Potential New Therapeutic Opportunities for CNS Diseases

The results of a recent Phase I trial testing the safety and tolerability of injecting an antisense oligonucleotide into the cerebrospinal fluid of people with ALS was recently published online in Lancet Neurology. The study was led by Dr. Timothy Miller of the Washington University School of Medicine and Dr. Merit Cudkowicz of Massachusetts General Hospital with support from Isis Pharmaceuticals as well as the ALS Association and Muscular Dystrophy Association. The trial found that delivery of an antisense oligonucleotide targeted against SOD1 (ISIS 333611) by intrathecal infusion was safe and well tolerated. This is good news for ALS as well as other CNS diseases. Isis Pharmaceuticals is invested in working with Drs. Miller and Cudkowicz to develop an SOD1 based antisense therapeutic, including improving the antisense oligonucleotide used for future studies. In addition, at the 23rd annual International Symposium on ALS/MND two different groups announced that they are independently working with Isis to develop antisense oligonucleotides therapies targeting C9ORF72. Stay tuned to the ALS Forum e-Newsletter for updates!

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