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HD InsightsMeet the Funder

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NAME: Helen Budworth, DPhil

TITLE: Senior Science Officer, California Institute for Regenerative Medicine

EDUCATION: BSc, Genetics and Microbiology, University of Liverpool; DPhil, Biochemistry, University of Oxford, UK

HOBBIES: Enjoys spending time outside on the beach and in the mountains

Dr. Budworth, recognized by HD Insights last year as a “Next Generation” HD Scholar, has recently become a Senior Science Officer at the California Institute for Regenerative Medicine (CIRM). She sat down with HD Insights to describe her own HD research and funding opportunities available through CIRM. The following is an edited transcript of the conversation.

HD INSIGHTS: Tell us about your scientific interest in HD.

BUDWORTH: I was a researcher at Lawrence Berkeley National Lab doing basic and translational research into the molecular underpinnings of HD, and working toward therapies that could ameliorate this condition.

HD INSIGHTS: What did you find through your research?

BUDWORTH: We were looking at the DNA repair mechanisms that act on the CAG repeat in HD, and investigating the role of oxidative DNA damage in initiating the CAG repeat expansion. Based on this research, we were investigating antioxidant therapies to counteract the effect of that oxidative DNA damage.

HD INSIGHTS: I noticed that you were one of the first Huntington’s Disease Society of America (HDSA) Human Biology Fellowship Award winners. Could you tell us about that experience?

BUDWORTH: I was funded in 2013 by HDSA to look at metabolic biomarkers in the blood of HD patients. Since blood is a very easily accessible bodily tissue with which to study progression of disease, we were looking to find not only things that could be detected in the blood, but that could be tracked with disease progression. We used a combination of molecular biomarkers, looking at gene expression levels through RNA, and looking at metabolites in the blood. We found distinct differences between the blood of HD patients and healthy controls. We showed that changes in fatty acid metabolism and the genes that regulate it were one of the signs of the disease.

HD INSIGHTS: You recently transitioned from Berkeley Lab to join the California Institute for Regenerative Medicine (CIRM). What prompted the transition?

Messages Image(529133913)BUDWORTH: CIRM is the California Stem Cell Agency, and they are working to accelerate stem cell treatments to patients with unmet medical needs. The mission of CIRM was something that really gelled with my own scientific mission and career direction. With the scientific office and the therapeutics team at CIRM, I am now able to contribute to a much broader range of projects, and much more translational and clinical projects, aimed at bringing results of scientific discoveries to the patients who need them. CIRM was funded by Proposition 71, which was passed in 2004 in California, with the goal of developing stem cell treatments and cell therapies for conditions that do not have other effective treatments. We aim to meet unmet medical needs using cell-based therapies. CIRM is looking to fund the most promising research projects at all levels, from discovery through to clinical-stage projects, to bring the science to patients.

HD INSIGHTS: What is CIRM’s interest in HD?

BUDWORTH: The neurodegenerative nature of the disease is one that stem cell researchers have been interested in for a number of years. CIRM has funded a variety of HD projects over the years, and we are looking to continue this. We want to enable cell-based therapies to progress through clinical trials then into the clinic, actually helping patients with HD. To date we have funded projects in the early stages, translational projects in HD, and some early stage clinical trials. Details of these can be found on the CIRM website (www.cirm.ca.gov). We know the history of HD, and understand the importance of having a new promising therapy come through into the clinics, so this is one of our priorities. We are hoping that stem cell therapy will be a real benefit.

HD INSIGHTS: Tell us about CIRM version 2.0, and how researchers who have been interested in stem cells can become engaged with CIRM.

BUDWORTH: CIRM 2.0 is a change in the way that we do business, aimed at introducing faster and more efficient systems for funding. This is part of our effort to accelerate the development and delivery of therapies for people who need them. We now have continuous funding cycles in the clinical stage, where each month, clinical applications are accepted and can be funded within a matter of months, something that used to take a lot longer. We have really streamlined our process for clinical stage awards. We also have translational awards that are offered every six months, and we also maintain our discovery program with some of those programs every six months and every year. CIRM 2.0 is intended to push and partner our projects to get them through what they need to do to bring therapies to patients.

HD INSIGHTS: I understand that CIRM is interested in seeing clinical trials of promising stem cell therapies for HD and other neurodegenerative conditions. Who is eligible to apply for research funding from CIRM?

BUDWORTH: We accept applications from researchers and from companies, any promising projects with stem cell therapies that are moving through translational and clinical phases. We have local requirements in that California-based organizations can use CIRM funds for all eligible project costs. Non- California-based organizations are also eligible to apply, but in those cases, the CIRM funding can be used for expenditures incurred within California. For example, in the case of a clinical trial, a clinical trial site within California would be funded for that work. More details about eligibility can be found on the CIRM website. Funding is based on the project, the promise of the stem cell therapy, and the unmet medical need. The CIRM website details all our funding programs. It also details projects we have funded to date, along with projects we are currently funding. The database can be searched by disease, so you can look up what we are doing in HD. When an application is submitted it will go through review, and successful applications will be managed and helped by the science office to assist the applicants to succeed.

HD INSIGHTS: Thank you and CIRM very much for your investment, and trying to identify new therapies for a wide range of conditions including HD.

About HD Insights

Our mission is to promote, disseminate, and facilitate research on Huntington’s disease. To fulfill this mission, we are guided by an outstanding editorial board that includes representatives from three continents, academia, industry, and the HD community.