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.


Angela Fleischman, MD, PhD

Repurposing a Nutraceutical in Myeloproliferative Neoplasms to Improve Disease Markers and Symptoms

UCI Chao Family Lab

Runners Up:

Contact us to learn more about any of these clinical repurposing trials.


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.


Tycel Phillips

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.



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:

Horizon Therapeutics supports repurposing researchRecordati Rare Diseases supports repurposing research


Funding support for the winning project comes from: 

Questions? Email Director of Scientific Affairs: Dr. Clare Thibodeaux at