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
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/
"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;
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.
About Cures Within Reach
Cures Within Reach (CWR) is a US-based philanthropic leader that improves patient quality and length of life by leveraging the speed, safety and cost-effectiveness of medical repurposing research, driving more treatments to more patients more quickly. CWR catalyzes research to facilitate and validate repurposing opportunities that create clinical impact. Through repurposing, CWR drives both market impact and health savings to patients and patient groups, from academia/researchers, with payers and the healthcare industry and with support from the government, philanthropy and others.
In 2019 alone, CWR began 6 new clinical repurposing research projects. CWR currently has a global portfolio of 20 repurposing research projects in 17 diseases at 15 institutions, as well as more than 180 repurposing research projects available for funding in a wide range of diseases on its CureAccelerator site. Visit www.cureswithinreach.org for more information.
Clare Thibodeaux, PhD