New Trk for an old protein

Short serum half-life, poor blood-brain barrier penetrability and limited diffusibility in CNS tissue all make brain-derived neurotrophic factor (BDNF) a less than ideal therapeutic. Indeed, the growth factor's track record in the clinic has been subpar, including a Phase III failure in amyotrophic lateral sclerosis.

Nevertheless, the protein's role in neuronal survival has kept researchers and companies looking for alternatives to delivering the protein, including preclinical programs for BDNF gene therapy,¹ BDNF-releasing cell grafts² and compounds that trigger expression and release of endogenous BDNF.^3,4...