FDA Set to Approve the First Gene Therapy Procedure

An FDA advisory panel voted unanimously this week to recommend that the FDA approve the first gene therapy procedure.  The procedure is for the treatment of B-cell acute lymphoblast leukemia in children that have resisted treatment by other methods.  Final FDA approval is expected by October.

The procedure involves removing millions of a type of white blood cells called T cells from the patient, genetically modifying them to enhance their ability to attack abnormal B cells (the type of cell that becomes abnormal in this type of leukemia), and then returning them to the patient.  So far the technique has been tried on 63 patients.  Fifty-two of them are still in remission (for an 82% remission rate); the other eleven died.  The very first patient to receive the treatment was 12-year-old Emily Whitehead.  She was treated 6 years ago and is currently cancer-free.

It would seem to be a no-brainer that this procedure should be approved.  However, although the FDA panel did recommend approval at this time, a word of caution is in order.  We still don't know how permanent alteration of a patient's T cells might affect the their health in the long-term.  To try to answer that question, patients who undergo the procedure will be entered into a registry and followed for at least 15 years.  But so far, at least, everything looks good.

Now for the bad news.  The procedure is likely to cost at least $300,000.