last month that reported for the first time that nearly 600 dubious stem cell clinics are peddling their unproven therapies across the country.  Their report supported the view that the stem cell field is rife with snake oil merchants. For the general public, which does not delve deeply into medical research, the perspective could well be described as, "You seen one stem cell therapy, you seen 'em all."

Does this mean that these clinics should be ignored and shoved quietly off into a corner in order to avoid besmirching legitimate efforts? Of course not. It certainly appears that the FDA and other  state regulatory bodies can do a better job. But stiffer regulations are not going to come anytime soon, despite an FDA hearing on the topic in September.

However, the situation DOES mean that folks like Knoepfler and Turner should continue to speak out along with many other scientists who do not want their efforts blackened by the snake oil men. Researchers can work with their institutions' PR departments to place op-ed pieces, find speaking engagements and gin up TV and radio interviews. Blogs, like the one produced by Knoepfler, can be started. The International Society for Stem Cell Research should revive its public education efforts to help patients and the general public understand the facts about stem cell research. It should also reinvigorate its warnings about dubious therapies, which were throttled back a few years ago, reportedly after legal threats were made.

California's stem cell agency has a special concern. It is trying to "de-risk" development of therapies  by a variety of means and lure biotech and Big Pharma into the stem cell game in a bigger way. This summer the agency is offering $75 million to entice a private partner into the state's first-ever, public-private partnership to create a stem cell cure.

The private sector has shied away from the stem cell business, as the stem cell agency's CEO, Randy Mills, has remarked on multiple occasions. One of the reasons involves the public climate and perception of the field. It is hard for companies to invest hundreds of millions of dollars or billions when the stem cell field is burdened with public perception and regulatory obstacles.

Selling the stem cell story in a realistic way is not necessarily an easy task. Nuances must be respected, but excitement is also needed. Balancing it all is a challenge for the men and women in the trenches. But without a good push, development of therapies will be slower, and, as Mills has noted, people will undoubtedly die who likely would have benefited from a more timely treatment.

A Scientist's Comment: Deal Directly with FDA Rather Criticizing It in the Media

The following comment on FDA regulation of stem cell research was submitted via email by a longtime scientist in the field, who asked to remain anonymous. 

"Many advertised  'stem cell' treatments use hematopoietic or blood stem cells which are run under a different set of regulations from pluripotent stem cells since they are umbrella-ed under regulations designed to cover blood transfusions and bone marrow transplants.  Pluripotent stem cells are very new, with many unknowns and the potential for triggering tumor growth.   In contrast, clinical use of blood stem cells has been ongoing for over 50 years with special regulations (or lack thereof) grandfathered in.   Unfortunately, the newly touted 'treatments' are not for homologous use of blood stem cells, are unlikely to be therapeutic and may even be dangerous. 

"The FDA has published new draft guidances addressing these issues that will be discussed in open forum this September.  I think it best to bring concerns about FDA regulations to these meetings and to initiate change by talking directly with the agency rather than criticizing them in the press or through Congress.  Like their oversight over the IRS and other federal agencies, Congress’s actions are counterproductive when they add broad, unfunded mandates while cutting funding at the same time.  But perhaps this is their way of shrinking the government and simultaneously dinging them for not doing their work.  A sure route to failure."

Do Stem Cells, Go to Jail

A Stanford law professor Monday told a group of stem cell scientists and businessmen and women in California that some of them would be subject to hard time in prison if they were sitting in South Dakota.

Henry Greely used the example to illustrate the "bizarre patchwork" of stem cell regulation in the United States, which varies widely from state to state. Greely said that some of the stem cell activities that some members of his audience are engaged in would be illegal in South Dakota.

Greely, who heads a California advisory panel on stem cell regulations, pointed out that regulations and patent law vary widely also from country to country, posing possibilities for confusion and "offshore production" of stem cell products.

Speaking to The Stem Cell Meeting in San Francisco sponsored by Burrill & Company, Greely indicated that a Stanford researcher collaborating with a British scientist could possibly be breaching Stanford research rules -- if the British scientist did not have the same set of research standards. Failure to abide by Stanford's rules could result in professional discipline at the university.

Hope for standardized regulations is dim in the short term, he indicated. Even if a new president in 2009 liberalizes federal stem cell research rules, variations will continue to exist from state to state unless Congress passes a strong new law regulating the science.

Greely said the best hope for something like universal acceptance of embryonic stem cell research would be a well-publicized cure. Then, he said, "political and moral objections will evaporate like the morning mist."

Meanwhile, Greely advised his audience to consult their attorneys, pay careful attention to details and pool information about stem cell research.