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2 January, 2019 - 10:53 By Tony Quested

Ahead of the curve, ahead of the cure

A large number of science & technology entrepreneurs worldwide set out to pick the low hanging fruit to woo investment in the hopes of making a fortune.

One can only salute the courage of Healx co-founders Dr Tim Guilliams and Dr David Brown, the celebrated inventor of Viagra, who eschewed convention and decided to tackle a huge area of unmet medical need by seeking solutions to the planet’s rarest diseases.

With major pharma players hanging by their fingertips to a patent cliff edge, Guilliams and Brown sidestepped the ‘high volume drugs for well known mass markets’ model and instead rallied to the cause of an estimated 350 million people worldwide living with thousands of illnesses that may have never been accurately diagnosed, let alone treated.

On every continent people of all ages had been waking up daily to the fear that no-one cared – and no-one cured. Healx has changed the paradigm and handed a real dose of hope to a previously anonymous army of sufferers with conditions no-one had ever heard of.

The Cambridge company is using Artificial Intelligence to re-purpose drugs to treat these rare diseases and in a relatively short timespan has achieved incredible success. Major funding has already been secured from global investors and the company continues to refine its platform to cover larger patient populations, an increasing number of rare disease areas and more territories across the planet.

As the FT eloquently put it recently: “The new AI-powered drug discovery company turns a $1 billion process, requiring 2,000 scientists into one that uses a team of 20 and a budget of $100,000 to help cure the world’s rarest diseases.”

According to Dr Guilliams 95 per cent of the 350 million people worldwide living with 7,000 rare illnesses don’t have a treatment. When the founders were researching technology that could lead to cures they drew on the experience of parents whose children suffered from rare diseases. One was Nick Sireau, who has two children with the same rare disease and managed to raise £10m to repurpose a weed killer as a treatment. It is now in phase three of clinical trials – the final phase of testing.

Another case involved Matt Might, whose son Bertrand was born with an ultra-rare disease that affected one in 18 million people. Thanks to Matt’s persistence, the NGLY1 deficiency from which Bertrand was suffering went from a completely unknown disease to one with multiple treatment options.

CEO Guilliams reflects that the founding duo set up Healx in 2014 with the ambitious aim of turning the entire pharmaceutical strategy on its head – and this week was named among the 100 fastest-growing companies in the UK.

He says: “The challenge in the past was that nobody could make the commercial case to develop drugs for rare diseases because it would take 10 to 15 years, cost $2bn and had a 95 percent failure rate, so you couldn’t justify spending all your time and resources on helping tiny disease populations.”

Healx leveraged advancements in machine learning and AI to cure rare diseases at twice the speed and a fraction of the cost.

Dr Guilliams said: “On the one hand, we look at all the rare diseases that we’ve curated and classified, and where we believe existing treatments could potentially help. On the other side, we’ve done the same exercise for all the drugs and nutrients. We then we let algorithms match those up.”

The company made the decision to initiate its investigations through dialogue with the patients or their parents who know the conditions best; this helps to accelerate the identification of potential treatments, says Dr Guilliams.

One of the areas Healx worked on was Fragile X, a rare neurological disease that causes a range of learning disabilities and cognitive impairment.

“We worked with a patient group and in about 15 to 18 months and with $100,000 we managed to identify candidates of existing drugs that worked pre-clinically and that are now ready to be tested on patients in the clinic,” 

Dr Guilliams says. “This used to take five years and cost tens of millions – but now we’re talking months and $100,000.”

Healx is now recruiting patients to start a trial in the US and, as the drugs are already safe and on the market, they can be accessed by patients much quicker.

Because the bioneurological diseases and rare cancers Healx is trying to cure are so complex, more than one drug is usually needed. 

Dr Guilliams says: “With cancer, you need to use a cocktail of drugs because it’s much more complicated – it’s not just one drug, one target.”

Of the 30 staff currently working at Healx, 25 are technology experts specialising in machine learning, bioinfomatics, cheminfomatics and natural language processing.

But Dr Guilliams says the technology alone is not enough. “I don’t think we’re at the stage yet where you can replace all the experts with AI – you still need your drug discovery experts, clinicians and patient voice.

“It’s a platform to provide the tools for optimal and fast decision making from people who really understand the disease and I believe it is absolutely disrupting the space.”

To date, Healx has helped bring two drugs to clinic and is now looking into 10 more as it starts to scale, with the ultimate ambition of helping people suffering from 100 rare diseases by 2025.

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