For years, the neuroscience industry has been plagued by a high failure rate of drugs in the clinical stages. Long drug discovery timelines, inability of drugs to cross the blood brain barrier, and challenges with the FDA have driven numerous biopharmaceutical companies to downsize their early-stage CNS drug development efforts or even abandon the CNS market, leaving patients with limited hope for cures to truly devastating diseases.
Recent events, however, have instilled some optimism in neurosciences.
“For years, the neuroscience industry has been plagued by a high failure rate of drugs in the clinical stages. Long drug discovery timelines, inability of drugs to cross the blood brain barrier, and challenges with the FDA have driven numerous biopharmaceutical companies to downsize their early-stage CNS drug development efforts or even abandon the CNS market, leaving patients with limited hope for cures to truly devastating diseases.
Development of therapies for central nervous system (CNS) diseases has lagged behind other therapeutic areas despite a growing need for new treatments. New drug development for CNS diseases poses unique challenges due to the brain’s complexity, an incomplete understanding of CNS disease biology, and of course, the blood-brain barrier (BBB)- a structural and functional roadblock to microorganisms, and large molecules that may be circulating in the bloodstream.
Recent events, however, have instilled some optimism in neurosciences. We have seen a few scientific breakthroughs that have resulted in biomarkers that can be used to de-risk early-stage programs. New therapeutic modalities have also emerged that can reach cells and tissues of interest, through the blood brain barrier. In addition, the FDA’s decision to approve Biogen’s Aduhelm™ (aducanumab) for the treatment of Alzheimer’s using the accelerated approval pathway has provided hope for biopharma companies and generated renewed interest in moving clinical candidates forward. The application of genetic profiling to CNS diseases has also revealed subpopulations which can be targeted specifically.”
