Ionis Study: mutated Huntingtin was lowered successfully in CSF of Huntington patients

The HD community learns that a phase I/II study of Ionis Pharmaceuticals showed a dose-dependent reduction of CSF mutated huntingtin (mHTT) concentrations after repetitive lumbar injection of oligosense-nucelotides in HD patients. This is an important step on the way to a future disease modifying therapy of HD. Whether this approach will results in clinical benefit and possibly slow the progress of HD will be investigated in an upcoming phase III clinical trial. While the results communicated today do not mean a cure for HD they are a hugh and very important milestone on the way to developing a successful therapy in HD.

Congratualtions to Frank Bennett and team at Ionis Pharmaceuticals for making this very promissing approach available to the HD community and to Sarah Tabrizi and the team of clinical investigators that successfully completed this important clinical trial!

Ralf Reilmann, MD, PhD, FAAN

Press-Release of Inois Pharmaceuticals - click here

Video message of EHA about first news - click here