Cure SMA’s Early-Stage Funding Leads to Big Pharma Investments in Promising Therapies

Cure SMA, an organization founded in 1984 to fund and invest in research for spinal muscular atrophy (SMA) treatments, also focuses on the day-to-day realities of patients and families, reaching about 4,000 SMA families every year through family support services with its 110,000-plus members and supporters.

Among its other milestones, Cure SMA has invested more than $59 million in research for spinal muscular atrophy and has funded two-thirds of the 15 ongoing drug programs for spinal muscular atrophy.

The organization’s main focus is early-stage funding for promising new therapeutic approaches and attracting larger investments from the industry and government, which is extremely important because the development process for one new drug, from initial scientific studies and clinical trials up to FDA approvals, can cost up to $1 billion.

This early investment strategy has already provided a few success stories. In the field of gene therapy, Cure SMA invested, through a series of grants, $845,000 in two different gene therapy programs led by Dr. Brian Kaspar, principal investigator in the Center for Gene Therapy at Nationwide Children’s Hospital.

Based on data generated by this funding, Kaspar’s programs were awarded an additional $4 million grant from the National Institute of Neurological Disorders and Stroke (NINDS). The technology was then licensed to another company to conduct clinical trials and an initial public offering (IPO) by AveXis raised more than $95 million to move the therapy to Phase 2 clinical trials.

The same process happened in the area of antisense oligonucleotides (ASOs). In 2013, Cure SMA awarded $150,000 to Dr. Chris Lorson and Dr. Arthur Burghes to aid in the development and optimization of an ASO that binds and interferes with the novel E1 sequence, a strategy that could improve the function of the SMN2 gene, also called the SMA “backup gene.”

Last year, results from this research revealed the team was able to increase the life span of SMA mouse models by four times and intermediate mouse models of SMA by seven times. Based on this encouraging data, Lorson was recently awarded two grants totaling $500,000 from MDA, GSF, and FightSMA, to continue the development of this ASO compound.

Finally, Cure SMA also awarded a grant in the area of muscle activators to Dr. Jeff Jasper at Cytokinetics to support the development of a small molecule that activates and protects damaged skeletal muscles. Cytokinetics has now partnered with Astellas Pharmaceuticals to bring this drug class, specifically CK-2127107, into clinical development. Following an investment of $55 million, the drug is now being tested in a Phase 2 clinical trial in adults and teens with spinal muscular atrophy.