Precision medicine offers a path to a better life for patients with diabetes, says ADA President, Medicine & Science Louis H. Philipson, MD, PhD, FACP, who delivered his presidential address Sunday morning at the Scientific Sessions.
Dr. Philipson discussed the future of precision medicine and what medical professionals and advocates can do now to help actualize its potential power. To illustrate that potential power, Dr. Philipson told the story of one of his patients at the Kolver Diabetes Center at the University of Chicago Medicine, where he is director.
Lily Jaffe was diagnosed with type 1 diabetes just after birth, but when she was six the Kolver Center team discovered that she had a mutation that responded to the sulfonylurea glyburide. She has not taken insulin in more than 12 years.
“That is the ultimate promise of precision medicine: Finding an inexpensive drug that exactly fixes a problem to simplify and improve treatment,” Dr. Philipson said. “It’s not a cure, it’s not without other problems, but it’s spectacularly better than what we had before.”
Precision medicine in diabetes requires a deep understanding of disease mechanisms and biomarkers as well as engagement with patients and clinicians to employ those biomarkers and genetic insights to realize best therapies and best outcomes, Dr. Philipson said. His roadmap to the future of precision medicine includes integrating insights from human islet and organ research as well as population-wide data.
Precision medicine’s greatest successes to date have come for patients with monogenetic diabetes, Dr. Philipson said. He called for expanded genetic testing and making it the standard of care as a way to not only identify patients with the more rare forms of diabetes, but also as a way to advance research for all types of diabetes, including the interplay between the environment and genetics that could lead to diabetes and cardiometabolic disease.
“Do these the insights into monogenic diabetes help most people with diabetes? Well, perhaps no, not directly. But they do illuminate key pathways, receptors, enzymes, and transcription factors that involve insulin, its synthesis and release, and its action that allow us to better understand these metabolic control systems in humans,” he said.
Research into diabetes precision medicine continues around the world. But that research requires more funding, said Dr. Philipson, who highlighted the work of the Pathway to Stop Diabetes® program to fund research to advance precision medicine. He also emphasized the importance of taking detailed family histories about diabetes, as that history can determine the value of genetic testing for the patient and other family members.
“I ask you to resolve to see every person with diabetes in your practice with nuance,” Dr. Philipson said. “Why does this person in front of me have diabetes? What about the family? Did you think about the family history? Can you incorporate such thinking right now? Starting to incorporate the key elements of precision medicine can be a transformational event for both you and your patients.”