FRAXA Research Foundation initially partnered with Healx in 2016 in a repurposing study to identify existing drugs with potential to treat Fragile X syndrome, using machine learning algorithms and computational biology. The study produced results, and so FRAXA and Healx launched a new round of studies to evaluate combinations of compounds, including both drugs and natural products.
With an initial grant from FRAXA in late 2016, University of Cambridge (England) startup Healx conducted studies to identify novel therapies for Fragile X syndrome, based on current drugs that are FDA-approved for other disorders, such as diabetes.
Sophisticated drug-matching technologies, which originate from the University of Cambridge, are used to identify hidden links between existing drugs and rare diseases. The most promising drugs are then tested experimentally in Fragile X models. In case an approved drug is indeed safe, the next step is clinical trials.
Given a successful result, doctors could then consider “off-label” prescription of the medicine or repurposing it to improve quality of life for Fragile X patients.
Results and Next Steps
Healx has reported a ranked list of the top 8 drugs with the most promising profiles:
While most of these drugs have been approved for general use for other indications, the results require validation. This is where the FRAXA-DVI comes in. Patricia Cogram, PhD, of the University of Chile has FRAXA funding to test new pharmaceutical leads in animal models of Fragile X. Dr. Cogram maintains colonies of Fragile X mice and fruit flies, conducts standardized tests of therapeutic compounds, and compares their effects in the animals to previously tested drugs. Dr. Cogram is testing the top matches now.
Because FRAXA has the established capability to test drugs in animal models quickly and cost-effectively, it should be possible to determine which drugs are the best candidates for clinical trials, and to move forward with those trials within the next year or two.
Phase II Study: Combinations
We are also moving forward with a second study with Healx to identify potential drug combinations for the treatment of Fragile X syndrome, from a library that includes both drugs and natural products. Combinations of drugs enable a multi-pronged attack on a disease, and the potential for synergy can mean lower doses, and fewer or less severe side effects as a result. In addition, with combinations the potential treatment space to be investigated is increased exponentially.
Healx’s technology platform uses an array of different drug matching methods, which will be applied for the identification of combination candidates. The lead candidates will then be validated experimentally in cell systems and in animal models, together with FRAXA-DVI and other academic or industrial partners.