Cautionary Tale: Tumors Caused by Neural Stem Cells

I usually avoid commenting on current events occurring in the field of regenerative medicine, primarily because I can’t keep up!  In this case,  I believe the recent publication by Amariglio et al, has FDA regulatory implications for the clinical development of therapies based on stem cells.  The under-lined portion of the title of the publication says it all:

Donor-Derived Brain Tumor Following Neural Stem Cell Transplantation in an Ataxia Telangiectasia Patient”

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13 Tips for Successful FDA Meetings

There are many reasons for meeting with the FDA during development of a regenerative medicine product. Some regenerative medicine products are more novel than others, meaning they are so unique they have never been studied in human clinical trials. Others may not be first of its kind, but may be used in novel clinical applications or be manufactured in new ways in comparison to competitor products. The nuts and bolts of preparing to meet with the FDA will be covered in a separate posting.

There are a number of opportunities for informal and formal communications with the FDA. For the purpose of this post, I am assuming you have requested a formal meeting with the FDA, perhaps to discuss the filing of an investigational new drug (IND) application to initiate human clinical studies.

Usually, to reach this point you will have expended a significant amounts of time and money. Therefore you don’t want to blow it by not being prepared to meet effectively with the FDA. Below is my list of tips to help the process go more smoothly:

  1. Remember, The FDA Wants You to Succeed
  2. Leave the “Dog & Pony” Show at Home
  3. Safety is the FDA’s Primary Focus
  4. Provide Clarification for FDA Identified Issues
  5. The FDA Will Have Questions, Lots of Them
  6. Designate a Spokesperson to Lead the Discussion
  7. Unity is a Virtue
  8. Take Time to Summarize
  9. Call in Early
  10. Call a “Time Out” if Needed
  11. Identify yourself
  12. You May Get the FDA’s Comments Early
  13. Overall, Remain Calm, Be Factual and Enjoy the Experience.

Read on, for my thoughts on how each of these tips can be used to ensure a smooth meeting with the FDA.

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What the Heck is a Regenerative Medicine Therapy?

My favorite working definition for regenerative medicine is any medical intervention that is intended to repair, replace, restore or regenerate (the 4 R’s) the cellular, tissue or organ systems in the human4Rs.jpg body. As described by one of my former FDA colleagues, Dr. Steven Bauer, cellular therapies represent one branch of regenerative medicine that on their own can fulfill the 4 R’s. However, it is clear that many other medical technologies will contribute to the field of regenerative medicine. See my post on how the FDA views the world of regenerative medicine.

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How Does the FDA View the World of Regenerative Medicine?

A major focus of the Regenerative Medicine Guru Blog is the development of new medical interventions based on regenerative medicine.

Therefore, it is important to understand how the ‘gatekeepers’ view this field. In the U.S., the Food and Drug Administration (FDA) is the key gatekeeper. You may be surprised to learn that there is not a specific regulatory pathway spelled out for regenerative medicine products.

Instead, the FDA’s approach is to examine each specific regenerative medicine product and determine the regulatory pathway based on the most important component(s) of the regenerative medicine product. To accomplish this, the FDA essentially conducts a thought experiment in which the regenerative medicine product is ‘de-constructed’ in order to identify the key components and ask the question, which component(s) are most important in providing benefit to the patient.

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