Groundbreaking Leukemia Treatment ‘a Entire Fresh Approach’ to Fighting Cancer

FDA approves CAR T-cell therapy, a state-of-the-art immune-therapy trial to target childhood leukemia. Mott is poised to suggest the treatment — one of a select few sites in the country on track to do so.

Maryam Rasheed was just seven years old when the cancer she had battled a year earlier returned with a vengeance, and treatment could no longer stop it.

The heartbreaking news came after several rounds of chemotherapy, radiation and a bone marrow transplant from her 4-year-old brother, Rashid, in the battle against acute lymphoblastic leukemia.

Maryam’s doctors in Detroit referred the Macomb Township family to University of Michigan C.S. Mott Children’s Hospital for one last chance at finding a cure.

“I told them we will attempt anything,” her mother, Asmaa Rasheed, remembers.

At that time, Mott was among a handful of sites in the country participating in a clinical trial testing an innovative treatment to immunotherapy, or cancer treatments that corset a patient’s own immune cells to treat disease. Maryam enrolled in the examine to attempt the therapy. About one hundred days later, a bone marrow test brought stunning news: She was cancer-free.

The Food and Drug Administration on Aug. Thirty officially approved the therapy that helped Maryam: CAR T-cell therapy, the very first gene therapy for leukemia to reach the market. Mott is poised to suggest the treatment — one of a select few sites in the country on track to do so.

The FDA’s stamp of approval is largely based on a multisite explore, which included Mott, and tracked outcomes of fifty patients who received CAR T-cell therapy in 2015-2016. Forty-one (82 percent) of patients went into remission, with sixty percent of patients surviving leukemia-free for at least six months after therapy. The outcomes are striking because all patients had leukemia that had relapsed or was resistant to other forms of therapy before the CAR T infusion.

“This is a dawn of a fresh and arousing era in cancer therapy,” says Gregory Yanik, M.D., clinical director of the Pediatric Blood and Marrow Transplantation Program at Mott.

“This fresh treatment has the potential to switch the face of cancer therapy for years to come, not just in childhood acute lymphoblastic leukemia but in other cancers in which a patient’s own T-cells can be collected, genetically modified and redirected to kill a patient’s tumor. This permits us to turn patients’ own cells into a powerful weapon to fight the disease — a weapon that does not rely on chemotherapy but takes a entire fresh treatment to attacking childhood leukemia.”

What is CAR T-cell therapy?

CAR T-cell therapy involves extracting millions of a patient’s own T-cells, a pivotal, disease-fighting white blood cell. The T-cells are turbocharged through bioengineering technologies that reprogram them to kill cancer cells. The technology essentially converts the patient’s cells into what scientists call “a living drug.”

In the clinical trial, T-cells were programmed to attack a protein (called CD19) found on the surface of a common type of acute lymphoblastic leukemia. The T-cells are given to patients through an intravenous infusion that takes less than thirty minutes. One dose of the drug can ruin up to 100,000 cancer cells.

Acute lymphoblastic leukemia makes up about a fourth of cancer diagnoses among children under age fifteen and is the most common childhood cancer in the U.S. Children with leukemia often react to standard treatment, but in fifteen percent of cases — like Maryam’s — cancer cells stubbornly stand against treatment or patients relapse.

Doctors are quick to note that while some patients had good responses to CAR T-cell therapy, those effects have sometimes been short-lived, and the treatment can be life-threatening. Side effects can include high fevers, enlargened blood pressure, rapid pulse and decreased blood flow to internal organs that require stays in the Intensive Care Unit.

Novartis is the very first drug company poised to suggest the therapy, but it will primarily limit use to a petite number of medical centers trained and approved to administer the drug. Mott is among a puny group of sites in the country that participated in the licensed clinical trial that led to the FDA approval and is finalizing certification as a treatment center. Patients receiving the treatment will be tracked for fifteen years in a national registry.

“We are pleased that our center was able to play a major role in enrolling patients for the CAR T-cell clinical trial and that we have the expertise and system in place to suggest this potentially lifesaving treatment to our patients,” says C.S. Mott’s director of pediatric oncology, Rajen Mody, M.B.B.S.

“At this time, only a petite fraction of patients will be considered for the fresh treatment,” Mody adds. “This therapy provides fresh hope for patients with terminal leukemia in which no other treatment has worked.”

Helping other kids

Maryam, who is coming in fourth grade this fall at Shawnee Elementary School, has been in remission for more than two years. Every few weeks, she receives infusions of IVIG (intravenous immunoglobulin), which involve purified plasma pooled from thousands of blood donors and give her antibodies to fight off germs. Patients on the therapy rely on these infusions to stay healthy, because the T-cell treatment not only kills leukemic B-cells but also healthy ones that protect against infections.

After losing her hair three times, spending long periods wearing face masks, falling behind in school because of treatment and spending months at a time hospitalized, Maryam is ultimately experiencing regular kid life, her mom says. She likes the beach, family vacations and draping out with friends.

Her mother says it was an emotional hurricane to go from considering hospice to hearing doctors say all signs of cancer were gone.

“It was like a desire,” Rasheed says. “We know there are no ensures the cancer won’t come back, but this treatment is the only reason she’s here now.”

She says Maryam, who was part of a precursor probe leading to the thicker licensed Novartis trial, hopes her practice contributes to skill doctors and researchers have about leukemia treatment.

“This treatment saved my daughter’s life and helped our family so much,” Rasheed says. “Maryam is old enough to understand that what she has gone through can help other kids. It makes her glad to know she can help doctors and other families who are going through this.”

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