Partnership For CMT2A
StarWise Therapeutics LLC based in Madison, WI has developed novel, safe HDAC6 inhibitors for use in the treatment of various neurological disorders. On the top of its list is the use of its new drugs in the potential treatment of the genetic disorder known as Charcot-Marie-Tooth disease, or CMT for short.
CMT is a progressive genetic disease of which there are many variants. CMT2 is a mutation in the sequence of the mitofusin 2 gene or MFN2. MFN2 is a protein that plays an essential role in the function of mitochondria which generate energy in the cells. In MFN2 patients, the energy produced by neuronal mitochondria is insufficient to sustain axons. As a consequence, axonal degeneration gradually occurs, causing muscle weakness, atrophy and deformities in the feet, legs, arms and hands. Those with CMT often lose the ability to walk and may become dependent upon assistive devices to remain mobile. Severe, chronic pain is common, and there is no cure.
StarWise Therapeutics and Waikato University in NZ have demonstrated that their key drug is able to restore sensory and motor function in the CMT2A mutant animals to performance levels akin to those of the wild type animals.
Allison Moore, CEO HNF & Alan Kozikowski, CEO StarWise
December 20, 2018 ~ NYC