Drug discovery converts what we have learned about the causes and biology of SMA through basic research into new drug candidates that can be tested in clinical trials.
It can be a long and difficult process. Failures happen often, so it’s difficult to predict which drugs will be successful. An estimated 80% to 90% of experimental drugs starting human clinical trials never gain FDA approval.
Our Approach to Drug Discovery
Our unique approach minimizes the most common challenges of drug discovery by building a diverse “pipeline” of drug candidates.
- We invest in a number of projects at a time, which means that if a drug candidate fails, several others can take its place.
- We fund projects that represent a variety of therapeutic approaches, attacking SMA from all sides.
- We provide seed funding for new projects. Traditionally, it has been difficult to get pharmaceutical companies to invest in research for rare diseases like SMA. By providing early-stage funding, we lower the risk and attract larger investments from industry and government as drug candidates move through the process.
- We are unbiased as we evaluate all possible treatment opportunities, and we prioritize, select, and manage our drug discovery projects through a Translational Advisory Committee, comprised of industry experts.
Our Achievements in Drug Discovery
Our diverse and broad-based approach has led to important breakthroughs:
- In December 2016, the FDA announced that it had approved Spinraza, a treatment developed by Biogen and Ionis, making it the first-ever approved therapy for SMA. Cure SMA provided the very first research funding for this program beginning in 2003.
- The approval of Spinraza is just the leading edge of a robust drug pipeline, with a breadth and depth that reflects our goal of treatments for all ages and types. Fifteen years ago, we had just two potential drugs in the beginning stages of preclinical discovery. Today we have our first-ever approved treatment, plus another 17 programs in development, including five in clinical trials.
- We have identified four different therapeutic approaches that show promise in treating SMA: replacement or correction of the mutated gene in SMA called SMN1, modulation of backup gene in SMA called SMN2, neuroprotection, and muscle protection.
- Important biotech and pharmaceutical partners are committed to SMA research. Today, 14 companies are engaged in SMA research, including some of the biggest pharmaceutical companies in the world.