Saturday, July 29, 2017

'Transformative" treatments deemed possible

Race underway to create gene therapies for breast, prostate, ovary, lung, pancreas cancers

Pharmaceutical companies and universities are in a race to develop "utterly transformative" gene therapies, according to a recent article by Denise Grady in The New York Times.

The story says "one of the big goals now is to get them to work for many [cancers other than leukemia, for which approval is expected soon], including those of the breast, prostate, ovary, lung and pancreas."

The radically new class of treatments, it also says, may "re-engineer and turbocharge millions of patient's own immune cells, turning them into cancer killers that researchers call a 'living drug.'"

Dr. Stephan Grupp
The Times piece contends that the new leukemia drug, called CAR-T therapy, "has been utterly transformative in blood cancers," according to Dr. Stephan Grupp, director of the cancer immunotherapy program at the Children's Hospital of Philadelphia, a professor of pediatrics at the University of Pennsylvania, "and a leader of major studies."

Although Grupp cautions that it will take at least five years to make an accurate determination, he maintains that if the treatment "can start to work in solid tumors, it will be utterly transformative for the whole field."

The treatment apparently is also being studied in conjunction with "glioblastoma, the aggressive brain tumor that Sen. John McCain was found to have."

Grupp says one particularly encouraging potential avenue of research with children involves earlier stages of disease "instead of very late, as rules now require," the Times story indicates. 

Earlier treatment, it reports him as believing, "might help some patients avoid bone-marrow transplant, a grueling, last-ditch treatment. Children with less advanced disease also tend to have milder side effects."

Other studies are underway to combine the new therapy "with immunotherapy drugs called checkpoint inhibitors, which help unleash the cancer killing power of T-cells" — which the story describes as the white blood cells "often referred to as the soldiers of the immune system."

One of the big problems with the new treatment — which "involves removing millions of…T cells…from the patient's bloodstream, genetically engineering them to recognize and kill cancer, multiplying them and then infusing them back into the patient" — is its cost factor.

The process, the story explains, is very expensive (at least $300,000 per treatment, other articles have said)  "because each treatment has to be made separately for each person."

The treatment was developed at the University of Pennsylvania and licensed to Novartis.

More information about cancer research 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 male caregivers.

Saturday, July 22, 2017

'Mismatch repair-deficient' genetic flaws studied

Tumor cell testing might show if new immunotherapy could target your particular cancer

The more gene mutations hidden inside your cancerous tumor, "the better chance your immune system has to fight back."

That's what Lauren Neergaard, an Associated Press medical writer, wrote recently after a newly approved landmark drug became "the first cancer therapy ever cleared [by the Food and Drug Administration] based on a tumor's genetics instead of the body part it struck first."

Keytruda, an immunotherapy, apparently might help those whose disease is among those with a common genetic flaw — called a mismatch repair defect — carried by seemingly unrelated cancers.

Genetic testing can show if a patient is a candidate for the precision immunotherapy.

Neergaard's story indicates that Johns Hopkins researchers estimate "about 4 percent of cancers are mismatch repair-deficient, potentially adding up to 60,000 patients a year."

Widely available tests to tell who's eligible for the immunotherapy cost between $300 and $600.

The flaw, Neergaard's article says, "is more common in colon, endometrial and gastrointestinal cancers but occasionally in a list of others." 
Dr. Bert Vogelstein

According to Dr. Bert Vogelstein, a cancer geneticist at Johns Hopkins, where the new use for the immunotherapy was discovered, the more mutations in a tumor, the greater the chance that at least one of them produces a foreign-looking protein that is a beacon for immune cells." 

More information on cancer research is contained in "Rollercoaster: How a man can survive his partner's breast cancer," a VitalityPress book I, Woody Weingarten, aimed at male caregivers.

Wednesday, July 12, 2017

Gene-changing leukemia therapy nears approval

Feds open door to okaying cancer-fighting treatment that genetically alters patient's cells

An FDA panel has given a unanimous vote of confidence to a gene-altering leukemia therapy.

According to a story by Denise Grady in The New York Times today, the Food and Drug Administration panel has unanimously recommended, following successful clinical trials, that "the agency approve the first-ever treatment that genetically alters a patient's own cells to fight [lethal] leukemia, transforming them into what scientists call 'a living drug' that powerfully bolsters the immune system to shut down the disease."

The agency is likely to accept the recommendation, Grady's story indicates, an action that would make Novartis' experimental treatment the first gene therapy to reach the market — ending in September a decades-long competition by researchers and drug companies.

"To use the treatment," the article continues, "a separate treatment must be created for each patient" — at enormous cost: between $300,000 and $600,000 for the requisite single infusion, according to analysts.

The CAR T-cell technique has multiple steps: Cells removed from the patient must be frozen, thawed and processed at a plant run by the manufacturer, frozen again and shipped back to the treatment center.

Severe side effects — including "fever, crashing blood pressure, lung congestion" — can potentially be life-threatening, but scores of patients have experienced long remissions and, in some cases, possible cures, the Times story maintained.

