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First ALS Patient Receives Isis-SOD1RX Antisense Treatment in Phase 1 Clinical Trial Isis Pharmaceuticals, Inc. has initiated a Phase 1 study of ISIS-SOD1Rx 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-SOD1Rx. ISIS-SOD1Rx is an antisense drug designed to inhibit the production of SOD1.  Antisense techniques are used to deactivate disease-causing or undesirable genes so that they cannot produce harmful or unwanted proteins. Read full story >>
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."   Read full story >>
Additional $7.5 Million for the ALS Research Program This weekend, the U.S. Senate appropriated an additional $7.5 million for the ALS Research Program at the Department of Defense (DOD), a 50% increase over last year!  The funding is in addition to the $6 million (20% increase) Congress appropriated for the National ALS Registry last week and also comes as Congress added another $2.5 million to support the Lifespan Respite Care Act, which helps to fund respite care programs in the states.   We would like to thank everyone who contacted their Members of Congress throughout the year in support of the ALSRP.  Your efforts led Congress to significantly increase its commitment to the ALSRP, something that was not done for most other disease research programs.  The legislation now heads to the President, who is expected to sign it into law. Read full story >>