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Experts wonder who could be helped after breakthrough new cancer drug – and when

Experts wonder who could be helped after breakthrough new cancer drug - and when
Written by admin_3fxxacau

In 2020, almost 340,000 people died from rectal cancer. Now a new treatment give hope to overcome the disease – and perhaps also for other forms of cancer – could be available within a year.

A recent trial, led by researchers at Memorial Sloan Kettering Cancer Center and published in the New England Journal of Medicine on June 5, produced almost unheard-of results: 14 out of 14 patients are in remission up to two years after treatment. Four others are still undergoing treatment

Best of all, the drug dostarlimab did not show any side effects or drawbacks unlike other treatments. “Radiation therapy is effective in removing the tumour, but it has a negative impact on the patient. Up to thirty percent of those who have surgery need colostomy bags,” Dr. Andrea Cercek, an oncologist at Sloan Kettering and co-author of the study, told The Post. ” [Radiation] can also lead to sexual dysfunction. They are improving but they are not functionally the same.

According to Cercek, the odds of all 14 case studies being in remission is one trillion to one. Based on this, she said, “I’m very optimistic about success.”

She hopes to see the drug approved by the FDA “as soon as possible.”

Andrea Cercek, an oncologist at Sloan Kettering, with patient Imtiaz Hussain, hopes the drug dostarlimab will be fast-tracked by the FDA.
Sloan Kettering Cancer Memorial

But Dr. Siddhartha Mukherjee, Pulitzer Prize-winning biologist, physician and author”Emperor of All Diseases: A Biography of Cancerwarns that it may be too early to break out the champagne.

“I was thrilled that this drug had such an incredible response rate,” Mukherjee told The Post. “I would like this to be repeated in a big trial. It’s the kind of result that makes people in our field excited but also worried.

He also warned that the treatment, which he said could be widely used within a year, was aimed at a very specific population.

Cancer experts say the drug dostarlimab could be useful with other forms of cancer - if patients have a certain gene.
Cancer experts say the drug dostarlimab could be useful with other forms of cancer – if patients have a certain gene.
NEJM

“The patients [in the study] have been carefully selected and are struggling with a specific form of cancer susceptible to this drug,” he said.

According to Cercek, all 18 case studies had a form of rectal cancer in which a genetic mutation prevents their cells from repairing DNA damage.

Cercek said only 5-10% of people with rectal cancer have this gene – which malfunctions in that it doesn’t do well at hiding from the drug. As a result, the drug, which strengthens the immune system, is very effective.

All of the patients in Sloan Kettering's study had some form of rectal cancer in which a genetic mutation prevents their cells from repairing DNA damage.
All of the patients in Sloan Kettering’s study had some form of rectal cancer in which a genetic mutation prevents their cells from repairing DNA damage.
NEJM

“Stomach, pancreatic, and bladder cancers have cells with these dysfunctions in similar percentages,” she said. “It can also work on those. Our plan is to expand and see if that’s the case. I think it’s very likely.”

Mukherjee said 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 selective targeting of patients is staggering.

“Exciting Potential”

“Many trials have shown response rates in patients using this family of drugs, but I haven’t seen a 100% response rate,” he said. “This renews 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.

Sascha Roth, now in remission, is amazed that she experienced no side effects with dostarlimab.
Sascha Roth, now in remission, is amazed that she experienced no side effects with dostarlimab.
Sloan Kettering Cancer Memorial

Trial participant Sascha Roth was days away from heading to Georgetown University Medical Center for rounds of chemotherapy — treatments that rarely work against mutated rectal cancer cells — when she visited Sloan Kettering for another opinion.

“They were going through amendments with the FDA for final approval [to run trials]Roth, now 41, told The Post. “Then I was told that I would be perfectly compatible with the treatment. Two months later, in December 2019, I was treated.

Treatment included a 60-minute infusion of dostarlimab every three weeks, for a total of nine doses. Roth said she didn’t experience any side effects and hoped for the best.

Patients Sascha Roth, Imtiaz Hussain, Avery Holmes and Nisha Varughese with researchers Drs.  Luis Diaz and Andrea Cercek.
Patients Sascha Roth, Imtiaz Hussain, Avery Holmes and Nisha Varughese with researchers Drs. Luis Diaz and Andrea Cercek.
Sloan Kettering Cancer Memorial

“After the first month and a half, the biopsies showed that the cancer was being eliminated. He just melted,” she said, still sounding like someone on cloud nine. “After six months, Dr. Cercek called me and asked if I was sitting down. She was looking at a CT scan and told me there was absolutely no cancer. I didn’t cry when I heard the news. Truth be told, I was shocked. But I jumped up and down.

As for what’s next, Cercek said, “We’re focused on rectal cancer, make sure we can get that approved and continue efforts to resolve other tumors. We also need to understand what’s going on. It is these tumors and the environments in which they live that makes them so susceptible to therapy.

Anticipating that researchers around the world will work on it, she said: “I think it will be the next big treasure hunt in medicine.”

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