Lung Institute Report: The FDA Hearing on Stem Cell Therapy Could Have Widespread Impact

Four Lung Institute staff members attended the FDA hearing on stem cell regulation on Sept. 12-13, and reported key takeaways from the event that could potentially have a major impact on the healthcare industry

TAMPA, Fla., Sept. 22, 2016 – On Sept. 12-13, Lung Institute staff attended the FDA public hearing: Request for Comments – Draft Guidances Relating to Regulation of Human Cells, Tissues or Cellular or Tissue-Based Products (HCT/Ps). The hearing was held on the campus of the National Institutes of Health, just outside of Washington, D.C., where the public was invited to comment on tissue and stem cell therapy regulation.

There are currently over 500 stem cell clinics on the United States, which has raised the question: Is there a need for increased regulation? Some might be surprised to learn the regulations discussed at the hearing can affect more than just stem cell clinics. If passed, the proposed regulation could impact procedures that are already approved and commonly performed, many of which are even covered by insurance. For example, the proposed regulation could affect procedures, such as breast reconstructive surgery, wound healing care, blood banking, orthopedics, cosmetic surgery and more.

“I think it is important that there be some control over the introduction and utilization of these new investigative technologies that have the potential to radically alter how we treat illnesses now and into the future,” said Jack Coleman, Jr., M.D., senior medical director of the Lung Institute. “Our organization applauds the FDA for not only recognizing this need, but also realizing that this is a different situation from areas of prior regulation. We are grateful to the FDA for the opportunity for public comment about oversight that could have potential for widespread impact on future patient care in the United States.”

The general consensus of the two-day hearing was that we need to change how we regulate HCT/Ps. The manner in which that is accomplished, however, was not agreed upon by all.

Minimal Manipulation and Main Function

A key topic was the degree to which tissue manipulation is acceptable. Currently, the proposed regulation would attribute one “main function” to body tissues, and body tissues could not be altered to serve any function other than the main function. For example, the regulation could affect breast reconstructive surgery after a mastectomy. The main function assigned to adipose, or fat tissue, is to provide structural support and cushioning, and the main function of breasts is lactation. Therefore, if a woman undergoes a mastectomy procedure due to breast cancer, breast reconstructive surgery that involves using tissue from other areas of the body having a different main function would be considered more than a minimal manipulation procedure. Consequently, it would be subject to new regulation because the adipose tissue and the breast serve two different main functions.

Pamela Vetter, Regulatory Policy Director at AlloSource, a nonprofit organization founded to responsibly develop and distribute life-saving human tissue donations, spoke about limiting adipose to one main function.

“Adipose contains both structural and non-structural components,” she said. “By focusing solely on the main function, the draft guidance lacks in a categorization of structural tissue, and by doing so, inappropriately states that isolated cells from structural tissues are not to be treated like cells, but rather as structural tissue. Such a narrow descriptor of an HCT/P in relation to FDA’s distinction between structural and non-structural tissue not only ignores scientific understanding of HCT/Ps, the individual tissues they’re comprised of and their various functions, but it also has the potential to impede access to clinical treatments.”

Presenters at the event proposed to return to the model of attributing “basic functions” to tissues. Tissues can have more than one basic function, and are often comprised of more than one tissue, they argued. The basic function approach recognizes that many tissues have more than one functionality. For example, adipose tissue not only provides structural support, it also has the function of paracrine signaling, which is a form of cell-to-cell communication in which cells send out signals that induce changes in nearby cells, altering the behavior or differentiation of those cells. In other words, adipose tissue can also have an anti-inflammatory and anti-apoptotic effect, meaning it can reduce tissue inflammation and keep cells from dying. Many presenters made the point that, by limiting the main function description of adipose tissue to structural support, policymakers are ignoring other major functions of the organ; namely, their ability to store energy and communicate with other cells for a healing effect.

