Healx deploys its AI in hunt for Friedreich's ataxia therapies

Tim Guilliams (R) and David Brown (L)

UK artificial intelligence specialist Healx has begun a partnership with two medical charities that aims to uncover new therapies for the neurodegenerative disease Friedreich's ataxia (FA). 

Healx has joined forces with Ataxia UK and the US-based Friedreich's Ataxia Research Alliance (FARA) on the project, and will use its AI-powered drug discovery platform Healnet to try to find already used drugs that could be repurposed as treatments for FA.

Healnet draws on biomedical research, patient group data, scientific literature and deep learning to create a database or "knowledge graph" of rare diseases that can be mined for drug-disease relationships.

It isn’t designed to come up with new molecules, but looks at the 4,000 or so existing drugs on the market, either alone or in combination, ranking those most likely to have an impact on a disease process. The objective is to start clinical testing within six to 12 months.

FA is a progressive form of ataxia, a group of disorders that affect co-ordination, balance and speech, and is the most common of the inherited forms caused by faulty genes. It's still rare however, estimated to affect around one in 30,000 people in Europe and one in 50,000 in the US.

FA is caused by a mutation in a gene that encodes a protein called frataxin and typically symptoms start to develop sometime before the age of 18, including clumsiness and frequent falls, difficulties with speech and swallowing, abnormal curvature of the spine and heart problems.

Symptoms get gradually worse over many years, shortening lifespan although some people with FA live into their 60s or older. At the moment there are no approved therapies for the disease, although some treatments are available to help manage symptoms.

Healx and the patient organisations intend to combine their expertise to try to find drugs that can tackle the underlying cause of FA, rather than just symptoms, in the hope of holding up or halting disease progression.

The biotech specialises in rare disease, and has developed a collaborative model with patient groups and medical charities as it believes getting their input early on increases the chance of finding new therapies.

This year, Healx has announced collaborations with PKD Charity to find to find therapies for polycystic kidney disease, and Mission: Cure to identify the world’s first treatments for chronic pancreatitis.

It is also working with the Foundation for Angelman Syndrome Therapeutics (FAST), Muscular Dystrophy UK, and the Children's Tumour Foundation on new therapies.