When Stephen Huff went for a test because of shortness of breath, he was 29 years old, a non-smoker and preparing for his wedding. Instead of an explanation for his symptoms, he received a diagnosis of stage four lung cancer. Tumors were found in his lungs, neck, spine, liver and brain.
The next study changes the direction of treatment. Analysis of more than 300 genes in a tumor sample revealed a change in ALK - a gene that is involved in the control of cell growth. So Huff was given alectinib, a drug that targets the specific genetic change.
“My initial study showed that almost every tumor either shrank or disappeared completely,“ Huff told “New Scientist“.From the organ to the mutation
This approach is called precision oncology. In traditional treatment, the type of cancer is usually determined by where it originated - the lung, bowel, skin or pancreas. Genomic profiling is about which change in DNA is causing the tumor to grow.
Prof. David Thomas of the University of New South Wales describes this change as a logical step because “cancer is fundamentally a genetic disease“. Instead of treating all patients with a tumor in the same organ, a drug is being sought that targets a specific mechanism in the cancer cells.
In a study of almost 4,000 patients with advanced forms of cancer, including lung, pancreas, brain, ovary, bowel and breast, more than a third had changes that allowed a recommendation for targeted therapy. Patients in whom a match was found between the mutation and the drug lived almost twice as long as those without such a match.
New opportunities in pancreatic cancer
One of the clearest examples is pancreatic cancer. According to Prof. Thomas, more than 30% of patients with this type of cancer who received an appropriate recommendation for treatment are alive five years later. In patients without a match, this proportion is 4%.
On August 26, 2026, the US Food and Drug Administration approved daraconrasib for adults with metastatic pancreatic cancer who have already received at least one systemic treatment. In a trial with 500 participants, the median overall survival was 13.2 months with the new drug compared to 6.7 months with standard chemotherapy.
The result does not mean that the drug is a universal solution. The approval concerns a specific group of patients, and the treatment has side effects, including skin and tissue toxicity, inflammation of the oral cavity, diarrhea and a risk of lung inflammation.
The approach also enters brain tumors
Vorasidenib - a drug that targets mutations in the IDH1 and IDH2 genes - is used in some brain tumors. In a phase 3 clinical trial in patients with low-grade gliomas, the median time without disease progression was 27.7 months with voracidenib compared with 11.1 months with placebo.
The data show a benefit for a specific group of patients, but do not prove that all brain tumors can be affected in this way. In many types of cancer, no mutation has yet been discovered for which there is an effective drug.
Genomic profiling in childhood
The Australian Zero Childhood Cancer program applies even more detailed analysis to children with cancer. The entire genome of the tumor, RNA, chemical changes in DNA and the genetic code with which the child is born are examined. In some cases, cancer cells are grown in the laboratory or in disease models to test different drugs.
In a study of 384 children, the two-year progression-free survival rate was 26% for those who received treatment recommended by the genomic analysis. With standard therapy, the rate was 12%. However, the program includes children with high-risk tumors, and the results cannot automatically be transferred to all oncological diseases.
The main obstacle to precision oncology remains access. Genetic testing can find a suitable target, but not everywhere has a paid test, an affordable drug or the opportunity to enroll the patient in a clinical trial. In addition, tumors can develop resistance and start growing again.
That's what happened to Huff five years after starting treatment. By then, a new drug against ALK - lorlatinib - was already available. Four years after switching therapies, he continues to work and lives with his wife and two children.
Sources: newscientist.com