Breakthrough in the treatment of the deadliest form of cancer

The pancreas performs vital functions in the human body: it produces enzymes for food breakdown and insulin, necessary for regulating blood sugar levels. However, this organ is the site of a disease that is statistically considered the most dangerous among all types of cancer.

Currently, survival rates for patients diagnosed with pancreatic cancer remain low. Less than 15% of them live five years after diagnosis. If, at the time of detection, the disease has already metastasized to other organs, the five-year survival rate does not reach even 4%.

Due to few and non-obvious symptoms, the disease is often diagnosed at late stages, and for several decades, no progress has been observed in its treatment. But this year, as reported by Forskning.no, results of clinical trials were presented at the world's largest oncology conference in the USA, which specialists call a serious step forward in treating this one of the most complex forms of oncology.

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Публикация от Dana-Farber Cancer Institute (@danafarber)

The international study, conducted in sixty clinical centers in the USA, France, Germany, Italy, Spain, and Japan, involved about 500 patients whose pancreatic cancer had already spread to other organs. All of them had previously undergone standard treatment. Patients were randomly divided into two groups: one continued to receive chemotherapy, while the other took the experimental drug daraxonrasib in tablet form.

Researchers assessed treatment efficacy based on median overall survival – an indicator considered more informative in oncology than average life expectancy, as it is less dependent on individual atypical cases – very short or very long lives. For patients receiving chemotherapy, it was 6.6–6.7 months, while for those taking daraxonrasib, it was 13.2 months.

The study showed not only greater efficacy of the new drug but also better tolerability. Due to side effects, only 1.2% of patients were forced to stop daraxonrasib treatment, while in the chemotherapy group, this was done by 11.2% of participants.

Mechanism of Action

As specialists explain, the new drug acts on a protein of the RAS family, which regulates cell growth and division. In 90% of patients with pancreatic cancer, a mutation affects the KRAS gene, as a result of which the corresponding protein remains constantly active and stimulates tumor growth. Daraxonrasib blocks this pathological signal and "switches off" the protein.

Specialists believe that such a targeted approach allows for significant control over the disease, even if the effect remains time-limited for now.

Currently, the new drug has not received registration in either the USA or the European Union. Nevertheless, expanded access programs are already being launched in the United States, allowing critically ill patients to receive it outside of clinical trials.

In Europe, the drug is currently only available within the framework of scientific projects. New clinical trials are planned in the near future, involving patients with newly diagnosed cancer. Scientists plan to compare the efficacy of monotherapy with the new drug and its combination with chemotherapy. The exact timing for the widespread introduction of the drug into practice has not yet been determined, as more data on its action at different stages of the disease is required.

Researchers suggest that the potential of daraxonrasib is not limited to pancreatic cancer alone. Since KRAS mutations are the driver of many oncological processes, researchers are studying the possibility of using daraxonrasib for other diagnoses, particularly in lung cancer. This offers hope for creating more effective treatment protocols for a wide range of patients in the future.

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