Last week, Kala Booth, a Huntington’s disease patient, caregiver, advocate, and the 4th known generation in her family to test HD positive appeared before the House Committee on Energy & Commerce subcommittee on neurodegenerative diseases. In her testimony, Kala shared the hardships her family has experienced through misdiagnoses, denial of disability benefits, and more, and laid out a series of requests for congress to pass the HD Parity Act, expand awareness of Huntington’s Disease, and to better account for the unique needs on the HD community in design of clinical trials.  

The committee heard courageous testimony not only from Kala, but also patient and family advocates from the Alzheimer’s and ALS communities seeking treatments, cures, and coverage for patients and families affected by neurological diseases. In anticipation of rapid change and development in therapeutic interventions for these and other terminal conditions, Stat News took a step back to determine what it means for clinical trials to be pro-patient, and the role of patient advocates in bringing about a brighter future for these dire diagnoses. Watch Kala’s testimony here and read the full article here. 

Reliable Resolution of Healthy and Mutant Huntingtin Protein  

Many HD therapies currently in development aim to treat symptoms by lowering levels of huntingtin protein (HTT) in affected individuals. Some of these treatments are selective to mutant huntingtin protein (mHTT), while others lower levels of both mutant and normal huntingtin, called wild-type huntingtin protein (wtHTT). In order to measure the effectiveness of any HTT-lowering drug, it is essential that researchers are able to quantify both wtHTT and mHTT in HD samples. While reliable methods exist to measure mHTT and total HTT in biological samples, wtHTT has posed a greater challenge. A study recently published in the Journal of Huntington’s disease from researchers at the University of British Columbia, including HDSA Berman-Topper fellow, Dr. Nicholas Caron, shared a novel method to directly compare wtHTT and mHTT levels. This technique will allow researchers to better evaluate effectiveness of potential new treatments for HD. 

Australian National HD Conference Recordings 

The Huntington’s Australian National Conference was held from May 25th through June 1st, 2021, in a virtual format. Sessions focused on four broad themes: quality of care, policy outcomes, consumer outcomes, and research. In the research session, Dr. Travis Cruickshank and Clement Loy presented an overview of clinical trials, and Drs. Rachel Harding and Leora Fox focused on how basic research is driving the development of novel therapies for HD. You can view all of the recorded presentations by following this link, clicking the “sign up” button on the right-hand side of the page, then entering some basic info. Make sure to choose the “HD Family” ticket type for free access to all the content.