Drug Research
Drug Discovery & Development

Karyopharm receives grant to further develop selective inhibitors of nuclear export compounds

Published 09 January 2014

Karyopharm Therapeutics, a clinical-stage pharmaceutical company has been awarded a grant from the National Multiple Sclerosis (MS) Society through its affiliate, Fast Forward, to support research on the potential of SINE compounds in inflammatory models of disease, including MS.

Finding ways to restore and protect the damaged nervous system is a key priority of the National MS Society's No Opportunity Wasted (NOW) research campaign to ultimately eradicate MS.

Karyopharm founder, chief scientific officer, and president Dr Sharon Shacham noted the company is excited about its continuing collaboration with the National MS Society, and with Mount Sinai with Dr. Patrizia Casaccia, who has dedicated herself to advancing research in MS and other important diseases.

"We believe SINE compounds may offer significant therapeutic benefits in a range of indications and look forward to exploring the potential application of SINE compounds in inflammatory diseases, including MS," Dr Shacham added.

SINE compounds inhibit Exportin-1 (XPO1 or CRM1), which mediates the export of approximately 220 different mammalian cargo proteins. In MS, toxic factors accumulate in the brain and spinal cord that may attack or destroy the myelin coating on a neuron's axon causing electrical signals to other nerve cells, muscles, and cells throughout the body to slow, thus leading to neurodegenerative symptoms.

Preclinical data suggest that SINE compounds may reduce inflammation and protect against these toxic factors in neurons, which could potentially reduce the perpetuation and progression of MS.

Dr Casaccia, Professor in the Departments of Neuroscience and Genetics and Genomics at Icahn School of Medicine at Mount Sinai, is the academic lead on the study. Her laboratory first identified nuclear export as a potentially important mechanism in neurodegeneration and conducted preliminary experiments using mouse models. Her experiments suggest that oral administration of a novel SINE compound to mice with ongoing paralysis of the tail and hindlimb may improve their walking ability.

Dr Casaccia will continue this research, in conjunction with Karyopharm, to further study the mechanism of action and safety profile of SINE compounds in inflammatory models and to gather data to potentially select a SINE compound as a candidate for early-stage clinical trials in human patients with MS.

Dr Casaccia underscored the potential of SINE compounds as a novel treatment for MS.

Source: Company Press Release