Anti-CD40 Ligand Therapy for Slowing Progression and Extending Life in Amyotrophic Lateral Sclerosis
Abstract
ALS Therapy Development Institute (ALS TDI) discovered that subtly modulating the immune system with an antibody targeting a protein called CD40 Ligand (CD40L) is protective in an animal model where mice develop amyotrophic lateral sclerosis (ALS). Disease progression in these "ALS mice" treated with the antibody was slower and the mice lived longer than usual. ALS TDI then discovered that the elements of the immune system that are targeted with this antibody are also disrupted in people living with ALS. Because of these discoveries, ALS TDI decided to build a new antibody drug, AT1501, that would target CD40L in humans. This was not simple because antibody drugs that have targeted CD40L in humans in the past were toxic. They caused platelets to clot in blood, which can be fatal. Here, ALS TDI presents AT1501, an anti-CD40LG antibody that is designed not to cause platelets to clot. This proposal is to fund work that would prove in non-human primates that AT1051 is safe enough to move into human Phase I ALS clinical trials. Successful completion of the work plan would enable the filing of paperwork with the Food and Drug Administration (FDA) necessary for initial human ALS clinical trials of AT1501. We hypothesize that AT1501 will be able to slow ALS disease progression and extend lives in people living with the disease.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Aug 07, 2017
- Source ID
- W81XWH1710057
Entities
People
- Steve Perrin
Organizations
- ALS Therapy Development Institute
- United States Army