Drug Repurposing: Shortening the Path to Successful Treatments

October 17, 2016

DrugRepurposingWordCloudDrug repurposing or repositioning has grown in importance recently due to significant advantages over traditional drug development. The approach is to find new uses for “old” drugs that have either been shelved or are being used for other diseases. A repurposed drug has already passed a significant number of toxicity and other tests, its safety is known and the risk of failure for reasons of adverse toxicology are reduced. More than 90% of drugs fail during development, and this is the most significant reason for the high costs of pharmaceutical R&D. In addition, repurposed drugs can bypass much of the early cost and time needed to bring a drug to market. Recognizing the need to speed the discovery process for ME/CFS patients, the CFIDS Association took a bold step forward into the drug repurposing arena.

In 2012 the Association awarded a grant to Biovista, a small but mighty Charlottesville, VA-based biotech with expertise in drug repurposing. Biovista used their state-of-the-art computational platform to evaluate more than 90,000 compounds together with 25,000 clinical outcomes and the more than 6,000 biomedical publications on ME/CFS. This approach looks for correlations in information leaving no stone unturned. The goal was to find one or more compounds that could be repurposed to safely and effectively treat ME/CFS. As a result of their search, Biovista has identified two candidate therapy approaches including a drug combination treatment.


“We are very excited to have reached this stage and look forward to furthering our understanding of this complex disease and developing a potential treatment that will improve ME/CFS patients’ quality of life,” said Dr. Andreas Persidis, Biovista’s CEO. “Our work is an example of things to come in terms of novel collaboration models that promise to accelerate treatment development and, through the drug repurposing approach, do so in a cost effective way,” Dr Persidis added. “This is a perfect example of how drug repurposing can slash the time and cost to bring new therapies to the patients who need them most,” says Biovista vice president for drug discovery Spyros Deftereos, MD, PhD.

The next step is to design a clinical trial to test if this drug combination is safe and effective in ME/CFS patients.  This is called a “proof-of-concept” clinical trial. When used alone these drugs have been shown to be safe and effective in the diseases they are indicated for; this proof-of-concept clinical trial is required to establish the safety and effectiveness of using these drugs in combination in ME/CFS patients. Biovista, the Solve ME/CFS Initiative and a clinical collaborator have begun designing the clinical trial by submitting the necessary documentation to the FDA – an essential part of the regulatory process for drug development. This gives us the opportunity to ask questions of the FDA so that we can optimize the clinical trial design for the best possible outcome before we submit our request for FDA’s approval to conduct the trial.center-home3

Upon approval from the FDA for the trial, we will complete all other due diligence steps and aim for 2014 to begin this proof-of-concept clinical trial. In just over two years we went from funding a new study to partnering in sponsoring a clinical trial. This is a first for ME/CFS and a sign of things to come- safe and effective treatments for ME/CFS. The Solve ME/CFS Initiative is uniquely positioned to foster the translation of scientific knowledge into meaningful impact for patients more quickly than ever before.

With your support, we will be able to shorten the path to safe and effective treatments for ME/CFS. Please consider making a donation in any amount that is meaningful to you.

Read the press release announcing the partnership