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FDA approves treatment that saved Philipsburg girl

by on September 07, 2017 9:54 AM

PHILIPSBURG — An experimental cell replacement procedure that saved the life of a now 12-year-old Philipsburg girl will now save thousands of young lives across the world.

On Aug. 30, the Food and Drug Administration approved a chimeric antigen receptor T-cell therapy for treatment of certain pediatric and young adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia.

This therapy was first used on 5-year-old Emily Whitehead, and since she was deemed five years cancer free just a few months ago, Emily, and her parents Tom and Kari, have been on a mission to make the procedure that saved her young life available to everyone facing childhood cancer. In July, the family, as well as a group of doctors and industry experts, presented testimony about the procedure to a panel seeking to push the method into the FDA hands for approval. That panel of 10 unanimously saw the advantages of the procedure and recommended it for approval.

The FDA, with the Whiteheads present, made it official last week.

“It was very emotional for us to finally have the CTL019 T-cell therapy FDA approved,” Tom Whitehead told Centre County Gazette. “It made us even more proud and amazed at how much Emily surviving her treatment is changing the world.”

The treatment, to be tradenamed Kymriah and carrying the generic name tisagenlecleucel, is sold by Novartis. The company said it will charge $475,000 for the one-time treatment.

Emily is a three-time cancer survivor alive today because of T-cell therapy. She was the first child patient in the world to receive the treatment in 2012, after relapsing following other treatments. Her initial diagnosis with acute lymphoblastic leukemia was made in 2010, at the age of 5.

Typically, children diagnosed with this type of leukemia have an 85 to 90 percent chance of being cured; however, Emily relapsed in October 2011. A bone marrow transplant was scheduled for February 2012, but Emily relapsed again just weeks before the transplant date. Her leukemia was so aggressive that doctors felt they had run out of options and recommended that she go home on hospice care.

But the Whiteheads were not ready to give up. They caught wind of a revolutionary new treatment that had just become available at Children’s Hospital of Philadelphia and enrolled Emily in a Phase I clinical trial to have her immune system trained to fight cancer. The process involved collecting her T-cells (a type of white blood cell), then genetically reprogramming them through the use of, interestingly, the HIV virus, to recognize and attack cancer cells. These modified cells were then infused back into Emily’s blood.

The treatment caused Emily to become very sick, and she spent the next two weeks in an intensive care unit, that first night beating the odds of survival of 1 in 1,000, according to the doctor in charge. After her treatment, Emily slipped into a coma, which was reversed by yet another experimental procedure — the introduction of an arthritis medicine called tocilizumab. Slowly, she recovered, and a few weeks later, the family, her doctors and, most importantly, Emily, realized she was cancer-free.

This spring, Emily celebrated her 12th birthday. She also celebrated being cancer-free for five years, a monumental date for cancer survivors.

Since Emily’s treatment five years ago, technology has advanced the procedure, which has now treated 200 pediatric patients in the United States. Doctors said the procedure has around a 90 percent success rate with an initial remission, and 55 to 60 percent of patients who underwent the treatment are now past one year of being cancer-free.

“Emily is feeling great and enjoying seventh grade,” Tom said. “Since the announcement, we have been getting messages from other parents from everywhere in the world looking for information on how to get their children enrolled.

“We have also been getting more requests than ever to inspire other by sharing or story,” he said.

In addition, the Whiteheads are preparing for the Believe Ball, scheduled for Oct. 28 at the Valley Forge Casino. There, several patients who received the treatment after Emily will meet for the first time. Money raised at this event will be used to help continue saving young lives.

The family is also busy with the production of a feature documentary film with academy award-winning director Ross Kauffman, who will visit the Whiteheads in Philipsburg in November for continued filming.

For more information, visit www.emilywhiteheadfoundation.org.

(Gazette correspondent John Dixon contributed to this article.)

 

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