The Dual Images of Stem Cell Medicine Highlighted in the Headlines

Google news stem cell headlines this week

A couple of notable headlines surfaced this week dealing with stem cell matters, capturing something of the dichotomy in a field that has been heralded for its possible production of miracle cures. 

One story told of a person who plays football for a living, Doug Baldwin, who is known as the $46 million man.  He decided to spend some time in England this summer receiving what he believes was a stem cell treatment to prevent his knees from deteriorating. It was the type of story that gives many others hope that some sort of stem cell therapy could cure a serious or fatal condition despite the fact that no such cure has been approved for widespread use in this country. 

The other story told of a modest crackdown by the Food and Drug Administration (FDA) on a couple of unregulated stem cell clinics in this country, a move that many believe was long overdue. More than 500 such clinics exist in the United States.  One of the California clinics identified by the FDA was using a restricted small pox vaccine as part of its treatment. 

Your average health care consumer could justifiably wonder, "What in the world is going on? Is this stuff safe or what?" Some scientists have been long frustrated by the dueling public faces of stem cell medicine. On one hand, stories of miracle cures involving professional athletes generate great attention and tend to create faith in the efficacy of stem cell treatments, although the therapies involved do not measure up to scientific standards. On the other hand, the FDA is now taking a more active role in policing dubious actors in this business.

Our average medical consumer might believe that the FDA action signals a new assurance of stem cell safety or is it the other way around? Is the stem cell glass half full or half empty?

Paul Knoepfler, a stem cell researcher at UC Davis, has been the bellwether for seven years regarding news about unregulated and dubious stem cell clinics. He once even went sort of undercover to attend a public marketing session produced by one clinic in the Sacramento area. 

Knoepfler wrote this week on his blog about the FDA action, its implications and the questions that remain. He said the FDA action was a "very big deal." 

But Knoepfler said six important questions remain:

• "How much further will the FDA go with actual enforcement actions?
• "Will the FDA define fat stem cells as a drug (or not) in upcoming guidances?
• "How will the FDA handle non-homologous use of bone marrow cells in the commercial sector?
• "Will the FDA work to deal with the growing problem of unapproved, amniotic stem cell offerings? 
• "What about networks of stem cell businesses?
• "Will other entities like state medical boards use this FDA action as a spring-board to get off the sidelines and take positive action too?"

Meantime, patients will continue to have to wade through conflicting claims, hype and the possibility of serious medical harm or death as they ponder whether they should undergo a stem cell treatment. 

UC Irvine Stem Cell Blog: Troubling New Federal Law on Stem Cell Research

The stem cell blog at UC Irvine this week published a piece that warned that a new federal law aimed at speeding commercialization of stem cell therapies is worrisome and could lead to harm to patients and damage the entire field of research.

The April 10 article was written by Navied Akhtar, a Ph.D. candidate in the biomedical engineering department.

Akhtar's topic was the 21st Century Cures Act. He wrote that the law, signed by President Obama and which had a broad range of supporters,

"...utilitizes evidence from clinical experience to 'help to support the approval of a new indication for a drug approved under [accelerated approval]' and 'to help to support or satisfy post approval study requirements.' The only requirements written in for accelerated approval past a 'reasonable likelihood' that there will be clinical benefit, is that one or both of two requirements are met: (1) That the manufacturer conducts studies after accelerated approval to verify the predicted effect on mortality or other clinical benefit and (2) That the manufacturer submits copies of marketing materials for the drug during the preapproval period." 

Akhtar said that the law 

"will allow for what is effectively anecdotal evidence to be used as actual evidence to support the requirement for accelerated approval. Furthermore, the verbiage surrounding the term 'surrogate endpoint' is loose at best. The idea of a surrogate endpoint is to produce a clinically relevant point in which to be able to measure the efficacy and safety of a drug. In the Cures Act, the definition of what constitutes a surrogate endpoint is left rather open-ended. It reads as such: "The term ‘surrogate endpoint’ means a marker, such as a laboratory measurement, radiographic image, physical sign, or other measure, that is not itself a direct measurement of clinical benefit, and is known to predict clinical benefit and could be used to support traditional approval of a drug or biological product; or is reasonably likely to predict clinical benefit and could be used to support the accelerated approval of a drug or biological product.' It is worrisome to write that this endpoint can be a marker that is not a direct measurement of clinical benefit, but is known to predict clinical benefit. This leaves open a large workaround for accelerated approval of drugs that may have no business being approved."

Akhtar goes on to cite cases of drugs that ultimately proved harmful even after clearing the usual approval process. 

In the case of novel stem cell therapies, he argued, serious mishaps or death because of loose federal standards could endanger progress in the entire field.