University of Vermont Medical Center hematologist Dr. Chris Holmes got a first look at CAR T-cell therapy several years ago when a patient of hers, a woman with multiple myeloma, traveled to Boston for the new cancer treatment.
The patient’s disease, which had not responded to other options, afterward went into remission. But the cost of traveling and staying in the city significantly strained her finances.
“At that point, we knew we had to get it here, because we can do the very same therapy,” Holmes said. “Then our patients can remain close to home.”
Today the innovative blood cancer treatment is available through the UVM Cancer Center, a partnership between the medical center and the UVM Larner College of Medicine.
For now, the center can provide CAR T-cell therapy for lymphoma and certain types of leukemia for adults. The program may expand to the treatment of multiple myeloma and childhood leukemia at a later date, said Cancer Center director Randall Holcombe, who’s also a medical college dean, at a virtual press event on Wednesday announcing the new program.
“This therapy truly is groundbreaking and has completely changed the lives of many patients,” said James Gerson, who was recruited to Burlington to start the treatment program. He came from the University of Pennsylvania, where the first such treatment was developed and went through clinical trials.
The treatment is also very expensive — around $400,000 on average. But only one course is needed, so for some patients, it would likely be less expensive overall than a cancer therapy given once a week for several years, Gerson said. In his experience, both private and public insurance should provide coverage if CAR T-cell therapy is used as a secondary or tertiary treatment option, currently what it is approved for by the U.S. Food and Drug Administration.
Gerson’s program at the cancer center is bringing CAR T-cell therapy to Vermont and northern New York for the first time. The FDA has approved the therapy for an increasing number of blood cancers over the last three years. The treatment has become available in many of the more populated parts of the country.
Until now, the closest places for Vermonters to receive treatment have been Dartmouth Hitchcock Medical Center in New Hampshire, several Boston hospitals and the academic medical center in Rochester, New York, Gerson said.
“Bringing this type of leading-edge care is the advantage of having an academic medical center in our region,” said Richard Page, dean of the UVM medical school and a member of the UVM Health Network board, at the press event.
The UVM Cancer Center anticipates treating about one patient a month at the outset, but the program can grow to meet demand. Gerson advised patients who think they may be eligible for the treatment to talk to their oncologists.
What makes the treatment unique is that a person’s own T-cells are genetically modified to make them more able to find and attack the cancer cells. T-cells, a type of white blood cell, are a key part of the body’s immune system.
Clinical trials have found that almost all patients see a partial retreat of their cancer. A significant portion — varying from 50% to 80%, depending on the cancer type — achieve complete remission.
Gerson saw those kinds of results in the almost 100 patients whom he treated personally at the Philadelphia academic hospital.
“For all intents and purposes, many of these patients with advanced cancer who had really no other treatment options ended up being cured from their cancer after receiving this treatment,” he said.
The treatment process takes three to six months, but is completed as outpatient, unless complications develop. The most intense visits occur over the course of a month, according to the medical center. In the first part of the treatment, a patient’s blood is circulated through a machine that separates out enough T-cells to send to a private lab for modification.
The decades-old technique, which uses a viral vector to insert sections of DNA into a cell’s protein production machinery, is common to research laboratories around the world. In this case, the new section of DNA directs the T-cell to build a protein on its surface called a “chimeric antigen receptor,” or CAR, that has been engineered to bind to other proteins on the surface of a cancer cell. The modified T-cells are then forced to multiply until they reach around 70 times their original volume.
“They’re eventually given back to the patient, sort of similar to a blood transfusion,” Gerson said.
UVM Medical Center has contracted with Kite Pharma — a subsidiary of California-based Gilead Sciences, which owns one of the FDA-approved processes — to perform the lab work.
Patients can experience side effects after the altered T-cells are reintroduced to the body related to having a “supercharged” immune system, Gerson said. The most common side effects are fevers, which can progress to become severe; low blood pressure; and issues with organ functioning. Serious cases are rare, as doctors now know how to both prevent the effects and treat them quickly if they start.
“Because we’re well aware of the side effects, we monitor patients very closely for it,” Gerson said.
Blood cancers account for about 10% of all cancers, according to the Leukemia & Lymphoma Society. They were diagnosed in about 186,400 people nationwide in 2021.
So far, CAR T-cell therapy has been less effective on so-called solid tumor cancers, such as breast, prostate or lung. Scientists are not yet sure why, and it is an area of active research that Gerson hopes to bring to UVM after the clinical program is fully established.
That would allow patients to participate in clinical trials of “products that are on the forefront of discovery, both in blood cancers as well as in solid cancer,” he said.