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Brainstorm Health: Huntington’s Disease Drug, Ascension-Providence Deal, Opioid Executions

Hello, readers! I hope you had a wonderful weekend. This is Sy.

An experimental injectable drug from Ionis Pharmaceuticals is one of the first to show potential in treating the root causes of Huntington’s disease, a devastating and fatal rare genetic disorder that causes nerve cells in the brain to break down. But the treatment is still in its earliest stages, researchers caution, and much larger clinical trials will need to be done to prove it can truly help reverse patients’ disease.

Ionis Pharmaceuticals stock remained relatively flat in Monday trading despite success in an early stage study of it’s Huntington’s drug, Ionis-HTTRx. The treatment is part of a new class of therapies that aims to “silence” certain genes in order to prevent the production of toxic proteins that can cause disease. Muted investor response aside, the results (called “groundbreaking” by lead researcher Sarah Tabrizi of the University College London) prompted Swiss pharmaceutical giant Roche to exercise its licensing option for Ionis-HTTRx.

“The key now is to move quickly to a larger trial to test whether Ionis-HTTRx slows disease progression,” said Tabrizi in a statement. That is, it remains to be seen whether the treatment’s reduction of the Huntington’s-associated toxic protein can slow or reverse the mental and physical decline wrought by the disease. (Other diseases present the same big question—for instance, whether or not eliminating amyloid plaque buildup in the brain can reverse or stave off the effects of Alzheimer’s disease.)

Still, the reduction in the protein is notable, according to Huntington’s specialists and patient advocates, especially since existing Huntington’s therapies only tackle its symptoms rather than its causes. And the general field of “gene silencing” has recently been on a tear given its promise to address genetic diseases at the genomic level. For instance, Alnylam Pharmaceuticals’ own patisiran has driven the company’s stock up more than 250% this year thanks to strong, later-stage clinical trial results.

Read on for the day’s news.

Sy Mukherjee


CAR-T drugs show long-lasting efficacy. Speaking of milestone drug development advances—a slew of companies presented impressive results for a new class of blood cancer treatments called chimeric antigen receptor T-cell (CAR-T) during the American Society of Hematology (ASH) meeting in Atlanta. As readers will know, CAR-T is the newfangled method of attacking cancer cells with re-engineered versions of patients’ own immune cells that home in on blood cancers. At ASH, CAR-T treatments from Celgene and Bluebird Bio for aggressive multiple myeloma and Novartis’ recently approved Kymriah for diffuse large B-cell lymphoma (DLBCL) were both effective and long-lasting.


Viagra goes generic. Bloomberg is out with a fantastic read on the history of Pfizer’s Viagra, the “little blue pill” that brought the company billions in sales over the decades and shifted the men’s sexual health paradigm in the United States. Viagra is going generic today following 19 years of exclusivity in American markets. Just how quickly did it take off once it hit? “Sales started almost immediately after the U.S. Food and Drug Administration approved the drug on March 27, 1998. In less than two months, Viagra was on the cover of Time magazine with this headline: “The Potency Pill.”


Merger mania continues: Ascension, Providence consider deal. The newest trend in health care M&A appears to be vertical consolidation, if CVS-Aetna and UnitedHealth-DaVita are any indication. Well, hospital operators Ascension Health and Providence St. Joseph Health are trying a more old-fashioned horizontal approach, according to the Wall Street Journal. A deal between the two firms would create the nation’s largest hospital chain with more than 190 hospitals, displacing current leader Hospital Corporation of America (HCA). (Fortune)

Execution by opioid? Two states (Nebraska and Nevada) are pushing to use the extremely powerful opioid drug fentanyl to assist in executions, drawing rebukes from death penalty critics and doctors who say such unproven new methods amount to human experimentation. States have had to come up with creative ways to carry out executions as an increasing number of pharmaceutical companies have refused to sell the traditional drugs used in death penalty cases for the purposes of eliminating a human life. That trend has materialized, in part, from investors who have pushed drug makers to stay out of the death business. Death penalty supporters argue newer methods are necessary given the dearth of the usual drug cocktails. (Washington Post)


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Produced by Sy Mukherjee

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