Carlsbad Doctors Seeks Cause, Treatment of Huntington's Disease, ALS

Dr. Frank Bennett works at Isis Pharmaceuticals in Carlsbad and helping pioneer the development of stem cell and gene silencing therapies for both of Huntington's Disease and ALS which are human neurodegenerative diseases.

Dr. Frank Bennett is the senior vice president of research at Isis Pharmaceuticals. Dr. Bennet has worked for the Carlsbad company for 23 years and is a Carlsbad resident as well. 

Carlsbad Patch asked Dr. Bennet about his job and work with Huntington's Disease and ALS.

Carlsbad Patch: How have you and the Isis team helped further the research for Huntington’s Disease/ALS?

The focus of Isis since its inception has been on novel therapeutic agents that target RNA. We use a technology called antisense oligonucleotides (ASOs) that uses short synthetic nucleic acid analogs designed to bind to a target RNA.  Our preclinical work on Huntington’s disease was performed in conjunction with Dr. Don Cleveland’s lab at UCSD.  In these studies, we evaluated our approach to reducing the production of the protein that causes huntington’s disease.  These data were very promising and provided the foundation for our drug discovery efforts.   We are now conducting the last of our preclinical studies that will allow us to select a drug to move forward in development. 

Carlsbad Patch: What are you and the team hoping to achieve in the next five years in terms of research with those diseases?

Next year, we hope to identify a drug to evaluate in human clinical studies for Huntington’s disease.  Once identified, we will conduct the preclinical studies that support a human clinical trial, which could start within 12 to 18 months after we identify a drug.   We also have an ongoing program for ALS.  We have already conducted a Phase 1 study in patients with a specific mutation leading to ALS.  In this study, we demonstrated that our drug was safe and well tolerated.  Over the next couple of years, we hope to identify a drug to move into development that would be more broadly applicable to ALS patients.  

Carlsbad Patch: Anything else?

Severe and rare diseases are an area where we feel that our technology can produce drugs where other attempts have failed.  We can do this because we work at the RNA versus protein level.  To this end, we have several ongoing programs, including a drug to treat Spinal muscular atrophy (SMA).  SMA is a debilitating disease and the leading genetic cause of infant mortality.  There are no treatments for these children.  We are currently evaluating a drug we discovered in a Phase 2 study in children with SMA.

Dr. Bennet along with Don Cleveland, Holly Kordasiewicz and the research teams at UCSD and Isis Pharmaceuticals were honored Nov. 16 at the Huntington’s Disease Society of America (HDSA) 12th annual Celebration of Hope Gala & Auction at the Manchester Grand Hyatt in downtown San Diego.

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