NextGen ALS is teaming up with Project ALS along with Weill Cornell Medicine & Ionis Pharmaceuticals

NextGen ALS is aligning with Project ALS as an initiative within their organization. Project ALS is a 501(c)3 non-profit that identifies and funds the most promising scientific research that will lead to the first effective treatments and a cure for ALS. Since 1998, Project ALS has recruited world-leading doctors and scientists to work together—rationally and aggressively—to understand the genetics and biology of ALS, develop new technologies and tools for drug discovery, and ultimately, deliver the first meaningful treatments to people with ALS.

Together with Dr. Giovanni Manfredi and Dr. Hibiki Kawamata Fujita at Weill Cornell Medicine, along with Ionis Pharmaceuticals, we have developed a highly qualified team of scientists in the field of Genetic ALS. This team is dedicated to medical and scientific research directly targeted toward developing treatments for Genetic ALS. Drs. Manfredi and Kawamata have already been collaborating with Ionis, and they have made a series of CHCHD10 ASOs that they are now testing to see which ones knock down the gene most effectively. After that they will test the most successful ones for tolerability, and if all goes well we will be testing the top choices at Weill Cornell by this summer!

Every dollar you donate to NextGen ALS will go directly to research and drug development for the rare form of ALS that has impacted generations of our family. For many years, our family knew that we had a "familial"—inheritable—form of ALS, but doctors could not identify the specific genetic cause. But times have changed: in the last decade, new technologies have allowed scientists to both identify the specific gene that causes our family's ALS—CHCHD10—and to develop new types of drugs, like antisense oligonucleotides (ASOs), that have shown promise in effectively treating other brain diseases. With Project ALS, we are assembling a team led by Drs. Giovanni Manfredi and Hibiki Kawamata (Weill Cornell Medicine) to take down CHCHD10-ALS. Your donation will immediately go toward studies in the Manfredi & Kawamata labs that aim to (1) understand what goes wrong in a brain cell with CHCHD10-ALS, and (2) rapidly test therapies that might slow, stop, and even prevent CHCHD-10 ALS.