Sophisticated therapy that enlists the body to fight cancers of the blood by priming a component of the immune system to strike a specific target is in the vanguard of innovative approaches to malignancies.
The emerging treatments — known as CAR T-cell therapy — are customized to each patient, whose T cells are isolated from a blood sample and then activated in a laboratory to zero in on a bull’s-eye.
T cells are key members of the immune system. Once prepped to seek a precise mark, the cells hunt it around the clock.
Two such therapies have been federally approved for certain forms of leukemia and lymphoma, though they come at huge cost. One of the products, developed for children and young adults, approaches $500,000. Some cancer centers are shunning the products, citing the exorbitant prices.
Stony Brook University Hospital doctors are developing their own CAR T-cell treatment and plan to offer it to patients through a clinical trial within three months — at no charge. They are not administering the other therapies, citing their expense.
Dr. Theodore Gabig, a hematologist and oncologist at Stony Brook Cancer Center, said the trial is designed to treat T-cell lymphoma, a form of cancer also referred to as non-Hodgkin’s lymphoma.
“This would be for refractory T-cell lymphoma, to get control of the disease and get them to a stem-cell transplant,” Gabig said. Refractory T-cell lymphoma is cancer that is not responding to therapy.
Doctors who are welcoming the two newly approved approaches as part of their treatment plans said they want to give cancer patients as many options as possible.
“We want to offer them because we want to take care of the patients in our community and give them the highest level of care,” said Dr. Ruthee-Lu Bayer, director of the adult stem-cell program at Northwell Health’s Cancer Institute in Lake Success.
She noted that the products are a form of immune therapy combined with cellular therapy.
“They call them living drugs,” Bayer said. “Scientifically, this is fascinating.”
Northwell is the farthest along of two Long Island cancer centers that plan to offer the therapy. NYU Winthrop Hospital in Mineola is the other.
Dr. Jeffrey Schneider, chief of oncology and hematology at NYU Winthrop’s cancer center, said there is no CAR T-cell program currently at his institution, but that he and his colleagues plan to have one in the near future.
CAR T-cell therapy, also known simply as CAR T, was pioneered by medical scientists Isabelle Rivière, Michel Sadelain and Renier Brentjens of Memorial Sloan Kettering Cancer Center in Manhattan. CAR T stands for chimeric antigen receptor T-cell therapy.
The team’s breakthrough discoveries led to clinical studies in this country and abroad in which pediatric and adult patients developed high rates of remission without the need for additional therapy. A remission rate of 93 percent was reported in a small study of one of the therapies. The treatments are not yet offered at Sloan Kettering’s satellites on Long Island.
An international study, published in a February issue of the New England Journal of Medicine, also reported high rates of remission for pediatric and young adult patients who had failed other therapies. CAR T cells, the study revealed, persisted in patients’ blood for 20 months.
The treatments involve some of the most groundbreaking research to address cancer, crossing multiple disciplines in science and medicine.
For example, the therapies are called individualized — or customized — because patients’ own cells are key ingredients. Activating patients’ T cells in manufacturers’ laboratories to recognize a cancer target, called CD19, involves genetic modification of the cells, a process that can take up to three weeks.
The first CAR T-cell therapy was approved by the U.S. Food and Drug Administration in August. Manufactured by Novartis, it goes by the commercial name Kymriah and costs $475,000. The therapy was designed for patients 25 and younger who have B-cell acute lymphoblastic leukemia.
In October, the FDA approved Yescarta, developed by Gilead Sciences Inc., for adults. It costs somewhat less, at $373,000, and is aimed at patients who have relapsed on conventional therapies for B-cell lymphoma.
Cancer specialists at Northwell “have not yet given our patients either of these FDA-approved products, but our facility has just undertaken a pretty extensive process to implement the therapy,” Bayer said.
Her colleagues at Northwell’s Cohen Children’s Medical Center, also in Lake Success, are taking steps to begin the treatment there.
“Not just any institution will be able to utilize these therapies,” Bayer said. “They will be regulated, like stem-cell transplantation.”
She cautioned that the treatments are “not a slam dunk.”
“I call them boutique products. They offer a line of treatment that we otherwise would not have,” Bayer said. “There are still questions about long-term durability, and the [research] follow-up has been short.”
For patients, the question is how to meet the price tag.
The high costs have raised questions among government and private insurers. Pharmaceutical companies offer patient-assistance programs that can cover some or all the costs of some therapies. Grants are available through nonprofit organizations.
Novartis and Gilead have vigorously defended their pricing in recent months, saying that their proprietary therapies are labor-intensive and highly personalized for each patient.
The treatments, both for children and adults, are infused only once.
Stony Brook’s Gabig said costs eventually may be dramatically shaved from CAR T-cell therapy by using a standardized cell line, as opposed to harvesting a patient’s own T-cells to create a customized product.
“It’s extraordinarily expensive the way they are designed now, being unique to each patient,” he said.
The Stony Brook CAR T-cell research is headed by two scientists, Dr. Yupo Ma and Dr. Huda Salman.
The science underlying CAR T-cell therapy is similar to that of the prostate cancer treatment known as a cancer vaccine, Provenge.
That treatment involves isolating patients’ dendritic cells, also components of the immune system, and priming them in a lab to seek out a specific target on metastatic prostate cancer cells.
Five years ago, that therapy was lambasted by critics as expensive at $99,000 for three treatments.
“That looks like a bargain now,” Bayer said.