2020 Reflections & 2021 Goals
For all its challenges, 2020 was a transformative year in many ways. As our country continues to navigate the current challenges, including and especially the widespread impact of the COVID-19 pandemic, we have been heartened to see an overwhelming response to the CACNA1A Foundation’s work.
We rocketed out of the gate last year: we unveiled our website, www.cacna1a.org, formed a Scientific Advisory Board, launched a Research Grant Program, secured a plan for a natural history study, and started building our Family Advisory Council. We also raised nearly $200,000! We are grateful for the continued support and contributions of our families, friends, doctors, and advisers. For all that we achieved last year, we have our sights set even higher in 2021!
This year, we are focused on continuing to raise awareness about CACNA1A; growing the CACNA1A community and our network of families, doctors, scholars, and advisers; accelerating research; and finding treatment options for this rare disease.
Collaboration For a Cure
The Foundation’s top priority this year is growing its ambitious research grant program. The program is intended to support translational research that will pave the way to clinical trials and ultimately find treatments and a cure for CACNA1A. With input from the Foundation’s Scientific Advisory Board, we have broken down our research agenda into five actionable steps.
Step 1. Find Patients – By raising awareness and advocating for our disease, we will build and strengthen our community.
Step 2. Natural History Study – The Chung Lab at Columbia University will conduct a robust natural history study to identify research needs and inform drug development.
Step 3. Build a BioBank – We will see a method of collecting patient samples that can be used to create animal models and iSPC lines with diverse phenotypes.
Step 4. Collaboration – We will encourage collaboration among the scientific community, including pharma and biotech, to simplify and quicken the research process.
Step 5. Translational Research – We will partner with qualified scientists who share our goal of research that brings about targeted treatments and a cure.
Additional Goals for 2021
In addition to growing the research program, the Foundation’s President and Board of Directors have set the following goals for 2021:
Expand fundraising efforts: As an all-volunteer family foundation, we rely on donations to support all of the great work we’re doing. Last year, the Foundation raised nearly $200,000. This year, we’re hoping to raise $300,000. Our first community-wide fundraiser, the CACNA1A Run/Walk/Roll, is scheduled for June 12, 2021. We also plan to achieve this goal by implementing several new fundraising and development strategies, such as helping families create personalized campaign and fundraising pages, creating new merchandise to sell on the website, and applying for research and operating grants.
Increase awareness of CACNA1A: We look forward to celebrating the first annual CACNA1A Awareness Day on March 19. Launching our website was an important step in raising awareness about CACNA1A and the Foundation, and expanding into social media, including Facebook, Instagram, and Twitter, has helped to elevate the Foundation’s profile. But we need to do much more. We will expand our social media presence significantly over the next year, particularly around telling our families’ stories. Additionally, we plan to increase our engagement with elected officials and policymakers at the local, state, and federal levels to advocate for more resources to support research and treatments.
Expand support for CACNA1A families: As we learn more about CACNA1A and fund research to find treatments and a cure, it is more important than ever to support CACNA1A families. This summer, we will host the world’s first CACNA1A Family and Scientific Conference. We will strive to do this by growing our educational resources for parents and families, including launching a regular educational webinar series, hosting moderated Zoom discussion groups and presentations by leading doctors, scholars, therapists, and more, and continuing to build our Family Advisory Council.
This is an ambitious agenda and achieving it will take all of us doing our part, including you! Thank you for being a part of the CACNA1A community. We truly appreciate your continued support of life-changing research, which gives families hope for their loved ones.