Viagra can teach us a lot about the treatment of rare diseases

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In June 1993, David Brown was exasperated. For eight years he was developing a drug to treat angina pectoris, but early clinical trials showed it didn’t have enough effect to make it commercially successful. His employer, Pfizer, gave him three months to reverse the project. Weeks later, Brown heard unusual news from a group of Welsh men involved in a clinical trial of the drug: They were having more erections than usual. Realizing that he might have a huge blockbuster on his hands, Pfizer shifted his clinical trials to focus on erectile dysfunction. “He went from death to number one in the Pfizer portfolio within two weeks,” Brown said. Approved in the United States in 1998, Viagra sold over $ 400 million (£ 237 million) in its first three months alone.

Now Brown wants to repeat this trick to find new treatments for rare diseases with his startup, Healx. Co-founded in 2014 with Tim Guilliams, the Cambridge-based startup uses artificial intelligence to identify existing drugs that have already been in clinical trials, then reuse them as treatments for rare diseases. More than 400 million people around the world live with such diseases, but because each affects a small number of people, pharmaceutical companies lack the financial motivation to develop new drugs to treat them. “The fastest, safest, and most cost-effective way to invent a new drug is to start from an old drug,” says Guilliams, CEO of Healx.

The idea for Healx came from a conversation Guilliams and Brown had at a Cambridge pub with Nick Sireau, whose two sons suffer from a rare genetic condition called alkaptonuria. Although there are around 7,000 rare diseases, 95% of them are untreated. When treatments are developed they can be extremely expensive. Zolgensma, a drug approved in the United States to treat spinal muscular atrophy, costs $ 2.1million (£ 1.5million) for a single treatment. The huge failure rate when it comes to developing new drugs increases costs. According to one estimate, only four percent of drugs developed make it through to approval.

Guillams and Brown want to massively improve those odds. At the heart of Healx’s approach is Healnet: an AI platform that finds links between drugs and disease. Using natural language processing, the platform extracts data from clinical trials, patents, electronic health records, genomic datasets and scientific articles to find drugs or combinations of drugs that match a particular disease profile. At the same time, scientists at Healx are creating and maintaining new datasets to fill knowledge gaps about new diseases. “You put that human mind next to a computer, and the computer helps the human mind to be more efficient, and the human mind helps the computer to be more efficient,” says Brown.

A rare condition that Healx works on is called fragile X syndrome; one of the most common causes of inherited learning disabilities. In 2017, she began to identify a number of reused drug combinations that showed promise in mouse trials, and now plans to start clinical trials in 2020. The company is also researching treatments for other diseases. rare, especially Pitt-Hopkins syndrome, Duchenne musculature. dystrophy and some rare cancers. Ultimately, Brown hopes that Healx’s work will not only find new treatments for rare diseases, but completely overhaul the way the industry finds new drugs. “I think we can also transform the whole drug discovery process,” he says.

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