After years as a practicing medical oncologist, a few diagnoses still make me shudder. Any new case of pancreatic cancer comes with a pang of dismay and helplessness. The disease can be especially unforgiving for patients: The effects are debilitating, the prognosis is grim and the treatments available are highly toxic and limited in scope.
But recently, pancreatic cancer research witnessed its moonshot moment. A drug called daraxonrasib — brand name Rasonque — was demonstrated in clinical trials to double the overall survival for patients with metastatic pancreatic cancer. On Wednesday, the U.S. Food and Drug Administration approved the pill for patients with this deadliest of cancers.
Decades of work, supported in part by the National Institutes of Health, have come to upend conventional beliefs about the susceptibility of KRAS.
“I have not seen anything like that before in trials we have run in pancreatic cancer,” Brian Wolpin, a medical oncologist at the Dana-Farber Cancer Institute who led the landmark study, told The New York Times. “I just keep repeating, ‘Wow.’”
This giant leap for cancer care and patients comes at a moment when cancer research — and scientific research in general — are flailing in the U.S. due to funding cuts by the Trump administration. The development of daraxonrasib, and its new way of counteracting tumors, is an opportune reminder of the limitless ability and circuitous process of cancer research. And the risks of losing its unrealized ideas and potential are unconscionable.
Daraxonrasib’s remarkable story centers on a smooth-surfaced protein found inside cells known as KRAS. When it is mutated or altered, it can fuel the growth of many types of cancers: pancreas, colon and lung. For years, the dogma was that drugs could not be designed against stopping KRAS because it was a “greasy ball” that appeared impregnable.
Yet decades of work, supported in part by the National Institutes of Health, have come to upend conventional beliefs about the susceptibility of KRAS. And while it has taken years of uneven progress, the chinks in KRAS’ armor have been discovered and manipulated to develop a drug like daraxonrasib that can target and disable it.
The NIH is the largest funder of cancer research in the United States, allocating billions annually to universities and grant awardees.
And now that KRAS has been unlocked, daraxonrasib may very likely be ushering in the beginning of a new era for greater, more impactful and less toxic drug development for many different cancers. As author and oncologist Siddhartha Mukherjee told Oprah Winfrey recently, “The way I think about cancer, all cancer, is it’s like climbing a mountain. And the first crampon you put into the mountain is very crucial, because it’s going to hold up the whole journey upwards. And in this case, the first crampon has been planted. From here on, we’ll know, you know, how does it become resistant? Can we make another medicine? Can we combine it with a third medicine?”
In short, pancreatic cancer is just the start.
But as this door to new consequential cancer drugs begins to open, it’s important to spotlight the Trump administration’s politicized NIH funding cuts, federal health firings and grant terminations that endanger this pipeline’s future.
The NIH is the largest funder of cancer research in the United States, allocating billions annually to universities and grant awardees. The basic science and cancer pathways discovered here undergird the work of pharmaceutical companies to develop therapeutic drugs. This was also seen in the story of daraxonrasib, where NIH-supported academics did the foundational legwork that allowed the pharmaceutical industry to stymie KRAS with a novel approach. In fact, 99.4% of new drugs approved by the FDA between 2010 and 2019 resulted from findings by NIH research.
“Pushing science forward will require reliable federal funding to make the next generations of these drugs. The recent funding cuts and uncertainty around if existing grants will be paid out is killing research at a time when we need it most,” Suneel Kamath, an oncologist at Cleveland Clinic, told me. “We’re studying better ways to treat pancreatic cancer to help people live longer with this terrible disease. There shouldn’t be anything controversial about that.”
Though Congress and courts have fortunately stood in the way of the deepest funding cuts, the downstream effects can already be felt through delayed treatments and lost scientific potential. For many cancer patients and families who are fighting daily for a few more quality months, this is an unthinkable ask.
The emergence of daraxonrasib teaches us that the timeline for lifesaving drugs is already so long and fraught with setbacks and progress. Obstructing that road further is wholly gratuitous, when at its end, there are boundless benefits that can be measured in lives prolonged and saved.
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