Precision Oncology Offers New Hope Against Cancer

Cancer treatment is entering a new era in which doctors can target the genetic mutations driving a tumor rather than relying solely on where the cancer develops. This approach, called precision oncology, is helping some patients with advanced cancers live years longer than previously expected.

Stephen Huff was just 29 and preparing to get married when he learned he had stage 4 lung cancer. Despite being a former professional baseball player who had never smoked, the disease had already spread to his brain, liver, spine and other parts of his body.

"I put on a brave front, but deep down I was terrified. I didn't know if I'd be here in six months," Huff told New Scientist.

At his mother's urging, Huff underwent comprehensive genomic profiling, a test that examines hundreds of genes in a tumor to identify mutations that may respond to targeted drugs.

The test revealed an alteration in a gene called ALK, allowing doctors to prescribe alectinib, a drug designed to block the cancer's growth signals.

"My first scan showed that basically every tumour site had either shrunk or gone away completely," Huff said.

The treatment controlled his cancer for five years before the disease began progressing again. By then, another targeted drug, lorlatinib, was available. His tumors responded again, and nine years after his diagnosis, Huff is married, has two children and reports feeling well.

Traditional chemotherapy attacks rapidly dividing cells, including healthy ones, often causing significant side effects.

Precision oncology uses genetic testing to identify specific abnormalities fueling a patient's cancer. Doctors can then select drugs designed to interfere with those abnormalities.

"Being able to identify exactly what makes a cancer tick is having an incredible impact," says David Thomas, a cancer researcher at the University of New South Wales in Australia.

An Australian study involving nearly 4,000 patients with advanced cancers found that more than one-third had genetic changes that could potentially be targeted with treatment.

Patients who received treatments matched to their tumors lived substantially longer than those who were not matched, although the study was not a randomized trial and cannot establish that genetic matching alone caused the difference.

The findings helped launch Australia's nationwide precision oncology program in 2023, offering genomic testing and treatment recommendations to patients with difficult-to-treat cancers.

Precision oncology is also opening treatment options for some of the most challenging cancers.

Pancreatic cancer. Genetic testing can identify a small group of patients whose tumors may respond to immunotherapy or other targeted treatments. Researchers are also developing drugs against KRAS mutations, which are common in pancreatic cancer.

Brain cancer. A targeted drug called vorasidenib can delay tumor progression in certain low-grade gliomas carrying IDH1 or IDH2 mutations. Its benefits do not extend to all types of brain cancer.

Blood cancers. Imatinib, one of the earliest targeted cancer drugs, transformed treatment for chronic myeloid leukemia. Long-term survival has improved dramatically since its introduction.

These advances do not mean every patient will benefit. Some tumors lack mutations that can be treated with existing drugs, while others eventually develop resistance.

Despite its promise, comprehensive genomic profiling remains underused in the United States. Research cited by New Scientist suggests that only about one in four Americans with advanced cancer receives this type of testing.

Cost, insurance coverage and access to specialized cancer centers can be barriers.

Patients diagnosed with advanced or recurrent cancer should ask their oncologist whether comprehensive biomarker testing could identify additional treatment options or clinical trials.

Testing is particularly important for certain cancers, including advanced non-small cell lung cancer, where the results can directly guide treatment.

However, finding a genetic mutation does not guarantee that an effective drug is available. Some targeted treatments are approved only for specific cancers, and others remain experimental.

Still, precision oncology is giving some patients new options when conventional treatments offer little hope.

"Life is really good; I'm just trying to cherish it for as long as I can," Huff said.