cureCADASIL and a team of world-renowned researchers have been awarded a $1M grant for their collaboration to drive progress for the CADASIL Community as part of the Chan Zuckerberg Initiative’s Patient-Partnered Collaborations for Rare Neurodegenerative Disease (CZI PPC) – a program that supports work by patient communities and scientists to accelerate research in the fight against rare diseases.
The CZI PPC award supports research centered on science that will close critical knowledge gaps and build a strong foundation for translational efforts, as well as engagement and communications activities to involve and inform the patient community.
CZI PPC-funded Patient Engagement Efforts
The $200K in funds awarded to cureCADASIL over the next 2 years will help strengthen patient engagement, drive enhanced educational and communications activities, and support the research team in their effort to make breakthroughs that will benefit the CADASIL community. Below is progress made toward our funded milestones.
A major milestone of the CZI PPC grant is the establishment of a Patient Advisory Board (PAB)—a dedicated group of individuals with direct experience with CADASIL who will provide invaluable insights into patient and caregiver perspectives, needs, and aspirations. The PAB will play a vital role in guiding our research team toward discovery and providing feedback to cureCADASIL on its patient engagement activities and associated materials. We extend our heartfelt gratitude to all our 2023 PAB members for their participation and thoughtful contributions.
Patient Advisory Board
Debarag Banerjee, PhD
Patient Advisory Board Chair
Ki Coale
Andria Burroso
Sheila Connor
Michael Kennedy
Sarah Vadnais-McDaniels, PhD
CZI PPC-funded Research Efforts
The coordinating research PI, Fanny Elahi, MD, PhD, Assistant Professor of Neurology, Neuroscience, and Pathology and Molecular and Cell-Based Medicine at the Icahn School of Medicine at Mount Sinai, will receive $800K over the next 2 years. Dr. Elahi will lead a multi-disciplinary team to build cellular models of CADASIL to reverse engineer the disease and discover new therapeutic targets. They will employ two complementary models of human brain vasculature created from patient samples to understand CADASIL’s early instigating events and disease progression. We are excited to share that tremendous progress has already been made and work is already underway in completing the Year 1 (2023) objectives, as shown in the Research Progress Tracker below.
Get Involved
In the CZI PPC’s unique funding program, CADASIL patients can participate in research at the bench rather than only in the clinic, making us partners in this truly translational work. Dr. Elahi and cureCADASIL need your help to complete the Year 1 goals and acquire materials to complete the Year 2-4+ milestones. What we need: blood samples and other standard assessments (brief medical exam, brain imaging, and memory tests) from individuals living with CADASIL and from healthy volunteers (family members, friends). We deeply appreciate your consideration and thank everyone who has already volunteered!
MEET THE TEAMS
cureCADASIL Team
Bertram Kasiske
cureCADASIL President
Jane Gunther
(Patient Organization PI)
cureCADASIL Research Director
Pedro De Lencastre
cureCADASIL Secretary
Debra Robinson
cureCADASIL Treasurer
Sandra Talbird
cureCADASIL Trustee
Stellate
Communications &
Patient Engagement Partner
Research Team
Fanny Elahi, MD, PhD
(Contact PI) ISMMS
Joel Blanchard, PhD
(co-PI) ISMMS
Towfique Raj, PhD
(co-PI) ISMMS
Shrike Zhang, PhD
(co-PI) BWH-HMS
One opportunity for the CADASIL community to learn more about these research efforts is our Patient-Investigator Meeting happening on June 24, 2023, as part of the United Leukodystrophy Foundation Family Conference. Representatives from both the CZI PPC Research Team and cureCADASIL will be in attendance to answer your questions and tell you about how you can get involved. Learn more and register for the conference below.