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- Federal regulators have approved a new CAR T cancer drug as a treatment for certain forms of multiple myeloma.
- Cancer experts say the approval of cilta-cel will provide more options for people who have not responded successfully to other treatments for multiple myeloma.
- CAR T treatments use a person’s genetically modified T cells to fight cancer.
The Food and Drug Administration (FDA) has考虑到绿灯to cilta-cel, a cancer treatment for people with pretreated multiple myeloma.
The drug will be sold under the brand name Carvytki. It’s manufactured byJanssen Pharmaceuticals, a company owned by Johnson & Johnson.
It’s thesecond such therapyapproved for multiple myeloma with the approval of Bristol Myers Squibb’sAbecmalast year.
“The FDA approval of Carvykti [cilta-cel] marks the first approved cell therapy for Janssen, a testament to our continuing commitment in oncology to deliver new therapeutic options and drive toward our vision of the elimination of cancer,”Mark Wildgust, PhD, vice president of global medical affairs for oncology at Janssen, told Healthline,
Cilta-cel is part of the expanding world of chimeric antigen receptor T-cell immunotherapy, also known as CAR T therapy.
CAR Temploys a person’s own genetically modified T cells to find and kill cancer.
Experts in the cancer treatment field hailed this latest FDA approval.
Dr. Joseph Mikhael, the chief medical officer of the International Myeloma Foundation and a professor at the City of Hope Cancer Center in California, told Healthline the approval of cilta-cel is a major step forward.
“We already have one CAR T-cell product approved, but with cilta-cel the response rate has tripled and it has shown to be more durable than other therapies,” said Mikhael.
He predicted that CAR Ts such as cilta-cel as well as others that are in the pipeline will have an increasingly large role in myeloma.
“Maybe even in earlier stages of the disease,” he said. “There is tremendous enthusiasm. Even a geeky hematologist like me, my jaw drops to the floor when I see what this treatment can do.”
Dr. Caitlin Costello, an oncologist at Moores Cancer Center at the University of California San Diego Health, told Healthline that CAR T offers at least durable remissions and cures in some cases, and allows for prolonged treatment-free intervals.
“This is particularly exciting for a cancer like multiple myeloma that has essentially required continuous and indefinite treatment to maintain remissions, which while successful, can have both clinical and financial implications,” she said.
The FDA approval was the result of clinical trial datapresentedat the American Society of Hematology annual meeting in Atlanta, Georgia, in December.
That trial showed 83 percent of participants achieved a complete response at a median follow-up of 22 months.
临床医生也报道,evalua的92%ble patients achieved minimal residual disease negativity with progression-free survival and overall survival sustained in those participants for more than 6 months.
People with multiple myeloma for whom at least three treatment regimens have stopped working face a median survival of less than a year with currently available treatments.
Dr. Sen Zhuang, the vice president of clinical research and development at Janssen Research & Development, the company that manufactures cilta-cel, said the clinical trials were hopeful news for people with this type of cancer.
“These data add to the growing body of evidence supporting the potential clinical benefit of cilta-cel in the treatment of patients with relapsed and/or refractory multiple myeloma, a population in need of new options,” Zhuang said in apress statementin December.
Dr. Thomas G. Martin, a principal study investigator and the director of clinical research at the University of California San Francisco’s Helen Diller Family Comprehensive Cancer Center, and principal study investigator, was also optimistic.
“[The study data] build upon previous results that show that a single infusion of cilta-cel resulted in durable responses and long-term survival across the study population, further confirming the potential of cilta-cel in offering patients and physicians a valuable new treatment option,” Martinsaidat the time.
CAR T therapies are creating a new paradigm for blood cancer treatments.
In October, the FDA
The approval, the second for Tecartus, was based on results of a clinical trial of more than 50 adults who were treated with the drug.
Within 3 months of receiving the treatment, more than half of the participants had no signs of cancer.
“We haven’t seen responses and durations of responses like this,”Dr. Bijal Shah, an associate member in the department of hematology at the Moffitt Cancer Center in Florida who was the clinical trial’s lead investigator,
CAR T treatments have had some serious side effects in the past, but medical experts say they are getting those under control.
“As for the cytokine release syndrome and neurotoxicity, these are well-known side effects of therapy that physicians have become very comfortable in managing. Ongoing evaluation of preventative approaches for CRS/neurotox as well, will potentially mitigate these side effects and allow for more broad eligibility for these treatments,” said Costello.
Here is a brief rundown of CAR T therapies that have been previously approved and are now available.
- Abecmafrom Bristol Myers Squibb is for adults with relapsed or refractory multiple myeloma after four or more prior lines of therapy.
- Breyanzifrom Bristol Myers Squibb is for adults with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy.
- Kymriahfrom Novartis is for adults with relapsed or refractory diffuse large B-cell lymphoma and young adults up to 25 years old with relapsed or refractory acute lymphoblastic leukemia.
- Tecartusfrom Kite, a Gilead company, is for people with relapsed or refractory mantle cell lymphoma. Tecartus is also for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia.
- Yescartafrom Kite, a Gilead company, is for people with large B-cell lymphoma or follicular lymphoma.