The CHDI Foundation is a single-disease funder that puts all of its support into accelerating therapeutic development for Huntington’s disease. HD is a fatal genetic disease that lacks treatments or a cure, but CHDI is working to change that.
Let’s take a closer look at the types of projects CHDI has been supporting and collaborating on lately.
Promising New Drugs
CHDI recently supported research for two experimental drugs that reduce inflammation associated with the disease and one drug that blocks the underlying cause. CHDI was joined by the National Institutes of Health and the Heredity Disease Foundation in this funding support.
Biomarkers for Advancing Research
Earlier this year, CHDI also supported Singulex, Inc. to use its Erenna Immunoassay System to develop biomarkers to further research for Huntington's disease.
"Quantifying disease-specific proteins in cerebrospinal fluid has been critical to the study and treatment of other neurodegenerative disorders, but until now mutant huntingtin protein has never been measured in CSF since it is present at such very low levels," said Douglas Macdonald, PhD, Director of Drug Discovery and Development at CHDI. "With this new assay we hope that mutant huntingtin will prove to be a useful biomarker for the upcoming trials of huntingtin-lowering therapeutics."
Genetics determines the onset and progression of Huntington’s disease, so much of CHDI’s funding goes toward gene study. For example, the foundation recently supported University of Florida Health researchers, who discovered that “the gene that causes the fatal disorder makes an unexpected ‘cocktail’ of mutant proteins that accumulate in the brain.”
CHDI also supported a clinical trial to test the safety of an experimental drug called ISIS-HTTRx. According to a press release, this was the first drug tested in patients that “targets the known cause of the disease: a toxic protein called mutant huntingtin, which slowly damages and kills neurons, leading to the progressive and ultimately fatal decline in mental and physical abilities that is the devastating hallmark of Huntington's disease.”
CHDI makes agreements with academic institutions to support the work of faculty members, post-doctoral fellows and technical staff. On a case-by-case basis, the foundation also considers support for graduate students. Here’s a quick overview of what the CHDI Foundation does:
- Funds and works with academic HD researchers at universities around the world.
- Staff scientists manage CHDI internal drug programs through collaborations with contract research organizations.
- Forms partnerships with biotech and pharmaceutical companies to develop potential drugs.
- Acts as a ‘collaborative enabler’ for any research group that wants to work on HD by making resources (reagents, antibodies, mouse models, clinical samples, etc.) freely available to the HD research community.
- Builds clinical capacity so that as soon as promising potential drugs are ready, CHDI and the wider HD research field can quickly progress to clinical trials.
Inquiries about proposals for clinical studies should be directed to CHDIClinicalQuestion@CHDIFoundation.org. Researchers, institutions or universities that have an idea for a preclinical research proposal should contact the most appropriate CHDI science director.
To learn more about this funder’s disease support, check out IP’s full profile, CHDI Foundation: Grants for Disease.