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The Readout Damian Garde & Meghana Keshavan

A major first for liquid biopsy, with caveats

For the first time, doctors have used a liquid biopsy test to detect and treat cancers in people with no symptoms of disease, a sizable advance for the nascent field, albeit with some limitations.

As STAT’s Kate Sheridan reports, a blood test from Thrive Earlier Detection detected twice as many cancers as standard screening alone in a study involving 10,000 healthy women between 65 and 75 years old. The study, published in Science, marks the first time liquid biopsies have been used with the explicit goal of guiding people’s medical care.

But the big potential benefit of liquid biopsy — catching cancers early, when they’re most treatable — wasn’t entirely supported by the data. About half the cancers Thrive’s test identified were already late-stage, suggesting the technology could still be improved.

Read more.

Some hope for off-the-shelf CAR-T

While CAR-T cancer therapy has proved life-changing for patients with certain blood cancers, the long and expensive process of engineering each person’s immune cells has limited the number of people who can get it. Early results from a small clinical study suggest a faster, ready-made alternative might be possible.

As STAT’s Sharon Begley reports, scientists in China crafted a CAR-T therapy using T cells from healthy donors and got a promising result in a trial on five patients with relapsed leukemia. Four months after treatment, four of the patients had barely detectable cancer. The fifth saw no benefit.

It’s too early to tell whether the off-the-shelf CAR-T, made by a company called Gracell Biotechnologies, will lead to the durable responses seen with first-generation therapies like Novartis’s Kymriah. But the initial data are “impressive,” said Yvonne Chen, a molecular geneticist at the University of California, Los Angeles.

Read more.

Biogen’s blockbuster drug could be in trouble

The success of Biogen’s spinal muscular atrophy treatment Spinraza has been among the few bright spots in a trying few years for the company. But new data from a competing medicine suggests diminishing returns in the future, a problem that could be exacerbated by the coronavirus pandemic.

Yesterday, partners Roche and PTC Therapeutics disclosed that their SMA treatment, risdiplam, led to a 93% one-year survival rate among patients with the most severe form of the disease. In a similar study, Spinraza kept about 84% of severe SMA patients alive after a year.

This, of course, is good news for patients. The risk to Biogen hinges on the fact that risdiplam is an oral treatment, while Spinraza must be injected into the spine. Roche’s drug, which is up for FDA approval in August, could become the treatment of choice for patients who’d prefer not to undergo those injections, and concerns over coronavirus might prompt them to switch off of Spinraza to avoid having to go to a doctor’s office.

Further compounding the issue is Zolegensma, Novartis's gene therapy, which could soon win approval as a treatment for patients with milder SMA. That means the choice for patients would be between a one-time dose of Zolgensma, an oral treatment with risdiplam, or an every-four-months spinal injection of Spinraza. Biogen could come out the loser.

How do you run a cancer trial in a pandemic?

The coronavirus pandemic has made regular doctor visits unthinkable, and for scientists trying to rigorously test new cancer treatments, that poses a serious threat to progress.

As STAT’s Andrew Joseph reports, doctors, patients, and drug companies are getting creative to keep studies rolling and protect the integrity of science. That means couriering drugs to patients homes, using telemedicine for check-ins, and changing study sites to minimize travel.

The stakes are high for everyone involved. Patients don’t want to go without a therapy that might be prolonging their lives. Companies don’t want to lose hope for a return on their research investment. And, in societal terms, it’s essential that the resulting data is interpretable in order to decide whether drugs should be approved and given to thousands of patients.

Read more.

More reads

  • Most Americans believe Trump hasn't made progress on drug prices. (STAT Plus)
    California fines more than a dozen drug makers for not providing drug pricing data. (STAT Plus)
  • Blueprint’s voyage ends in disaster. (Evaluate Vantage)
  • Hospital demand for hydroxycholoroquine to treat Covid-19 patients is waning. (STAT)

Thanks for reading! Until tomorrow,

Wednesday, April 29, 2020

STAT

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