Scleroderma doesn’t come with an easy roadmap, but expert guidance can make all the difference.
Your Scleroderma Questions, Answered is a monthly Q&A column featuring responses from the SRF’s Chief Medical Officer, Dr. Gregory Gordon, who shares guidance grounded in decades of clinical expertise.
In this edition, he explores how artificial intelligence is beginning to reshape drug discovery and scleroderma research—and why many clinical trials focus specifically on patients in the earlier stages of the disease.
Q: Are researchers using artificial intelligence to accelerate scleroderma research or treatment discovery?
A: There is growing interest in using AI to help discover new medical treatments, including for scleroderma. While still in its early stages, AI has the potential to transform the drug discovery process.
Currently, the most common application of AI is virtual screening. Traditionally, drug discovery begins with identifying a protein or enzyme that is malfunctioning and causing disease symptoms. Researchers search for molecules that can interact with it and correct the problem. This process has historically been slow and trial-and-error.
AI accelerates this process by rapidly screening thousands of potential compounds and identifying the most promising ones. These molecules can then be tested in animal models, saving significant time and resources. In the future, AI may go even further, actually designing molecules specifically tailored to target the malfunctioning protein or enzyme.
AI is also being applied to other aspects of drug development, such as optimizing clinical trial design and noting potential safety signals earlier than human reviewers can. The goal is to conduct clinical studies that are faster, safer, and require fewer participants.
Q: Why do some clinical trials exclude patients who have had scleroderma for more than five years?
A: In many clinical trials, only patients who were recently diagnosed are included. This is because many diseases change over time, both in the rate of progression of the disease and the symptoms experienced. For a clinical trial, it is important to have a relatively homogenous population, meaning a group of patients in similar stages of the disease and experiencing similar symptoms. This makes it easier to detect differences in disease activity during the study between those receiving the experimental drug and those receiving a placebo.
In addition, there are specific reasons for limiting disease duration in scleroderma studies. Early-stage scleroderma is characterized mostly by inflammation, while later stage disease is dominated by fibrosis. Most clinical studies focus on enrolling one of these two populations, depending on how the experimental drug is expected to work.
