Family of Juno patient speaks out: 'He died for greed.'

Max Vokhgelt died from the symptoms that stemmed from his car-t therapy. (andrew spear for stat)

We know how many patients have died in the CAR-T immunotherapy trials conducted by Juno Therapeutics.

What we haven't heard: Who these people were — and how their families are coping. Today, STAT Plus tells the story of the first patient to die from side effects in Juno's ROCKET trial.

Two days after getting an infusion of Juno-engineered T cells, 24-year-old Max Vokhgelt saw his fever spike to 105.2 — a sign that his physicians saw as a positive, because for CAR-T therapy to work, it has to nearly kill you. When the fever subsided, Max was optimistic.

But then the signs of neurotoxicity kicked in, and a few days later, Max was declared brain dead. The company didn't say anything publicly and continued to test the therapy until two more patients died. After a brief FDA hold, Juno tinkered with its formula and then resumed the trial. Two more patients have since died.

The Vokhgelt family’s grief is mixed with anger and a thirst for answers.

“First, I believed Max had died for science, but now I think he died for greed,” his father told STAT contributor Rob Waters.

Big Pharma wants to be a better neighbor

The scienciest corners of Cambridge, Mass., have long since been colonized by the world’s biggest drug makers, driving up rents for startups and changing the makeup of the community. Now, some major tenants say they’re trying to better engage with the natives, including academics.

“I want to re-introduce Novartis,” top scientist Dr. Jay Bradner said Friday at a sort of open house in the company’s expansive research campus in Cambridge. “We have one of the most gorgeous temples of science in the world. We need to open the door to our neighbors.”

But a firm as large as Novartis can’t just go full open-source, Bradner said, noting that even his PowerPoint had been vetted by the company’s lawyers. In other words, being an “intergalactic corporate structure” has its limitations.

The same goes for Johnson & Johnson, said Robert Urban, who runs that company’s collaboration-minded innovation division. Being the world’s largest health care company frees J&J to do things startups can only dream of, “but it’s also a burden, because what it means to management is, ‘Don’t touch a thing,’” Urban said.

Meanwhile, in Trumpland

Remember Jim O’Neill, the regulation-averse investor rumored to be on President-elect Donald Trump’s short list for FDA commissioner? Turns out he might just have been a red herring to drum up support for Dr. Scott Gottlieb, a well-regarded ex-FDAer who carries conservative bona fides but doesn’t seem to harbor ambitions to completely subvert agency dogma. At least, that's one theory that's been kicking around D.C.

Elsewhere in palace intrigue, Dr. Francis Collins, longtime head of the NIH, said he’d like to stick around, if Trump will have him, which some top Republicans strongly recommend.

The president-elect said little about NIH in his campaign and ensuing transition, beyond jokingly suggesting that conservative radio host Michael Savage ought to lead it. Congressman Andy Harris of Maryland has nominated himself for the position but is widely seen as a bit of a long shot, having never held a job that compares to leading the NIH.

Then again, this transition is proving stubbornly hard to handicap.

Nature's pick: The best science of 2016

The lionization continues for CRISPR/Cas9, which has been called out as one of the top notable advances of 2016 by Nature. CAR-T immunotherapy, too, made the cut — right in time for expected FDA filings from Novartis and Kite Pharma.

But the big kids on the science block don't get all the glory. Among other highlights of the year:

Scientists now have a complex way to build antibodies that neutralize the virus. It's still too complicated to be industry-ready, but it’s a start towards building an HIV vaccine.
How does cancer metastasize? Here’s one clue: Looks like tumor cells enter the bloodstream by triggering endothelial cell death, creating an escape hatch in the circulatory system so they can spread to new tissues.
Researchers are finally starting to understand the genetic underpinnings of the excessive synaptic pruning that goes on in schizophrenia and other neuropsychiatric disorders.

Check out the rest here.

Monday, December 12, 2016