Brainstorm Health: John McCain’s Cancer, Marijuana and Breast Milk, Esperion Cholesterol Drug

August 27, 2018, 9:15 PM UTC

Good afternoon, readers. This is Sy.

As you’ve likely heard, Sen. John McCain, former presidential candidate and a larger-than-life figure in American politics, passed away Saturday at age 81 following a battle with an aggressive form of brain cancer.

There have been plenty of tributes to and reflections on the Arizona Republican’s political career and record of public service. Those will, no doubt, continue to proliferate. But it’s also important to home in on the insidious disease that claimed McCain’s life—an aggressive form of brain cancer called glioblastoma multiforme (GBM, or just glioblastoma, for short).

A glioblastoma diagnosis, statistically speaking, amounts to a death sentence. Brain and nervous system cancers in general are deadly. In fact, just about one in three of the approximately 24,000 Americans diagnosed with such cancers in 2018 are likely to still be alive five years later, according to the National Cancer Institute (NCI).

Glioblastoma specifically makes up 16% of all brain and nervous system cancers—and the numbers are even more dire in these cases, says the American Cancer Society. If the disease manifests between the ages of 20 and 44 (a relatively rare occurrence), there’s a 19% chance of survival five years after diagnosis; among those 45 to 54, that drops to 8%; and for older Americans aged 55 to 64, the five-year relative survival rate is a dismal 5%. McCain was in his 80s when his diagnosis was publicly revealed. (Beau Biden, son of former Vice President Joe Biden, also died of the disease, but at the age of 46.)

Just why is the prognosis so dire? A lot of it has to do with just how aggressive this specific kind of tumor is, and the limitation of buzzsaw approaches such as surgery, chemotherapy, and radiation.

Dr. Duane Mitchell, a physician and professor of neurosurgery at the University of Florida, had this to say in The Conversation on Monday:

“An additional characteristic of GBM is the invasive nature of the disease. GBM tumor cells essentially crawl away from the main tumor mass and embed themselves deep within the normal brain, often hidden behind a protective barrier known at the blood-brain barrier,” Mitchell writes. “This invasive feature means that while neurosurgeons can often remove the main central tumor mass of a GBM, the invasive finger-like projections protrude into other areas of the brain. The distant islands of tumor cells that have migrated away cannot be effectively removed by surgery.”

Thus, the normal courses of treatment for other kinds of cancers may not prove effective against such a clever and malevolent foe. At the same time, the complexities of the brain’s structure—and its critical role in, well, everything—have made it so that we have yet to see the same kind of progress against glioblastoma that we have in so many cancer, such as immunotherapies and gene-based treatments for skin, lung, and blood cancers.

But scientists have sounded some (very, very early) notes of optimism in recent years. “[G]ene therapy, highly focused radiation therapy, immunotherapy, and chemotherapies utilized in conjunction with vaccines” are being tested in experimental glioblastoma trials, the American Association of Neurological Surgeons notes—while adding the sobering note that these early-stage technologies have only boosted patients’ survival rates by a median of three months.

Read on for the day’s news.


A "new kind of neuron." You're supposed to aspire to learn something new everyday. So how about learning about a new kind of brain cell? Science reports that researchers have discovered "a new kind of neuron" that is present in people but seems to be missing in mice. It's unclear exactly what these bushy, brain-y tangles do—but they could provide some insight into why human brains differ from rodents (an important issue in drug development). (Science)


Esperion's cholesterol drug triumphs in trial. Will it matter? Shares of biotech Esperion rose 5% in Monday trading on the strength of clinical trial results for its cholesterol-busting drug, which cut levels of "bad" cholesterol (LDL-C) by 35% when combined with Merck's Zetia. That's a considerable improvement over placebo or either of the treatments in solitary; but, if eventually approved, the question is whether or not physicians will flock to the therapy over cheaper, generic alternatives. Companies like Amgen and Sanofi have created treatments that drastically slash LDL but faced pushback over their high price tags; Esperion claims that its combo would be cheaper and thus more appealing. (Reuters)

Novartis cancer immunotherapy nabs European approval. Novartis' pioneering cancer gene therapy Kymriah has won approval in the European Union. It's a significant development since the treatment, which involves re-engineering patients' own immune cells to fight their blood cancers, comes with an eye-popping price tag, and the EU's drug pricing watchdogs are notoriously stingy. Novartis will still have to work out the pricing and payment details in various EU countries (Kymriah comes with a list price of $475,000 in the U.S.).


A new study raises flags on marijuana use and breast feeding. Researchers from the University of California, San Diego have conducted a (small) study suggesting that THC—the main psychoactive, high-inducing substance in marijuana—can remain in the breast milk of nursing mothers up to six days after use. There are a whole bunch of caveats in the study, which only involved 50 women and concluded that only small amounts of THC remained. But the scientists say it raises questions about the potential effects of marijuana consumption by pregnant or nursing mothers on their children. (USA Today)


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