Stem-cell therapy feels Food and Drug Administration's pinch

Denver Post, Jason Belvins, April 12, 2010

62-year-old Hal Kaye had an injection of his own stem cells into his injured ankle, the results were so astonishing that he no longer needed surgery. The treatment, developed by a Broomfield doctor, has been applied to over 500 patients in the United States, primarily for orthopedic conditions, and involves extraction of bone marrow stem cells, expansion of the cells, and subsequent readministration.Three years after administration of the stem cells, Hal Kay says "I can walk anywhere now. It's been an incredible recovery." Mr Kay no longer needs to use his cane and has reported a significant improvement.

Unfortunately the treatment has resulted in concerns by the Food and Drug Administration.In 2008 the FDA sent the Broomfield doctor a letter stating that since the procedure was not approved according to regulator channels such as a Biologics License Application (BLA). Typically cell therapy falls into the category of a "biologic" and therefore requires 2 successful Phase clinical trials before it can be sold to the general population. The doctor was asked by the FDA to provide a response detailing "steps you have taken or will take to address the violations."

While the doctor claims to have responded to the FDA, no formal reply to his response was provided he stated. His position is that a person's own stem cells, despite being expanded in tissue culture in a laboratory, are not a drug.

Currently the doctor has assembled a team of colleagues and will be meeting with the FDA to present their position that stem cell therapy using cells from the same patient should be regulated as a medical practice and not as a new drug.His argument comes in part from the example of in vitro fertilization, an area of medicine that is regulated by a peer-reviewed panel but not by the FDA.

Interestingly, not all doctors have followed the approach of going against the FDA's position. Companies such as TCA Cellular Therapy from Louisiana are applying to the FDA for clinical trials and are currently conducting controlled experiments in order to obtain approval through the regular channels. This is despite the fact that they are using stem cells from the same patient.

In the opinion of most biotechnology companies we discussed with, the outcome of the discussions with the FDA will be of great importance.Regulation of autologous therapies has been around for decades. Before stem cells became popular, the use of patient's own immune cells such as dendritic cells or T cells also required FDA approval. This has set up the current paradigm for developing cell-based therapies.In our opinion the use of patient's own stem cells without expansion may in some situations be acceptable for performance without FDA review, however, expanding cells in tissue culture is a very complex and difficult procedure. It will be important for the FDA to regulate this since there are numerous possibilities for non-pure cell products being used if the industry is unregulated.