Prediction: Price of drug therapies will skyrocket

New FDA-approved cancer gene therapy to cost $475,000 for a single course, reports say

Are pharmaceutical companies gouging the public?

Very possibly, as evidenced by a new gene therapy for childhood leukemia that will cost almost half a million dollars for a single course.

Reports yesterday about Federal Drug Administration approval for Novartis' Kymriah, which is the first gene therapy for cancer that it's okayed, say it will cost at least $475,000.

Although some oncologists have described the CAR-T drug as revolutionary, critics fear the price tag could pave the way for future drug costs to skyrocket.

According to a story by Catherine Ho in the San Francisco Chronicle, the way the treatment works is this: A patient's cells are extracted from his or her blood, then sent to a site where they'll be genetically modified. The altered cells are then re-infused into the patient's body.

The FDA this week approved Kymriah as a treatment for childhood B-cell acute lymphoblastic leukemia, a cancer of the blood and bone marrow that's the most common childhood cancer.

The National Cancer Institute has estimated 3,100 patients up to 20 years old are diagnosed with the disease annually. Although the survival rate is 90 percent, that figure drops to as low as 10 percent for those who relapse after a bone marrow transplant. It is that subset of patients who are eligible for cell therapy, whose rate of success is said to be between 70 and 80 percent.

Only a few hundred children and young adults are expected to be treated with Kymriah each year.

Bruno Strigini

A Guardian story, supplemented by Associated Press reporting, said that Bruno Strigini, Novartis' head of oncology, has explained that the high cost of the treatment is an attempt to balance patient access to the drug with ensuring a return on the company's investment.

The Guardian piece also cited a Novartis statement to the effect that the company "was collaborating with Medicare on a plan in which the government would only pay for the treatment if patients responded to them by the end of the first month."

Side effects of the drug include high fevers, diarrhea, vomiting, delirium, loss of balance, and difficulty speaking and understanding.

More information about the exorbitant costs of cancer treatments can be found in  "Rollercoaster: How a man survive his partner's breast cancer," a VitalityPress book I, Woody Weingarten, aimed at male caregivers.

Aggressive, 'just-in-case' treatments unnecessary

New test can shield breast cancer patients from unneeded treatments, study indicates

A new tumor test can help identify which breast cancers don't need extra treatment.

According to a recent KQED morning edition piece by Joe Neal and Patti Neighmond, "more and more studies are showing many small, early tumors don't present a danger."

The public-radio report, under the rubric "Shots: health news from NPR," indicated that a study published in JAMA Oncology suggested that it's possible "to distinguish fairly precisely between 'ultralow-risk' tumors that are unlikely to cause problems and those that are more aggressive and likely to spread."

Which translates into some patients being able to, according to the piece, "avoid unnecessary treatments."

The MammaPrint diagnostic test, used by researchers in the United States and Sweden, apparently can measure a tumor's genomic "fingerprint" and compare it with survival time after a tumor is removed.

The NPR story reported that the researchers said "they were able to pinpoint patients who had a very low risk of death from breast cancer — even up to 20 years after the first diagnosis."

Dr. Laura Esserman

The piece quoted Dr. Laura Esserman of the University of California, San Francisco, the lead author of the study, as saying what it means is that "we don't have to be aggressive upfront and treat you with everything, just in case."

The test results, it continued, "build on findings of a 2016 report using the same test that  showed 46 percent of women with certain genetic profiles could actually skip chemotherapy with little consequence to their long-term survival."

According to Esserman, it appears that about 20 to 25 percent of tumors being diagnosed today may be ultralow-risk and not require treatment after surgery.

The tumor test, which isn't covered by all insurance companies, is not cheap. It costs $4,200.

Many more details about much more research on breast cancer can be found in "Rollercoaster: How a man can survive his partner's breast cancer," a VitalityPress book I, Woody Weingarten, geared to caregivers.

Smaller studies can mean missed side effects

Pharmaceutical companies can't find patients for trials of experimental cancer drugs 

Dr. Peter Bach

Too few patients are available to cover all the clinical cancer drug trials being conducted.

That, at least, is the conclusion of a recent story by Gina Kolata in The New York Times.

Her article notes that the problem "is caused partly by companies hoping to rush profitable new cancer drugs to market, and partly by the nature of these therapies, which can be spectacularly effective but only in select patients."

There currently are more than 1,000 immunology trials underway, for example.

But Kolata says "immunotherapy trials have proliferated so quickly that major medical centers are declining to furnish patients to them. The Yale Cancer Center participates in fewer than 10 percent of [those] it is asked to join." 

The writer cites words of Dr. Peter Bach, director of the Center for Health Policy and Outcomes at Memorial Sloan Kettering Cancer Center: "I think there is a lot of exuberant rush to market. And we are squandering our most precious resource — patients."

Dr. Roy Herbst

She also quotes Dr. Roy Herbst, the Yale center's chief of medical oncology, to the effect that the companies sponsoring the trials aren't addressing new research questions but are merely "trying to get proprietary drugs approved."

Some companies are working on experimental drugs that attack and block mutations that tumors need to grow and thrive. The mutations sought by those targeted therapies, however, are extremely rare so pharmaceutical companies "may be forced to undertake a worldwide search for subjects that can last for years."

