Cure SMA Releases Updated Overview of Research Model

When Cure SMA first started funding research thirty years ago, we had to build up from virtually nothing. It’s no easy task. Drug development relies on volume to be successful—just one or two options are not enough. The vast majority of drug candidates end in failure. To arrive at just one FDA-approved drug for SMA, we need to start with thousands of compounds in the pipeline. On average, only 10% of drugs that make it as far as clinical development will ultimately receive FDA approval.

Getting the funding and attention we need to find a treatment and cure for SMA is an uphill battle. Our research strategy is about volume, balance, and leverage—promoting a breadth of options, pursuing a diversity of approaches, and positioning SMA as an attractive investment for companies.

Though challenges remain, we’re seeing tangible results from our approach. Through strategic investments, we’ve grown the pipeline to an unprecedented level of breadth and diversity. Fifteen years ago, we had just two potential drugs in the beginning stages of preclinical discovery. Five years ago, we had 12. Going into 2015, we have 17, including seven in clinical trials—just steps away from FDA approval.

We’ve created a new booklet, Advancing Research Toward a Treatment and Cure, to help introduce the research process. The last few months have seen several exciting announcements of new research grants and new clinical trials. As these announcements are made, we’re evaluating where each of them fits into the big picture of research, and how each might contribute to our overall goal of a treatment and cure for SMA. This booklet explains more about that big picture.

You can download a PDF of our new booklet, or send us an email at [email protected] to request a printed copy of the booklet.

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