The race to cure DMD hits a snag

Solid Biosciences, a company founded to halt the muscle-wasting Duchenne muscular dystrophy, has disappointing news from its first clinical trial: The firm’s gene therapy, designed to boost production of a key protein, performed well below expectations in a small study.

As STAT’s Matthew Herper reports, the three patients treated in Solid’s trial produced only small amounts of that protein, called dystrophin. Investors — and Solid’s management — had hoped to see much better results from the study, making this morning’s disclosure a stumble for the early-stage therapy.

But the process is hardly over. The patients in question received the lowest planned dose of Solid’s treatment, and three more are yet to be treated. And Solid, which is racing with Sarepta Therapeutics to develop a gene therapy for DMD, believes it’s still in the game.

“We believe we will remain competitive,” Ganot said. “I don’t think we ever stopped being competitive. I think the competitiveness of the situation will be determined by, of course expression. But also the quality of the protein. But also the ability to manufacture it at scale.”

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An idea to stop cancer before it starts

Some of the biggest names in biotech venture capital are lining up behind a company coming out of stealth mode today. That company, New York-based HiberCell, announced it has raised $60 million and plans to develop therapies targeted at the unique genetic signatures of “dormant disseminated tumor cells” — that is, cancer cells that spread throughout the body even before a tumor is detectable.

“We believe that dormant disseminated tumor cells really are central to preventing cancer metastasis and relapse,” Alan Rigby, the company’s co-founder and president, told STAT’s Sharon Begley.

HiberCell plans to target both solid tumors, especially breast and prostate, and blood cancers. It also calls itself the first company exclusively focused on the detection and treatment of dormant tumor cells.

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Has ‘precision medicine’ gone too far?

With the millennium came the mapping of the human genome and some utopian predictions that, in just a decade’s time, decoding DNA would usher in a new era of medicine. That didn’t exactly happen, and the ethos that promised it, called genetic reductionism, may be blinding scientists to the complexities and potential of biology.

As a pair of doctors write in STAT, the dogma of precision medicine — that genetics is the be-all, end-all of understanding disease — has stolen focus and funding from other ideas and blunted debate in the halls of science.

“While we are occasionally told that we are Luddites or nihilists (generally without much debate of the merits of our position),” they write, “the most frequent communications we receive have been along the lines of ‘I agree with you, but can’t speak up publicly for fear of losing my grants, alienating powerful people, or upsetting my dean.’ This atmosphere cannot be good for the culture of science.”

Read their argument here and weigh in for yourself.

A possible step forward for Alzheimer’s diagnostics

As the drug industry flails in its efforts to develop new Alzheimer’s treatments, scientists have struggled on a related front: coming up with blood tests to either diagnose the disease in asymptomatic patients or predict which healthy people will develop it.

But yesterday brought a sliver of good news: Researchers said that blood levels of 10 proteins did a decent job of identifying patients who had high enough brain levels of beta-amyloid, a marker of the disease, to be classified as having preclinical Alzheimer’s.

The hope is that the findings could give drug companies a desperately needed tool to identify patients for clinical trials.

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The race to cure DMD hits a snag

An idea to stop cancer before it starts

STAT webinar: Is the new Congress ready to lower drug costs?

Has ‘precision medicine’ gone too far?

A possible step forward for Alzheimer’s diagnostics

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