Thursday, October 12, 2017

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, where we keep you on top of the latest in biotech. For more in-depth coverage of biopharma, subscribe to STAT Plus. And don't forget to tune in at 3:30 p.m. ET today for a live chat about Spark Therapeutics' day in front of an FDA advisory panel. Register here.

Maybe don’t try biotech at home


This man would like to convince you that do-it-yourself pharmaceuticals are safe. (BIZ HERMAN/STAT)

Meet Michael Laufer, a 38-year-old biohacker so fed up with the cost of prescription medicines that he’s crafting an Anarchist Cookbook for drugs. That means tabletop recipes for an ersatz EpiPen, a bootleg Daraprim, and even bathtub Sovaldi.

“To deny someone access to a life-saving medication is murder,” Laufer told STAT’s Charles Piller. And “an act of theft [of intellectual property] to prevent an act of murder is morally acceptable.”

Needless to say, health professionals strenuously and vociferously recommend against taking DIY pharmaceutical products. But Laufer, a flamboyant figure who does not hesitate to compare himself to Gandhi, remains dedicated to what he calls “an emancipation of knowledge.”

Read more.

Getting to the root of CAR-T toxicity

CAR-T is a potent new anti-cancer tool, sure, but the associated side effects can wreak havoc on a patient's body. From raging fevers to severe brain swelling, CAR-T comes with a measure of risk.

Researchers at Fred Hutchinson Cancer Research Center are working to minimize that danger. And now they've unearthed some clues on how to identify the patients at most risk for reactions like neurotoxicity and cytokine release syndrome — as well as tantalizing hints as to why they occur in the first place.

Read more on STAT Plus.

MannKind's Hail Mary fundraise

Face it, MannKind: Afrezza is not working. That's the advice from STAT's Adam Feuerstein, who notes that the inhaled insulin has failed to attract a critical mass of interested patients — or prescribing physicians. 

The company is burning through cash, but thanks to the biotech bull market, saw its share price rise anyway. So it took advantage of the moment, and this week managed to raise $58 million through sale of stock. 

But to what end? 

Read more on STAT Plus.

How are we doing against cancer?

Relatively well, according to some leading experts in the field.

“We have entered a new era,” Tyler Jacks, director of MIT’s Koch Institute for Integrative Cancer Research, said at a STAT panel yesterday.

By that he means the days of relying solely on surgery and chemotherapy have given way to immuno-oncology and a bramble of acronyms like PD-1 and CAR-T that have proved to extend patients’ lives.

Some of the biggest remaining hurdles are practical, not scientific. Only a fraction of the nation’s cancer patients actually get the new drugs that could treat their disease, N-of-One CEO Chris Cournoyer said. It's a problem fueled by unequal access, a lack of physician education, and the slow adoption of diagnostic technology.

Read more.

More reads

  • Join STAT's Adam Feuerstein for live updates of today's FDA panel discussion of Spark Therapeutics' gene therapy. (STAT Plus)
  • Despite a positive late-stage trial, Merck is not going to file its good cholesterol-boosting drug for FDA approval. (Press release)
  • The rise and fall and rise again of 23andMe. (Nature)
  • Ardelyx's constipation drug succeeds in late-stage trial. (Reuters)

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Thanks for reading! Until tomorrow,

Damian & Meghana

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