As clinical research, treatment and care move away from a one-size-fits-all approach, personalized medicine has the potential to improve treatment efficacy, reduce healthcare costs, and save lives, since precision diagnostics allow for a more proactive approach.

Combining the knowledge of an individual’s genetic profile with an understanding of disease mechanisms, doctors can avoid trial and error and recommend treatments most likely to be effective given a person’s unique biology. 

Here are some exciting clinical trials and research approaches at the forefront of precision medicine:

Processing Multi-Omic Data with AI and Machine Learning

Precision medicine wouldn’t be as advanced as it is today without the power of AI and machine learning, which enables the processing and harmonization of massive amounts of genetic information. By evaluating data from millions of individuals, AI tools can help detect trends and assist in the selection of biomarkers that can lead to precision medicine approaches.

An upcoming study for Extensive Stage Small Cell Lung Cancer (ES-SCLC) supported by the National Cancer Institute (NCI) plans to advance a biomarker-driven approach to treatment. By choosing tailored treatments based on a patient’s molecular subtypes, including tumor and immune profiles, the study aims to match patients to more effective therapies and accelerate drug development.¹

Gene Editing Therapies for Sickle Cell Disease

Sickle Cell Disease (SCD) affects approximately 100,000 people in the United States. People living with SCD suffer from episodes of intense pain resulting from the blockage of blood flow. In addition to chronic pain, people with SCD may experience stroke, lung problems, eye problems, infections, and kidney disease.²

There is a dire need for solutions to ease the burden of this condition. An investigational therapy called BEAM-101introduces single-base changes in the promoter areas of the γ-globin genes HBG1 and HBG2, utilizing adenine base editors to disrupt the BCL11A repressor and activate fetal hemoglobin (HbF) expression. This treatment could reduce the symptoms of SCD by increasing HbF in red blood cells over sickle hemoglobin.³

Personalized Screening for Breast Cancer

A new, personalized approach to breast cancer screening could improve detection and intervention. MyPeBS is a randomized, open-label, multicentric study researching the impact of a risk-based breast cancer screening strategy.³

By calculating a polygenic risk score with DNA from saliva, then combining the data with other risk factors like family history and breast density, trial participants will undergo screening for four years. Over a 15-year period, outcomes like incidence of breast cancer will be monitored. If the study advances detection and results in better outcomes, it may mark a significant change in how breast cancer is diagnosed and treated.⁴

Wearable Devices and Machine Learning to Improve Sleep Health

In recent years, wearable devices, such as smart watches and rings, have been used to track sleep data in individuals, which can lead to behavioral changes that promote better sleep.

Now researchers are developing models that may prevent poor sleep. Using algorithms trained on data from 80,811 adults in the UK, researchers examined sleep efficiency through analysis of various sleep and activity parameters. The research demonstrated that sleep patterns in preceding days may predict poorer sleep quality, as well as higher activity in certain pre-sleep periods. This predictive, personalized approach may help people take interventive measures to improve sleep.⁵

Personalized medicine is moving toward revolutionizing prevention, intervention, diagnosis, and care for many diseases and conditions—but there is much work to be done. Many of these approaches require more research to determine efficacy. The high cost of some personalized treatments may impede the delivery of care. Public and private investment in research is critical to ensuring these approaches reach the patients who need them most.

1. https://www.biospace.com/press-releases/bostongene-announces-collaboration-with-swog-cancer-research-network-to-drive-personalized-approaches
2. https://www.nhlbi.nih.gov/health/sickle-cell-disease
3. https://www.clinicallab.com/top-5-clinical-trials-shaping-medicine-in-2025-28300
4. https://clinicaltrials.gov/study/NCT03672331
5. https://academic.oup.com/sleep/advance-article-abstract/doi/10.1093/sleep/zsaf113/8121042