(Bloomberg) -- Multiple myeloma is the drug industry’s next bet for a novel genetic therapy that has given dying patients with other forms of blood cancers a new shot at life.
While it’s too early to tell whether it will yield the same powerful results, companies are rushing in. Celgene Corp. and Bluebird Bio Inc. are in the lead, but pharmaceutical heavyweight Johnson & Johnson vowed this weekend to accelerate its effort. More than a half dozen of the gene-altering therapies are in development, all using the same target: a protein called BCMA.
“There is a lot going on -- BCMA is crazy,” said Henry Fung, vice chairman of hematological malignancies at Fox Chase Cancer Center in Northeast Philadelphia. “Everyone is targeting the same thing.”
It’s easy to see why there is so much excitement. Multiple myeloma is one of the most common types of blood cancer, with about 30,000 Americans diagnosed each year. The first two genetic therapies to reach the market last year, for much smaller groups of hard-to-treat pediatric leukemia and diffuse large B cell lymphoma, have put 39 percent to 62 percent of patients into deep and long-lasting remission. Doctors believe some patients may be cured.
Early data presented at the American Society of Hematology meeting in San Diego this week showed that the genetic therapy known as CAR-T can also spur complete responses in multiple-myeloma patients, but there’s no proof yet that the results will be powerful enough to keep the cancer at bay.
“It’s clear the therapy can work,” said Fred Locke, a medical oncologist and leader of the immune cell therapy initiative at Moffitt Cancer Center in Tampa, Florida. “Will it be a cure for multiple myeloma? I don’t know. The data isn’t suggesting that to me at this point.”Malignant Cells
The challenge with multiple myeloma is its biology of malignant cells that invariably return, forcing doctors to cycle patients from one toxic treatment to the next. The hope is that CAR-T therapy will perform better because it involves engineering the patient’s own infection-fighting cells to destroy the cancer.
“If you look at lymphoma trials, patients in complete remission at three months have a very good chance of remaining in complete remission for two years and beyond,” Locke said. “We don’t know that for myeloma.”
The cancer has returned in many myeloma patients who initially showed no signs of disease after CAR-T treatment -- even with highly sensitive testing methods that can detect one cancerous cell in a million. In contrast, in the earlier leukemia and lymphoma studies, those are the patients doctors believe are most likely to be cured.
Still, there was promising data at the hematology meeting, stemming from studies of the sickest patients who may be most difficult to treat.
Celgene and Bluebird’s experimental CAR-T therapy bb2121 is “leading the pack in the overcrowded BCMA space,” said Biren Amin, a health care analyst at Jefferies LLC. Ten of 12 patients treated with a next-generation version of the compound initially responded to treatment, and nine continue to benefit.
A different compound from Celgene, JCARH125, was able to maintain a higher number of engineered cells in the patient’s bodies, which could mean it will work for longer periods.J&J Move
J&J put its rivals on notice with plans to push deeper into CAR-T with technology it licensed from Chinese partner Nanjing Legend Biotech, a unit of GenScript Biotech Corp. Data presented on their first compound, LCAR-B38M, showed 88 percent of patients responded to treatment, with 74 percent being in complete remission.
Other contenders include Allogene Therapeutics Inc., which is seeking regulatory approval to study an off-the-shelf CAR-T that could be used for any multiple-myeloma patient -- current CAR-Ts are tailored to each patient.
Companies including Amgen Inc. and GlaxoSmithKline Plc are developing related drugs that target the BCMA protein, found in myeloma cells. They could be easier to use than the bespoke CAR-T treatments, though they may not be as potent, said Fung, the blood cancer expert from Fox Chase.
“Who is going to be winning, who is going to be cured, it may take five or 10 years before we know,” Fung said.
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