Rectal Cancer “Vanished” in All 18 in Clinical Trial: An “Astonishing” and “Unheard-Of” Result

(p. A18) It was a small trial, just 18 rectal cancer patients, every one of whom took the same drug.

But the results were astonishing. The cancer vanished in every single patient, undetectable by physical exam, endoscopy, PET scans or M.R.I. scans.

Dr. Luis A. Diaz Jr. of Memorial Sloan Kettering Cancer Center, an author of a paper published Sunday [June 5, 2022] in the New England Journal of Medicine describing the results, which were sponsored by the drug company GlaxoSmithKline, said he knew of no other study in which a treatment completely obliterated a cancer in every patient.

“I believe this is the first time this has happened in the history of cancer,” Dr. Diaz said.

Dr. Alan P. Venook, a colorectal cancer specialist at the University of California, San Francisco, who was not involved with the study, said he also thought this was a first.

A complete remission in every single patient is “unheard-of,” he said.

. . .

Dr. Kimmie Ng, a colorectal cancer expert at Harvard Medical School, said that while the results were “remarkable” and “unprecedented,” they would need to be replicated.

The inspiration for the rectal cancer study came from a clinical trial Dr. Diaz led in 2017 that Merck, the drugmaker, funded. It involved 86 people with metastatic cancer that originated in various parts of their bodies.

. . .

Tumors shrank or stabilized in about one-third to one-half of the patients, and they lived longer. Tumors vanished in 10 percent of the trial’s participants.

That led Dr. Cercek and Dr. Diaz to ask: What would happen if the drug were used much earlier in the course of disease, before the cancer had a chance to spread?

. . .

Perhaps, Dr. Cercek and Dr. Diaz reasoned, immunotherapy with a checkpoint inhibitor would allow such patients to avoid chemotherapy, radiation and surgery.

Dr. Diaz began asking companies that made checkpoint inhibitors if they would sponsor a small trial. They turned him down, saying the trial was too risky. He and Dr. Cercek wanted to give the drug to patients who could be cured with standard treatments. What the researchers were proposing might end up allowing the cancers to grow beyond the point where they could be cured.

“It is very hard to alter the standard of care,” Dr. Diaz said. “The whole standard-of-care machinery wants to do the surgery.”

Finally, a small biotechnology firm, Tesaro, agreed to sponsor the study. Tesaro was bought by GlaxoSmithKline, and Dr. Diaz said he had to remind the larger company that they were doing the study — company executives had all but forgotten about the small trial.

Their first patient was Sascha Roth, then 38.

. . .

Soon, she was scheduled to start chemotherapy at Georgetown University, but a friend had insisted she first see Dr. Philip Paty at Memorial Sloan Kettering. Dr. Paty told her he was almost certain her cancer included the mutation that made it unlikely to respond well to chemotherapy. It turned out, though, that Ms. Roth was eligible to enter the clinical trial. If she had started chemotherapy, she would not have been.

. . .

After the trial, Dr. Cercek gave her the news.

“We looked at your scans,” she said. “There is absolutely no cancer.” She did not need any further treatment.

“I told my family,” Ms. Roth said. “They didn’t believe me.”

But two years later, she still does not have a trace of cancer.

For the full story, see:

Gina Kolata. “Study on Rectal Cancer Results in Complete Remission.” The New York Times (Tuesday, June 7, 2022): A18.

(Note: ellipses, and bracketed date, added.)

(Note: the online version of the story has the date June 5, 2022, and has the title “A Cancer Trial’s Unexpected Result: Remission in Every Patient.” The online version of the article says that the title of the print version was “Rectal Cancer Drug Trial Results in Complete Remission.” But my National edition of the article had the title “Study on Rectal Cancer Results in Complete Remission.”)

The paper mentioned above in The New England Journal of Medicine is:

Cercek, Andrea, Melissa Lumish, Jenna Sinopoli, Jill Weiss, Jinru Shia, Michelle Lamendola-Essel, Imane H. El Dika, Neil Segal, Marina Shcherba, Ryan Sugarman, Zsofia Stadler, Rona Yaeger, J. Joshua Smith, Benoit Rousseau, Guillem Argiles, Miteshkumar Patel, Avni Desai, Leonard B. Saltz, Maria Widmar, Krishna Iyer, Janie Zhang, Nicole Gianino, Christopher Crane, Paul B. Romesser, Emmanouil P. Pappou, Philip Paty, Julio Garcia-Aguilar, Mithat Gonen, Marc Gollub, Martin R. Weiser, Kurt A. Schalper, and Luis A. Diaz. “PD-1 Blockade in Mismatch Repair–Deficient, Locally Advanced Rectal Cancer.” New England Journal of Medicine 386, no. 25 (June 23, 2022): 2363-76.

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