12 Sep

Update on a IONIS Phase I study in Huntington’s Disease
The purpose of this study is to investigate the safety, tolerability and activity of the investigational drug IONIS-HTTRx in patients with early stage Huntington’s Disease.
Huntington’s Disease (HD) is caused by a mutation in the huntingtin gene. A portion of the gene is expanded and subsequently produces an expanded huntingtin protein. Mutant huntingtin interferes with cell processes and disrupts the overall function of the cell to cause the symptoms of the disease.
The new drug manufactured by IONIS pharmaceuticals, IONIS-HTTRx blocks the production of the huntingtin protein by binding to huntingtin RNA, telling the cell to destroy it. It is hoped that reducing the levels of the mutant protein in this way will slow or halt disease progression. Pre-clinical studies with IONIS-HTTRx in animal models have been promising and the purpose of this trial is to test the safety of the drug in humans.

The drug is delivered by injection into the lower back, known as an intrathecal injection (a form of ‘lumbar puncture’). Treatment by this method will mean that the antisense drug is delivered directly to the cells of the brain that are affected by the expression of the huntingtin protein.
The trial is “blinded” which means the patient and the trial doctor will not know whether the patient is given IONIS-HTTRx or placebo. The placebo is an injection that does not contain any active drug. Each patient will receive four doses of IONIS-HTTRx or placebo by an intrathecal injection, with doses four weeks apart. After the last dose, patients will be followed for several weeks to monitor the safety and activity of IONIS-HTTRx.
The study period includes a 13-week period during which they receive the study drug, followed by a 15-week observational period. More information contact Lucy Collins on 01223 331160

We would like to thank everyone for their interest in the trial and for patients volunteering. The trial is going well and as of today there are no safety concerns.