According to a story from fortune.com, the French drug startup Pharnext, led by CEO Daniel Cohen, recently made an announcement not to long ago that made its stock shares rocket through the roof. In a Phase III clinical trial, the company’s experimental product PXT3003 showed positive results as a treatment for Charcot-Marie-Tooth disease, a rare neuromuscular disorder that currently has no cure.
AI and Drug Repurposing
The results from this trial were encouraging and appear to indicate that PXT3003 was actually capable of reversing some of the effects of the disease, something which no prior therapy has been able to do. Just as remarkably, PXT3003 is not a brand new compound; it is actually a combination of three different drugs that are commonly used for different purposes. The company was able to use AI technology in order to come to the conclusion that these three drugs, when used together, could be a useful treatment for Charcot-Marie-Tooth disease.
This is a prime example of drug repurposing, the process in which drugs that have already been approved for use are reassessed and used to treat another disease as well. The approach is becoming more popular, particularly in the rare disease drug development arena, and the utilization of AI could help make the process more efficient and effective than ever.
There are several reasons why drug repurposing is appealing. First and foremost, testing a drug that has already been approved and developed makes the regulatory process more streamlined and completely cuts out the time and diligent research that is necessary in order to get an all-new medication ready for testing and approval. This also translates into money saved as well. Ultimately, the process can be quicker and less costly than developing a brand new drug when everything goes as planned.
The major challenge with drug repurposing is figuring out which drugs actually have a chance to work. This is where AI can play a major role. This technology was able to select three currently used drugs as the active components of PXT3003, including sorbitol (a common laxative), naltrexone (often used to treat opioid addiction), and baclofen (a common muscle relaxant). Apart, these drugs would probably have no impact on Charcot-Marie-Tooth disease, but together, the have synergistic effects.
AI-style programs still have some limitations because they are dependent on their knowledge of a programmed, finite set of rules. In medicine, all of these rules are still unknown. However, Pharnext was still able to assemble a model of Charcot-Marie-Tooth disease using as many known parameters as possible and based on the results of the trial, they were successful. While the utilization of AI in this way still has a long way to go, the activities of Pharnext are hopefully an encouraging glimpse into the future of drug repurposing.
