The Biggest Cancer News Of 2017? 'CAR-T Cells, Hands Down'

As 2017 and our series, "This Moment In Cancer," draw to a close, I asked a handful of local experts: What was the biggest cancer news of the year?

They all said roughly the same thing.

• "That would have to be CAR-T cells, hands down," replied Mara Bloom, director of the cancer center at Massachusetts General Hospital. "This is a breakthrough therapy and patients — all of us here at Mass. General — are really excited about it."

• "2017 saw two different CAR-T therapies approved," answered Dr. Bruce Johnson of the Dana-Farber Cancer Institute, current president of the 45,000-member American Society of Clinical Oncology. More broadly, he said, the biggest cancer news is that immunotherapy — treatments that harness a patient's own immune system, including CAR-T — have "come of age.'"

• The advent of CAR-T therapies as "the first gene and cellular therapy for cancer" stands "head and shoulders" above other developments in 2017, said Dr. Glenn Dranoff of Novartis, which makes one of the two CAR-T treatments approved this year.

The next question, for all non-specialists, is naturally "What is CAR-T cell therapy, again?"

Reporter Karen Weintraub wrote a vivid story this year on CAR-T treatments that featured Judith Wilkins, a hairdresser in Woburn, who has a type of cancer called B-cell lymphoma. Wilkins compares CAR-T cells to the old 'Pac-Man' computer game: "It's like a Pac-man in my body," she said. "The cells just cruise through your blood and identify the bad B cell and just eats it."

Karen wrote in October:

This week, the U.S. Food and Drug Administration approved the second therapy in this class, called Yescarta, to treat advanced B-cell lymphomas, like Wilkins’. The first CAR-T drug, Kymriah, received approval in August to treat an advanced form of childhood leukemia. There are nearly 200 clinical trials exploring CAR-Ts currently registered with the federal government.

Both therapies — and others like them still in the works — are tailored to each patient and re-engineer their body’s immune system to attack their cancer. The T in CAR-T refers to T cells, key soldiers of the immune system that normally attack invaders.

Eventually, CAR-T therapies are expected to help a large percentage of patients whose leukemia, lymphoma or multiple myeloma has resisted first-line treatments. Studies suggest that about 80 percent of Yescarta patients get some improvement from the treatment, with about 30 percent keeping those benefits long-term.

Only a few thousand people can benefit from CAR-T treatments at this point, and right now, they only work against blood cancers, not solid tumors. But this is looking like only the beginning for CAR-T cell therapy, and more broadly, cancer treatments that use the patient's own cells.

The downsides: Some patients get serious side effects that land them in intensive care units. And the price tags are steep. The CAR-T treatments are heading upward toward half a million dollars: One is about $370,000 and one is $475,000.

On the other hand, these could be one-and-done treatments, Dr. Dranoff said, "where patients can get their own cells that have been genetically altered as a single treatment, and then achieve long-term benefits without requiring additional therapy."

'An Extraordinary Coalescence'

To pan out a bit, the progress on CAR-T treatments is part of broader progress on immunotherapy. Dr. Johnson of Dana-Farber said he uses the term "coming of age" because "we've been seeing remissions — and remissions that are lasting" in patients followed beyond three years.

He points to the example of patients with advanced melanoma, long considered an incurable and deadly disease, who were treated with a combination of immunotherapy drugs.

"Patients are getting complete remissions, and they're lasting for years, unlocking what we don't commonly talk about: a possibility of curing advanced cancer," he said.

Cancer specialists normally eschew the word "cure," and when I asked Dr. Johnson about using it, he allowed, "It's a little early. But one of the first things you have to do is see people surviving without their cancer for several years, and we're seeing follow-up data from trials that are out beyond three years."

Not to get too rosy here. Millions of people worldwide, and more than 600,000 Americans, are still dying of cancer every year. Cancer news tends to focus on just a few pockets of progress, and immunotherapy is only helping a minority of patients at this point.

Dr. Johnson, a lung cancer specialist, said that in lung patients who get immunotherapy, about half see no improvement, while about one quarter are alive two years out. Still, he said, even those mixed results are noteworthy: "For a person who treats people in the clinic with these agents, you can have a very different conversation with people," he said, "and for us, that's an incredible advance that we've seen in 2017."

To end with a plug for science, Dr. Dranoff at Novartis, a longtime leader in cancer immunotherapy research here in Boston, said this about the dawn of this CAR-T era:

This represents four decades of basic research in genetics, virology, gene therapy, immunology, cancer biology and clinical investigation. It's an extraordinary coalescence of learnings that have now all been integrated into a therapy that's transforming the lives of cancer patients who previously had no meaningful chance of achieving long-term survival.