Cure SMA Grants $304,000 to Columbia University Professor for SMA Drug Discovery

Cure SMA recently granted a $304,000 drug discovery award to Livio Pellizzoni, assistant professor of Pathology & Cell Biology at Columbia University for a new research project with potential to treat spinal muscular atrophy (SMA).

Pellizzoni and collaborators at Northwestern University have identified an altered cellular pathway in SMA patients, which may contribute to disease development. SMA is caused by a deficiency of a motor neuron protein called SMN (survival motor neuron.)

Using SMA mouse models, researchers found that the SMN deficiency results in the activation of the specific pathway, called p38αMAPK .

The new project, “Pharmacological Inhibition of p38αMAPK as a Candidate Therapeutic Approach for SMA”, will seek to find if the inhibition of the p38αMAPK pathway can help treat the disease.

Clinical trials in Alzheimer’s and other neurological diseases are currently evaluating an orally available p38αMAPK inhibitor. But the newly funded project will examine the efficacy of the compound in an SMA mouse model in hopes that results could lead to pre-clinical and clinical development of the compound for SMA patients.

Early clinical research mainly focused on increasing the production of SMN by correcting or replacing faulty SMN1 or by modulating SMN2. But evidence shows that several processes, systems and pathways such as p38αMAPK, influence the disease. These alternative approaches could be used in combination with treatments that directly target SMN levels to attack SMA from all sides, providing a better chance for comprehensive, effective treatment.

A consequence of the strategy is greater focus toward developing combination compounds for SMA in order to treat all types and stages of the disease. Approaches focused on enhancing SMN may be heightened by mixing them with compounds that target cellular pathways which contribute to motor neuron death or muscle dysfunction –  independently of SMN.

Cure SMA explored the subject with researchers during its 2014 and 2015 SMA Researcher Meetings, as noted in SMA’s Researcher Meeting Summary.

The summary also includes information  about research for related disorders. Experts maintain that similar work in ALS, Parkinson’s and Alzheimer’s disease could accelerate the development of treatments for SMA.

A news release from Cure SMA said the grant is part of the organization’s recent distribution of $704,000 toward several drug discovery projects.