This year, February 29 is Rare Disease Day, which is an annual event in which patient organizations around the world raise awareness of rare diseases through activities and special programming such as charity runs, documentary screenings, panel discussions, art contests and much more.
Many rare diseases, very few treatments
There are approximately 7,000 rare diseases out there,
affecting more than 300 million people—and yet, nearly 95% of them have no
treatment options. Precisely because rare diseases are uncommon, public
knowledge of them is typically low, a problem that also contributes to lack of
funding for treatments and cures. The fact that drug discovery and development
is an extremely costly process, and that most new drugs fail, also contributes
to the rarest diseases being left behind.
But drug repurposing is one method that has emerged as a
way of finding treatments for rare diseases because it is less costly, since it
involves taking already-approved drugs and identifying other diseases that they
A drug repurposing Datathon tackles a rare
Non-profit organizations CuresWithinReach and Mission: Cure have both been working on drug repurposing to identify drug candidates for treating chronic pancreatitis, a rare condition that affects roughly one million people worldwide. Last year, Elsevier and the Pistoia Alliance partnered with these organizations to run a drug repurposing Datathon with the goal of identifying repurposable drug candidates for chronic pancreatitis.
Participants in the Datathon used Elsevier’s Entellect
platform to access Elsevier datasets like Reaxys Medicine Chemistry, ResNet and
PharmaPendium (or their own datasets). The result of their work was the
identification of FOUR drug candidates. The next step will be a clinical trial
conducted by Mission: Cure.
“The problem-solving and teamwork focused on chronic pancreatitis were inspiring,” said Mission: Cure’s Megan Golden about the datathon in this recent RELX Annual Report. “We look forward to taking the promising candidates to the next step where we hope they will help us find effective treatments for this difficult, rare disease.”
Register for our drug repurposing webinar
This is a really promising development in the rare disease space, and if you would like to learn more about the Datathon, the latest on the progress of the clinical trial, information about predictive analytics for drug repurposing and much more, I hope you will check out the webinar that I am co-hosting with Megan Golden on February 27 at 10AM EST!
Register for free to join us for “Drug repurposing for rare diseases: an integrated data driven approach.”