Controversial Stem Cell Therapies Get Congressional Backing
A bi-partisan medical research bill will speed up the FDA's approval process
On Wednesday, the House of Representatives overwhelmingly approved a massive piece of bi-partisan legislation called the 21st Century Cures Act. It’s a $6.3 billion bill that would fund wide-ranging medical research, such as the Obama administration’s “cancer moonshot” and BRAIN initiative, and speed the FDA’s approval of new drugs.
One of the most controversial measures would accelerate the regulatory review of stem cell therapies. Proponents of these treatments celebrate that the legislation could grant patients quicker access to potentially life-changing therapies. But critics fear that treatments that haven’t been sufficiently evaluated could put unsuspecting patients at risk and leave regulators with little recourse.
Stem cells can transform into any of the hundreds of types of cells. Though stem cells occur naturally in the body, scientists can today distill them from a person’s own tissues (often fat) to treat medical conditions. At hundreds of clinics across the U.S. and abroad, patients can pay thousands of dollars for clinicians to re-inject stem cells into the body that could, in theory, treat diseases such as Alzheimer’s, Parkinson’s, multiple sclerosis, muscular dystrophy, spinal injuries, and blindness.
But most treatments haven’t yet been proven effective by double-blind clinical trials, the gold standard of medical research, and many clinics are operating with minimal regulation from the Food and Drug Administration (FDA). Though the FDA has some requirements for how the stem cells must be stored and treated before they can be administered, the clinics have avoided regulation in part because the cells used in the therapies are from patients’ own bodies, Stat News wrote in early 2016.
To stem cell therapy proponents, the FDA should sanction fewer hurdles for patients to access these therapies. On clinic web sites and in some journalistic or scientific articles, it’s not hard to find reported success stories—quadriplegic patients who regained movement in their limbs, blind patients that can now see thanks to stem cells. But there are also (some confirmed, some alleged) horror stories that have concerned critics. A stroke victim developed an aggressive spinal cancer after his treatment, The New York Times wrote. Patients with poor vision who went blind altogether, according Nature News.
For now, stem cell therapies are only available through these loosely regulated clinics. But proponents, which include clinicians, some scientists, and some patients, would like to see them accessible to more people, in a greater number of medical facilities across the country, and possibly even covered by insurance. For that to happen, the treatments would need to be approved by the FDA.
The Cures Act would accelerate the pace at which the FDA could approve stem cell therapies. The bill, updated days before the House vote, contains language that would allow stem cell treatments, which are a part of a growing field called regenerative medicine, to be fast-tracked by the FDA, granting them provisional approval if they seem promising. That means they could become available to patients more quickly, with less scientific vetting than previously required.
Experts are still figuring out exactly what all this would mean in practice. Some of the language about stem cell therapies seems to be derived from an earlier, now-defunct bill called the REGROW Act. But in response to critics, the current language is actually much less extreme in speeding up review, says Paul Knoepfler, a biologist and stem cell expert at the University of California, Davis School of Medicine. Still, he says, some may oppose that the current version may still curtail much-needed FDA oversight.
Some of wording in the bill means that approved therapies could come to market without completing randomized, double-blind clinical trials. For example, the treatments could be approved on the basis of “real world” data collected from patients once the treatments are already available. “This is a particularly important point because we hear a lot these days, in the regenerative medicine field, about individuals in trials having very positive outcomes. But one or even a handful of patients’ outcomes with no controls don’t teach us anything meaningful,” Knoepfler says.
In other words, without strict scientific standards in place, it may not be possible to say that a treatment is as safe or effective as its backers claim, even if it seems to be working well. That would also limit the FDA’s ability to intervene if a treatment is not performing as well as anticipated.
Writing in the New England Journal of Medicine this week, FDA officials defended the agency’s slower approach, noting that studies “have not reliably demonstrated the effectiveness of stem-cell treatments even in some of the most systematically studied conditions, such as heart failure and graft-versus-host disease.”
The Cures Act could go to vote in the Senate as early as Monday or Tuesday of next week, according to a spokesperson from the office of Fred Upton, the Michigan representative who presented the bill in the House. If that happens, the Cures Act could be on the president’s desk before the end of the year.