CA color bar

CA Live w circleR 2018 v2

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.


 CA logo graphic only

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"

 CA Live Rare Winner


Still Ready to Fund: 


Baldassarri for website

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.

Logo AOUS Siena web


Winthrop Kevin headshot for web

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.



Wong Stephen Cropped copy

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.

HM Cancer Center v1


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:

                                                    Horizon Logo Full Color RGB M01 UPDATED 2019      RRD LOGO tagline TM CMYK Black


Thank you to the Judy Hirsch Foundation for philanthropic support:  

JHF Logo

Questions? Email Director of Scientific Affairs: Dr. Clare Thibodeaux at

CA logo graphic only

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

Click here to sign up for our eNewsletter today!

Please include your Full Name* and Email Address*


If the link above doesn't work, please contact our Admin support at with your Full Name and Email Address to sign up!


Cures Within Reach

134 N. LaSalle, #1130
Chicago, IL 60602

All funds donated to Cures Within Reach may be tax deductible for federal income tax purposes. 

Partnership for Cures DBA Cures Within Reach is recognized by the IRS as a 501(c)3 tax-exempt not-for-profit organization, tax ID 20-3620169.


Sunday the 20th. Copyright 2018 Cures Within Reach.