"Without additional funding, we may have to make a difficult decision: aggressively move forward with only one of these discoveries, or move forward more slowly with all of them."
- Dr. Fernando Vieira, ALS TDI Chief Executive Officer and Chief Scientific Officer
To the ALS Community,
Thanks to your support, the recent progress in our lab has resulted in two very promising discoveries – both of which could lead to the development of new ALS drugs for clinical testing.
The problem is that right now, our current budget cannot support rapidly advancing both of these programs. We know you need treatments as quickly as possible, but we could soon face a hard choice. Will you donate today to help us advance both of these promising discoveries as quickly as possible?
Help Move These Discoveries Forward Quickly
Our data are compelling enough to justify the immediate aggressive investment to move each of these discoveries forward:
- Discovery of a Series of Protective Drug-like Molecules: For the first time at the ALS Therapy Development Institute (ALS TDI), our scientists have been able to apply human induced pluripotent stem (iPS) cells to discover a related collection of drug-like molecules that are protective against cell death in both iPS cells and in the leading animal model of ALS.
- Discovery of a New Drug Target – Type I PRMTs: Our scientists have explored how C9ORF72 mutations, the most common known cause of ALS, cause neurons to die. That dedicated effort revealed that inhibiting Type I PRMTS, a family of cellular enzymes, might be protective against C9ORF72 ALS progression – and there is reason to believe that the target may be relevant for other forms of ALS.
Along with needing to fund the advancement of these discoveries, we must also maintain funding of our earlier-stage drug discovery efforts, because we know that more treatments will be needed to meet the needs of everyone with ALS.
Without additional funding, we may have to make a difficult decision: aggressively move forward with only one of these discoveries, or move forward more slowly with all of them.
Lately, I have been talking with many community members about drugs that are in clinical trials. We discuss the hard truths – not all treatments advanced into clinical testing succeed, and even those that do succeed will not be effective for everyone.
Research at ALS TDI must continue in order to identify more treatments. With your continued support, we will not stop until we develop treatments for everyone with ALS, but your action is needed now. If you are able, please send a gift today to ensure that we can rapidly move forward with these promising discoveries and get us closer to finding effective treatments.
Fernando Vieira, MD
Chief Executive Officer and Chief Scientific Officer
To donate, click here.