Small study shows that experimental drug makes rectal cancers vanish in 100 percent of the cases

A small rectal cancer clinical trial has had an unexpected result — remission in every patient.

According to a story by Gina Kolata in recent editions of The New York Times, the cancer vanished in each of the 18 patients, "undetectable by physical exam, endoscopy, PET scans or MRI scans." 

Dr. Luis A. Diaz Jr.

Dr. Luis A. Diaz Jr. of Memorial Sloan Kettering Cancer Center, who published a paper on the trial in the New England Journal of Medicine, is quoted as saying he knew of no other study in which a treatment completely obliterated a cancer in every patient. "I believe this is the first time this has happened in the history of cancer" he says.

That view is confirmed by Dr. Alan P. Venice, a colorectal cancer specialist at the University of California, San Francisco, was wasn't involved in the study. That kind of record, he says, is "unheard of."

The patients entered the study with the expectation that they might face chemotherapy, radiation and, most likely surgery that cold result in bowel, urinary and sexual dysfunction — and require colostomy bags. All 18 were pleasantly surprised to find that no such treatments were necessary.

Dr. Andrea Cercek

"There were lots of happy tears," Dr. Andrea Cercek, an oncologist at Memorial Sloan Kettering and a co-author of the paper, is quoted.

The immunotherapy drug the patients were given every three weeks for six months — dostarlimab, a checkpoint inhibitor produced by GlaxoSmithKline, which sponsored the clinical trial — costs about $11,000 per dose. "It unmasks cancer cells, allowing the immune system to identify and destroy them," the story says.

The study's authors indicate that although the earliest patient to complete the trial is more than two years post-treatment, many have only been involved for six months, and all patients will be monitored for at least five years.

Dr. Julie Gralow

A follow-up story by Kim Bellware and Lenny Bernstein in The Washington Post notes that the study's results marked "the first time immunotherapy alone eliminated the need for chemotherapy, radiation or surgery."

The Post story quotes Dr. Julie Gralow, chief medical officer and executive vice president of the American Society of Clinical Oncology, as commenting that "I'm excited when you see such a dramatic response. It gives me hope we can find such a dramatic [treatment] for other cancers, too." 

More information about clinical trials can be found in "Rollercoaster: How a man can survive his partner's breast cancer," a VitalityPress book that I, Woody Weingarten, aimed at male caregivers.

$373,000 per regimen for new treatment

FDA gives its okay to a second gene therapy that can send blood cancer into remission

The FDA has approved a new gene therapy for adults with aggressive non-Hodgkin's lymphoma.

According to a recent story by Denise Grady in The New York Times, Yescarta was okayed for patients who've undergone two regimens of chemotherapy that failed.

The Times article notes that the therapy "is part of the rapidly growing field of immunotherapy, which uses drugs or genetic tinkering to turbocharge the immune system to fight disease. In some cases the treatments have led to long remissions."

The therapy, which turns cells into "living drugs," is made by Kite Pharma.

Approval by the Federal Drug Administration was its "second in a radically new class of treatments that genetically reboot a patient's own immune cells to kill cancer."

The first was Kymriah, made by Novartis. It was okayed in August "for children and young adults with an aggressive type of acute leukemia."

That one costs $475,000 per dose, although the company has said "it will not charge patients who do not respond within the first month after treatment."

Yescarta isn't quite as expensive — $373,000 per treatment.

Dr. Frederick L. Locke

Dr. Frederick L. Locke, a leader of a study of Yescarta (and a specialist at the Moffitt Cancer Center in Tampa in blood cancers), is quoted as saying, "We're excited. We think there are many patients who may need this therapy."

Grady's story says "about 3,500 people a year in the United States may be candidates for Yescarta," which is intended "to be given once, infused into a vein, and must be manufactured individually for each patient."

Side effects, as with many therapies, can be daunting.

Life-threatening issues that stem from the Yescarta treatment can include "high fevers, crashing blood pressure, lung congestion and neurological problems," the story indicates.

According to Grady, two patients in the study that led to the drug's approval, "died from side effects," although doctors — through training and experience — apparently "have learned to manage them better."

The study, which was conducted at the Dana-Farber Cancer institute and Brigham and Women's Cancer Center in Boston, involved 111 patients at 22 hospitals.

"Initially," Grady's piece delineates, "54 percent [of the 101 who received Yescarta] had complete remissions…Another 28 percent had partial remissions [and] after six months, 80 percent…were still alive. With a median follow-up of 8.7 months, 39 percent of the 101 were still in complete remission — a much higher rate that achieved with earlier treatments — and 5 percent still had partial remissions."

Dr. Caron A. Jacobson

Says Dr. Caron A. Jacobson, who helped do the study: "You're really seeing people get their life back. After a couple weeks in the hospital and a couple weeks at home, they go back to work. On its face, it's quite remarkable and revolutionary."

The treatment, the Times explains, "was originally developed at the National Cancer Institute, by a team Dr. Steven Rosenberg led. The institute entered an agreement with Kite in 2012, in which the company helped pay for research and received rights to commercialize the results."

The drug giant Gilead reportedly bought Kite two months ago for $11.9 billion.

"In just several decades, said Dr. Scott Gottlieb, the FDA commissioner, "gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer."

A look at many treatments and clinical trials can be found in "Rollercoaster: How a man can survive his partner's breast cancer," a VitalityPress book I, Woody Weingarten, aimed at caregivers.