On March 26, the FDA published “The Voice of the Patient” (FDA’s) based on responses from over 200 HD patient advocates who attended the Patient-Focused Drug Development Initiative. Board member Daniel Medina and I attended that meeting in Washington D.C. This was a historical moment for HD families, and it is our hope that our voice had an impact on putting HD clinical trials on fast-track. We made a passionate appeal to the FDA panel to include HD in the fast-track clinical trials category, just as they have done for other devastating diseases. Here is the link to the report. http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM491603.pdf
April 27, we held our first HD-CARE Symposium with guest speaker Dr. Ray Dorsey, of the University of Rochester, New York. Also presenting were Dr. Leslie Thompson, Dr. Neil Hermanowicz, Dr. Peter Donovan, and representatives from CIRM and Americans for Cures. We honored patient advocate Julie Rosling with the HD-CARE Patient Advocate Award for her many years of being a voice for the HD community. We had maximum attendance and the program was truly information and inspirational for all stakeholders.
May 1st, we participated in the OC Marathon for the fourth year. Thank you, Karen Thorburn, for chairing this very successful event.
June, we attended the International Society of Stem Cell Research Conference in San Francisco. ISSCR is an independent nonprofit organization and the voice of the stem cell research community.
July, HD-CARE honored the Thompson HD Bioinformatics Team with the first Everest Award for their work ethic, bringing to mind the incredibly difficult ascent of Mt. Everest. The bioinformatics Team routinely masters concepts, methods and systems that have arduously steep learning curves, hence the name of the award, the HD-CARE Everest Award.
August, we presented at the CIRM SPARK Program summer program in Berkeley.
Also in August, we honored Griselda Barbosa with the HD-CARE Champion Award for her leadership in advocating for Huntington’s disease families.
September, we held a Patient Advocate presentation at Chapman University for Dr. Milton Greenburg’s pre-med students.
October, UCI HD-CARE members Frances Saldaña, and Daniel Medina, and UCI HD research presenter Dr. Jack Reidlin presented at an HD Symposium hosted by the Biology Club and the Pan African Stem Society at California State University, San Bernardino..
December, HD-CARE attended the World Stem Cell Summit in Palm Beach, Florida as Ambassadors for Americans for Cures. One of the highlights of the Summit were the HD research presentations by Dr. Jan Nolta of UC Davis and Dr. Leslie Thompson of UC Irvine. Frances Saldaña hosted the “Huntington’s Disease Experts Round Table” with participation from Dr. Leslie Thompson, Dr. Sidney Golub, Americans for Cures Ambassador Selena Gallardo, Daniel Medina, David Saldaña, David C. Levinger, Chief Executive Officer, Aura Capital Partners Asutosh Yagnik, authors of the 21st Century Cures Act.
Also in December, the HD-CARE Board of Directors hosted a mixer to thank all the players in the Thompson HD lab for their commitment to finding a treatment for HD. Dr. Frank LaFerla, the Co-Director of UCI MIND that houses HD-CARE attended and provided remarks. Dr. LaFerla is also the Dean at the Hana and Francisco J. Ayala School of Biological Sciences, Chancellor’s Professor and former Chair of the Department of Neurobiology and Behavior.
LEEP: Collaboration between patient advocate organizations is exemplified by the success of the HD Law Enforcement Educational Program (LEEP), which was initiated by HD-CARE in in 2014 in an effort to educate police officers about Huntington’s Disease. Collaborating in this initiative along with HD-CARE were Help4HD, HD Reach, HD Drug Works, and HD Lighthouse. Thank you, Help4HD for implementing the program and taking it nationally.