Press Release: St. Jude Researcher Wins CureAccelerator Live! for Rare Diseases

St. Jude Researcher Wins CureAccelerator Live! for Rare Diseases

Clinical Trial for Ultrarare Hemophagocytic Lymphohistiocytosis Receives Funding to Repurpose Existing Blood Cancer Treatment

June 10, 2019

Kim E. Nichols, MD, representing St. Jude Children’s Research Hospital won Cures Within Reach’s CureAccelerator Live! for Rare Diseases philanthropic pitch event on June 6 in Philadelphia. 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. CureAccelerator Live! brings together stakeholders from pharma, biotech, academia, medicine, philanthropy and patient groups to showcase repurposing projects in need of funding. Held in partnership with Global Genes’ RARE Drug Development Symposium with the UPenn Orphan Disease Center, attendees voted for the winning repurposing clinical trial. This CureAccelerator Live! event focused on clinical trials that use repurposed therapies to treat an unmet medical need impacting patients with rare diseases.

Attendees heard from three clinician-researchers representing academic medical centers from the US and Italy. Dr. Nichols’s project, “Repurposing a Blood Cancer Drug to Treat an Immune Disorder in Children,” will study the FDA-approved drug ruxolitinib in a prospective 10 patient clinical trial to test whether treatment with a ruxolitinib-containing regimen could control inflammation and thus improve the outcome for children with hemophagocytic lymphohistiocytosis (HLH). If successful, this project may provide proof-of-principle of the potential benefits of the proposed ruxolitinib-containing regimen and allow St. Jude to move forward with a larger pivotal Phase II clinical trial. St. Jude Children’s Research Hospital and Dr. Nichols (www.stjuderesearch.org/site/lab/nichols) will receive up to $50,000 in funding from Cures Within Reach to support this research.

“HLH is a devastating disease with many patients dying despite current treatment options. Thanks to the support of Cures Within Reach, we will now be able to test whether a novel treatment approach that repurposes the drug ruxolitinib will prove effective for children with HLH,” said CureAccelerator Live! winner Nichols, Division of Cancer Predisposition Director at St. Jude Children’s Research Hospital. “We are very grateful for Cures Within Reach’s support and dedication to helping better the lives of patients with rare diseases such as HLH.”

“All of our finalists’ clinical repurposing projects have a real opportunity to improve the quality and length of life for patients living with a rare disease. It is inspiring to see these researchers present their work and share the possible impact for patients if successful,” said Barbara Goodman, President & COO at Cures Within Reach. “We are thrilled to support Dr. Nichols on her winning clinical trial for HLH patients.”

The other finalists who presented their repurposing projects at CureAccelerator Live! included:

• Margherita Baldassarri, MD, University of Siena: Repurposing a Generic Diuretic to Treat Alport Syndrome
• Stephen Wong, PhD, Houston Methodist Research Institute: Repurposing a Heart Failure Drug for Pediatric Brain Cancer

Expert Panelists representing pharma, clinicians, academia and patient advocacy helped attendees make a more informed voting decision, and included representatives from:

• BioMarin Pharmaceutical
• EveryLife Rare Disease Foundation
• Horizon Therapeutics
• Orphan Disease Center at University of Pennsylvania
• Recordati Rare Diseases

Cures Within Reach is grateful for industry support from Horizon Therapeutics and Recordati Rare Diseases, and philanthropic support from the Judy Hirsch Foundation for this CureAccelerator Live! event.For more information about CureAccelerator Live!, please visit cureswithinreach.org.