For cancer patients on experimental immunotherapy drugs, it’s been an emotional week

It’s been a roller coaster week for cancer patients betting their lives on the experimental field of immunotherapy.

A pioneer in that field, the Seattle biotech company Juno Therapeutics, announced late last week that three young leukemia patients testing one of its treatments had died of swelling in the brain. Federal regulators abruptly halted the trial.

Then, on Tuesday, an encouraging announcement: Federal regulators had swiftly concluded that a tweak to the trial protocol would make it safe for Juno to start enrolling new patients again.

Two days later, Juno quietly filed a financial document revising its earlier statements to acknowledge that a total of four young patients, not three, had died during its clinical trials testing a family of immunotherapy treatments known as CAR-T. Three of the deceased patients had received a specific CAR-T treatment in recent months; the fourth had been enrolled in an earlier trial of a different therapy.

All that made it an emotional week for cancer patients testing other immunotherapies, which have delivered spectacular results — but only for a minority of patients. STAT asked a few patients, their loved ones, and their advocates how they were feeling about the news. Here’s what they had to say:

‘My Hail Mary’

Stephen Estrada, a 30-year-old hairstylist from Denver, was running out of options to fight his advanced colon cancer last year when he joined an experimental trial that he calls “my Hail Mary.”

He now gets infusions every three weeks of a therapy combining a checkpoint inhibitor — a type of immunotherapy that takes the brakes off the immune system — and a monoclonal antibody, engineered to attack defects in cancer cells. His tumors are shrinking, and he’s back to working out at the gym most days.

Estrada called the Juno patient deaths “heartbreaking,” but the news didn’t make him second-guess his choice to join his own trial.

“I think that most people in our shoes would still take that chance at a potential cure or a longer life, even if it meant possible death” from an unexpected side effect of a new treatment, he said.

Estrada said he was excited to hear that the Food and Drug Administration had cleared Juno to start the trial again because he believes in the CAR-T approach, which involves removing a patient’s immune cells, rewiring them to seek and destroy cancer cells, and then infusing them by the billions into the patient’s blood.

“I know that as far as the CAR-T trials go, if my drug fails me, I, of course, want to be able to jump onto that,” he said.

Estrada’s message for other cancer patients taking or considering immunotherapies: “I’m hoping that people will realize that just because people died doesn’t mean we should stop — or that they should be scared.”

‘It does affect you’

Nicholas Wilkins, an 18-year-old from Virginia, stopped responding to chemotherapy three years ago, a decade after first being diagnosed with leukemia. So he joined a trial at Children’s Hospital of Philadelphia testing another CAR-T immunotherapy being developed by Novartis. After a one-time infusion, he’s doing well and planning to go to college.

Learning about the Juno patient deaths last week was tough on Nicholas’s mother, Lisa Wilkins, who is deeply grateful that CAR-T has helped her son.

“Hearing of so many other patients that don’t respond — it does affect you,” she said. “In some respects, I feel guilty when I hear of kids that don’t do well or that relapse, because you want everyone to do well. It’s kind of like the survivor complex.”

‘It’s scary’

Rachel Lefebvre, a 43-year-old psychologist from Florida, had exhausted most of her options to fight her advanced colorectal cancer before she started an early-stage safety trial last month of an immunotherapy being developed by Merck. It uses specific proteins, called immunocytokines, to stimulate the immune system.

She’s felt some pain and fatigue after receiving two infusions of the drug. An early scan suggested that her tumors looked bigger, but it’s not clear whether that’s due to inflammation or actual tumor growth.

Hearing about the Juno patient deaths was “scary,” she said. “We don’t know what some of these experimental drugs can do and we don’t have a lot to compare it with.”

But she’s still going to stick with her trial. “I’m willing to do anything that has a chance of keeping me here for as long as possible,” she said.

‘Time is not on our side’

Linda Cegelski, a 50-year-old corporate training director from Charlotte, N.C., had been struggling with chemotherapy to fight her inoperable advanced colorectal cancer. Last year, she joined an experimental trial for a checkpoint inhibitor. (The immunotherapy drug she’s testing, which is sold by Bristol-Myers Squibb as Opdivo, is already approved to treat several other types of cancer.)

Eight months after joining the trial, Cegelski is feeling good, though she’s not back to work full-time. Learning about the Juno patient deaths was hard, but she was encouraged to hear that the trial had restarted so quickly.

“Time is not on our side,” she said. Hearing that regulators gave Juno the green light, she said, is a “kind of reassurance that I’m doing the right thing.”

‘Don’t tip the apple cart’

Juno’s CAR-T trials are just one of many treatment approaches within the umbrella category of of immunotherapy, which aims to marshal the body’s immune system to attack cancer.

Linda House, an advocate for cancer patients, thinks it’s key to make those distinctions clear.

“I just think that we all have to be very careful in messaging so that we don’t tip the apple cart and create a sense of panic in patients who are on a therapy that has been well-tested, deemed to be safe by the FDA, et cetera,” she said. “You can’t paint all of the immuno-oncology drugs with the same brush.”

Many patients seem to recognize those distinctions: House, president of the headquarters of the nationwide Cancer Support Community, said the group hasn’t heard from any patients concerned or fearful in the wake of the Juno patient deaths.