If tissues are attributed various basic functions, presenters argued, the basic functions could serve as a basis of measurement to determine if a procedure is considered a minimal manipulation procedure.

The general consensus was that there are currently two categories under which tissue utilization can fall: a drug or a medical procedure. If tissue is considered a drug, the typical path is 10-20 years of research and clinical trials, which could cost billions of dollars, before put into practice. The other option, under current regulation, is that tissue utilization is a medical procedure, and therefore, exempt from FDA regulation. Rather, it is subject to respective state Department of Health and medical board oversight.

Leigh Turner, Associate Professer at the University of Minnesota Center for Bioethics, spoke out against investigative clinical application of stem cells and called for strict regulation. “The out-of-control marketplace for stem cell interventions needs effective regulatory oversight. I, therefore, hope the draft guidances are more than stage props, and this hearing is more than public theater. When patient safety and public health are at stake, the FDA must do more than function as a paper tiger. It is time for action.”

An Overwhelming Number of Positive Patient Testimonials

Several patients provided testimonies at the event about how investigative stem cell therapy has had a positive impact on their lives. It is worth noting that there was not one negative patient comment. Many patients argued that they didn’t have 10-20 years to wait for a new treatment to be approved, or that they may not survive if their current treatments were suddenly taken away. Patients for Stem Cells was in attendance, and argued against FDA control over cells from their own body.

One patient, 30-year-old Julie Cerrone of Pittsburgh, Penn., told the panelists how a stem cell procedure changed her life after suffering from avascular necrosis, a condition where bone dies due to lack of blood supply. “I’d walk into these top, top, top leading doctors with such hope, and I’d leave just completely defeated with my family, in tears because no one would give me a solution,” she stated. “I found my own solution though, and that was a Regenexx stem cell procedure. And in March 2015, I went and had the procedure done on my left femur bone. Three months out, I was off all of the pain meds I had been living on three to four times a day for three years at that point. Six months out, 40 percent of my bone had regenerated, and I was able to get off the crutches that I was on for three and a half years. And a year out, 60 percent of my bone regenerated. Today, I stand in here, unassisted, a year and a half out.” Today Cerrone is a yoga instructor and wellness coach.

Choosing the Right Path to Acceptance

Many presenters argued that the two current pathways of identifying stem cells as either a drug or medical procedure don’t work for stem cell therapy. It makes sense, they said, to add another approval pathway that doesn’t impede progress of these types of therapies, but does add quality measures to ensure patient safety.

According to a post on Patientsforstemcells.org, Dr. Andrew von Eschenbach, former FDA director, has stated, “Breakthrough technologies deserve a breakthrough in the way the FDA evaluates them. Take regenerative medicine… after proof of concept and safety testing, a product could be approved for marketing with every eligible patient entered in a registry, so the company and the FDA can establish efficacy through post-market studies.”

With the development of a registry, organizations would be responsible for providing and monitoring their patient outcomes data. A registry would provide patients with a public portal through which they may research clinics before scheduling treatment and would also provide oversight of clinical organizations by holding them accountable for their outcomes data. While the FDA might not have the manpower to oversee such regulation, other boards might be able to take on that responsibility. Those who believe that stem cell therapy falls under the practice of medicine call for regulation by a medical board, the likes of which already exist for hospitals and outpatient surgery centers. Medical board oversight would ensure consistency among things such as treatment, sterilization protocols and training.

“The FDA had a genuine desire to have honest opinions of their proposals discussed openly,” said Dr. Coleman. “The FDA, Medicaid community and patients all realized that there is a very fine line between regulating progress so that it can proceed in a safe manner, and over regulating so as to impede progress to the point of stifling it. A good example of this was brought up more than once of bone marrow harvesting for cancer treatment. Initially, the morbidity and mortality rates were so high that, by today’s regulations, it would have been abandoned. But, today it is standard of care for many cancers, so it is very important that we all work together to try to get this right the first time for the sake of our patients.”