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First ALS Patient Receives Isis-SOD1_RX Antisense Treatment in Phase 1 Clinical Trial

Isis Pharmaceuticals, Inc. has initiated a Phase 1 study of ISIS-SOD1_Rx in patients with an inherited, aggressive form of Lou Gehrig’s disease also known as familial amyotrophic lateral sclerosis (ALS).  Approximately 20 percent of all familial ALS cases are caused by a mutant form of superoxide dismutase, or SOD1.  The ALS Association and the Muscular Dystrophy Association are providing funding for the development of ISIS-SOD1_Rx.

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First U.S. Stem Cells
Transplanted Into Spinal Cord

ALS Association Chief Scientist Lucie Bruijn, Ph.D., was interviewed by CNN regarding Thursday’s announcement about the first ALS patient to receive transplanted stem cells.

Dr. Bruijn was quoted as saying, “This is the first study to see if the invasive injection into the spinal cord is safe for the patient.”

She went on to say, “Our biggest hopes for stem cells is to significantly slow the progression of the disease.”

To read the full story, click here.

New “Antisense” Experimental Therapy
Clinical Trial Announced

A new experimental therapy using an approach known as antisense, in which a drug is designed to shut down the RNA (Ribonucleic acid) that is responsible for the production of disease-causing proteins, is being prepared for a clinical trial in people with a familial form of ALS later this year. The clinical trial follows research funded by The ALS Association through TREAT ALS (Translational Research Advancing Therapy for ALS), our research pipeline that funds and facilitates the development of treatments for ALS based on important laboratory findings.

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