How AI could transform cancer treatment and medicine

Researchers are now using artificial intelligence to help tailor cancer treatments and uncover overlooked uses for existing medicines. Other systems can search microscopic images or identify subtle biological signals that people could easily miss.

Some of these technologies have already helped patients. Others remain in clinical trials or research labs, so we need to be careful about separating promising science from treatments you can actually receive today.

Still, what researchers are accomplishing would have been difficult to imagine a few years ago. Here's where AI is already changing medicine and what you should know before trusting it with your health.

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CANCER VACCINE PREVENTS DEADLY MELANOMA FROM RETURNING OR SPREADING IN LANDMARK TRIAL

One of the biggest recent developments comes from Moderna and Merck. On Aug. 19, the companies announced positive topline results from a Phase 3 melanoma trial. The study tested intismeran autogene, also known as V940 or mRNA-4157, alongside Keytruda. The trial enrolled 1,137 people with high-risk melanoma that surgeons had completely removed. The combination met its primary endpoint for recurrence-free survival. It also met a key secondary endpoint measuring distant metastasis-free survival.

Merck and Moderna said this was the first positive Phase 3 readout for an individualized neoantigen therapy. It was also the first positive Phase 3 result for an mRNA-based cancer therapy. That sounds complicated, but the basic idea is fascinating. Researchers start with a sample of a patient's tumor. They analyze its unique mutations and use an algorithm to select targets that may help the immune system recognize the cancer. The resulting individualized therapy can encode up to 34 neoantigens. Moderna has also said the V940 program uses integrated AI algorithms during the development process.

The company then creates an mRNA treatment based on the selected targets. You may have seen this approach called a personalized cancer vaccine. Moderna and Merck currently describe intismeran as an individualized neoantigen therapy. The goal is to train the immune system to recognize characteristics unique to that patient's cancer.

There is plenty of reason for excitement, but there is also an important limitation. Merck and Moderna have announced only topline results from the Phase 3 trial so far. The companies plan to present the full findings at an international medical meeting and share them with regulators. The study also continues to track overall survival.

Earlier results offer additional context. In a smaller Phase 2b study with longer follow-up, intismeran plus Keytruda reduced the risk of recurrence or death by 49% compared with Keytruda alone. It also reduced the risk of distant metastasis or death by 59%. Those earlier results came from a much smaller patient group. That makes the larger Phase 3 trial an important step forward. Still, intismeran remains investigational. The FDA has not approved intismeran as a melanoma treatment.

Developing a new medicine can take years. Another group of researchers is asking a different question: What if a useful treatment already exists? Dr. David Fajgenbaum co-founded the nonprofit Every Cure to pursue that possibility. According to Every Cure's 2025 annual report, about 18,000 recognized diseases exist worldwide. The organization says roughly 4,000 have FDA-approved medications. That leaves an enormous number of diseases with limited treatment options.

Every Cure uses AI to scan biomedical knowledge and look for connections between existing medicines and other diseases they might potentially treat. The organization says its system can generate tens of millions of predictions in less than a day. Researchers then examine the most promising possibilities.

The federal Advanced Research Projects Agency for Health, or ARPA-H, is backing this approach through a project called MATRIX. MATRIX uses machine learning and artificial intelligence to predict which FDA-approved drugs could potentially treat other diseases. Researchers then validate promising candidates through laboratory or clinical work. AI does not prove that a drug will work for another illness. Instead, it can help researchers decide where to look next. That could dramatically narrow an otherwise enormous search.

Fajgenbaum has seen firsthand what finding a new use for an existing drug can mean. Kaila Mabus developed multicentric Castleman disease at 13 and became severely ill despite chemotherapy. In 2020, her doctors tried ruxolitinib, a drug already used for certain blood disorders but not FDA-approved for Castleman disease. She began improving within months and was declared in remission in January 2021. AI did not identify her treatment, but her case shows why Every Cure wants to use AI to uncover promising drug-disease connections much faster and on a far larger scale.

At Columbia University Fertility Center, artificial intelligence has taken on a very different challenge. Researchers developed the Sperm Tracking and Recovery system, known as STAR. It combines high-speed imaging with an AI detection model and microfluidics.

STAR was designed for patients with azoospermia or cryptozoospermia, conditions where sperm may appear absent or exist in extremely small numbers. The system examines a semen sample far more thoroughly than a person could reasonably do by hand. STAR can capture and process about 1.1 million images every hour. Its AI model examines frames for possible sperm cells.

When the system confirms one, a microfluidic mechanism isolates the cell. Doctors may then use the recovered sperm for fertility treatment or freeze it for later use. In one validation sample, embryologists searched for two days without finding sperm. STAR found 44 sperm in about an hour. That is exactly the type of repetitive search where AI can shine. A human eye can get tired. A computer can keep examining frame after frame.

NEW BLOOD TEST DETECTS 90% OF AGGRESSIVE PROSTATE CANCER CASES, BEATING CURRENT SCREENINGS

This technology has moved beyond a research demonstration. Columbia says STAR achieved its first reported pregnancy in March 2025. The couple involved had spent nearly two decades trying to conceive. STAR found and recovered sperm that conventional examination of the same sample had missed. The pregnancy later resulted in a healthy delivery.

