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[MINNEAPOLIS] A novel therapy from Bluebird Bio Inc and Celgene Corp for patients nearing death from an aggressive form of blood cancer appears to get more effective over time, wiping out signs of disease in more than half of those treated in a small trial.
A single infusion of the personalised therapy known as bb2121 generated a response in all but one of the 18 multiple myeloma patients treated at the highest dose, according to the study presented at the American Society of Haematology meeting in Atlanta. After nine months, 56 per cent were in remission. That's an improvement since May, when an earlier look at the data found 27 percent of the patients experienced a complete response.
"Most of these patients were ready for hospice, and then this came along," said Jesus Berdeja, director of myeloma research at Sarah Cannon Research Institute in Nashville, and one of the first doctors to use the approach. "These are just spectacular results in a patient population with a median expected survival of four months or less." The treatment, known as a CAR-T, involves removing the patient's own immune system cells, tweaking them to attack the cancer, and infusing them back into the person. The first two CAR-Ts, from Novartis AG and Gilead Sciences Inc, were approved this year in the US to treat certain forms of blood cancers. The Bluebird-Celgene therapy targets a different protein, BCMA, found on myeloma and plasma cells.
In the bb2121 study, the improvements extended as long as 15 months for patients who had previously tried all existing therapies, including stem cell transplants, and seen their cancer return.
Minimal residual disease, a measure of tiny levels of cancer cells found using the most sensitive laboratory tests, was present in only one of the 10 patients tested. The responses have persisted for more than a year in some patients, according to James Kochenderfer from the Center for Cancer Research at the National Cancer Institute, the study's senior author.
Longer study in more patients is needed before any conclusions about the therapy can be drawn, the researchers said. Not everyone improved, and among those who did the benefit wasn't always durable. After six months, 81 per cent of patients hadn't had their cancer worsen, which fell to 71 per cent after nine months, the study found.
Out of an extended group of 21 patients, 15 developed cytokine release syndrome, a potentially deadly immune system response to the therapy, and five experienced neurotoxicty, including one 46-year-old woman who had a severe reaction.
Bluebird, of Cambridge, Massachusetts, and Celgene, based in Summit, New Jersey, are starting to enroll patients in a larger study known as pivotal that is used to seek regulatory approval, with a goal of 94 patients. They will be followed for six months after the one-time infusion.
It would be possible to fully enroll the study within a few months and have the therapy available in about a year, said Mr Berdeja, the director of myeloma research.
There are about 30,000 new cases of multiple myeloma diagnosed each year in the U.S., according to the American Cancer Society. As many as 100,000 people may be living with refractory disease that hasn't responded to previous treatment, Mr Berdeja said.
Bluebird and Celgene aren't the only ones looking to treat multiple myeloma by targeting the BCMA protein.
A rival CAR-T from Novartis and the University of Pennsylvania posted promising results at the haematology meeting, showing eight of 10 patients given a high dose responded, including one entering remission. A separate first-in-human study showed GlaxoSmithKline Plc's GSK2857916, a traditional biotechnology medicine that targets BCMA with an attached chemotherapy drug, generated a response in 21 of 35 patients.
"All of these data indicate BCMA is an attractive target in refractory myeloma," said lead author Adam Cohen, director of Myeloma Immunotherapy at Penn's Abramson Cancer Center.