A medical first in Philadelphia: new research released Sunday shows that a leukemia treatment that’s been successful with adults is also working for aggressive pediatric leukemia.
7-year-old Emily Whitehead is lucky to be alive.
“It was scary. Really scary,” said Emily.
Emily was diagnosed with the most common childhood cancer, acute lymphoblastic leukemia. Standard treatments didn’t work and her cancer came back.
“We knew that the chances go down below 30 percent if you relapse of you making it through,” said Tom Whitehead, Emily’s father.
Emily was the first to get an experimental new treatment at The Children’s Hospital of Philadelphia.
“We’ve treated the first couple of patients and we’ve been blown away by the results. They’ve been very exciting,” said Dr. Stephan Grupp, Director of the Center for Childhood Cancer Research at The Children’s Hospital of Philadelphia.
He is leading the new study for patients like Emily, collaborating with researchers at the Hospital of the University of Pennsylvania. They’re testing a genetically engineered T cell therapy, that’s also working for adults with chronic lymphocytic leukemia.
“We collect cells of the immune system from a patient, so we use the patient’s own cells. We put in a new gene in those cells that makes the cells go after cancer cells and then we put those cells back in the patient,” said Dr. Grupp.
The treatment isn't fun. A sign that it is working is the patient becomes extremely ill, as her immune system goes into overdrive. Oncologists call this "shake and bake."
Emily is one of 12 patients who received the new treatment. It wiped out her cancer within in a couple of weeks. Eight other patients had similar results. Some of them have been cancer free for more than two years, for Emily it’s been six months.
“We don’t know until we treat more patients and we have longer follow up, whether there’s a potential for curing these patients,” said Dr. Grupp.
A cure is what Emily and her family are hoping for. For now, they’re just happy she’ll be home for Christmas, the first time in two years.
“The doctors and scientists and staff here gave us Emily back, and that’s the best gift of all,” said Tom.
Researchers hope this new treatment reduces or replaces the need for bone marrow transplantation, which comes with risks and offers only a limited chance for cure.