The ALSETF is about bringing treatments in late development (post-Phase 2) to ALS patients. Right now there is no hope for the majority of living patients because over 50% do not qualify for clinical trials. For a newly diagnosed patient, the odds of living to see a drug approved which is starting trials at the same time is about 10% (actually much less considering the historical approval rate of ALS treatments). However, with recent advances into the nature of ALS, certain drugs have been developed which show real promise for treating at least a subset of PALS. More are planned to enter trials in the United States very soon. We at ALSETF mean to get that hope ASAP to PALS who are currently living.
ALSETF is a 501C3 non-profit organization with the mission to partner with government and industry agencies to enable Expanded Access Programs. Expanded Access Programs (EAPs) are FDA authorized programs that permit the use of yet-unapproved drugs under medical supervision, in specific cases where those drugs are in late stages of development and have shown preliminary evidence of safety and efficacy. EAPs are only for immediately life threatening conditions for which no effective approved therapies exist. We have open communication with the FDA’s Office of Neurology products for guidance on EAPs involving investigational drugs for ALS. We also maintain open discussion with clinical leaders on the best practices for EAPs, as well as with certain pharmaceutical companies with drugs in trial and in the pipeline.
Our focus right now is to raise up to $5M to help fund certain costs of an EAP such as upgrading manufacturing to clinical-grade, production quantity necessary for fulfilling EAP demand, etc. These are all issues which can currently prevent a pharmaceutical company (especially the small start-ups likely to take a chance on ALS) from accommodating a large EAP. We believe the financial issues can be solved and that the drugs being talked about with excitement in the ALS community can be brought to those who don’t qualify for trials now, while they are still living.