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CureAccelerator Live! for Rare Diseases 

June 6, 2019 in Philadelphia, PA

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.

 

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Congratulations to the winner of CureAccelerator Live! for Rare Diseases: Kim Nichols, MD, St. Jude Children's Research Hospital "Repurposing a Blood Cancer Drug to Treat an Immune Disorder in Children"

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Finalists: 

 

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Margherita Baldassarri, MD, University of Siena "Repurposing a Generic Diuretic to Treat Alport Syndrome" (click to see project summary)

Alport syndrome is a rare, inherited disease that affects the kidneys, and it can also impact the eye and inner ear. Many patients experience end-stage renal failure at some point. Podocytes are a specific type of cell in the kidney, which have a predominant role in Alport syndrome onset. Loss of podocytes through the urine seems to be the first sign of disease progression. Amiloride is a diuretic used to treat congestive heart failure, high blood pressure and low potassium levels, and it has been previously proven to have significant role in reducing podocyte damage. Building on our preliminary results and other published evidence, we will test the safety and efficacy of low-dose amiloride treatment in 12 Alport Syndrome patients, and determine if amiloride can slow the progression of disease. We will also perform biochemical tests, both before and after Amiloride treatment, in order to identify biological pathways involved and any differences between responders and non-responders.

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Kim Nichols, MD, St. Jude Children's Research Hospital "Repurposing a Blood Cancer Drug to Treat an Immune Disorder in Children" (click to see project summary)

Hemophagocytic lymphohistiocytosis (HLH) is a rare, poorly understood disorder of the immune system characterized by recurrent episodes of hyperinflammation. Currently, the treatment for HLH is aimed at quieting the overactive immune system with drugs such as steroids and etoposide (a type of chemotherapy). While this approach is effective in some patients, these drugs can cause significant side effects with less- than-optimal results. In addition, with the high morbidity and mortality associated with HLH, there is a pressing need to develop more effective therapies. We have found that the FDA-approved blood cancer drug ruxolitinib decreases the signs of disease and enhances survival in mice, and now want to determine whether ruxolitinib is safe and effective in human patients, specifically in children. We’ll start with a 10-patient clinical trial, and the data provided will form the foundation for a larger pivotal Phase II clinical trial.

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Kevin Winthrop, MD, MPH, Oregon Health and Science University "Repurposing a Leprosy Drug to Treat a Rare Lung Infection" (click to see project summary)

Nontuberculous mycobacterial (NTM) lung disease is caused by a bacterial infection. The current treatment for NTM includes a triple antibiotic drug combination taken for 18-24 months at a time. After stopping therapy, unfortunately NTM has a high risk of relapse or reinfection, and many patients must begin therapy again. Treated patients frequently experience debilitating side effects, and many patients delay the start of antibiotic treatment due to these risks. Clofazimine is FDA-approved to treat leprosy, and offers an orally available treatment option for NTM patients. Although clofazimine has been used off-label to treat NTM, the efficacy and safety is unclear as clofazimine has not been evaluated in the context of a randomized controlled trial. Accordingly, we are conducting a multi-center randomized, placebo-controlled Phase II study of clofazimine in the treatment of NTM.

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Stephen Wong, PhD, Houston Methodist Research Institute "Repurposing a Heart Failure Drug for Pediatric Brain Cancer" (click to see project summary)

Medulloblastoma is the most common malignant brain tumor in children. While a majority of medulloblastoma patients are cured with current standard therapy, survivors face significant long-term after-effects. Furthermore, for patients who fail standard treatments, there are currently no effective second-line therapies. The need for new treatments for medulloblastoma remains pressing. We used a novel bioinformatics approach to identify repurposed drugs with activity against medulloblastoma. This approach identified the well-characterized cardiac drug, digoxin, as a potential candidate for a new treatment. Based on preclinical results demonstrating the effectiveness of digoxin against medulloblastoma, we hypothesize that digoxin can be safely administered to children with medulloblastomas and other central nervous system (CNS) tumors. This clinical trial will define and describe the safety and anti-tumor effects of digoxin in children with recurrent or refractory CNS tumors.

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CureAccelerator Live! for Rare Diseases is presented in partnership with

                     global genes logo registered 1 2  and its  GG ODC Rare Drug Development Logo cropped

The Rare Drug Development Symposium is a partnership of Penn Medicine Orphan Disease Center and Global Genes

UPenn ODCAttending the Rare Drug Development Symposium? Your ticket includes entry to CureAccelerator Live! for Rare Diseases. 

 

Thank you to our industry partners for their support:

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Thank you to the Judy Hirsch Foundation for philanthropic support:  

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Questions? Email Director of Scientific Affairs: Dr. Clare Thibodeaux at clare@cureswithinreach.org


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Don't miss our first CureAccelerator Live! of 2019, focused on the Developing World. Click here for those event details.

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