A new technique has achieved a long-sought goal — giving a patient’s own immune system the lasting ability to fight cancer. While the leader of the research team does not use the “cure” word, the new treatment provides hope for patients with leukemia and, down the road, perhaps other forms of cancer.
Emma Whitehead has been bounding around the house lately, practicing somersaults and rugby-style tumbles that make her parents wince.
It is hard to believe, but last spring Emma, then 6, was near death from leukemia. She had relapsed twice after chemotherapy, and doctors had run out of options.
Desperate to save her, her parents sought an experimental treatment at the Children’s Hospital of Philadelphia, one that had never before been tried in a child or in anyone with the type of leukemia Emma had. The experiment, in April, used a disabled form of the virus that causes AIDS to reprogram Emma’s immune system genetically to kill cancer cells.
The treatment nearly killed her. But she emerged from it cancer-free and seven months later is still in complete remission.
She is one of nine leukemia patients who are cancer-free after being treated with genetically-altered versions of their own immune cells, giving strength to a promising new approach for treating the blood cancer.
The trial includes 12 patients in total and bolsters findings from 2011. Then, scientists from the University of Pennsylvania in Philadelphia reported that two of the first three patients treated showed no traces of the malignancy after getting the therapy. Today’s results were presented at the American Society of Hematology’s annual meeting in Atlanta.
For Walter Keller, 59, who had failed every other treatment for his chronic lymphocytic leukemia diagnosed in 1996, the regimen meant he’s been in remission since his treatment in April. Before the therapy, “I thought I had a year to live,” he said.
“I feel better than I have in a long, long time,” said Keller, of Upland, Calif., in a telephone interview. “I’m excited because I think this will help a lot of people.”
The scientists, led by Carl June, a professor of pathology and laboratory medicine at the Abramson Cancer Center at the University of Pennsylvania and a study author, used genetic engineering to manipulate white blood cells extracted from the patients. The researchers reprogrammed the cells to specifically target the leukemia cells and reinjected them into the patients.
Similar approaches are being tried at other centers, including the National Cancer Institute and Memorial Sloan-Kettering Cancer Center in New York.
“Our goal is to have a cure, but we can’t say that word,” said June, who leads the research team at the University of Pennsylvania. He hopes the new treatment will eventually replace bone-marrow transplantation, an even more arduous, risky and expensive procedure that is now the last hope when other treatments fail in leukemia and related diseases.
CLL is a slow-growing cancer that starts from white blood cells in the bone marrow and interferes with the production of healthy blood cells. The condition leads to complications such as immune deficiencies and swollen lymph nodes. The disease strikes about 16,000 adults each year and 4,600 die from it, according to the American Cancer Society.
The disease is treated with chemotherapy. Another approach is bone marrow transplant in which chemotherapy is first given to kill diseased cells then replaced with healthy ones from bone marrow extracted from the patient or a donor.
Keller underwent chemotherapy and then a stem cell transplant in 1998 and 1999. He was in remission for 5 and a half years. He then underwent a different chemotherapy regimen and got a second stem cell transplant. The cancer came back again in 2010, and he had no options left. He was one of 10 adult patients treated so far.
“No patient who had complete remission has lost it,” said Keller’s doctor, David Porter, director of blood and marrow transplantation at the University of Pennsylvania’s Abramson Cancer Center. “It’s unusual to have a treatment that can work to this magnitude and until now, this duration.”
Three adults with chronic leukemia treated at the University of Pennsylvania have also had complete remissions, with no signs of disease; two of them have been well for more than two years, said Dr. David Porter. Four adults improved but did not have full remissions, and one was treated too recently to evaluate. A child improved and then relapsed. In two adults, the treatment did not work at all. The Pennsylvania researchers were presenting their results Sunday and Monday in Atlanta at a meeting of the American Society of Hematology.
Despite the mixed results, cancer experts not involved with the research say it has tremendous promise, because even in this early phase of testing it has worked in seemingly hopeless cases.
“I think this is a major breakthrough,” said Dr. Ivan Borrello, a cancer expert and associate professor of medicine at the Johns Hopkins University School of Medicine.
Dr. John Wagner, director of pediatric blood and marrow transplantation at the University of Minnesota, called the Pennsylvania results “phenomenal” and said they were “what we’ve all been working and hoping for but not seeing to this extent.”
A major drug company, Novartis, is betting on the Pennsylvania team and has committed $20 million to building a research center on the university’s campus to bring the treatment to market.
Herve Hoppenot, president of Novartis Oncology, called the research “fantastic” and said it had the potential — if the early results hold up — to revolutionize the treatment of leukemia and related blood cancers. Researchers say the same approach, reprogramming the patient’s immune system, may also be used eventually against tumors like breast and prostate cancer.