Small clinical trial surprises researchers: Patients all see their cancer go into remission

Something that never happens just did: Every patient participating in a small clinical trial for a new therapy for rectal cancer went into remission.

Something that never happens just did: Every patient participating in a small clinical trial for a new therapy for rectal cancer went into remission.

The cancer vanished in every patient. It could not be detected with physical exams, endoscopy, PET scans or M.R.I. scans. 

“I believe this is the first time this has happened in the history of cancer,” Dr. Luis A. Diaz Jr. of Memorial Sloan Kettering Cancer Center told the New York Times.

Diaz, an author of a paper published June 5 in the New England Journal of Medicine and presented at the American Society of Clinical Oncology annual meeting, said he knew of no other study in which a treatment completely obliterated a cancer in every patient.

Just as surprising, none of the patients experienced clinically significant complications.

The study was designed to assess the efficacy of dostarlimab, a checkpoint inhibitor, a drug that blocks proteins called checkpoints, which sometimes prevent T cells from killing cancer cells. Earlier research overseen by Diaz suggested that checkpoint inibitors could be effective in fighting cancers  that have a gene mutation that prevents cells from repairing damaged DNA.

In the latest study, patients took a dose of dostarlimab every three weeks for six months. The goal was to enable patients’ immune systems to destroy the rectal cancer cells.

The therapy was a significant break from standard treatment, which typically involves chemotherapy, radiation and surgery. The Times article suggests that several pharmaceutical companies declined to get involved in the trial, concerned that the delay in traditional treatment might allow the participants’ tumors to grow.

In the end, that didn’t happen. Memorial Sloan Kettering Cancer Center announced that “all 14 [patients] who initiated treatment on the trial and have had at least six months of follow-up achieved a clinical complete response with no evidence of tumor.”

The research once again demonstrates that sometimes the best therapy is one that empowers the body to heal itself.  Several of the companies Methuselah Foundation supports are working on therapies that aim to do just that. 

These findings are certainly promising, but much more research is needed to confirm the results and assess the effectiveness of checkpoint inhibitors on different kinds of cancer.  Plus, we need to assess the long-term effects on study participants.  Have their cancers been cured, or subsided? 

Having shared all the caveats, it’s still good news and another step in our march towards longer, healthier lives.