FDA Invites Comment on Pending Stem Cell Therapy Regulations

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The U.S. Food and Drug Administration (FDA) will hold a one-day public hearing on four recent draft guidelines regarding the regulation of human cells, tissues, or cellular or tissue-based products (HCT/Ps). Critics of the proposed regulations warn that they curtail the medical use of autologous cell therapy and biologics, and their future potential as treatments for a range of diseases.

The meeting, Public Hearing; Request for Comments – Draft Guidances Relating to the Regulation of Human Cells, Tissues or Cellular or Tissue-Based Products takes place on April 13, 2016, at the FDA’s White Oak Campus in Silver Spring, Maryland, from 8:00 a.m. to 5:00 p.m. It will provide stakeholders an opportunity to discuss the FDA’s proposed regulation of HCT/Ps regarding four areas: homologous use, same surgical procedure exception, minimal manipulation, and adipose tissue.

The FDA states that the draft guidances were issued in response to stakeholders’ requests for guidance on the regulatory agency’s current views as to how manufacturers, establishments, distributors of HCT/Ps, and healthcare professionals can meet criteria in agency regulations applying to HCT/Ps.

The FDA says it will consider information it obtains from the hearing in the finalization of these guidelines.

The meeting’s purpose and scope is to enable the FDA to obtain comment and feedback, both general and specific, from a broad range of stakeholders, including HCT/P manufacturers, tissue establishments, biological and device product manufacturers, healthcare professionals, clinicians, biomedical researchers, and the public. The FDA seeks comment on the scope of each guidance, including particular topics covered, specific questions posed, and whether the recommendations for each topic are sufficiently clear and consistent within and across documents to provide meaningful guidance. It is also interested in additional issues on which stakeholders would like guidance.

The FDA recommends that presentations exclude discussion of products outside the definition of an HCT/P, such as platelet rich plasma, and asks that presenters coordinate comments when possible to allow for the widest range of perspectives within the meeting’s allotted time.

Stem cell therapy advocates say the guidelines described in an FDA draft document titled Human Cells, Tissues, and Cellular and Tissue-Based Products from Adipose Tissue: Regulatory Considerations; Draft Guidance for Industry are restrictive, proposing that adipose procedures be regulated as a drug through the FDA directly. Such regulation, they argue, might prevent countless patients who stand to benefit from cell therapies, such as those with multiple sclerosis, from receiving them. The FDA draft was published in the Federal Register on Dec. 24, 2014.

The draft document provides sponsors, clinicians, and other establishments that manufacture and use adipose tissue with recommendations for complying with the regulatory framework for human cells, tissues, and cellular and tissue-based products (HCT/Ps). Within that framework, the FDA considers connective tissue, including adipose tissue, to be a structural tissue.

“To label someone’s own cells as a drug is absolutely ridiculous. Using one’s own stem cells has been shown to be extremely safe in clinical settings through a multitude of studies. To classify one’s cells as a drug would be a complete disservice to the public population increasing the cost of healthcare in America significantly,” says Joe Perricone, director of Patient Advocacy at StemGenex, on the regulations.gov website.

AACTlogoThe Alliance for the Advancement of Cellular Therapies (AACT), a global organization of scientists, researchers, physicians, administrators, device manufacturers, and patients, dedicated to ethical, efficacious, and expeditious advancement of cell therapies, has launched a robust effort to have an overwhelming “grassroots” presence at the FDA public hearing to advocate for retention of cell therapies in the practice of medicine.

The AACT’s stated mission is to establish best practice guidelines and clinical translation of biologics within informed regulatory oversight, and to act as an advocate for the safe deployment of cell therapies to treat a range of diseases. The group does so by supporting the training of cell therapy providers, increasing public awareness, and facilitating patient access to cell therapies.

AACT identifies six areas of concern regarding the FDA draft proposals, and suggests their adoption could result in:

1) Reversal of current surgical practices that have been in use for years.

2) Significant delays in the introduction of new life-changing (life-saving and/or quality of life improving) therapies.

3) Continued pressure for U.S. citizens to seek stem cell therapies in offshore healthcare facilities.

4) Limitations on the physician’s ability to incorporate potentially life-changing therapies into a patient’s treatment plan.

5) Loss of a leadership role for American scientists and physicians in the generation of new clinical treatments for diseases for which there now are no reasonable treatment options.

6) Inequality of access to life-changing therapies based on a patient’s financial resources.

