Health
Millions Suffer from Untreatable Rare Diseases – AI Offers Hope
United States: Presently almost more than seven thousand individuals are affected by rare and undiagnosed diseases all over the world.
Despite the fact that each of these conditions is extremely rare as a genetic disorder, the total burden of these diseases is immense due to the fact that they afflict some three hundred million people worldwide.
However, while only five to seven percent of these conditions have a drug that has been approved by the Food and Drug Administration (FDA), the majority of these patients continue to be either untreated or undertreated.
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Millions Suffer from Untreatable Rare Diseases – AI Offers Hope. Credit | Shutterstock
It remains one of medicine’s toughest tasks to create new drugs, but one AI tool can bring new therapies from existing drugs, bringing light to underserved patients and physicians, news.harvard.edu reported.
The AI model, referred to as TxGNN, is the first such model designed to find drugs for rare diseases and disorders with no treatments.
From the existing cocktails for over 17,000 diseases, many of which have no known cure, it pinpointed drug candidates. To date, this is the biggest number of diseases that a single AI model is capable of diagnosing.
The researchers say that this is an indication that the model can be applicable to even more diseases other than the 17,000 that it was tested on in the experiments.
To date, just 5–7% of rare and neglected diseases have an FDA-approved drug. The rest remain either untreated or undertreated. Now a team of scientists developed #TxGNN which predicts drug candidates for conditions with limited or no treatment options.https://t.co/KqJoEnWBiq
— Genetic Engineering & Biotechnology News (@GENbio) September 26, 2024
More about the research
The work, described on Wednesday in Nature Medicine, was done by members of the Harvard Medical School. The tool has been made freely available by the researchers and they want clinicians-scientists to use it in their quest for therapies for diseases with either no or few treatment solutions.
The new tool has two key attributes a first one that lists the treatment candidates with likely side effects as well as a second one that provides a rationale for the decision, news.harvard.edu reported.
In total, the tool found drugs for nearly 8,000 out of 17,080 diseases, including untreatable diseases, from FDA-approved medicines and experimental drugs currently under clinical trials.