Cure SMA Announces $315,000 Grant to California Institute of Biomedical Research

ELK GROVE VILLAGE, Ill., March 2, 2015 /PRNewswire/ -- Cure SMA recently announced a new $315,000 drug discovery grant to the California Institute of Biomedical Research (CALIBR). This funding is an extension of a spinal muscular atrophy research partnership that began in 2012, with a $700,000 Cure SMA grant to Dr. Peter G. Schultz and his team for their drug discovery program, "Optimization of Small Molecules that Increase SMN2 Levels for the Treatment of Spinal Muscular Atrophy."

Survival motor neuron (SMN) protein is critical to the function of the nerves that control muscles. Individuals with spinal muscular atrophy don't properly produce this protein at high enough levels, due to a mutation in the survival motor neuron gene 1 (SMN1). Individuals with SMA do have one or more copies of survival motor neuron gene 2 (SMN2), the SMA "back-up gene." SMN2 makes only a small amount of functional SMN protein, so researchers are looking for ways to prompt this gene to make more protein.

One very promising therapeutic approach is small molecules, which are chemicals that can treat or cure a disease. The Schultz group previously discovered several different compound classes that enhance SMN protein levels. Cure SMA's funding to CALIBR has been focused on turning these compounds into drug candidates through a process called medicinal chemistry.

Progress to Date

"As with all our drug discovery grants, this program is overseen by the Cure SMA Translational Advisory Council, a committee of drug discovery and SMA experts," said Jill Jarecki, PhD, Cure SMA's research director. "Funding for this project is provided after achieving a series of predetermined milestones, which are reviewed and approved by a sub group of the TAC convened specifically to oversee this program."

To date, the research team has completed the following milestones:

(1) They have demonstrated that compound treatment elevates SMN protein levels in a severe mouse model of SMA; (2) They have identified new related compounds, called analogs, that are more effective at increasing SMN levels; and (3) They have identified new analogs that more effectively reach the brain.

Recently, funding for this project was expanded to include a collaborative effort with Dr. Chien-Ping Ko of the University of Southern California. Currently, Dr. Ko and the team are testing CALIBR's compounds for benefit in a severe mouse model of SMA. They will be assessing weight gain, motor function, survival, and motor neuron morphology at synapses in the central nervous system and at the muscle.

Next Steps for New Funding

The new goals of the project, which will also be funded through a series of predetermined milestones, focus on continuing to optimize compounds in order to identify a drug candidate suitable for human testing. These goals include demonstrating a survival benefit in a severe model of SMA, further enhancement of brain exposure, and optimization of safety and selectivity of the lead compounds.

These optimized compounds will hopefully lead to a clinical development candidate. The candidate would then undergo the series of studies required for an Investigational New Drug (IND) application, the first step to obtaining FDA approval for a human clinical trial.

"We're enthusiastic about the progress we've made, and delighted to continue our partnership with Cure SMA," said Dr. Peter Schultz. "Their leadership in and commitment to research leads the way in spinal muscular atrophy. We look forward to continuing to move this program toward the clinic."

Leading the Way to a Treatment and Cure

"Developing a treatment and cure for SMA is the driving force behind Cure SMA, and the goal of our research model," said Jarecki. "While there is real promise in the research landscape right now, which gives us great reason for hope, we know that much work remains to be done. There's a pressing need for continued and growing investment in drug discovery, to respond to the complexities of the process and lead into clinical trials. This is why we aggressively invest in promising programs like the CALIBR program."

Funding for the CALIBR program is supported by a special gift from The Michael and Chandra Rudd Foundation. Funding for the USC expansion is supported by a special gift from The Dhont Family Foundation.

PRLog ID: www.prlog.org/12430932

SOURCE Cure SMA