Cancer.  The dreaded word.

When that diagnosis is provided, the patient knows they are in for surgery, radiation therapy or chemotherapy.  Or, all of the above.

Not that these mean one will be cured.  Often, the best folks can hope for is a few months to a few years of continued life.

Which brings up a new modality- and a (limited) clinical trial- that is exciting many clinicians.

Drs. Andrea Cercek and Luis Diaz (Memorial Sloan Kettering),along with 26 compatriots at Sloan Kettering, with four more from Yale University, published results from their short trial in the New England Journal of Medicine.

The study involved 14 patients diagnosed with early stage rectal cancer.  (It was sponsored by the biotech firm, Tesaro, which has been gobbled up by GlaxoSmithKline.)   After 6 months of treatment with a monoclonal antibody, the team found the tumors had vanished.

Let that sink in for a moment.  The tumors vanished! The regions where knotty, discolored tumors existed now manifest smooth, pink tissue.  (Biopsies and scans found no traces of the cancer, either.)   (NOTE:  This short study does not prove that these patients will remain cancer free for the rest of their lives.  But, it is a first for when everyone on the trial manifests the same fantastic results.)

Which also means that no rounds of chemotherapy, radiation treatment, or surgeries are planned for these 14 folks.   (There are four others still undergoing treatment- but seem to be presenting these same miraculous results.)

A total of nine doses of dostarlimab (every three weeks, via IV, intravenous administration), an immunotherapy drug that targets a special cell cancer protein.  (This has already been approved for endometrial cancer under the FDA accelerated approval process.)

Why target that protein?  Because when it is expressed, it instructs the patient’s immune system to withhold its response; in other words, the body doesn’t attempt to fight off the cancer cells.

And, this is not important for just rectal cancer.  (Rectal cancer treatment often leaves the patient a mess- bowel/bladder dysfunction, sexual impotency, infertility.)  Even though the sample size was small, the patient census was diverse in age, race, and ethnicity.

But, the key is these patients all carried a mismatch-repair-deficiency (MMR)- which means the body can’t normalize or repair abnormalities as cells divide.  This occurs only for about 10% or less of rectal cancer patients, and it also means they would be resistant to chemotherapy.

It also means this therapy can be extended to other cancers, where the patient manifests the mismatch-repair-deficiency.  These include cancers of the pancreas, stomach, bladder, breast, endometrium, or prostate.

This could be the start of something really great.

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