Shepherding New Stem Cell Therapies Through the Valley of Death

2011 BMT Tandem Meetings
E. Donnall Thomas Lecture
Malcolm Brenner, MD, PhD
Baylor College of Medicine
Texas Children's Hospital
The Methodist Hospital

Objectives: Dr. Malcolm Brenner of Baylor University Medical Center/Texas Childrens Hospital presented the 2011 E. Donnel Thomas lecture. After providing a history of Cell and Gene Therapy (CAGT), referencing his development in the field, and mentioning stem cell transplantation, he initiated a detail discussion complex biological therapeutics (CBT) which he showed did not fit the classic pharmaceutical model. CBT has a very difficult pathway for development of clinical applications. Various CBT probes in order to be successful must be shown to be better, simpler, cheaper, and safe. The present regulatory environment can be very difficult to traverse. While strong regulatory control is essential, it must be made less complex. Young clinical investigators are needed to “shepherd a new CBT through the valley of death” so they can emerge successfully on the other side. CBT has very favorable pharmacoeconomics. They need a fast track orphan drug status.