Case Study: Consortium Explores KPT-350 as a Potential Treatment for ALS

The ALS drug KPT-350 was one of the first of a dozen therapeutic options to emerge from the Target ALS Innovation Ecosystem and is now poised for clinical trial thanks to the power of collaboration. Shortly after its launch, Target ALS forged and funded one of the world’s biggest consortium, with eight labs offering complementary expertise. The consortium sought to better understand the pathobiology of C9orf72 ALS, the most common form of familial ALS that is also associated with ~7% of sporadic ALS cases.

Consortium members identified nucleocytoplasmic transport (NCT) as one of the important mechanisms involved in C9orf72 ALS. After reviewing these findings, Target ALS connected consortium leaders with Karyopharm Therapeutics, a biotechnology company that was already targeting NCT for cancer treatment through a drug called KPT-350. Target ALS fostered a cross-sector collaboration to validate the use of KPT-350 to target NCT in ALS. As a result, KPT-350 advanced rapidly as a potential therapy for ALS and was acquired by neuroscience pioneer Biogen for translation into a clinical trial.

From start to finish, our unique Innovation Ecosystem helped to bring together the right partners and accelerate ALS research that would have otherwise taken many years to advance. A new treatment for ALS is within reach.