On the other hand, "re-engineering cells for treatment sometimes take four months," it noted, leaving some patients "so sick that they died before their cells came back."

Dr. Carl H. June
Dr. Carl H. June, an immunology professor and leader of the University of Pennsylvania research team that developed the treatment and licensed it to Novartis, has labeled the altered cells "serial killers."

One cell purportedly can destroy up to 100,000 cancer cells.

The panel's recommendation specifically dealt with leukemia "that has resisted treatment, or relapsed, in children and young adults ages 3 to 25" — for patients who typically had a bleak prognosis.

The clinical trials, according to a story by Laurie McKinley in The Washington Post yesterday, took place "in almost a dozen countries," with results of 83 percent of patients going into remission.

That story also indicated that "researchers are exploring CAR T-cell therapy's use for multiple myeloma and chronic lymphocytic leukemia [and] are also tackling a far more difficult challenge — using the therapy for solid tumors in the lungs or brain."

Meanwhile, Dr. Stephen Schuster, a Penn oncologist and leader of a lymphoma study, is quoted by the Post as saying, "We're saving patients who three or four years ago we were at our wit's end trying to keep alive."

Details on a variety of cancer therapies can be found in "Rollercoaster: How a man can survive his partner's breast cancer," a VitalityPress book I, Woody Weingarten, aimed at male caregivers.

Friday, July 7, 2017

Cell experiment shows 'extraordinary' promise

Can new gene therapy change patient's own blood into a cancer killer? Quite possibly

Turning your own blood into cancer killers may now be possible.

According to a new study reported recently by the Associated Press, more than a third of very sick lymphoma patients showed no sign of the blood cancer six months after a single treatment of an experimental gene therapy.

Findings of the study, the story by Marilynn Marchione indicated, were made by the treatment's maker, California-based Kite Pharma, which purportedly "is racing Novartis AG to become the first to win approval of the treatment, called CAR-T cell therapy," in the United States.

The treatment could become the nation's first approved gene therapy, the AP article indicated.

Side effects appear to be manageable.

There are risks involved, of course. "Three of the 101 patients in the study died of causes unrelated to worsening of their cancer," the story said, "and two of those deaths were deemed due to the treatment," which was developed at the National Cancer Institute and licensed to Kite.

Dr. Roy Herbst
One independent expert, Dr. Roy Herbst, cancer medicines chief at the Yale Cancer Center, was quoted as calling the results "extraordinary" and "extremely encouraging."

He suggested, though, that follow-up studies were needed to make sure the benefit doesn't wane over a longer period of time.

New research and treatments are an integral part of "Rollercoaster: How a man can survive his partner's breast cancer," a VitalityPress book I, Woody Weingarten, aimed at male caregivers.

Saturday, July 1, 2017

Rare malignancies of immune system studied

Implants may bring a new disease to cancer patients with mastectomies, N.Y. Times says 

Can breast cancer implants give a woman who's had a mastectomy another cancer?

According to a recent article by Denise Grady in The New York Times, the answer is a definite yes.

The new cancer, it indicates, may not be breast cancer "but a rare malignancy of the immune system —  caused by the implants used to rebuild her chest."

The disease, anaplastic large-cell lymphoma, is a cancer that's "been linked to implants with a textured or slightly roughened surface, rather than a smooth covering," the piece says.

Although the federal Food and Drug Administration first reported a link between implants and the disease in 2011, an FDA update in March linked nine deaths to the implants and helped raise awareness, Grady's article notes.  

The FDA has also received 359 voluntary reports of implant-associated lymphoma from doctors or patients around the world.

That number, the story says, "is expected to rise as more doctors and pathologists recognize the connection between the implants and the disease."

As of now, however, no implants have been recalled — and "what's inside the implant, silicone or saline, seems to make no difference."

Between 2000 and 2016, the number of U.S. breast augmentations rose 37 percent — "and reconstruction after mastectomy rose 39 percent," Grady writes.

"Annually, nearly 400,000 women in the United States get breast implants, about 300,000 for cosmetic enlargement and about 100,000 for reconstruction after cancer, according to the American Society of Plastic Surgeons," the story adds.

Lymphoma, if detected early, is often treatable and rarely fatal — although its symptoms "usually include painful swelling and fluid buildup around the implant. Sometimes there are lumps in the breast or armpit."

Dr. Mark W. Clemens II
In cases with bad outcomes, the Times quotes Dr. Mark W. Clemens II, a plastic surgeon and expert on the disease at the University of Texas MD Anderson Cancer Center in Houston, "it was usually because they were not treated or there was a major delay in treatment, on the level of years."

Clemens believes that as late as 2015, "only about 30 percent of plastic surgeons were routinely discussing the cancer with patients," the Times piece says.

The newspaper also notes that what causes the disease isn't known. "One theory," Grady's story postures, "is that bacteria may cling to textured implants and form a coating called a biofilm that stirs up the immune system and causes persistent inflammation, which may eventually lead to lymphoma."

Clemens noted that researchers are also looking for mutations that might contribute to the disease.

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