Remarkable Drug Trial Ends With All 18 Patients Cancer-Free: The results of the Phase II trial were published in the New England Journal of Medicine

Gizmodo via MSN ^ | 06/06/2022 | Ed Cara

[SeekAndFind]

Acancer treatment has shown astounding results in a small clinical trial. All of the treated patients, who had a specific form of mid-stage rectal cancer, have since experienced complete remission. Though the findings are based on a sample size of just 18 people, they could hold important implications for treating these particular cancers.

The results of the Phase II trial were published over the weekend in the New England Journal of Medicine . The study involved researchers at the Memorial Sloan Kettering Cancer Center as well as Yale University, and it was sponsored by the pharmaceutical company GlaxoSmithKline.

The trial enrolled volunteers diagnosed with stage II or III rectal cancer, meaning their tumors had begun to grow larger and spread to nearby parts of the body. Their cancer was also determined to be caused by a particular mechanism known as a deficiency in mismatch repair.

Cancers can form for many different reasons. But sometimes, our cells develop mutations that hinder them from being able to fix errors made when DNA is copied within them. These errors can then eventually lead to cancerous cells.

The researchers theorized that their treatment, a lab-made antibody called dostarlimab, might be able to help this subset of patients. It works by inhibiting a protein known as programmed death receptor-1 (PD-1) found in many cancer cells. This inhibition then allows the immune system to recognize the cancer cells as harmful and target them for destruction. The drug was developed by GlaxoSmithKline, and it was given an accelerated approval by the Food and Drug Administration last year for cases of endometrial cancer linked to a mismatch repair deficiency.

The patients were given a dose of dostarlimab every three weeks for six months.

[philippa]

Interesting, yes. Not conclusive. We’ve seen so many promising drugs over the years that haven’t planned out.

[philippa]

It’s a start, but too small to be conclusive. There needs to be a larger trial.

[LilFarmer]

I am leery of any drug ending in “mab” as many of them were developed using humanized mice (including this drug). List is here: https://en.wikipedia.org/wiki/List_of_therapeutic_monoclonal_antibodies

Some of these mice are humanized with fetal tissue/fetal stem cells and it is not always easy to find out which ones. Here are some:

https://www.fetalindustry.com/post/aborted-baby-organs-used-to-humanize-mice-for-covid-19-vaccine-and-therapeutic-testing

[LilFarmer]

Under “Ethical Statement”
Humanized NOG-EXL mice (highly immunodeficient NOG mice) expressing human GM-CSF and human IL-3, engrafted with CD34 human stem cells

http://www.anaptysbio.com/wp-content/uploads/Preclinical-characterization-of-dostarlimab.pdf

[JustOneStarfish]

By definition, a phase II trial is a small number of people.

Pre-clinical is animal studies/lab studies. Phase I is determining how large a dose is SAFE in humans (not effective, just SAFE). Phase II is determining if it is EFFECTIVE at levels below the phase 1 safety margins.

There doesn’t need to be “another trial.” This (or any other dug with similar profiles) just continues on through the next phase of testing. If it is shown to be effective in Phase II trials, it moves to Phase III. Phase III is expanding to a much larger group. They don’t just look at a few monkeys or petri dishes and then release this or any other drug on a huge number of people.

There is value and relevance in results of EVERY phase of testing, including failure levels. This drug shows a lot of promise on a small, well-controlled group of people.