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  • Writer's pictureDr. Robert A. Nagourney, MD

Treatment for Stage 4 Pancreatic Cancer May Be at Hand

It’s Just Probably Not What You Are Being Offered


Few diseases strike greater fear into the hearts of patients than pancreatic cancer, with over 62,000 new diagnoses and 49,000 deaths in 2022. The overall five-year survival of 11% falls to 1-2% for advanced disease (Stage III&IV), the most common presentation.


A headline recently echoed by PANCAN showing an improvement in 5-year survival from 11% to 12% rings a bit hollow when patients with advanced disease realize that they still confront an 88% likelihood of death from their disease.


It is evident that better ways to treat this disease are needed, and our laboratory can offer hope to those with the most advanced stages. We are now seeing more and more advanced pancreatic cancer patients as they come to recognize the futility of available therapies for advanced disease.


One such patient arrived on October 31st, 2022. With symptoms worsening over several months, he became jaundiced and required the placement of a stent for bile drainage. A biopsy confirmed pancreatic cancer and the major cancer center where he obtained an opinion recommended the “gold-standard” chemotherapy combination FOLFIRINOX.


He and his son came to my office to discuss options. He had a large pancreatic mass, extensive liver metastasis, deep venous thrombosis (DVT), and bilateral pulmonary emboli (blood clots that had traveled to the lung), which are common in advanced disease. I must admit, in retrospect, it was unclear to me whether I would be able to offer assistance.


Recognizing that there was no time to lose, I immediately referred him to my hepatobiliary surgeon, who conducted a surgical biopsy the next day.


The results were very instructive; first, the patient was not sensitive to the FOLFIRINOX regimen that had been offered. Second, he showed activity for new classes of drugs that target P53 and MEK/ERK, associated with mutations that are common in this cancer.


What was striking was the activity for novel 3-drug combinations that would not even be on the radar screen for most oncologists. Yet, this was the exact combination that he needed and despite it not being widely used, each of the individual drugs are approved for this disease.


The patient was too ill to wait. With pain, weight loss, nausea, venous thromboses, pulmonary emboli, and a rapidly deteriorating performance status, we crafted his treatment schedule immediately and began therapy one week after our first meeting.


As one might imagine, he had a bit of a tough time, even on reduced doses. The only good news after the first two weeks was that he was still alive.


Then, with the completion of his third cycle of two weeks on and one week off, he returned to my office literally a “new man”. Gone was the pain, nausea, weight loss, pallor, and the sense of doom. Here instead was a man who was relaxed, comfortable, and looking healthy. Though he had hair loss from one of the drugs, he was eating well, gaining weight, free of pain, and back to normal daily activities.


I marveled that barely six weeks after meeting a man who I felt might be too ill to treat, his liver function tests were normal and his CA 19.9, the most useful measure of this disease, had fallen from 186,000 to 27,000, dropping roughly 50% with each cycle.


Our conversation in less than two months had gone from consideration of hospice care and pain management to whether he might be allowed a glass of wine or beer, our ability to taper the anticoagulant Lovenox, and his desire to return to the gym.


There are several messages we can take away from this. The first is that every patient deserves the chance to get better. The second is that the major centers are offering drug combinations that are simply not effective enough for their indiscriminate use. The third is that patients must be offered the treatment that is right for them. Finally, our ability to craft a multi-drug combination enabled this patient to receive a life-saving therapy without undue toxicity that was definitely not FOLFIRINOX.


Pancreatic cancer is no different from other diseases; there are patients who will do well and those who will not. Today’s major institutions and “so-called” patient-support organizations lump everyone together, hoping their average patient/average outcome dictum will prevail.


Patients must take charge of their own disease and get the treatment that works for them. There are no good or bad diseases, just good or bad treatments. Patients must be selected for good treatments and avoid bad treatments. That’s the mission of the medical oncologist!


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