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Chicago hospitals gearing up to offer breakthrough treatment for pediatric cancer

By Lisa Schencker – Contact Reporter | Chicago Tribune | Aug 30, 2017 | See Original Article Here

[COMMENT: So nice to see a new treatment being prepped for pediatric use so quickly. Hopefully this is a sign of things to come for the Rhabdo community – Alan]

University of Chicago Medical Center and Lurie Children’s Hospital could be among the first hospitals in the country to offer a new, breakthrough pediatric cancer treatment approved Wednesday by the U.S. Food and Drug Administration.

The treatment, which uses a patient’s own modified cells to battle a form of acute lymphoblastic leukemia, is the first gene therapy approved for use in the U.S. Acute lymphoblastic leukemia is the most common childhood cancer.

Its $475,000 price tag is drawing jeers from some advocacy groups, but many are also excited about the treatment’s potential to save lives.

Here’s how the CAR-T cell therapy works: Immune cells called T-cells are collected from a patient’s blood and genetically engineered to fight the leukemia. Those supercharged T-cells are then put back into the patient’s body, where they kill the cancer cells. The therapy came out of a collaboration between Switzerland-based Novartis and the University of Pennsylvania.

The treatment is intended to help the roughly 15 to 20 percent of children and young adults with the disease who don’t respond to initial treatment or whose cancer returns after initial treatment, according to the FDA.

Until now, children with those tougher cases of the disease had low survival rates, said Dr. John Cunningham, chairman of the department of pediatrics at University of Chicago Medicine Comer Children’s Hospital. In a clinical trial, more than 80 percent of patients who underwent CAR-T cell therapy went into remission, according to the FDA.

“In my 30 years of being an oncologist, that’s the most dramatic difference I’ve ever seen,” Cunningham said. “I’ve never seen something so spectacular.”

University of Chicago Medicine has been participating in a clinical trial of the treatment, which will be marketed under the brand name Kymriah.

More than 3,000 people under age 20 are diagnosed with acute lymphoblastic leukemia each year, according to the National Cancer Institute, and the new treatment is designed for the most common form of it.

For now, only a select group of hospitals across the country will be allowed to offer Kymriah commercially because of its potentially severe, life-threatening side effects, including high fever, flu-like symptoms, infections and low blood pressure. The FDA is requiring hospitals and clinics that offer the therapy to be specially certified and have another newly approved drug on hand to treat some of those side effects.

Representatives from University of Chicago Medicine and Lurie Children’s Hospital confirmed Wednesday they are working with Novartis to be part of a network of hospitals offering the therapy commercially.

Cunningham said University of Chicago Medicine could potentially begin offering the treatment to patients within weeks. Lurie could also begin offering it in coming weeks, said Dr. Reggie Duerst, clinical director of Lurie’s stem cell transplant program.

Novartis spokeswoman Dana Cooper said in an email Wednesday she could not provide the names of hospitals that will offer the therapy commercially in Chicago. Novartis is aiming to have at least 20 medical centers certified to offer the treatment within a month, she said. The company hopes to have 32 centers certified by the end of the year.

Novartis said Wednesday it will charge $475,000 for the therapy, and it’s working with insurers to make sure they provide coverage for patients. She said the price is below current standards of care, such as the cost of some stem cell transplants.

Novartis believes the $475,000 price will “support sustainability of the health care system and patient access while allowing a return for Novartis on our investment,” she said.

But the Campaign for Sustainable Rx Pricing criticized the price tag. “While science behind Kymriah is revolutionary, the business decision to price it at nearly half of a million dollars per treatment is not,” it said in a statement. “Kymriah’s price tag is simply a continuation of the pattern of sky-high launch prices that spins further out of control each year.”

The group did, however, say it’s excited about the therapy’s potential to save lives.

Some patients, such as Sam Tinaglia, of Park Ridge, have already seen the difference it can make.

Tinaglia was diagnosed with the disease when he was 5 years old, but he relapsed after initial treatment. He endured about eight years of chemotherapy and a bone marrow transplant before Cunningham, his doctor, helped him take part in a clinical trial of the treatment in Philadelphia in 2015.

Like many patients, he suffered severe side effects from the therapy. He had high fevers, memory loss and a seizure, said his mother, Suzie Tinaglia. But within about a week in the clinical trial, those symptoms cleared. He’s been in remission ever since.

Sam, now 18, graduated from Maine South High School this year, and his parents dropped him off at the University of Illinois, Urbana-Champaign, for his freshman year about two weeks ago.

“We never thought he would even be able to go to college,” Suzie Tinaglia said. “You just go back to your life and your body heals itself. It’s really amazing.”

Suzie Tinaglia said Novartis covered the costs of her son’s treatment because he was part of a clinical trial.

Several other companies have also been conducting clinical trials for CAR-T cell therapies.

Twitter @lschencker

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