FDA, at Last, Turns Its Attention to Stem Cell Therapies for PF and Other Chronic Ills

News Commentary

In the absence of conclusive scientific data about the outcomes of stem cell therapy in people with serious and chronic diseases — from multiple sclerosis and Alzheimer’s to pulmonary fibrosis — the hundreds of clinics offering these therapies across the U.S., and the thousands worldwide, rely mainly on anecdotal reports from patients undergoing stem cell treatments.

The approach isn’t necessarily invalid. Patient reports of improvements in their disease, similar to testimony given (and distributed via a recent press release) by a pulmonary fibrosis patient treated in March 2015 at the Lung Institute in Tampa, Florida, are undoubtedly true for the period they cover.

But that specified, and usually limited, period is part of the problem, experts say. As Scientific American reported in June 2016, in the article “Unproved Stem Cell Clinics Proliferate in the U.S.,” more than 550 clinics coast-to-coast provide a wide variety of stem cell therapies to pulmonary fibrosis, idiopathic PF, and other patients in the U.S. today, but the “booming market” is not yet matched by “systematic data about patients’ long-term outcomes — positive or negative.” (An exception, the journal noted, is widely accepted bone marrow stem cell treatments for certain cancers and blood disorders.)

For this reason, among others, the U.S. Food and Drug Administration (FDA) will be holding a public hearing Sept.12–13 at the National Institutes of Health Campus in Bethesda, Maryland, to solicit expert and general public opinion on four draft guideline documents the FDA is preparing to regulate the use of “human cells, tissues, and cellular and tissue-based products.” In other words, potentially regulating the variety of currently unregulated stem cell therapies.

Those who cannot take part in the actual meeting are welcome to follow it in real-time via a webcast on the FDA webpage regarding the hearing. The public is also invited to comment on “the particular topics covered, the particular questions posed, whether there are additional issues for which guidance would be helpful, and whether FDA’s recommendations … are sufficiently clear and consistent … to provide meaningful guidance to stakeholders.” Anyone planning to attend the FDA meeting needs to register by June 1 via this link; registration is not required to view the webcast and offer comments.

Interest in stem cell therapies is huge among people with chronic diseases for understandable, even obvious, reasons: unlike medication, stem cells hold the promise of attacking a disease at its core, and the hope of arresting — and possibly reversing — disease progression. It offers hope of treatment that goes beyond the continuous taking of medications, each with their own disease target areas and side effects. (Stem cell therapies can have side effects as well, but apparently not in all patients and, as of now, their long-term effects are largely unknown.)

For this reason, as columnist and multiple sclerosis patient Ian Franks reported in a recent column on the FDA hearings and stem cell treatments (“Welcome or Not, Focuses on Stem Cell Treatments“), some patient advocates will be urging fast approval of these therapies and a patient registry to monitor outcomes.

“Patients will never get these treatments if they have to go the traditional double-blind placebo-controlled trial route,” Sammy Jo Wilkinson, founder of Patients for Stem Cells, told Franks in the column. “That takes 10 years and $1 billion. There’s got to be a middle ground, where you don’t shut off treatment, you just keep track of it.”

According to Scientific American, the FDA hearing is actually a delay of a hearing originally planned for April. The agency was so overwhelmed with interest from the public, health professionals, businesses, and others that it canceled that meeting, and rescheduled it as the two-day event now set for mid-September.

If the “proposed guidelines are enacted,” Scientific American said, “the FDA would likely bring more of these clinics under its purview and regulate offerings in the same way as biological drugs.”

But that’s exactly what some patients and a number of healthcare practitioners do not want — a decade-long delay in a treatment that is potentially showing promise, and one intended for people who rarely have a decade or so in their disease course to spare.

Many others, however, urge caution, including the Pulmonary Fibrosis Foundation, which argued in a May 2015 statement that “patients should be skeptical” until the safety and effectiveness of stem cell therapy has been “rigorously documented.”

Clinical trials into stem cell therapies for pulmonary fibrosis and other diseases are underway, but in early and yet inconclusive stages. The result is a flood of anecdotal testimony supporting stem cell therapy, like that from the pulmonary fibrosis patient, identified only as  Joseph O., treated at the Lung Institute last year.

Joseph’s story, indeed, is attractive. As he said in the release: “My oxygen level is [now] steady at 98-99, where it was in the 80s before. I haven’t seen oxygen in over six months. Haven’t touched it. I was on oxygen 24 hours a day, and now, zero.”

What’s more, the treatment may have brought about disease remission. Again, according to the release, CT scans of Joseph’s lungs taken three weeks before a follow-up visit to the Lung Institute showed no signs of inflammation (how much time had passed between this visit and the 2015 therapy was not specified in the August release, but it couldn’t have been much more than a year). “My pulmonologist is blown away,” Joseph said, adding that he himself felt “like a million dollars.”

As the Institute said in its release: “While some researchers are pushing for more research over clinical practice, many patients are sharing stories about how stem cells have essentially saved their lives.”

Both sides will undoubtedly be part of the Sept. 12-13 FDA hearing, and take part in offering insights as registered speakers. How the FDA will weigh such anecdotal evidence against still inconclusive scientific research results, and how it will look at the various types of stem cell therapies being offered for differing diseases, is not yet known — but, at least, an approach is finally coming into view.