Dear Project ALS family,

If you’d asked me 26 years ago whether I’d still be working at Project ALS—whether Project ALS would still be chasing down medicine and cures, I’d have told you that you were nuts. I’d have told you that I was nuts.

I mean how long does it take the world’s smartest people to figure out a brain disease? Just yesterday I heard on the radio we can now create AI avatars of our loved ones to chat with…after they die. That’s right: we can pursue virtual relationships with our beloved…post-mortem. Clever, right? Brilliant use of our collective intelligence. Meanwhile, back on the planet earth…life-threatening diseases like ALS? Yes, we’ve made significant progress, but we’re not there yet.

In 1998, when my sister Jenifer was diagnosed with ALS at age 35, doctors told her to max out her credit cards and see paris. She was going to die because ALS was always fatal. That’s when Jenifer and some of us started Project ALS, to recruit the smartest in the world to collaborate…work together toward milestones…share data openly. We wanted researchers to (1) pry open the black box of ALS—to understand how and why the disease happens, and (2) develop medicine for ALS, based on the clues drawn from that black box.

Project ALS was the first non-profit to hew to this business model. We recruited talent to ALS research as if we were a sports franchise. We built a young winner. Project ALS was first to get competitors to work together toward specific goals. Results? We now know of over 50 genes that contribute to ALS…we know of molecular and cellular pathways that we can target rationally and effectively with candidate therapies…Project ALS pioneered the first efforts within stem cell biology to generate patient-derived models of ALS These tiny models—ALS in a dish–are tools now used worldwide for drug discovery.

Aggressive, rationally pursued science is–by its nature–hard…slow…not sexy, and…100% necessary. Yes, 26 years is a long time, but we are moving closer to better medicine.

Project ALS will not stop until its new therapeutic candidates, including prosetin, currently in phase 1 for ALS, reach people living with ALS. Gene modulators known as ASOs, several of which started with Project ALS researchers and funding, are showing real promise. Right now, at the Project ALS therapeutics core, our team is optimizing new ALS compounds for clinical trial. Project ALS is also leveraging AI…to screen drugs faster and with greater accuracy than before.

Project ALS isn’t going anywhere. Rather we’ll go everywhere, until als is slowed and stopped in our lifetimes. I’d say about Project ALS what my sister Jenifer said about life: “it’s not about letting go, but reaching.” 


-Valerie Estess | Director of Research