Chan Zuckerberg Initiative launches new program to support rare disease patients

REDWOOD CITY, CALIF. — Today, the Chan Zuckerberg Initiative (CZI) announced a new program aimed at supporting and lifting up the work that patient communities are doing to accelerate research and drive progress in the fight against rare diseases. With guidance from patient communities, rare disease experts, and advocacy organizations, this new program — the Rare As One Project — will help communities of patients, researchers, and clinicians work together to advance progress against their diseases and scale these efforts through capacity-building services and up to $4.5 million in initial funding.

Rare disease is anything but rare: as many as 7,000 rare diseases affect 400 million people worldwide. In the U.S., a rare disease is defined as a condition that affects fewer than 200,000 people. The vast majority of these diseases are not well known, and fewer than five percent have any FDA-approved therapy. The knowledge and learnings of patients suffering from these diseases are key to driving breakthroughs in research and treatment, but a lack of funding and infrastructure to support such patient-led research is holding that progress back.

“Patients are a critical part of the solution to finding cures for their diseases — especially when it comes to rare diseases, the majority of which are not well understood,” said Priscilla Chan, Co-Founder & Co-CEO of CZI. “We hope that by partnering with the rare disease community, we can help support patients and patient-led organizations to make progress that not only delivers breakthroughs in their disease areas — but that accelerates broader research discoveries.”

In the first phase of the Rare as One Project, CZI is inviting patient-led, 501(c)(3) organizations to apply for a new, two-year grant opportunity to support the development and launch of research networks in their rare disease area — in partnership with clinicians and researchers. Through the Rare As One Network Request for Applications (RFA), CZI will provide up to 10 patient organizations with funding, training, community mentorship, and capacity-building services, and in turn, ask that they share feedback with and learn from one another, and identify how to best address the most pressing needs across a spectrum of rare diseases. Read our Medium post to learn more.

“A small but growing number of rare disease patient organizations are making progress in their disease areas with remarkable success,” said Tania Simoncelli, CZI Science Policy Director and Rare As One Project lead. “These efforts have resulted in the development of new disease models, diagnostic criteria, drug targets, clinical trials, and — in some cases — new approved therapies in remarkably short timeframes. Our goal is to scale these successes more broadly across the rare disease community.”

Since December 2017, CZI has made several learning grants in the rare disease space. They include:

• Rare Cancer Research Foundation, Pattern.org: $249,600 (2018)
  • Pattern.org enables rare cancer patients to direct excess tumor tissue to cancer research. This grant supports Pattern’s efforts to expand its functionality and capacity to engage patients directly in research.
• Castleman Disease Collaborative Network: $406,640 (2019)
  • The Castleman Disease Collaborative Network accelerates research to find a cure for Castleman disease and improve survival for all patients. This grant supports the development of software tools and infrastructure that will enable other patient organizations to create and facilitate patient-led collaborative research networks.
• Global Genes: $1,249,480 (2019)
  • Global Genes is a global non-profit advocacy organization for individuals and families fighting rare and genetic diseases. This grant supports the development and dissemination of educational materials and interactive workbooks that will assist rare disease patients and advocacy organizations in their understanding and activation to create patient-led research groups and data registries.
• Rare Genomes Project, Broad Institute of MIT and Harvard: $749,287 (2019)
  • The Rare Genomes Project enables patients with undiagnosed, suspected genetic conditions to participate in genomic research as partners, in order to turn genomic data into clinically meaningful answers. This grant will support piloting new approaches to engagement, recruitment, and the return of diagnoses to rare disease families.

The Rare As One Network RFA will accept Letters of Interest starting at 9 a.m. Pacific time Tuesday, June 11, 2019 until 5 p.m. Pacific time on Tuesday, July 23, 2019. For more information and application instructions, please visit CZI’s website. For administrative and programmatic inquiries, technical assistance, or other questions pertaining to this RFA, please contact [email protected].

About Chan Zuckerberg Initiative

Founded by Dr. Priscilla Chan and Mark Zuckerberg in 2015, the Chan Zuckerberg Initiative (CZI) is a new kind of philanthropy that’s leveraging technology to help solve some of the world’s toughest challenges — from eradicating disease, to improving education, to reforming the criminal justice system. Across three core Initiative focus areas of Science, Education, and Justice & Opportunity, we’re pairing engineering with grant-making, impact investing, and policy and advocacy work to help build an inclusive, just and healthy future for everyone. For more information, please visit www.chanzuckerberg.com.