First European HD patients has been dosed with Uniqure’s gene therapy 

First week of February another exciting step in HD research was taken, as the first two European patients were given Uniqure’s gene therapy in the ongoing Phase I/II trial.  The drug is named AMT-130.   Uniqure started the trial in the United States last year and is happy to be able to dose the first European participants as it has taken some time to identify the right participants and prepare the brain surgery this treatment requires. 

The surgery was done at The Interventional Neurotherapy Center (INC) at Mazowiecki Szpital Bródnowski Hospital in Warsaw.  This is a highly specialized hospital and currently the only place where they can perform brain surgery assisted by MRI.  This means that the doctors during the operation can follow the brain surgery with advanced imaging.  A great advantage, as this is the first trial where HD patients receives gene therapy administered directly into the brain.  

Since 1995, our center at the Institute of Psychiatry and Neurology (IPiN) has offered genetic testing and clinical care to Polish patients with Huntington’s disease,” said Dr. Grzegorz Witkowski M.D. Ph.D., IPiN principal investigator. “Our patients have been very interested in the potential for a one-time treatment to stop progression of the disease and, given the recent setbacks in Huntington’s disease research it means a lot to the Polish HD Community to be able to enroll the first patients in this first EU gene therapy trial.

The European Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease will explore the safety, proof of concept, and dosing in 15 total patients with early manifest Huntington’s disease.   Five of the 15 will receive a low-dose, followed by a group of nine patient that will be given a higher-dose . All patients will be dosed with AMT-130. 

Participants will be recruited from clinics in Poland, UK and Germany.  AMT-130 is only administered once.  The study consists of an initial 6-month post-treatment study period followed by long-term follow-up for five years.

You can read more about Uniqure’s program in Huntington’s disease and the AMT-130 trial here.