On Thursday, March 21st, Roche/Genentech released a Community Statement about changes to the GENERATION-HD1 study, the clinical trial testing huntingtin-lowering drug RG6042. Essentially, they have decided to change the protocol to make it less taxing for families and health professionals: participants will have lumbar punctures (spinal taps) to receive the drug every other month instead of every month.

A protocol change to a pivotal drug study is a big deal, and a relatively rare occurrence. This one is very exciting, because it means that Roche/Genentech have reason to believe that less frequent dosing has just as positive an effect on huntingtin levels. This evidence comes from the ongoing “open-label extension” study, where the original participants in the early safety trial have continued to receive drug for at least 9 months. We still don’t know how RG6042 will affect symptoms, but GENERATION-HD1 is designed to test that. The trial will take a brief pause (a few weeks to months) to reorganize, but will move forward as planned, with less stress on participants.

HD Buzz did a fabulous job covering the study changes, so check out their article if you want more details. Gene Veritas also blogged about the news this week.

It’s also worth mentioning that business-driven sites are also reporting on the info – meaning that news around these trials is spreading beyond the HD community and sparking the interest of investors. Take their predictions with a large grain of salt, but wider awareness and more interest from industry is always a good thing for a rare disease field.


Anniversary of Discovering the HD Gene

Let’s not forget that recent advancements in genetically-based therapies for HD would not have been possible without the discovery of the HD gene – which was reported in the journal Cell on March 26th, 1993. It’s a fitting milestone to recognize as clinical trials move forward and more companies with HD gene therapies begin to enter the clinical space.


Software to Evaluate Driving Skills in HD Patients

Australian researchers recently published a study that tested driving skills software called DriveSafe DriveAware in people with HD. It’s an easy evaluation tool using an ipad that helps link cognitive skills with safe driving ability, and it has been used in the elderly and for people with cognitive impairment. DriveSafe DriveAware is not a replacement for a hands-on driving test, but it’s a good way to get a basic prediction on whether a person might need to think about having a driving assessment. The study was conducted in 26 people with different stages of HD, from asymptomatic to symptomatic, and it found that people with more advanced HD were less likely to get a predictive “pass” on the test. This is a potentially useful tool, but the program will require some more tweaking to be truly useful and predictive for Huntington’s disease.