Cure SMA is pleased to announce that we have expanded our research collaboration with the California Institute for Biomedical Research (CALIBR) to include a collaborative effort with Dr. Chien-Ping Ko of the University of Southern California.

At Cure SMA, we know that no single group can develop a treatment for SMA alone—it requires collaboration. One of our goals is to foster collaboration between research groups, and the extension of the CALIBR project to the Ko lab at USC is a prime example.

This partnership first began in 2012, when we awarded $700,000 to Dr. Peter G. Schultz and his team at CALIBR to support their drug discovery program, “Optimization of Small Molecules that Increase SMN2 Levels for the Treatment of Spinal Muscular Atrophy.”

One promising therapeutic approach to SMA is small molecules, which are chemicals that can treat or cure a disease. The Schultz group previously discovered two different compound classes that enhance SMN protein levels. SMN protein is critical to the function of the nerves that control our muscles, and individuals with SMA don’t properly produce this protein.

Our funding to CALIBR has been focused on turning these compounds into a useable drug through a process called medicinal chemistry. 

Currently, the team is testing the compounds in a severe mouse model of SMA, one of the final steps in the current project. This testing will be done in the laboratory of Dr. Chien-Ping Ko. Dr. Ko and his team will be assessing a number of important features in these mice, so they can determine how well the compounds are working.

We are excited to reach this stage of the project. Positive data will provide the rationale to initiate the studies needed for an Investigational New Drug (IND) application, which is the first step to obtaining FDA approval for a human clinical trial.

About Our Drug Discovery Funding

Developing a treatment and cure for SMA is the driving force behind Cure SMA. To achieve this goal, we aggressively invest in drug discovery projects like the CALIBR project. Drug discovery converts basic research ideas into new drug candidates that can be tested in clinical trials.

Our unique approach minimizes the most common challenges of drug discovery by building a diverse “pipeline” of drug candidates. Ten years ago, we had just one potential drug in the pipeline. Five years ago, we had four. Today we have over a dozen, including five now in clinical trials. Two-thirds of these programs have been funded by Cure SMA.

About Chien-Ping Ko, PhD, University of Southern California

Dr. Ko (pictured above) received his PhD in physiology from Washington University in St. Louis, and did postdoctoral training in neurobiology at the University of Colorado and then the National Institutes of Health.  Since 1981, Dr. Ko has been a faculty in the Section of Neurobiology, Department of Biological Sciences, University of Southern California, Los Angeles, CA.  Dr. Ko studies how the connections between motor neurons and muscle form, function, and are properly maintained.