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The Readout Damian Garde

Pfizer and Sangamo see hope in hemophilia data

An experimental gene therapy for hemophilia from Pfizer and Sangamo Therapeutics is showing promise: In a small, early trial, the one-time treatment boosted factor VIII, a protein that helps form blood clots and is deficient in people with hemophilia. Three patients who received the highest dosage saw their factor VIII levels return to normal, and their bleeds cut down to zero. 

The trial was tiny — just 10 patients — but the magnitude of success  suggests that the treatment could prove effective in later trials, and that the companies could join players like BioMarin and Spark Therapeutics in the race for curative options.

Read more.

Proposals to keep CAR-T side effects under control

One of the more dangerous aspects of CAR-T therapy is its potency: As  engineered T cells proliferate quickly in the body, aimed at killing off tumor cells, they can also wreak havoc on patients by producing extraordinary levels of inflammation. 

But a pair of new studies suggests that a molecular on/off switch for CAR-T therapy could be effective in taming the cells: A small molecule drug, dasatinib, can rapidly, precisely, and reversibly halt CAR-T function in mice. 

Read more. 

Can the U.S. beat Canada's drug prices? 

President Trump said Friday that he planned to issue an executive order that would ensure U.S. costs for prescription drugs are capped at the lowest price paid by either a manufacturer or a developed country.

“Why should other nations — like Canada — but why should other nations pay much less than us?” Trump asked. “We’re working on a favored nations law where we pay whatever the lowest nation’s price is.”

It's not entirely clear whether Trump was referring to existing pricing proposals — including an international plan index — or something new entirely.

Read more.

A live chat on Alzheimer's research

Researchers have been studying Alzheimer’s disease for decades, and still have precious little to show for it — at least in terms of treatments that would halt or stave off the disease. You may have questions. You may also have questions about a meeting right around the corner: The Alzheimer’s Association International Conference, which kicks off July 14 in Los Angeles. 

STAT Plus subscribers are in luck. STAT’s very own Matt Herper and Sharon Begley will be hosting a subscriber-only live chat at 11 a.m. ET on July 18. You can sign up to participate here

More reads

Thanks for reading! More tomorrow,

Damian

Monday, July 8, 2019

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