Ultimately, FRAXA went as far as a patient group could go towards developing a treatment for Fragile X. Despite 22 years of good progress, an effective drug had still not been found. FRAXA approached Healx to collaborate in late 2016. Its CEO, a clinician from America, was attracted by Healx’s fresh approach to drug discovery and was willing to try something new.

Healx’s ‘big data’ approach has proved to be very quick and cost effective when compared to traditional drug development. It takes less than half the time and 3% of the cost of traditional drug development to reach FDA approval. Different drug combinations can be tested together by a computer before testing in animal models. New pathways and targets, previously unknown to researchers, can also be identified.

Healx was initially attracted to FRAXA’s cause because it was a well-developed patient group that possessed a good model system in which drugs could be tested. The members were also open minded and willing to help Healx design clinical trials if a drug was found and offer insight into why past clinical trials were unsuccessful (as they had worked with the pharmaceutical companies who ran them). FRAXA also knew its community well and what really mattered to them.

Healx at first needed to know what symptoms the drugs were trying to treat. FRAXA was instrumental in this role. Fragile X can be a complicated disease with multiple symptoms. Healx wanted to make the algorithms prioritise finding drugs that treated the symptoms that mattered the most to the patients and families.  Research showed these to be:

  • Intellectual disabliilty 
  • ADHD, hyperactivy 
  • Anxiety, sensory issues 
  • Autistic behaviours 
  • Seizures

 Once the aims were identified Healx gathered as much biological data as it could for its algorithms to use. They searched through public data bases and found 19 potential sources. But only the data in 3 was deemed to be of high enough quality for use.

 Once the biological data was gathered…. 

  1. The algorithms looked at the data and assessed which drug’s expression profiles would counterbalance the aberrant gene’s expressions profiles for this specific diseas
  2. The data from all these different sources was collected into a knowledge graph (a representation of all the knowledge gathered and how it links together in a complex web). This acted as a map on which drug-gene-symptom-disease interactions could be traced on. Every possible drug was guided along it to see if it had the potential to help relieve one or more symptoms of the disease.

Once the computer had analyzed the data, a list of compounds that may relieve the symptoms of Fragile X was produced. Then a team within Healx, made up of pharmacologists with previous drug discovery experience, combed through the list. They eliminated compounds that would not work for biological reasons the algorithms had not been programmed to consider.

 From this, 8 drugs were shown to be promising. The computer showed 3 of them treated all symptoms FRAXA had define as very important to patients and their families. One of the drugs was a well-established anti-inflammatory drug used to treat arthritis. No one had previously thought about applying it to treat Fragile X.

 The 3 most promising compounds were tested on the mice model and the most effective drug is now in Phase 2a clinical trials.