In 2020, nearly 340,000 people died from rectal cancer. Now a new treatment giving hope for beating the disease—and maybe for other forms of cancer as well—could be available within a year.
A recent trial, conducted by researchers at Memorial Sloan Kettering Cancer Center and published in the New England Journal of Medicine on June 5, produced results that are all but unheard of: 14 out of 14 patients are in remission for up to two years after treatment . Four others are still undergoing treatment
Even better, the drug dostarlimab has shown no side effects or downsides — unlike other treatments. “Radiation is effective in eliminating the tumor but it impacts the patient negatively. Up to thirty percent of those who undergo surgery need colostomy bags,” Dr. Andrea Cercek, an oncologist at Sloan Kettering and a co-author of the study, told The Post. ” [Radiation] also can result in sexual dysfunction. They get better but they are not functionally the same.”
According to Cercek, the likelihood of the 14 case studies being in remission is a trillion to one. Based on that, she said, “I am very optimistic about the success.”
She hopes to see the drug approved by the FDA “as soon as possible.”
But Dr. Siddhartha Mukherjee, a biologist, physician and author of the Pulitzer-Prize winning “Emperor of all Maladies: A Biography of Cancer,” cautions that it might be too soon to break out the Champagne.
“I was elated that this drug has such an incredible response rate,” Mukherjee told The Post. “I would like to see it repeated in a large trial. It is the kind of result that makes people in our field excited but also apprehensive.”
He also cautioned that the treatment, which he believes could be in wide use within a year, was targeted for a very specific population.
“The patients [in the study] were carefully selected and are dealing with a specific form of cancer that is receptive to this medication,” he said.
According to Cercek, all 18 of the case studies had a form of rectal cancer in which a gene mutation inhibits their cells from repairing DNA damage.
Cercek said that only 5 to 10 percent of people with rectal cancer have this gene — which malfunctions in that it does not do well at hiding from the medication. As a result, the drug, which amps up the immune system, is very effective.
“Stomach, pancreas and bladder cancer have cells with those malfunctions in similar percentages,” she said. “It can work on those as well. Our plan is to expand and see if it does. I think it is very likely.”
Mukherjee said that the type of drug used in the study, known as a checkpoint inhibitor, is not new — checkpoint inhibitors have been around since 2014 and prevent cancer cells from hiding from the immune system — but the select targeting of patients is stunning.
“Many trials have shown response rates in patients using this family of drugs, but I have not seen a 100 percent response rate,” he said. “It rejuvenates the idea of using this family of drugs in other cancers. The potential for it to work with other cancers at this response rate is very exciting.”
Trial participant Sascha Roth was days away from checking into Georgetown University Medical Center for rounds of chemotherapy — treatments that rarely work against the mutated cells of rectal cancer — when she went to Sloan Kettering for another opinion.
“They were going through edits with the FDA for final approval [to run trials],” Roth, now 41, told The Post. “Then I was told that I would be a perfect match for the treatment. Two months later, in December 2019, I was being treated.”
Treatment entailed a 60-minute infusion of dostarlimab every three weeks, for a total of nine doses. Roth said she felt no side effects and hoped for the best.
“After the first month and a half, biopsies showed that the cancer was being eliminated. It just melted away,” she said, still sounding like a person on cloud nine. “After six months, Dr. Cercek called and asked if I was sitting down. She was looking at a scan and told me that there is absolutely no cancer. I didn’t cry upon hearing the news. Truthfully, I was shocked. But I did jump up and down.”
In terms of what’s next, said Cercek, “We focus on rectal cancer, make sure we can get this approved and continue with the efforts to solve other tumors. We also need to figure out what it is about these tumors, and the environments they live in, that makes them so sensitive to the therapy.”
Anticipating that researchers around the world will be working on it, she said, “I think it will be medicine’s next big treasure hunt.”