That does not mean STAR will work for everyone. Columbia currently reports that sperm are found in about 28% of patients who previously received an azoospermia diagnosis. The center says about 20% of mature eggs fertilize with STAR-recovered sperm. Around 18% of those fertilized eggs develop into good-quality embryos for transfer or freezing.

Those rates are lower than typical IVF or ICSI. The patients using STAR often face especially difficult fertility problems, which helps explain the difference. Even so, the technology shows how finding one tiny biological clue can completely change the options available to a patient.

Researchers at the University of Hong Kong are exploring another possibility. Their AI-based tool, called CardiOmicScore, analyzes molecular information found in blood. The researchers used large-scale data from the UK Biobank. Their system examined 2,920 circulating proteins and 168 metabolites. It also incorporated genomic information.

CardiOmicScore uses deep learning to estimate the future risk of six cardiovascular diseases. Those include coronary artery disease, stroke and heart failure. The system also evaluates atrial fibrillation, peripheral artery disease and venous thromboembolism. Researchers found that the approach improved risk prediction when combined with standard clinical information. In some cases, CardiOmicScore could flag elevated risk as much as 15 years before symptoms appeared.

Think about what that could eventually mean. Instead of discovering cardiovascular disease after symptoms develop, doctors might get a warning while there is still more time to intervene. However, CardiOmicScore remains a research development. You cannot walk into your doctor's office today and request it as a routine screening test.

At UCLA, scientists are taking another approach to personalized cancer treatment. They create tiny laboratory-grown replicas of patient tumors called organoids. Researchers can expose those organoids to different drugs and monitor what happens. Their platform combines 3D bioprinting with advanced imaging and artificial intelligence. AI helps researchers process the large amount of imaging data generated as the organoids respond to treatment. The system can track thousands of individual organoids.

That allows researchers to examine how different parts of a tumor respond to various drugs. This could be valuable because cancer can behave differently from one patient to another. Even cells within the same person's tumor can respond differently to treatment. Eventually, researchers hope this type of technology could help identify therapies that better fit an individual patient's cancer. For now, UCLA continues to develop and validate the platform.

The possibilities for AI in medicine extend beyond blood samples and microscopes. Researchers are also studying what computers can learn from the way we speak. A Perspective published Sept. 4 in npj Digital Medicine examined voice biomarkers for ALS and Parkinson's disease.

Neurodegenerative diseases can cause measurable changes in speech. Researchers believe AI could potentially analyze those changes and help monitor disease progression. For ALS, the authors see particular potential in tracking changes that affect speech and swallowing. However, this field remains early.

At the time of publication, no speech or voice-derived endpoint for ALS or Parkinson's disease had received qualification from the FDA or European Medicines Agency. One ALS speech analytics platform has received FDA Breakthrough Device designation. That status can help speed regulatory review, but it does not amount to FDA marketing authorization. Researchers see real potential here. The clinical proof still has more catching up to do.

PATIENTS REMAIN CANCER-FREE NEARLY 3 YEARS AFTER RECEIVING EXPERIMENTAL IMMUNOTHERAPY

You may encounter AI in your healthcare without ever opening an AI chatbot.

A laboratory could use it while analyzing a tumor. A fertility clinic might use it to search for something the human eye missed. Researchers can also use AI behind the scenes to find treatments worth investigating. The key question for you is how much evidence supports the specific technology being used.

A university research project sits at a very different stage from a medical device that has gone through clinical testing and regulatory review. You should also understand how much human oversight remains involved. AI can help doctors process information and identify patterns.

Your healthcare decisions still deserve qualified medical judgment based on your individual circumstances.

That is why asking a few questions can help when AI becomes part of your care.

Medical AI can be useful. You still deserve to know how it affects you.

Find out what role the technology plays in your care. Does it analyze information for a doctor? Does it flag something for additional review? The phrase "AI-powered" can cover a wide range of technology, so ask for a simple explanation.

Ask whether a doctor, specialist or laboratory professional checks the AI's findings before anyone makes a decision. Human review becomes especially important when a result could affect treatment or diagnosis.

Ask whether the FDA has cleared or approved the technology when regulatory authorization applies. Also find out what type of research supports it. Early research can show promise while still leaving important questions unanswered.

Medical AI may rely on sensitive health information. Ask how your provider stores that data and who can access it. You can also ask whether your information may be used to improve or train an AI system. For more on transparency around artificial intelligence in healthcare, see our CyberGuy guide on what patients should know about AI disclosure. This article provides general information and does not replace advice from your healthcare professional.

What I find so interesting here is how AI can help doctors and researchers see things that would be incredibly difficult to find on their own. A system can search more than a million microscope images in an hour looking for a single sperm cell. Another can sift through huge amounts of medical research to find a possible new use for an existing drug. Researchers are even developing cancer treatments around the unique mutations inside one patient's tumor. That is pretty remarkable. But I also think we have to be careful not to let the excitement around AI move faster than the science. The melanoma Phase 3 results are encouraging, but we still need to see the complete data. Several of the other technologies in this article remain experimental or available only in limited settings. For me, that is where this gets really interesting. AI may help doctors find answers faster and uncover possibilities they might otherwise miss. What I want to see next is how often those discoveries translate into treatments that actually make people healthier and improve their lives.

If AI uncovered a treatment your doctor had never considered, how much evidence would you need before you felt comfortable trying it? Let us know by writing to us at CyberGuy.com.

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