One strong woman — joplin resident receives new cancer treatment news antiemetic drug names

The U.S. Food and Drug Administration in October approved Yescarta, a one-time, custom-made CAR-T treatment for aggressive lymphoma in adults. CAR-T treatment uses gene therapy techniques not to fix disease-causing genes but to turbocharge T cells, immune system soldiers that cancer can often evade.

The process calls for T cells to be filtered from a patient’s blood and reprogrammed to target and kill cancer cells; hundreds of millions of copies are then grown. Returned to the patient, all the revved-up cells can continue multiplying to fight disease for months or years. That’s why these immunotherapy treatments are called “living drugs.”

“(The) approval of Yescarta is a very significant advance for lymphoma patients and for the cancer community as a whole,” Louis J. DeGennaro, president of the Leukemia Lymphoma Society, said in a statement.

“Immunotherapy is dramatically changing the way we approach blood cancer treatment.”

The treatment, from Kite Pharma, which became a subsidiary of Gilead Sciences two months ago, is for patients with three types of aggressive, or fast-growing, large B-cell lymphoma. The most common type accounts for about a third of the estimated 72,000 new cases of non-Hodgkin lymphoma diagnosed each year.

Yescarta, also known as axicabtagene ciloleucel, was approved for patients who have already been treated with at least two cancer drugs that either didn’t work for them or eventually stopped working. At that point, patients are generally out of options and only have about a 10 percent chance of even temporary remission of their cancer, said Frederick Locke, director of research for the Immune Cell Therapy Program at Moffitt Cancer Center in Tampa, Florida.

In a key test during trials, Yescarta was given to 101 patients. About 72 percent saw their cancer shrink, and about half showed no sign of disease eight months later. The therapy is still working in most study participants, so the average duration of its effects isn’t known yet.

McCulley’s battle with cancer happened swiftly and suddenly following a tonsillectomy in March. She told her doctors at Mercy Hospital Joplin about some lingering discomfort and swelling in her neck, and pathology tests revealed that she had lymphoma.

The following month, she started one form of chemotherapy that consisted of six treatments, one every three weeks. When a scan later found evidence of cancer still in her body, she embarked on a second form of chemotherapy. That, too, failed to kill all of the cancerous cells.

When McCulley, a former administrator with Webb City schools, received word that the lymphoma was actually spreading, which began to affect the nerves in her head and her range of motion, she started 10 days of radiation. In her spare time, she began researching alternative cancer treatments. What were the options, she wondered, when standard treatments didn’t work?

She had already been reading up on Yescarta when her oncologist recommended her as a candidate for it. A test to determine whether she would be eligible for the treatment through The University of Kansas Hospital ended in temporary heartbreak when she was initially denied because of her bloodwork. She would later tell her nurses that she had no hope left.

McCulley left for Kansas City immediately after that phone call, and she was ultimately approved to become the first person in this region to participate in the new treatment. Nurses harvested 287 milliliters of her T cells during a four-hour procedure on Nov. 28 and sent the collection to a laboratory in California to be modified with a special cancer-fighting gene.

After a two-day round of chemotherapy was ordered to kill any remaining white blood cells in McCulley’s body, it was finally Day Zero. A total of 68 milliliters of the modified T cells (the rest was saved by the lab in case a second dose is needed) was slowly dripped into her port on Monday, a process that took only about half an hour. The small bag that was hooked up to the machine looked normal, but everyone in the hospital room seemed to know just how lifesaving those cells might turn out to be.

Joseph McGuirk, director of hematologic malignancies and cellular therapeutics at the hospital, said one re-engineered T cell can kill thousands of cancer cells. It’s a major reason why Yescarta, he said, is “remarkably and profoundly different” from traditional cancer treatment options for patients with McCulley’s form of lymphoma, who typically face poor survival rates.

McCulley is the first to receive Yescarta at the hospital, but there are “many more (patients) to come,” McGuirk said. Referrals and inquiries about the treatment have come in from patients and doctors from as far away as England, Qatar and Pakistan, he said.

Yescarta is a potential game-changer in the cancer world, but it isn’t without its drawbacks. The treatment costs $373,000 per patient, according to its maker. McCulley knows she is one of the lucky ones: Her insurance provider, Optum, will cover the procedure.

Just days before McCulley received her treatment, Bloomberg News reported that five people had received the treatment at one of the cancer hospitals authorized to administer it in the U.S. and that waiting lists had grown to at least 200 people, shrinking only as some very sick patients died. Doctors at those cancer centers told the news organization that they blame holdups in getting the treatment paid for by Medicare and Medicaid, as well as some of the U.S.’s largest insurers.

“The biggest issue has been insurance, particularly with Medicare and Medicaid,” Michael Bishop, director of the cellular therapy program at the University of Chicago Medicine, told Bloomberg. “There’s no billing codes for this. It’s been difficult, to be very blunt.”

Yescarta also isn’t a benign treatment. Three people died in clinical trials after getting the treatment, which can cause serious or possibly fatal side effects such as cytokine release syndrome, which leads to high fever and flu-like symptoms, and neurologic toxicities. Other side effects include inflections, low blood cell counts and a weakened immune system, according to the FDA, which is requiring the manufacturer to do a long-term safety study and train hospitals to quickly spot and handle those reactions.

By Friday morning, just four days after the modified T cells were reintroduced to her body, McCulley was “doing beautifully,” McGuirk said. She developed cytokine release syndrome about 24 hours after the procedure, and she had a fever nearly all week, but her spirits have been high and she hasn’t needed ICU care, he said.

McCulley was to remain hospitalized for at least seven or eight days because that’s when side effects would likely be strongest. After she is released from the hospital, she will stay at the American Cancer Society’s nearby Hope Lodge through Day 30 for near-daily check-ins with her nurses.

McCulley’s older sister, Georgia Burleson, is confident that she will pull through. The Denver, Colorado, resident is serving as her sister’s 24/7 caregiver until McCulley’s husband can arrive from Joplin, where he is committed this time of year to portraying Santa Claus at Northpark Mall.