HSG 2015 Round-Up: Community

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Community: HSG’s Strategic Initiatives, Clinical Research, and HD Families

HSG 2015 was about community – the HSG member community, the sponsor community, and perhaps most importantly, the HD family community. Huntington Study Group has set a strategic initiative to be an invaluable research partner to these three communities. For Members, HSG will identify and develop the next generation of investigators and develop the existing human capital. For sponsors, HSG will expand the capabilities of the HSG research network and improve the efficiency of conducting clinical trials. For families, HSG will investigate new therapies for HD, expand the pipeline of therapies in development, and investigate, communicate, and educate about HD research and care.

Many local HD families had the opportunity to join the HSG 2015 attendee’s Saturday’s Clinical Research Symposium which kicked off with a lesson “Life Hacks: Dealing with Huntington’s Disease” by Erika Bjorklund Pope. On the list of life hacks:

  • Accept what cannot be changed
  • Laugh
  • Find inspiration in others
  • Focus on your own breath
  • Don’t waste your energy Accept strength regardless of the source Stay optimistic

Mesenchymal Stem Cells (MSCs) were presented as possible option for treatment of HD because of their neuroprotective factors. Kyle Fink’s presentation on UC Davis research featured MSCs keeping the degenerating cells in the brain alive longer to slow progression and modulating the microenvironment of the diseased brain. MSCs act as transplant cells capable of turning into neurons into the damaged area. The stem cells must be acceptable to the patients’ immune system and make functional connections with the host cells.

Dr. Juan Sanchez-Ramos spoke about the promises, obstacles, and solutions that could come about from the use of nanoparticles in gene silencing. Studies show that nanoparticles can get into the brain and be distributed, and delivering a meaningful, functional payload. Toxicity in nanoparticles can be controlled, and nanoparticles can be delivered for a meaningful length of time.

Identification of genetic modifiers that contribute to the variation of HD symptoms was the focus on research presented by Dr. Jong-Min Lee. The research identified two genome-wide significant loci harboring modifiers of age at onset of motor signs. Work will continue on identifying the functional variant and gene responsible for the modifier effect at from many candidates at each chromosomal level. The discovery of genetic modifiers and delineation of underlying mechanisms will assist in the development of therapeutics based on target processes that are already validated to be disease modifying in patients with HD.

MC2_8747Results from the 2CARE trial were shared by Dr. Andrew McGarry. This multicenter, randomized, placebo-controlled study was designed to test the hypothesis that high-dosage CoQ would slow the progressive functional decline of patients with early stage HD. Statistically significant differences were not seen between treatment groups in the primary or secondary outcome measures, and the data do not justify the use of CoQ as a disease modifying treatment in HD.

Claudia Testa presented on the First-HD and ARC-HD trials focusing on the safety, efficacy, and tolerability of SD-809. First-HD focused on treatment of motor symptoms exclusively with SD-809, a deuterated form of tetrabenzine. Deuterium is a nontoxic, naturally occurring form of hydrogen that when substituted at key positions on a molecule, can alter drug metabolism and pharmacokinetics. Conclusions from First-HD indicate that SD-809 effectively reduced chorea with an impressive safety and tolerability profile. Indications include effective treatment with good tolerability and overall benefit to patients with HD. The objective of ARC-HD was to evaluate the safety and efficacy of switching patients from stable does of tetrabenazine directly to SD-809. Conclusions from ARC-HD indicate that patients with HD can safely and rapidly convert from tetrabenazine to open label SD-809. There was continued control of chorea with a possibility of improving chorea control based on minimal adverse affects.

The HSG Family Day, a hallmark of the HSG meeting experience was facilitated by the Huntington’s Disease Society of America Central Florida affiliate in conjunction with the University of South Florida HDSA Center of Excellence.  Caregiver education specialist Jimmy Pollard led HD families in an engaging and lively discussion on the essential need for routines in daily HD caregiving. HD families also learned about the special safety issues in the home and strategies for addressing those safety issues. Laura Moore guided families through techniques to cope and manage with loss.

HSG 016Save the Date!

Join HSG in the Music City in 2016! The Tenth Annual HSG meeting will take place November 3-5, 2016 at Opryland USA in Nashville, Tennessee.