August 31, 2017 8:30 AM

Groundbreaking Leukemia Treatment ‘a Whole New Approach’ to Fighting Cancer

FDA approves CAR T-cell therapy, a state-of-the-art immune-therapy trial to target childhood leukemia. Mott is the only certified treatment center in Michigan and one of a select few in the country to offer it.

Maryam Rasheed was just 7 years old when the cancer she had battled a year earlier returned with a vengeance, and treatment could no longer stop it.

MORE FROM THE LAB: Subscribe to our weekly newsletter

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 try 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 approach to immunotherapy, or cancer treatments that harness a patient’s own immune cells to treat disease. Maryam enrolled in the study to try the therapy. About 100 days later, a bone marrow test brought stunning news: She was cancer-free.

The Food and Drug Administration on Aug. 30 officially approved the therapy that helped Maryam: CAR T-cell therapy, the first gene therapy for leukemia to reach the market. Mott is the only certified treatment center in Michigan and one of a select few in the country to offer it.

The FDA’s stamp of approval is largely based on a multisite study, which included Mott, and tracked outcomes of 50 patients who received CAR T-cell therapy in 2015-2016. Forty-one (82 percent) of patients went into remission, with 60 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 new and exciting era in cancer therapy,” says Gregory Yanik, M.D., clinical director of the Pediatric Blood and Marrow Transplantation Program at Mott.

“This new treatment has the potential to change 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 allows 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 whole new approach 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 techniques that reprogram them to kill cancer cells. The technique essentially transforms the patient’s cells into what scientists call “a living drug.”

SEE ALSO: Balancing Treatment and Toxicity for AML Patients

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 30 minutes. One dose of the drug can destroy up to 100,000 cancer cells.

Acute lymphoblastic leukemia makes up about a fourth of cancer diagnoses among children under age 15 and is the most common childhood cancer in the U.S. Children with leukemia often respond to standard treatment, but in 15 percent of cases — like Maryam’s — cancer cells stubbornly resist 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, increased blood pressure, rapid pulse and decreased blood flow to internal organs that require stays in the Intensive Care Unit.

Novartis is the first drug company poised to offer the therapy, but it will initially limit use to a small number of medical centers trained and approved to administer the drug. Mott is among 13 sites in the country that participated in the licensed clinical trial that led to the FDA approval. Patients receiving the treatment will be tracked for 15 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 offer 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 small fraction of patients will be considered for the new treatment,” Mody adds. “This therapy provides new hope for patients with terminal leukemia in which no other treatment has worked.”

"This is a dawn of a new and exciting era in cancer therapy."
Gregory Yanik, M.D.

Helping other kids

Maryam, who is entering 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.

SEE ALSO: Doctors Unleash New Weapon to Fight Pediatric Neuroblastoma

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 finally experiencing regular kid life, her mom says. She enjoys the beach, family vacations and hanging out with friends.

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

“It was like a dream,” Rasheed says. “We know there are no guarantees 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 study leading to the bigger licensed Novartis trial, hopes her experience contributes to knowledge 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 happy to know she can help doctors and other families who are going through this.”