15-08-08(10:39:56)
Authors:
Stewart, GR
Institutions:
Medtronic Neuromodulation, Minneapolis, MN 55432
Title of abstract : The Unraveling of Cell Therapy for Parkinson’s Disease: Where do we go from here . . . why gene therapy trials should be listening . . . and why didn’t we learn from trophic factors?
Abstract text:
Starting in the early 1990s, biotechnology brought great promise to the treatment of neurodegenerative disease with the development and clinical translation of neurotrophic factors. Unfortunately, all these programs, ranging from CNTF for ALS to GDNF for Parkinson’s disease (PD), ended in failure, disappointment and frustration. Likewise, a wave of cell therapy has moved through the clinic, most notably for the treatment of PD, and has suffered a similar fate. Now, we are in the midst of a third wave of biotechnology in the form of gene therapy, with several ongoing trials in PD as well as other neurologic disorders.
Of interest is to look across this tremendous outpouring of scientific discovery and clinical research and ask the obvious question: what is going wrong? This talk will quickly outline the commonalities among these different forms of therapy that may have contributed to clinical failure, and look at some ways to help insure a better outcome in the future. But even if there is success in translating cell or gene therapy for PD, a second realm of concern is to examine the inherent risks and limitations in their commercialization, turning cells into pills as it were. Cell therapies, in particular, must navigate a series of barriers, from societal pressures to manufacturing to reimbursement, if they are to succeed medically and financially.
