There is currently no approved treatment for Fragile X Syndrome-this is the most common cause of inherited intellectual disability. Currently, these treatments are aimed at controlling symptoms, from anxiety to obsessive-compulsive disorder, or in rare cases, seizures. But a new clinical trial is about to be conducted in a British start-up company. 

On Wednesday, drug discovery startup Healx announced that it has received FDA research new drug status for phase 2a clinical trials of a repositioned drug for Fragile X syndrome (more on this later). According to the Clinicaltrials.gov registration, the study will begin in January 2022, and the study will evaluate the impact of non-steroidal anti-inflammatory drugs on Fragile X virus. 

Healx is one of many startups that have invested in the use of artificial intelligence for drug development and identification. So far, it has raised approximately US$67 million in total funding-the company recently completed a US$56 Series B financing in October 2019. Healx's investors include Atomico, Balderton Capital, Amadeus Capital Partners, Intel, Global Brain, btov partners and angel investor Jonathan Milner. 

Healx's platform Healnet uses machine learning to capture data on existing drugs and potential disease targets to find new connections. Healx's platform can receive structured public or private data, or data in other forms, such as data in scientific publications. Part of Healx's approach is to use natural language processing to access data described in scientific literature. 

"All of this goes into the knowledge base, and then this is basically the power of our algorithm that can be used in the drug matching process," Healx's Chief Medical Officer, Dr. Anthony Hall, told TechCrunch. [Algorithm is] Looking for the connection between disease and drug, and then we can use it for preclinical and clinical trials. " 

The development of drugs based on artificial intelligence is becoming a larger part of the field of artificial intelligence. According to Stanford University’s 2021 Artificial Intelligence Index Annual Report, between March 2020 and 2021, the funding for artificial intelligence-based drug development soared to US$13.5 billion, which is 4.5 times the total funding in 2019. Although some of them may be affected by the competition to develop COVID-19 vaccines and drugs, there are still some important financing rounds (insilico Medicine, for example, the company completed a $255 million Series C financing in June). Others have managed to strike deals with large pharmaceutical companies. 

Currently, Healx focuses on rare diseases. In addition to Fragile X, the company also has potential drug treatments for Pitt Hopkins syndrome in clinical planning, and 11 drug candidates are in the preclinical development stage.

"We have many other projects in the pipeline," Hall said. 

Throughout the process, Healx also worked closely with FRAXA, a non-profit research foundation focused on Brittle X. Michael Tranfaglia, FRAXA's chief scientific officer and medical director, pointed out that Healx and FRAXA have collaborated even before the clinical trial process, when patient organizations are usually involved in the research. 

"The unusual thing here is that we are involved in the whole process," he told TechCrunch. "We have been providing companies with a lot of information that helps manage these different data sets." 

So far, Healx has focused on identifying drugs that already exist, rather than developing new drugs—although Hall said the development of new drugs will not be excluded in the future. 

Research on existing drugs (sometimes called drug repositioning) is far from unheard of, especially in the field of rare diseases. It reduces development costs and has been proposed as a way to initiate research on rare diseases. These rare diseases are often overlooked because they cannot guarantee the same profit return as treating common diseases. 

As pointed out in a review paper in Trends in Pharmaceutical Science in 2021, reused drugs can reach patients within 3 to 12 years, while new drugs usually take 10 to 15 years. 

The difficulty with drug reuse is that, in the past, it depends to some extent on luck. A 2011 paper pointed out that the next frontier in drug reuse is "the ability to operate in a systematic and rational way, not accidentally discovered." 

Healx's method happens to be artificial intelligence. 

Healx's clinical trial of a potentially Fragile X drug will be the company's first clinical trial of a repositioned drug. But it plans to conduct more powerful experiments in the near future. Specifically, Healx sees this trial as the first phase of adaptive clinical research that the startup hopes to conduct. The research will continue to test more drugs that have been identified by Healnet as drug candidates and verified by preclinical testing.  

Hall explained that there are already plans to add two more drugs to the trial — the company did not name the drugs they are testing because of concerns that they may stimulate off-label use. He did explain that these three drugs work together on three different mechanisms of action associated with Fragile X Syndrome. 

Ultimately, the trial will evaluate the effects of these drugs on two broad categories of endpoints in the Fragile X study: cognitive function and anxiety-based endpoints. 

It should be noted that in the field of autism research (fragile X patients are usually also diagnosed with autism), finding a cure rather than a treatment is no longer as important as it used to be. For some people, autism is just another way of being in the world, not something that needs to be cured in the first place. Other arguments suggest that it is society, not biology, that prevents people who are neurologically differentiated from living a happy and "normal" life. 

Studies have shown that treatment and having an environment that meets their needs are often helpful for people with Fragile X. Drug-based treatment does play a role in controlling symptoms such as hyperactivity or anxiety. Nevertheless, as Tranfaglia pointed out, some current symptom management drugs may not work well or cause uncomfortable side effects. 

For example, children treated for ADHD may find that medications increase their anxiety, he said. 

"This is a huge mole game, you look for a symptom, and then treat it with medication, but eventually it will cause side effects in another area and make things worse," he said.

Future research on three different drugs may feel that it has many moving parts. As Hall said, Healx's method is "multiple shots." Tranfaglia pointed out that these multiple injections also fit the view that treating rare diseases such as Fragile X may require more than one drug. 

"Ultimately, we hope to find the best two or three drug combinations for each of these 7,000 rare diseases, which can be made from one or 200 repurposed drug candidates," Tranfaglia said . 

This research is still only the first step. But this will be a test of the larger trend in the treatment of rare diseases: existing drugs may make a difference, but we have not yet found the tools for them.