In a presentation at a U.S. Food and Drug Administration (FDA) public hearing earlier this month, the chief scientific officer of StemGenex Medical Group, Steven A. Brody, MD, PhD, said succinctly: “Stem cells have arrived and have captivated the scientific and medical communities. With this excitement comes responsibility and with this responsibility comes regulation.”
The purpose of the FDA hearings, held at National Institutes of Health (NIH) facilities in Bethesda, Maryland, on Sept. 12–13, was to obtain comments on four Draft Guidances of Human Cells, Tissues and Cellular and Tissue-Based Products relating to the regulation of adult stem cell therapies from a broad range of stakeholders, including healthcare professionals, clinicians, biomedical researchers, patients, tissue establishments, biological and device product manufacturers, and the public.
The FDA asked for feedback on the scope of its draft guidelines, ranging from the particular topics covered and questions posed, to the identification of additional issues not covered but for which guidance would be helpful, and whether the FDA’s recommendations for each topic were sufficiently clear and consistent to provide meaningful guidance.
The nearly 500 event attendees heard presentations from approximately 90 speakers, many whom advocated relatively open access to adult stem cell therapies. But others maintained that adult stem cells should be regulated as a drug, to allow for the rigorous scientific screening that new drugs are required to go through, a process that evaluates both their safety, efficacy and proper dosing so as to limit potential harm and unknown outcomes to patients receiving them
Paul Knoefpler, a stem cell researcher at the University of California, Davis, is a strong advocate for strict FDA regulation. On his blog, The Niche, Knoefpler writes: “As a scientist who studies and worries a great deal about the tumorigenicity of stem cells, I personally think the FDA is doing its job of being extremely careful about the safety of stem cell-based drugs and the need for compelling pre-clinical safety data.”
In another blog entry, Knoefpler — who began his blog in 2009 while recovering from prostate cancer surgery — also argues that stem cell therapies are indeed the equivalent of drug therapies. “Stem cells are drugs and possibly permanent ones,” he writes in a June post. “Yeah, stem cells can be extremely unusual drugs, but they are often drugs even if some argue they aren’t. … Unlike other drugs, once a patient receives a stem cell drug, it will not necessarily simply go away like other drugs because a stem cell drug consists of living cells that often behave in unpredictable ways. What this means is if the stem cells are doing bad things your doctor has no way to stop it. You have no way to stop it.”
Knoefpler adds: “I am as excited as anybody about the potential of stem cells to treat a whole bunch of diseases and injuries, but they are not some kind of miracle cure for everything. When a doctor offers to inject some kind of stem cells into a patient … we just do not know at this point if it will do any good with the exception of bone marrow transplant.”
A drug approval process, however, could delay patient access to stem cell therapies for years, StemGenex argues, noting that the regulatory pathway to market authorization for a new drug, under favorable circumstances, can take some 10 to 15 years and a cost of tens of millions of dollars.
StemGenex, based in La Jolla, California, specializes in regenerative medicine and adult adipose stem cell therapy, emerging fields of medicine which, the company says, can help people with unmet clinical needs. Physicians at its treatment center offer stem cell therapy procedures to applicable patients requesting them. StemGenex is also currently sponsoring five clinical trials in patients with multiple sclerosis, Parkinson’s disease, rheumatoid arthritis, chronic obstructive pulmonary disease (COPD), and osteoarthritis, evaluating quality of life changes in people with these diseases who have received adipose tissue-derived stem cell treatment.
In his presentation, Brody, who specializes in regenerative and restorative medicine, and who has expertise in endocrinology and metabolism, gynecology, and reproductive medicine, asked the FDA to consider three changes, in particular, as it proceeded with finalizing the new guidances:
- Recognize the structural and non-structural properties of adipose tissue
- Review and expand the definition of minimal manipulation
- Regulate human cells, tissues, and cellular and tissue-based products in the same manner as blood tissue products, and within the existing comprehensive standards of organizations such as the American Association of Blood Banks (AABB)
“Organizations such as the AABB are ideally suited to safely guide and regulate the use of adipose-derived stem cells,” Brody said in a press release. “The AABB established regulatory framework would require stem cell treatment centers to meet standards that would ensure public safety.”
StemGenex’s position on stem cell therapy regulation is that amending the draft guidances as related to Brody’s recommendations would constitute a positive step in providing medical professionals with the ability to offer their patients a properly regulated but promising treatment option, particularly in instances where there is little hope.
Ed Tobias, an MS patient and columnist for Multiple Sclerosis News Today, also takes a look at this month’s FDA stem cell hearing in his column,”Packed House at FDA Stem Cell Hearing,” published today.
As Tobias and the FDA note, public comments into stem cell therapy and its regulation are welcome from the interested public — which certainly includes patients, family members and caregivers — through the close of Tuesday, Sept. 27, on an FDA website for comments (click on “Comment Now” blue button in the upper right corner).
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