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Dear ALS Community,
Earlier this month, at the International Symposium on ALS/MND, I watched a presentation from AL-S Pharma announcing that AP-101—a therapeutic antibody targeting misfolded SOD1—had succeeded in Phase 2b and is now advancing toward Phase 3 clinical trials.
Our team at ALS TDI helped make that molecule what it is.
Years ago, we partnered with a European biotech called Neurimmune and executed key parts of the preclinical optimization work that shaped AP-101 into something ready for human trials. It was painstaking, unglamorous science—the kind that rarely makes it into newsletters.
SOD1 mutations cause some of the most aggressive forms of familial ALS. They ravaged my late best friend, Sean Scott, and generations of his family. When our scientists were running those experiments, we had Sean’s family and families like his at top of mind. We held hope that the approach could benefit people with sporadic ALS, as well. AL-S Pharma maintains that hope and intends to advance AP101 into human trials in both SOD1 familial ALS and in sporadic ALS.
We don’t know if AP-101 will succeed in its Phase 3 development just like we couldn't know when we began collaborating with Neurimmune whether this program would ever advance this far. We only knew it was worth trying.
Here's what I want you to understand: if you gave to ALS TDI between 2013 and 2018, you helped fund this work. Not because we asked you to support a specific antibody program - we rarely mentioned it - but because your generosity gave us the flexibility to pursue promising science wherever it led. That is the power of the support you provide.
Progress in research is nonlinear. We cannot predict which programs will break through or when. But we can build a pipeline worth believing in, and right now, ours includes another lead program called TDI-1831 that may help people with rare ALS-causing mutations, and a versatile platform for delivering precision mRNA therapeutics directly to the brain and spinal cord.
I don't know which of these will advance to Phase 3 clinical development or beyond. But I know the work is worth trying. And I know it's only possible because of you.
If you're able to make a gift this year, it will go directly toward the science that could one day reach someone's best friend, someone's parent, someone's child.
Make a year-end gift
With deep gratitude,
Fernando Vieira, M.D.
CEO & Chief Scientific Officer
ALS Therapy Development Institute