Press Release: University of California, Irvine Researcher Wins CureAccelerator Live! for Rare Diseases

University of California, Irvine Researcher Wins CureAccelerator Live! for Rare Diseases

Clinical Trial in Myeloproliferative Neoplasms Receives Funding to Repurpose Nutraceutical Used for Acetaminophen Overdose

June 15, 2020

Angela Fleischman, MD, PhD representing University of California, Irvine won Cures Within Reach’s CureAccelerator Live! for Rare Diseases virtual philanthropic pitch event held on June 11. Cures Within Reach is a leading global nonprofit focused on improving patient quality and length of life by leveraging the speed, safety and cost-effectiveness of medical repurposing research. Held in partnership with Global Genes’ RARE Drug Development Symposium with the UPenn Orphan Disease Center, attendees heard from five clinician-researchers representing academic medical centers from across the US and voted for the winning repurposing clinical trial.

Dr. Fleischman’s project, “Repurposing a Nutraceutical in Myeloproliferative Neoplasms to Improve Disease Markers and Symptoms,” will study N-Acetylcysteine, a low cost nutraceutical and its impact on reducing thrombosis, inflammation and other symptoms. If successful, this project may lead to an inexpensive therapy to reduce symptom burden for myeloproliferative neoplasm (MPN) patients and provide the basis for larger studies on thrombosis reduction. The University of California, Irvine and Dr. Fleischman (http://www.mpnlab.org/) will receive up to $50,000 in funding from Cures Within Reach to support this research.

“This funding will support a clinical trial that could potentially provide MPN patients with a low-cost, low-risk treatment that impacts all three of the major issues affecting MPN patients,” said Dr. Fleischman. “I’m so grateful for Cures Within Reach and its funding partners for this support.”

“The speed to impact and cost-effectiveness of all five finalists are so encouraging. Each clinical repurposing project has a real opportunity to improve the quality and length of life for patients living with a rare disease, and we hope to find funding for all of them,” said Barbara Goodman, President & COO at Cures Within Reach. “As a start, we are thrilled to support Dr. Fleischman on her winning clinical trial to impact the MPN community.”

The other finalists included:

• Susanna Park, MD, PhD from University of California, Davis presenting “Bone Marrow Stem Cell Therapy for Vision Loss“
• Tycel Phillips, MD from University of Michigan presenting “Repurposing Venetoclax to Improve Patient Response in Mantle Cell Lymphoma“
• Kathleen Sakamoto, MD, PhD from Stanford University presenting “Niclosamide for the Treatment of Pediatric Acute Myeloid Leukemia“
• Alix Seif, MD, MPH from Children’s Hospital of Philadelphia presenting “Preventing Relapse After Bone Marrow Transplant in Pediatric Acute Lymphoblastic Leukemia with a Personalized Treatment”

Expert Panelists representing industry, academia and patient advocacy helped attendees make a more informed voting decision, and included representatives from CML Advocates Network; Global Genes; Horizon Therapeutics; Recordati Rare Diseases and the University of Kansas Cancer Center. Cures Within Reach is grateful for philanthropic support from Goldman Philanthropic Partnerships, industry support from Horizon Therapeutics and Recordati Rare Diseases, and additional support from the Judy Hirsch Foundation for this CureAccelerator Live! event.