Gene Therapy and Initial FDA guidelines

We feel a responsibility to keep our donors informed on the programs we have funded and continue to fund. It is the critical funding that you provide that enables The Sophia’s Cure Foundation the ability to support these programs. Recently there has been some confusion as to what is necessary to advance Gene Therapy  from the lab to the patient. We have released a note recently to update the SMA community:

Based upon initial talks with the FDA, the FDA believed the proof-of concept studies were adequate from Dr. Kaspar and Team’s proposal that was sent to them and they did not require a pig model of the disease for testing to move into clinical trials.  What would be required are a few additional studies to determine how much virus for dosing, as the FDA requested a dose escalation trial.  Then, Dr. Kaspar and Team would have to perform a toxicology and biodistribution study in mice to answer whether the doses given are truly safe in mice, and where the virus goes after delivery.  I think we all agree that additional models, including the pig model are good things to test a therapeutic in, but the simple answer is, It is not necessary for Dr. Kaspar to get his gene therapy into clinic based on the first call he and his team had with FDA.  The Sophia’s Cure Foundation will continue to fund the SMA gene therapy program from Nationwide Children’s hospital/OSU based upon guidance from the  Food and Drug Administration and the requirements they  deem necessary to move towards application for human clinical trials.

There seems to be some confusion as to the necessity of a larger animal species in order to advance Dr. Kaspar’s gene therapy from the lab into the human population. Based on FDA guidelines a larger animal study was not needed as they were pleased with proof of concept in initial talks. A larger animal species such as a Transgenic SMA Pig or SMA Non-Human Primate would certainly benefit SMA research in general. We encourage the development of  new models that will give us a better idea of how a therapy could possibly  benefit the patient population. However it is important to understand that developing a larger animal species is not needed for AAV9. Based upon the rigorous timeline that has been projected, creation  of larger animal models would not fit into that schedule.

March 03, 2011

“Dear SMA Community,

Once again thank you for your continued support for our Gene Delivery Program. We are all very pleased with the initial discussion with the Food and Drug Administration regarding moving our gene delivery for SMA patients to human clinical trials.  I want to take a short opportunity here to clarify a point of whether we needed to have proof of concept or testing in a large species of SMA, such as the pig model currently in development to move into the clinic.  Based on our preliminary call with the FDA,  we do not have to test our therapy in other models such as the pig to move forward towards human testing.  The guidance from FDA was to perform the formal safety testing in mouse studies only. We were encouraged by their suggestions and guidance and are currently gearing up with some additional small scale studies to get us to this point.  We will certainly update the community as we progress our clinical gene delivery program for SMA patients.

The pig model  is a long term vision for not only gene therapy but also drugs, oligos, and stem cells for the treatment of SMA. The development of this larger model of the disease will provide a very powerful tool in our toolbox for understanding SMA and developing new and improved therapies.   Therefore, I remain enthusiastic and in full support for developing additional models to study SMA and look forward to the continued momentum in research and therapeutic development in SMA.”  Dr. Brian K. Kaspar, Ph.D. Associate Professor The Research Institute at Nationwide Children’s Hospital The Ohio State University

Thank you for your continued support, we strive to give the most accurate up to date information regarding SMA Research.


About Catherine Gaynor

Mom to beautiful Sophia.... SMA Type 1

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