Amyotrophic Lateral Sclerosis (ALS) Treatment, AT-1501, Enters Clinical Trial

Posted February 19, 2019
Steven Perrin, Ph.D., ALS Therapy Development Institute

Steven Perrin, Ph.D., ALS Therapy Development Institute
Dr. Steven Perrin

The Amyotrophic Lateral Sclerosis Research Program (ALSRP) supports innovative and high-impact research into preclinical development of therapeutics for ALS, also known as Lou Gehrig’s Disease, a devastating, neurodegenerative disease.  Unfortunately, current treatment strategies offer only a modest improvement in patient survival and little relief from the debilitating symptoms of ALS.

ALSRP investigator, Dr. Steve Perrin, Chief Executive Officer of the ALS Therapy Development Institute (ALS TDI), had previously utilized ALS mouse models to evaluate treatments that targeted the protein, CD40 Ligand (CD40L).  Dr. Perrin’s group had correctly predicted that blocking CD40L would slow progression of ALS by preventing the activation of a pro-inflammatory immune response.  This finding led to Dr. Perrin’s fiscal year 2016 (FY16) ALSRP Therapeutic Development Award to assess the pharmacokinetics and toxicology of a new ALS antibody therapeutic, AT-1501, designed to block CDL40 activity in non-human primates.  In this pre-clinical trial, AT-1501 successfully prevented the molecular signaling that normally activates an inflammatory response in ALS.  Furthermore, AT-1501 did not cause harmful clotting which had been a problem with other CDL40 blocking drugs.

In response to the successful outcomes of Dr. Perrin’s FY16 award, as well as further development efforts by ALS TDI, the Food and Drug Administration granted Orphan Drug Designation and Investigational New Drug approval of AT-1501 in April 2018.  In addition, Anelexis Therapeutics, LLC has provided funding for a Phase I clinical trial to determine the safety of the AT-1501 as an ALS treatment strategy and to analyze the drug’s pharmacokinetics.  This trial has begun and is enrolling eight individuals with ALS in addition to healthy controls.  According to Dr. Perrin, seeing a lead candidate from his research team reach people with ALS is a good sign that we are moving closer to ensuring patients living with ALS can get the treatment they need.



Public and Technical Abstracts:  Anti-CD40 Ligand Therapy for Slowing Progression and Extending Life in Amyotrophic Lateral Sclerosis

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Last updated Monday, January 3, 2022