This week, we announced the winners of the 2019 HD Human Biology Fellowship. These young scientists were chosen for their well-designed projects that focus on understanding Huntington’s disease as it occurs in humans. Below are their names and the basic questions they will focus on in their HD research. 

  • Danielle Larson, MD, Northwestern University Feinberg School of Medicine: Could remote “telemedicine” visits with a neurologist be just as helpful as in-person visits for an HD patient?  
  • Osama Al Dalahmah, MD, PhD, Columbia University: How do astrocytes, the brain’s support cells, change over the course of HD to become harmful versus helpful? 
  • Vilija Lomeikaite, PhD candidate, University of Glasgow: Can we improve the methods to detect an increase in CAG repeats in certain cells of the body and brain as HD progresses? 
  • Ricardo Mouro-Pinto, PhD, Massachusetts General Hospital and Harvard Medical School: Can we develop drugs to stop CAG repeats from expanding further in HD?
  • Saul Martinez-Horta, MsC, Sant Pau Hospital, Barcelona: When HD patients begin to have difficulty thinking, what kinds of changes are happening in the brain and blood?

A summary of these five proposals can be found here. We look forward to hearing about the progress of these exciting research projects. 

Community Statement from Roche/Genentech

This week, Roche/Genentech announced in a worldwide community statement that they will be expanding the number of participants in the GENERATION-HD1 trial of huntingtin-lowering drug RG6042 from 660 to 801. The main goals of this change are to make the study larger, stronger, and more inclusive. The additional participants will be recruited from outside of the United States, including a new site in China. All sites in the US have finished recruiting, and with extraordinary speed, which is great news for the progress of the trial.

Resources on HD from the ASGCT

The American Society of Gene and Cell Therapy has recently created a set of resources around Huntington’s disease, including a short video explaining genetic therapies that are currently in clinical trials or will be moving to clinical trials soon. It’s a good way to visualize and better understand the ASO drugs that are being developed by Roche/Genentech and Wave Life Sciences, as well as the viral gene therapies that will be tested by UniQURE and other companies.