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Dr. Gregory Yanik, clinical director of the Pediatric Blood and Marrow Transplantation Program at C.S. Mott Children's Hospital in Ann Arbor, works with Maryam Rasheed of Macomb Township. Maryam was part of a clinical trial using gene therapy to successfully treat her leukemia.(Photo: Sophie Masson/Michigan Medicine)

The U.S. Food and Drug Administrationapproved on Wednesdaythe first-ever gene therapytotreat children and young adults withleukemia.

Called Kymriah, but better known as CAR T-cell treatment, the therapy is being hailed by doctors as revolutionary. Itinvolves genetically modifyinga patient's own T-cells, which thencantarget and kill a form of acute lymphoblastic leukemiacells.

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 patients own T-cells can be collected, genetically modified and redirected to kill a patients tumor," said Dr.Gregory Yanik, clinical director of the Pediatric Blood and Marrow Transplantation Program at the University of Michigan's C.S. Mott Children's Hospital. Mottwas one of a few hospitals nationally to take part inclinical trials of the treatment.

"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, Yanik said.

The CAR T-cell treatmentoffers new hope for children like Maryam Rasheed, 10, of Macomb Township.

Maryam was diagnosed with B-cell acute lymphoblastic leukemia at age 4, when her family was seeking refuge from religious persecution in Turkey, said Maryam's mother, Asmaa Rasheed.

Maryam Rasheed (right) with her brother, Rashid, and sister Samantha. Maryam, 10 of Macomb Township, survived acute lymphoblastic leukemia.(Photo: Rasheed family photo)

"My country is Iraq," Asmaa Rasheedsaid. "It wasnt safe. We are Christian. It was so hard over there in Baghdad. We run away to Turkey.

"We take her to hospital the first timebecause ... she stopped eating, stopped walking, stopped talking. We bring her to emergency. The doctor decided to take her bone marrow to do tests. Then the results came back, and she have leukemia."

Maryam underwent her firstchemotherapy treatment in Turkey.

"Over there, it was so hard," Rasheed said. "The doctors dont speak English over there. We know English a little bit. We speak Arabic."

Maryam Rasheed of Macomb Township undergoes treatment for acute lymphoblastic leukemia. She is now in remission.(Photo: Rasheed family photo)

Rasheed stayed with her daughter for two months in the Turkish hospital. A few months later,the Rasheed family was able to immigrate to the U.S. and settled in Michigan.

But Maryam's cancer returned. She was treated at Children's Hospital of Michigan with more chemotherapy and radiation. In 2013,her younger brother, Rashid, proved to be a match for a bone marrow transplant.

Still, the cancer wouldn't relent.

The Rasheed family learned of a clinical trial for CAR T-cell therapy under way atMott. It was the family's last chance,Rasheed said.

Maryam Rasheed, 10, of Macomb Township holds up her arms joyfully. She's surrounded by her sister Samantha (left), brother, Rashid, and baby sister Annabell.(Photo: Rasheed family photo)

"There was nothing to do," her mother said."In Detroit, there was chemo, radiation, bone marrow transplant. It returned back three times. She lose her hair three times. It was so hard for her and my family."

She remembers the date Maryam started the clinical trial at Mott: Dec. 17, 2014. Maryam spent Christmas and her seventh birthday in the hospital.

"I think we waited like 100 days,I dont remember exactly, and they did a bone marrow test, and the medicine, it work!" Rasheed said.

"It was like a dream, you know, like light coming from far away when youre in the dark. Theres nothing else we could do. But the CART-cell was like a shining light from far away."

Maryam has been in remission two years, andis starting fourth grade next week at Shawnee Elementary School in Macomb Township.

"Now, shes start her life, and doing everything a little kid is doing," said Rasheed, who says she hopes the treatment helps other children, too.

So does Yanik.

"Acute lymphoblastic leukemia is the most common form of cancer in children, accounting for approximately25% of all childhood cancers," Yanik said. "This particular therapy utilizes a childs own immune system to target their leukemia."

Theclinical trials focused on the 15% to 20% ofchildren whoseB-cell acute lymphoblastic leukemia had either relapsed or who had residual leukemia cells in their bone marrow after treatment.

"Historically, such patients would have an estimated cure rate of approximately 10%," Yanik said. "The two trials were groundbreaking. In the most recent trial, 52 of 63 patients with childhood leukemia successfully entered complete remission with this therapy."

Novartis Pharmaceuticals Corp. got the FDA approval for the gene cell therapy, whichinvolves drawing blood from childrenwith B-cell acute lymphoblastic leukemia. The T-cellsin the child's blood are thenshipped to a lab where they are genetically engineered so theywillseek outa particular protein in the leukemia cells and attack. Patients are then infused with the modified blood, and the T-cells go to work to find and kill the leukemia.

The New York Times reported Wednesday that the therapy will cost $475,000 for the initial treatment, with additional treatments administered at no cost.

Although 83% of the children in the clinical trials for CAR T-cell therapy went into remission, Yaniksaid it's too early to tell howcurative treatmentswill prove in the long run. And, its use will be limited to only a few medical centers in the U.S.

"The University of Michigan is the only site in the state and within this region that is licensed to administer these cells for childhood leukemia," he said.

Offering the treatment at a large medical center like U-Mis essential, said Dr. Rajen Mody,a pediatric oncologist at Mott, because of the severity ofpotential side effects.

"It can cause serious side effects, especially within the first 21 days," said Mody, who is Mott's director of pediatric oncology. "Patients can have high fevers, bleeding complications, trouble breathing, infections. ... Thats why a hospital like the University of Michigan is the ideal place. ... Patients who undergo this treatment are usually so sick after an infusion of the CAR-T cells, that they can't be safely treated at smaller hospitals."

Dr. Rajen Mody, a pediatric oncologist at the University of Michigan's C.S. Mott Children's Hospital.(Photo: University of Michigan)

Yanik is hopeful that successful treatment with CAR T-cell therapy in children with leukemia will open the door for similar therapies targeting other cancers.

"Aseparate CAR T-cell trial targeting diffuse large-cell lymphoma was recently completed with the results in that clinical trial now under review at the FDA," he said. That trial alsoincluded adult patientsat the University of Michigan.

Mody called the gene therapy revolutionary.

"This is clearly a life-saving and potentially curative therapy," he said."Its being tested in other types of leukemia and solid tumors. Its too early to say whether its going to work as well for other cancers.... We are not there yet."

Still, he said, it's made all the difference for Maryam and her family.

"She was one of the lucky ones coming from Iraq, and with all the things she has survived. And then coming here and surviving this,... she clearly has some goodluck.

"I think she should do very well. Patients who actually survive the first six months and still have CAR T-cells detected in their systems tend todo very, very well."

Contact Kristen Jordan Shamus: 313-222-5997 or kshamus@freepress.com. Follow her on Twitter @kristenshamus.

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First gene therapy to treat cancer gets FDA approval; UM only Michigan hospital to use it - Detroit Free Press

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