After surgery, radiation and three different therapies, Scott Goedeke faced a harsh reality: The cancer that first appeared on the roof of his mouth had spread to a lymph node in his neck. So the 58-year-old health consultant agreed to an experimental treatment that involved using his own cells to destroy them.
“I have to do this,” he remembered thinking at the time.
His medical team at Siteman Cancer Center in St. Louis Sampled and identified a tumor the cells that could attack that disease and propagated it in a laboratory. Billions of cells were then reintroduced into his body in the hopes of recharging him his body's natural defenses and overwhelms the cancer.
Six weeks later the tumor had shrunk significantly.
Now this unique cancer therapy has reached an important milestone. On Friday, the U.S. Food and Drug Administration approved its use to treat adults with skin cancer such as Goedeke melanoma, a melanoma that has spread or cannot be removed with surgery after other approaches failed. Although the disease is rare, cancer experts say the FDA's approval could mark the beginning of a powerful new weapon against much more common tumors.
Shares of Iovance Biotherapeutics, the California-based company that makes the therapy, rose more than 30 percent on Tuesday, the first full day of trading after FDA approval.
“The concept that the The FDA has now recognized that you can use a patient's own cells as a living drug to treat their disease, and that to me is a very exciting step forward,” said Steve Rosenberg, a senior researcher at the National Cancer Institute who pioneered the work The therapy that has been newly approved since the 1980s has achieved this. “It is the beginning of a new era in the development of a new approach to cancer treatment,” he said, describing ongoing research into more common deadly tumors such as breast, pancreatic and colon cancer.
Scientists have been working for decades to develop cancer treatments that use the body's own immune system to detect and destroy malignant tumors. Breakthroughs have been made in this area in recent years, including drugs that help the body recognize and attack cancer cells. Another tool, approved by the FDA in 2017, extracts the body's cancer-killing cells and programs them to attack a specific type of blood-based tumor.
The new individualized therapy, called tumor-infiltrating lymphocytes, harnesses the body's relatively small number of T cells that see a tumor as a threat and produces a laboratory-grown army of them. Of the 73 patients treated in a clinical trial, 31.5 percent had their tumors reduced or disappeared after cell treatment, the FDA said.
The FDA approval “represents the culmination of scientific and clinical research efforts leading to novel T-cell immunotherapy for patients with limited treatment options,” Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, said in a statement on Friday.
Iovance Biotherapeutics has priced the Amtagvi-branded therapy at $515,000 per patient. The FDA approval is “the first step toward realizing Iovance's ambition to usher in the next generation of cell therapy by bringing this breakthrough to patients with advanced solid tumors,” Frederick Vogt, Iovance's interim chief executive, said in a statement Friday.
The FDA's action is also a signal to the pharmaceutical industry that there is a commercial path to success, said Jason Bock, chief executive of CTMC, a company spun out of the MD Anderson Cancer Center that contracts with biotech companies to help with the disease To help produce cell therapies. He said approval for “one of the most complex therapeutics we have ever tried to develop” “took 40 years.”
Goedeke, the cancer patient, underwent chemotherapy to prepare his body for cell therapy. He remembers the day his cells were infused back into his body — Jan. 25, 2023 — at Siteman Cancer Center. They came in a special container. The procedure lasted about half an hour and was followed by further infusions to help the body get the new cells going.
After being discharged from the hospital, Goedeke met with his oncologist to discuss the results of his most recent cancer scan. These visits were always stressful and Goedeke's wife accompanied him.
George Ansstas, a Washington University oncologist at Siteman, broke the news six weeks after therapy: Goedeke had “responded” to the experimental therapy.
“I don’t think I can emphasize enough the relief and elation,” Goedeke said. “We couldn’t get to the car fast enough to deliver the news.”
Of the FDA's approval, he said, “I'm very happy that other patients have access to it.”