Rare Diseases
While Cures Within Reach is disease-agnostic, we are dedicated to improving the quality and length of life for patients living with a rare disease through the speed and cost-effectiveness of medical repurposing.
Our events and research within the Rare Diseases Repurposing Community all focus on connecting our many stakeholder groups together in our shared goal: driving more treatments to more patients more quickly through repurposing.
Funding Opportunity RFPs for Rare Diseases:
- Clinical Repurposing Research to Impact Veterans/Military 2026: accepting budgets of up to $75,000 to a U.S.-based institution for a repurposing clinical trial in any disease area impacting veterans or active military, such as mental health, PTSD and brain injuries. LOI SUBMISSIONS DUE MAY 29, 2026.
- Repurposing Research for Chicago 2026: accepting budgets of up to $125,000 for clinical repurposing trials from Chicago area research institutions in one or both of the tracks listed below. LOI SUBMISSIONS DUE MAY 8, 2026.
- Track 1 – Investigator Groups: clinical repurposing trials in any unsolved disease led by an Early Stage Investigator or an underrepresented researcher.
- Track 2 – Patient Groups: clinical repurposing trials impacting specific patient groups, such as health disparities, pediatrics, rare diseases and/or veterans issues (including mental health, PTSD and brain injuries).
- ReGRoW: Funding for Underresourced Researchers in Low and Lower-Middle Income Countries 2026: accepting budgets of up to $70,000 for clinical repurposing trials in any disease led by an underresourced researcher in any LMIC, as defined by the World Bank. LOI SUBMISSIONS DUE APRIL 10, 2026.
More information on our Funding Opportunities / RFPs page.
Our ongoing rare disease trials:
Current Rare Disease Trials
Rare Diseases: Completed Trials
(in the past 5 years)
Rare Diseases: Press Releases
Success Stories
Improving a Vitamin D Defect with Rifampin, a Common Antibiotic
Improving Blood Cancer Outcomes with IFN-gamma After Stem Cell Transplantation
Repurposing a Transplant Drug Saves Kids with ALPS, a Rare but Deadly Blood Disorder
Finding a New Use for Thalidomide in Multiple Myeloma Patients
Using a Tumor Ablation Device in Pancreatic Cancer Patients
Events
Diversity of Clinical Trial Researchers and Patients: A Repurposing Opportunity?
November 2021
Virtual
A dialogue on the dual opportunities of clinical trial diversity to impact health disparities and/or improve minority leadership in scientific research.
Real World Data Through a Patient’s Lens
October 2021
Virtual
A conversation on Real World Data (RWD) and case reports from clinicians and researchers.
CureAccelerator Live! for Pediatric Rare Diseases 2021
June 2021
Virtual
Four finalists from institutions across the US and Chile competing for up to $50,000 to fund their repurposing clinical trials, all focused in pediatric rare diseases.
Repurposing for Pediatric Patients: Best Practices and Lessons Learned
November 2020
Virtual
A patient-focused conversation on the opportunities that repurposing drugs, devices and other treatments provide for the pediatric patient population.
How Patients Impact the 505(b)(2) Regulatory Process
October 2020
Virtual
A patient-focused conversation on the 505(b)(2) regulatory process and how patients, caregivers and patient advocacy groups can engage with both industry and regulators to support the drug development process, share their real-world perspectives for clinical trial planning and represent the patient view throughout the drug approval process.
Cure Accelerator Live! for Chicago 2020
September 2020
Virtual
Four finalists from the greater Chicago area competing for up to $50,000 to fund their repurposing clinical trials.
CureAccelerator Live! for Rare Diseases 2020
June 2020
Virtual
Five finalists from the US competing for up to $50,000 to fund their repurposing clinical trials impacting rare disease patients.
The Future of Patient Care in Acute Myeloid Leukemia (AML) and Other Blood Cancers
September 2019
Palo Alto, CA
A patient education event providing insight into current approaches to treating and living with AML and into potential future AML therapies.
CureAccelerator Live! for Rare Diseases 2019
June 2019
Philadelphia, PA
Three finalists from the US and Italy competing for up to $50,000 to fund their repurposing clinical trials impacting rare disease patients.
CureAccelerator Live! for Oncology 2017
November 2017
Washington, DC
Five finalists from institutions across the US competing for up to $50,000 to fund their oncology repurposing clinical trials.