It took Pfizer three years, for example, "to locate 50 lung cancer patients who carried a rare aberration called ROS1, found in just 1 percent of patients."

Many new trials involve a limited number of patients, which can be risky.

Bach, according to Kolata, maintains that "the smaller the study and the shorter its duration, the more likely that what looks like an effect in a trial might simply be the result of chance" — which "leaves some of us evidence geeks wondering if it works."

Dr. Scott Ramsey

The writer also quotes Dr. Scott Ramsey, an oncologist at the Fred Hutchinson Cancer Research Center, that "in tiny studies, serious side effects can be missed."

Details of many 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.

Can 40 percent of cancers be prevented?

Vast majority of cancer-causing mutations due to random DNA errors, new study indicates

"Two-thirds of cancer mutations are due to random DNA copying errors, study says."

That's the bulk of a slightly scary headline at the top of a recent "To Your Health" section of The Washington Post website.

In a story by Laurie McKinley, the study — published in "Science" magazine — cancer-causing mutations occur when normal cells divide. 

Humans, according to the piece, "have trillions of cells, which are constantly regenerating by dividing and making new cells. But each time DNA is copied, the scientists said, an average of three random mistakes will occur. While most are harmless, a small number affect genes that will promote cancer."

Bert Vogelstein

The Post article indicates that two Johns Hopkins University researchers — Bert Vogelstein, a cancer geneticist, and Cristian Tomaetti, a mathematician — were able in their study to separate the randomness factor from the other main contributors to cancer: inherited genes and environmental factors such as smoking and obesity.

Cristian Tomasetti

Results, after analyzing  "genome sequencing and epidemiological data for 32 types of cancer," assigned the mutations thusly: 66 percent, DNA-replication mistakes; 29 percent, environmental factors; 5 percent, heredity.

Variations depended on what kind of cancer they were talking about. According to the story, for example, "random DNA-replication mistakes account for about 77 percent of critical mutations in pancreatic cancer, and virtually all childhood cancer."

But "more than two-thirds of the mutations in lung cancer were due to environmental factors, mostly smoking."

McGinley's piece noted that "the new research builds on a 2015 study that highlighted the role of 'bad luck' — random DNA errors — in developing cancer. That study drew vehement protests from some cancer physicians and researchers who worried it would encourage people to take a fatalistic approach to cancer rather than trying to reduce their cancer risk by maintaining a healthy weight, exercising regularly, eating a good diet and avoiding cigarettes."

The Hopkins researchers have claimed "their earlier work was widely misinterpreted," the article continued — and emphasized that the new study "was consistent with estimates that 40 percent of cancers can be prevented."

Vogelstein insisted said that "science needs to find better ways to detect cancer early, when there is a good chance of curing it."

According to the story, critics of the 2015 study "disliked the 'bad luck' explanation and complained that the study was limited to the United States and didn't include the most common cancers — breast and prostate."

Insights into cancer mutations — especially BRCA1 and BRCA2 genes — 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.

Are 16 unregulated chemicals putting you at risk?

Contaminants in drinking water may cause cancer, pregnancy problems and other risks

A new study shows that 16 cancer-causing chemical contaminants are filling California's water supply.

According to a Patch.com article by Autumn Johnson, hundreds of harmful contaminants have been found across the American water supply that also can cause "developmental issues in children, problems in pregnancy and other serious health conditions."

The subhead of the website piece warns, "What's legal isn't necessarily safe when it comes to your drinking water."

In California as well as other states, it says, the following contaminants have been detected above health limits or guidelines in some water districts:

Chromium (hexavalent), which is linked to cancer and liver damage; total trihalomethanes (TTHMs), which are connected with bladder and skin cancers plus fetal development issues; bromodichloromethane and dibromochloromethane, both of which are linked to cancer and harm to fetuses; chloroform, connected to cancer risks and fetal development issues; dicloroacetic and trichloroacetic acids, both of which are linked to cancer as well as reproductive or pregnancy difficulties; uranium, arsenic and radium-228, all of which have cancer links; nitrate and nitrite, which are linked to cancer and fetal development issues; haloacetic acids (HAA5) and fluoride, which may cause cancer; selenium, which can decrease thyroid hormone production; 1,2-dibromo-3-chlororopane (DBCP), which can cause sterility in men and well as cancer; and tetrachloroethylene (perchlorothylene), which can cause cancer and pollute soil and groundwater.

Nneka Leiba

The Patch story adds that the study, which was released today, contends that chemicals "above health-based lists, or health guidelines, [can be found] in the water of more than 250 million Americans, said Nneka Leiba, director of Healthy Living Science at the Environmental Working Group, or EWG, and independent nonprofit organization that release a detailed account of the contaminants."

Laws often permit utilities "to allow these dangerous chemicals to pollute our waters," Johnson's story continues.

The story quotes Leiba as saying that about 160 contaminants out of the 250 in the nation's drinking water are unregulated, "a big concern because…that means [they] can be present in our water at any level — and be legal."

In many cases, however, a simple water filter — although not 100 percent effective — can help protect consumers.

More information on cancer-causing chemicals 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.