Thursday, December 1, 2016

The Readout by Damian Garde & Meghana Keshavan
Welcome to The Readout, where we keep you on top of all the biotech news. For more, visit STAT. On Twitter: @damiangarde@megkesh, and @statnews.

Hacking a personalized cancer vaccine

Neoantigen vaccines are the quintessential  precision medicine: They’re cancer vaccines designed to rev up an individual’s immune system to kill a tumor.

The trouble is, they’re pretty tricky to optimize. Each neoantigen vaccine involves sequencing the 3 billion base pairs of a patient's normal cells — then comparing them to the 3 billion base pairs that make up his tumor. 

So the Parker Institute for Cancer Immunotherapy has just launched a collaboration between 30 companies and academic institutions — including the Broad Institute, Genentech and Bristol-Myers Squibb — to improve neoantigen analysis by using bioinformatics. The group has a tech-tastic acronym: TESLA — short for Tumor neoantigEn SeLection Alliance, of course. 

The goal, in essence, is to streamline the algorithms that analyze tumor DNA and RNA — to predict the neoantigens that are most likely to be found on each patient’s cancer and that are most easily targeted by the immune system. Since 30 organizations are involved, a hefty amount of data sharing is involved — but each institution's contributions will be anonymized, to help protect proprietary approaches.

“It fits with the whole hacking concept, if you go back to Sean Parker’s vision of the world — all these people get together and hack out the best way of doing something,” Fred Ramsdell, vice president at the Parker Institute, told STAT.

The CAR-T plot thickens

Bluebird Bio is jumping full steam into the CAR-T race; yesterday, it presented early but positive data with some rewired T cells.

Bluebird’s CAR-T therapy had a positive effect on seven of the nine multiple myeloma patients who were ready for evaluation in a Phase 1 trial. All six patients who received higher doses of Bluebird's treatment saw their cancers shrink, and two were left with no traces whatsoever after four and six months.

On the all-important safety side, Bluebird reported no side effects that limited dosing and no serious cases of neurotoxicity or inflammatory response.

It’s early days yet for the trial, which will eventually enroll 50 patients. Patients could relapse over time, and it’s worth remembering that the serious safety concerns that may sink CAR-T therapy from Juno Therapeutics didn’t emerge until later-stage trials. 

But the results are a clear positive for Bluebird. The company's prospects in CAR-T appeared to dim last year when partner Celgene pared back on the collaboration and struck a deal with rival Juno. But Celgene hung onto the rights to Bluebird’s myeloma CAR-Ts, and the company now appears to have a clear path forward.

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Should trial volunteers get to see their data?

The answer seems obvious: If you participate in a clinical trial, any measurements of you are inherently yours.

But some scientists and patients caution that raw data in the hands of nonexperts can be unwieldy at best and medically misleading at worst. And it’s unclear whether many patients even care; in a few recent pharma-sponsored trials, volunteers were given access to their data and few took advantage of it.

Read more.

Clinical validation for shrooms?

Bohemian music fests and navel-gazing forest jaunts may no longer be the exclusive domain for magic mushrooms.

Two studies published today in Journal of Psychopharmacology examine the effects of this currently taboo hallucinogen in about 80 cancer patients. They’re finding that psilocybin — the psychoactive ingredient in magic mushrooms — shows improved, and lasting, well-being and life satisfaction in more than 80 percent of participants. 

Though dosing needs to be carefully supervised by medical professionals, Dr. Stephen Ross, who led one study at NYU, said he saw “dramatic clinical changes.”

STAT’s Bob Tedeschi chatted with trial participant Karen Pappas, who was diagnosed with lung cancer in 2013 and has felt a great deal of anxiety and depression as a result. Her reaction? 

“It was joyful,” she said. “And it has changed my life.” 

Read more.

More reads

  • The 21st Century Cures Act passed the House by a wide margin and is due for a Senate vote next week. (STAT)
  • AstraZeneca inks big biobucks deal with Bicycle Therapeutics. (Fierce Biotech)
  • FDA Commissioner Robert Califf pushed back on the notion that the agency is too slow in approving new stem cell treatments. (STAT)
  • Valeant Pharmaceuticals' efforts to sell off its stomach drug business have broken down. (Wall Street Journal)

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Thanks for reading! Until tomorrow,

Damian & Meghana

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