The Laura's Hope Fund was established to honour Laura Evans, who passed away from Huntington Disease at a young age. The goal of this Fund is to advance research in Canada and internationally to accelerate the development and testing of potential new treatments for Huntington Disease.  In recent years this Fund has worked with the Huntington Study Group, headquartered in Rochester, New York, to fund original studies to test novel therapeutic agents in individuals with Huntington disease and to cultivate new investigators for future Huntington Study Group clinical trials.  

UDCA/TUDCA-HD

For example, a proof-of-concept study lead by Dr. Penelope-Hogarth of Oregon Health & Science University in Portland, Oregon is underway with regard to Ursodiol/Tauroursodeoxycholic acid  (UDCA/TUDCA-HD). Tauroursodeoxycholic acid (TUDCA) is a bile acid synthesized in the liver by the conjugation of taurine to ursodeoxycholic acid (UDCA).  It is thought to function as an anti-apoptotic agent in HD, evidenced by studies in toxic cell models and both toxic and transgenic rodent models of the disease.  Ursodiol is a commercially-available exogenous form of UDCA, the precursor of TUDCA, and it has been targeted as one of several high-priority drugs for development by the SET-HD initiative (Systematic Evaluation of Treatments in HD), a collaborative endeavor by the NIH and the Huntington’s research and lay communities. As a drug approved by the FDA, available in an oral formulation, and with a compelling scientific rationale for its use in HD, the drug seems a natural choice for study.

UDCA / TUDCA-HD has shown promise in toxic and transgenic cellular and animal models of HD and has an established dosing, safety, tolerability and efficacy profile in humans with hepatobiliary disorders. However, important gaps exist in the understanding of the pharmacokinetics / pharmacodynamics of the compound, particularly in patients with normal gastrointestinal function, and no human data exists for its therapeutic use in neurodegenerative disorders.  Thus developing the methodology to assess the CSF penetration of the compound, obtaining a preliminary safety and tolerability profile for the drug at standard doses in HD subjects, and determining the relationship, if any, between the available serum measures of bile acids and CSF concentrations in that dose range will be essential before moving into larger studies.