There are four known types of stem cells: Adult Stem Cells, or those taken from the adult human body; Embryonic Stem Cells, which are harvested from human embryos; Fetal Stem Cells, those derived from aborted human fetuses; and Induced Pluripotent Stem Cells (iPSCs), which also come from the human body. Use of embryonic and fetal stem cells is highly controversial for moral and ethical reasons.

AACT believes that most (if not all) of the benefit of stem cells can be realized with the use of non-embryonic stem cells, and its position is that the very strict regulations associated with stem cells in clinical medicine should be applied in a progressive manner based on safety issues. Some stem cell therapy providers, such StemGenislogo2as La Jolla, California-based stem cell treatment center StemGenex, use only adult stem cells in treating a wide range of diseases, including MS. Representatives of StemGenex, including the company’s founder and CEO, affiliated physicians, staff, and clients — all of whom believe in the safety and efficacy of stem cell treatment — will attend the FDA public hearing.

AlexanderRFounded in 2007 by Rita Alexander, the company’s CEO and also its first stem cell patient, StemGenex has been a pioneer in facilitating stem cell therapy worldwide. As a stem cell advocate, Alexander’s mission continues to focus on changing the lives of people diagnosed with degenerative illnesses.

BioNews Services reached out to StemGenex for the company’s perspective on the FDA’s proposals, and received the following response:

“We are passionately committed to helping people with unmet clinical needs achieve optimum health and better quality of life through stem cell treatment,” says Alexander. “Our goal is to have our voice heard, so that we can help the FDA put forth guidelines for medical professionals to safely administer adipose derived stem cell treatment, here at home, in the United States.”

StemGenex says it continues to champion stem cell treatment, and the public’s right to access the healing benefits of their own cells. Ms. Alexander and Mark Youssef, MD, have formally requested an opportunity to speak at the hearing and are awaiting further details. StemGenex also welcomes the hearing as an opportunity to align with other professional, scientific, and medical organizations to advocate on behalf of the many patients who have sought and continue to seek relief from disabling conditions. The company and its representatives see this platform as an opportunity for U.S. physicians working in this field to take a stand for the sake of their patients.

The FDA says it will do its best to accommodate requests, and will determine the amount of time allotted for each oral presentation. On or before Feb. 5, the agency says that it will notify presenters of their scheduled times and make available a meeting agenda available on this FDA link. Once informed, confirmed presenters need to submit an electronic copy of their presentation by March 11, 2016, to CBERPublicEvents@fda.hhs.gov.

A link to a live webcast of the public hearing will be available through this link as it is taking place. A video recording will be posted through the same FDA link and remain accessible for one year.

Registration to attend or present at the April 16 hearing closed on Jan. 8, when all available spaces filled. The FDA has reopened the comment period to allow interested persons time to submit relevant comments or information electronically or on paper through April 29, 2016. Details can be found at www.regulations.gov/#!documentDetail;D=FDA-2015-D-3719-0001. Because these comments will be made public, the FDA states that individuals are solely responsible for ensuring that their comments do not include any confidential information they or a third party may not wish to be posted, such as medical information, Social Security numbers, or confidential business information. All information in the body of a comment submitted will be made available on www.regulations.gov.

The U.S. Food and Drug Administration (FDA)
Alliance for the Advancement of Cellular Therapies (AACT)
U.S. Stem Cell


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  1. Judith P. Hicks says:

    This is yet another example of government overreach. My initial RRMS diagnosis was made in 1999, and after only two more exacerbations I was diagnosed with secondary progressive MS about 8 years later. My own stem cells are not a drug, unlike all the others I have been taking for all these years. The researchers who have the experience and expertise in this field should be allowed to work unfettered and unhampered by government regulators. I would GLADLY be a participant in their studies.

  2. Phantom says:

    FDA works for BIG PHARMA not for the safety of the public. Trials are showing promise in too many areas so the FDA needs to crack down??? Lets make it a drug and get it caught up in trails for the next 2 decades. (We want to be sure it is safe is just a way to say we need companies to make billions on it first…) This county is in big trouble for so many reasons. This is just another example of government not working for the best interest of the people.

  3. Janel Mitchell says:

    I was diagnosed in 2006 although we’ve been trying to find out what was wrong with me since I was a teenager. It took me going completely blind and loosing the feeling on the right side of my body to get me to the right Doctor. After 10 years of try this and that I’m at the point where they are keeping me comfortable until…When their voice trails off I know what that means. And if there is something out there that can help me then please make it available! I used to be a go getter! I worked 2 jobs and wanted to take over the world. Now I just want to get thru Christmas dinner without crying from the pain. If this can help me get up and do things for myself again then by all means let’s do it! I’m of no use to anyone laying in bed 24/7.
    Janel Mitchell

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