Cure SMA-Funded Researchers Receive NIH Grant

Cure SMA-funded researcher Peter Schultz has been awarded a four-year NIH grant of just over $2 million to support his research into small molecules to treat SMA. Cure SMA has funded the work of Dr. Schultz and his team at the California Institute of Biomedical Research (Calibr) through three drug discovery grants in 2012 and 2015, with the total amount of funding approaching $1 million.

Individuals with spinal muscular atrophy don’t properly produce survival motor neuron (SMN) 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. Our funding to Calibr has been focused on optimizing these compound classes.

With this experience in hand, Dr. Schultz and his team have been able to secure a new NIH grant. With the additional funding from the NIH, he will be able to carry on his work on identifying and developing small molecule drug candidates for SMA.

The Importance of Leverage

It can cost upwards of $1 billion to bring a therapy through the development process to FDA approval. This level of investment requires collaboration between academics, industry, government, and families. Cure SMA brings all of those groups together, breaking down roadblocks that might delay important developments.

One of our most powerful tools in breaking down those roadblocks is our “seed funding” approach. We provide early-stage funding for promising new ideas and novel treatment approaches. In doing so, we lower the risk and attract larger investments from industry, government, and other funding sources as drug candidates move through the process.

In addition, we are working to increase the number of new therapies in the SMA pipeline, to insure we have treatments for all ages and stages of SMA. Attracting investment from government, pharmaceutical companies, and other organizations provides the resources necessary to accomplish these goals.

The NIH grant to Calibr is another example of the impact of this seed funding strategy, along with other recent examples in gene therapy, antisense oligonucleotides, and muscle activators.

Our thanks to Dr. Schultz and the team at Calibr for all their hard work on this program. In addition, our thanks to the many generous families and donors who have supported the Cure SMA grants that have allowed us to leverage new investments in SMA research.

Funding for the Calibr program is supported by a special gift from The Michael and Chandra Rudd Foundation.

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