Press Release: Finalists Announced for Pediatric-Focused CureAccelerator Live! for Rare Diseases 2021 Competition

Finalists Announced for Pediatric-Focused CureAccelerator Live! for Rare Diseases 2021 Competition

Virtual Pitch Event on June 10 Features 4 Pediatric Clinical Repurposing Projects

April 27, 2021

Cures Within Reach, a leading global nonprofit focused on repurposing research as a fast track to impacting patients, is holding CureAccelerator Live! for Rare Diseases, its unique philanthropic pitch competition, virtually on June 10, 2021. The event this year, focused on pediatrics, is being held in partnership with Global Genes, who is working with UPenn Medicine Orphan Disease Center on the 2021 RARE Drug Development Symposium on June 10 – 11, 2021.

Researchers and clinicians from 4 institutions from across the US and from Chile will present their clinical repurposing research projects to community members representing industry, academia, medicine, philanthropy and patient groups. The winning project will be selected by attendees to receive up to $50,000 in funding. The 4 finalists presenting their clinical trial projects are:

• Ignacia Fuentes, PhD of DEBRA Chile: Repurposing a Common Antioxidant Nutraceutical, N-Acetylcysteine, for Wound Treatment in Butterfly Skin
• Nancy Gordon, MD of UT MD Anderson Cancer Center: Enhancing Treatment Response in Recurrent/Metastatic Osteosarcoma with Hydroxychloroquine
• Joseph Rower, PhD of University of Utah: Repurposing Valganciclovir to Treat Cytomegalovirus-Induced Hearing Loss
• Joyce Teng, MD, PhD of Stanford University: Treating Vascular Malformations in the Face, Head and Neck using Trametinib

“As a longstanding Foundation dedicated entirely to enhancing the lives of children through pediatric research and innovation, we recognize both that rare diseases are an area of great unmet need and that repurposing presents a unique opportunity to bring safe, effective treatments to patients more quickly,” said Charlene Mancusi, Associate Director of the Charles H. Hood Foundation. “It is an honor to work with Cures Within Reach to identify and support repurposing research projects that could expedite clinical breakthroughs for the pediatric population and catalyze meaningful impact on child health.”

“Testing already approved therapies has always been an important strategy in finding treatments for rare diseases, and it is even more critical in pediatrics,” said Clare Thibodeaux, PhD, Director of Scientific Affairs at Cures Within Reach. “Cures Within Reach has a successful history of catalyzing pediatric repurposing research, and the innovative clinical trials to be featured at CureAccelerator Live! for Rare Diseases on June 10 highlight this opportunity. The attendees will be faced with a difficult voting decision, as all 4 projects have the potential to improve the quality of life for rare disease children and their families.”

Registration for the virtual RARE Drug Development Symposium is open now: for more information, agenda and registration, visit: https://globalgenes.org/event/advocacy-symposium/. For more information on these finalists and the CureAccelerator Live! for Rare Disease competition, visit bit.ly/CWRcalrare2021.

Funding for the winning pediatric rare diseases project is from philanthropic partner the Charles H. Hood Foundation. Additional event support from the Rare Diseases and Pediatrics Community members: Brand Institute, EdgeOne Medical, Goldman Philanthropic Partnerships, Healx, Horizon Therapeutics, the Judy Hirsch Foundation, Medidata, PhRMA, Recordati Rare Diseases, Takeda Pharmaceuticals and TerSera.