CureAccelerator Live! for Rare Diseases 2020
CureAccelerator Live! for Rare Diseases
Virtual: June 11, 2020 from 3:30-5:30pm ET
CureAccelerator Live! is our philanthropic pitch event where up to 5 PIs are the finalists presenting their clinical repurposing trials for up to $50,000 in funding and attendees select the winning project.
ANNOUNCING THE 2020 WINNER!
Angela Fleischman, MD, PhD
Repurposing a Nutraceutical in Myeloproliferative Neoplasms to Improve Disease Markers and Symptoms
Runners Up:
Contact us to learn more about any of these clinical repurposing trials.
FUNDED:
Susanna Park, MD, PhD
Bone Marrow Stem Cell Therapy for Vision Loss
An 8-patient clinical trial investigating the safety and efficacy of autologous stem cell transplant to treat vision loss from the hereditary condition, retinitis pigmentosa.
FUNDED:
Tycel Phillips, MD
Repurposing Venetoclax to Improve Patient Response in Mantle Cell Lymphoma
Evaluating the long-term efficacy of adding the chronic lymphocytic leukemia drug venetoclax to lenalidomide plus rituximab treatment in 28 patients with newly diagnosed mantle cell lymphoma, to improve patient outcomes.
Kathleen Sakamoto, MD, PhD
Niclosamide for the Treatment of Pediatric Acute Myeloid Leukemia
Determining the safety and efficacy of combining the generic anti-tapeworm drug niclosamide with standard chemotherapy in 16 children with relapsed/refractory acute myeloid leukemia.
FUNDED:
Alix Seif, MD, MPH
Preventing Relapse After Bone Marrow Transplant in Pediatric Acute Lymphoblastic Leukemia with a Personalized Treatment
Determining the feasibility and safety of identifying 30 children with acute lymphoblastic leukemia (ALL) at risk of relapse following a bone marrow transplant and treating them with either blinatumomab (used to treat relapsed ALL), daratumumab (approved for multiple myeloma), or standard of care to prevent relapse.
CureAccelerator Live! for Rare Diseases was part of the two-day, virtual
on June 11 – 12, 2020, in partnership with
The Rare Drug Development Symposium is a partnership of Penn Medicine Orphan Disease Center and Global Genes
Cures Within Reach is proud to hold this event in support of our Rare Diseases Community. In addition, we are grateful to our industry partners for their support:
Funding support for the winning project comes from:
Questions? Email Director of Scientific Affairs: Dr. Clare Thibodeaux at clare@cureswithinreach.org