BioCentury
ARTICLE | Distillery Therapeutics

Neurology

February 15, 2017 5:57 PM UTC

Patient sample, rat and mouse studies suggest inhibiting CYPA could help treat ALS. Levels of CYPA were higher in CSF from sporadic ALS patients, a mouse model of familial ALS or spinal cord tissue samples from a rat model of familial ALS than in patients with unrelated neurological diseases or normal rodents, respectively. In co-cultures of primary mouse astrocytes and spinal neurons from the mouse model, a CYPA inhibitor tool compound increased motor neuron survival compared with no treatment. In the mouse model, systemic CYPA knockout increased numbers of motor neurons in the lumbar spinal cord compared with normal CYPA expression. Also in the model, intracerebroventricular infusion of the CYPA inhibitor increased numbers of motor neurons in the lumbar spinal cord, motor function and survival compared with vehicle. Next steps could include identifying and testing additional CYPA inhibitors in models of ALS...