A new cancer treatment that could eventually replace chemotherapy and bone marrow transplants — along with their debilitating side effects — soon will be offered at two of Tampa Bay's top-tier hospitals.
The U.S. Food and Drug Administration in August approved the first ever Chimeric Antigen Receptor Therapy, or "CAR-T cell therapy," for children and young adults up to age 25 suffering from leukemia and other blood and bone cancers. And just this week, the agency approved the same immunotherapy for adults with large B cell lymphoma, a form of non-Hodgkin's lymphoma.
Johns Hopkins All Children's Hospital in St. Petersburg will be the first and only pediatric hospital in Florida to become a certified treatment center. Moffitt Cancer Center in Tampa also will be among the first in the country to offer the same therapy on adults with blood cancers like B cell lymphoma.
CAR-T uses white blood cells from the patient's immune system and re-engineers them in a lab to target and wipe out cancer cells. Specialists draw the cells from the patient's blood and "re-program" them to go after blood and bone marrow-type cancer cells instead of the flu or any other bacteria or infection they would normally attack, said Dr. Frederick Locke, a principal investigator for the experimental therapy at Moffitt.
During the laboratory process, scientists work with receptors, which are molecular structures in cells that tell them what to do based on messages they receive in the bloodstream. In this instance, they add a chimeric antigen receptor, or CAR, to each T-cell. The CARs are programmed to target a specific protein, called CD19, found on cancer cells.
Then the newly trained T-cells are infused back into the body to do their job.
"T-cells normally circulate in our blood. They hang out in the lymph nodes and help fight infection," Locke said. "Some recognize the flu, others recognize tetanus. So we retrain the gene to express a specific molecule on the T-cell, which tells it to activate a fight when it sees only a cancer cell."
FDA Commissioner Scott Gottlieb called it "a new frontier in medical innovation" with "the potential to transform medicine."
Locke treated 101 cancer patients with CAR-T during a trial at the Moffitt Cancer Center in Tampa over the last two years. Eighty two percent of those cases responded well to the treatment, and 44 percent are still in remission at least eight months later, he said.
B-cell lymphoma is an aggressive, fast-moving cancer that accounts for nearly 30 percent of all newly diagnosed cases of non-Hodgkin lymphoma in adults in the United States, according to Moffitt. Locke and his team will present one-year follow-up results at the American Society of Hematology Annual Meeting in December.
The new therapy is a game-changer for leukemia patients, said Dr. Peter Shaw, deputy director of Johns Hopkins All Children's Cancer & Blood Disorders Institute. While children diagnosed with leukemia normally have high rates of remission, a small percentage do not respond well to chemotherapy and radiation.
Benjamin Gilkey, a 7-year-old from Sarasota, was one of those cases. Diagnosed with acute lymphoblastic leukemia in 2014, "Benji" and his family began a difficult, nearly three-year journey with the hope of remission at Johns Hopkins All Children's. Benji was considered a "very high risk" patient because he was slow to respond to treatment. He eventually developed an extreme toxicity to one of his chemotherapeutic medicines, according to his mother, Laura Gilkey, and suffered a relapse. He died in February.
"I can still picture the hospital rooms there. Imagine having the therapy right there in the hospital, it would eliminate so much time needed for trips for trials," Laura Gilkey said. "It will draw more people to Johns Hopkins just for this kind of treatment. We were ready to go to Philly for Benji to partake in the same type of treatment, but then he relapsed. We were ready to go anywhere."
Shaw says CAR-T could have helped Benji and other rare high-risk children who don't respond well to chemo and other more traditional cancer treatments. He expects the therapy to draw patients from all over the southeast.
"We hope to be up and running in the next few months because we see this as a potential magic bullet for the highest of high-risk patients with no other option," Shaw said. "These are the kind of patients where their prognosis is an end-of-life issue. Now we know how to save them."
But like any treatment, CAR-T doesn't come without some side effects.
Patients are at risk for high fever and inflammation since their immune systems have been re-trained to fight just cancer cells. Low blood pressure, acute kidney injury and decreased oxygen are some other common side effects.
But for Dimas Padilla, the CAR-T side effects were far easier to handle than another round of chemotherapy, or another bone marrow transplant.
Padilla was diagnosed with a form of non-Hodgkin lymphoma in 2011. After undergoing chemo, he was in remission for about a year before the cancer came back. The second time around, he had a painful bone marrow transplant. After a two-year period of remission, the cancer came back a third time at an aggressive stage-four status.
"They gave me six months to live the third time around," said Padilla, a father of two young daughters who lives in Orlando. He said he had large tumors growing around his lymph nodes that pressed his vocal chords to the point that he couldn't talk. "My oncologist called Moffitt and got me into the trial because the chemo wasn't working," he said.
During the CAR-T treatment, he experienced fevers topping 105 degrees. Padilla remembers shaking and trembling a lot and having some temporary memory loss. He spent two days in the intensive care unit after his treatment, but said he would do it again.
"The bone marrow transplant was the worst. You feel sick for so long. With CAR-T, it was intense for maybe a week," Padilla said. "It took a little while to feel normal again, but I've been in remission for a year. CAR-T gave me my life back. This is going to be the cure for cancer."
Shaw says more studies need to be done on CAR-T, but he sees the potential in this treatment to one day replace chemotherapy for patients with specific types of cancer.
"It's very new and exciting," Shaw said. "There are new studies that show how it can target other cancers as well, and more research is in the pipeline. There is a possibility that it could be a better treatment than what we've been using for many years."
Johns Hopkins All Children's was chosen as one of about 20 pediatric hospitals in the country to debut this new program through a partnership with Novartis, the multinational pharmaceutical company administering pediatric CAR-T treatments under the name, Kymriah. Staff are already preparing a lab and hiring new technicians in the hospital's bone marrow transplant department for when the time comes, Shaw said.
At Moffitt, meanwhile, successful clinical trials using the same therapy on adults have yielded unprecedented success rates, Locke said. Moffitt created its own department for CAR-T at its center in Tampa and has hired physicians and technicians to run it, he said. The center will administer the adult approved therapy, called Yescarta, through a partnership with Kite Pharma, Inc.
"As many as 10,000 patients a year could benefit from this therapy," Locke said. "Moffitt has made it a top priority. We're such a high-volume center in Tampa, and there are many people who need it. Luckily it's worked for a number of them who have tried it already. About 50 percent of patients had a complete disappearance of their lymphoma."
Contact Justine Griffin at [email protected] or (727) 893-8467. Follow @SunBizGriffin.