Long considered one of the most formidable diagnoses in medicine, pancreatic cancer could finally see the opening of a new therapeutic avenue. Until now, treatment options were rare and ineffective, and decades of unsuccessful clinical trials had finally convinced part of the scientific community that the biological obstacles were insurmountable.
A turning point now seems to be emerging with an experimental drug, daraxonrasib, currently being evaluated by the American health authorities. This treatment is the first to demonstrate a significant improvement in the survival of patients suffering from this particularly aggressive cancer. Its mechanism targets a key protein, involved not only in the majority of pancreatic tumors, but also in many lung and colon cancers, among the deadliest.
Some specialists no longer hesitate to speak of a major advance, potentially comparable to the arrival of immunotherapy around fifteen years ago. The discovery of this treatment was not a walk in the park, reveals an article in the New York Times which looks back on the researchers’ journey punctuated with failures.
At the heart of this revolution is the KRAS protein, identified for several decades as an essential driver of tumor growth. Described as a “greasy ball” because of its smooth and difficult-to-target surface, it was deemed undruggable, that is, inaccessible to any medicinal intervention. All attempts to neutralize it had so far failed.
The situation has begun to evolve thanks to the work of several teams of researchers, supported by public and private funding. In 2013, a key discovery showed that there was actually an exploitable flaw in this protein. At the same time, other scientists have developed an innovative approach based on molecular glues, capable of linking proteins together to deactivate KRAS.
Imperfect treatment
These advances have enabled biotechnology companies to design new treatments, including daraxonrasib. This medication works by blocking the KRAS protein even when it is active, thereby interrupting the signal for cancer cells to proliferate. Initial trials showed a reduction in tumors in some patients, with side effects considered generally manageable.
In an advanced clinical trial involving patients with metastatic pancreatic cancer already treated with chemotherapy, daraxonrasib increased median survival to more than 13 months, compared to less than 7 months with traditional treatments. A notable result for a disease in which only 3% of affected patients survive up to five years when the cancer is disseminated.
However, the treatment remains imperfect. It does not cure the disease, its effectiveness decreases over time and not all patients respond to it. In addition, its side effects – fatigue, digestive disorders, skin rashes – can be serious. But in the absence of truly effective alternatives, it represents tangible hope.
For patients, this hope translates into reality. Some people are now living well beyond initial predictions thanks to this experimental treatment. If no promise of recovery is made, the prospect of additional years of life, once unimaginable, is now possible.
While dozens of similar drugs are now in development, researchers remain cautious but optimistic. This breakthrough may mark the start of a new era in the fight against some of the most difficult to treat cancers.
