Improving therapeutic potential of GDNF family ligands

The last decade has been a frustrating time for investigators who had envisioned major advances in the treatment of Parkinson’s disease using neurotrophic factors. The first trials of glial cell line–derived neurotrophic factor for treating Parkinson’s disease were very promising. Later blinded control trials were disappointing, not reaching the predetermined outcomes for improvement in motor function. Consideration of the problems in the studies as well as the biology of the neurotrophins used can potentially lead to more effective therapies. Parkinson’s disease presents a multitude of opportunities for the cell biologist wanting to understand its pathology and to find possible new avenues for treatment.