I’ve spent my career working on personalized and precision medicine and have seen outstanding advances in the field which continue to be accelerated by the use of AI. However, most of my friends don’t know what that really means or how it can benefit them when they become patients – in spite of doctors encouraging the use of genetic testing as the field of precision medicine continually evolves.
In a 2023 AMA interview for “What Doctors Wish Patients Knew”, Dr. Jordan Laser, MD and chair of the Personalized Medicine Committee for the College of American Pathologists, said “the field is constantly expanding as to where the appropriate clinical application of genetic testing exists, so I would always encourage someone to ask about genetic and genomic testing for any disease.”
So, what does precision or personalized medicine mean?
Precision medicines are developed for specific subgroups of patients who can usually be identified using genetic or genomic testing for biomarkers developed as companion diagnostic tests. These drugs are generally more effective in the subgroups of patients, and patient subgrouping improves clinical trial efficacy. Personalized medicines take that a step further, and are fully individualized to the patients.
What advances have been made so far?
Thus far, we’ve seen the biggest advances in precision medicine in oncology. Herceptin (trastuzumab) was the first drug approved (1998) with required biomarker testing; it’s only approved in HER2-positive breast cancers. Since then, we’ve seen an explosion of approvals in oncology and a shift toward developing these drugs with companion diagnostics. The importance of this was famously emphasized when Merck beat Bristol Myers Squibb (BMS) to be a first-line non-small cell lung cancer therapy because they developed Keytruda with a companion diagnostic testing for PD-L1, whereas BMS developed Opdivo for a broad population and failed to show benefit compared to chemotherapy.
Drug makers have also started to develop precision medicines for autoimmune disease, though we’re still waiting on approved drugs that use biomarkers other than auto-antibodies, which will expand the subtypes of autoimmune disease which can benefit from precision medicine.
Additionally, the field has expanded to include personalized medicines such as Kymriah and Yescarta in oncology, which take a patient’s own T-cells and tailor therapies based on these, and similar approaches are in clinical trials for autoimmune disease developed by companies like Kyverna and Cabaletta.
AI continues to drive advancements in precision medicine and awareness
In 2024, precision/personalized medicines made up 38% of new molecular therapeutic approvals, bringing us to a full decade of having at least a quarter of drug approvals be in this category. This is striking because just over a decade ago, these approvals made up less than 10%.
The root of developing precision medicines is using AI to find patient subgroups. Although we will never use biomarkers or health data to target advertising, I’m thrilled to see the use of AI at the GTM stage of drug development. I’ve both heard about and experienced the need for self-advocacy as a patient, and I believe AI can help my friends (and others!) become aware of novel therapeutics so they can ask their doctors about them.