A 7-year-old American girl who was so sick in April that her friends and family gathered to say their final goodbyes is now laughing, running, jumping and living proof that an experimental new treatment may offer the greatest hope yet for cancer treatment, according to results being presented by her doctors this week.
“This is a whole new way of treating cancer, and the idea is that we’re redirecting the cells of the immune system against the cancer,” said Dr. Stephan Grupp, who is leading the study at Children’s Hospital of Philadelphia, in an interview with CBS News.
The girl, Emma Whitehead, is one of a dozen patients with advanced leukemia to get the experimental treatment developed at the University of Pennsylvania.
Four of them including Emma are in complete remission. Five others have shown improvement.
Emma was diagnosed with acute lymphoblastic leukemia when she was five years old.
By last April she had relapsed twice, according to the New York Times, and her doctors had few remaining options other than the experimental treatment which uses a disabled form of the AIDS virus to kill cancer cells.
It had only been tried a few times, and never on a child.
Doctors take the T cells that fight infections from the patient’s body, use the AIDS virus to alter them so they can attack the cancer cells, and inject them back into the patient.
A similar treatment has been done before, with mostly unsuccessful results.
“Unfortunately, cancer is really good at dodging T cells,” Grupp said to the Centre Daily Times.
This time, working with Grupp, researchers from the Perelman School of Medicine at the University of Pennsylvania decided to grow the T cells outside the patient’s body until there were enough of them to be effective.
“So instead of trying to stimulate or trigger a rare cell in somebody’s body by a vaccine, you give them the end product of that, which is a billion cells that are engineered to recognize what you want them to recognize,” said Michael Kalos, one of the researchers and director of the Translational and Correlative Studies Laboratory at the Perelman School of Medicine.
While the cells do battle, already sick patients become even sicker. In Emma’s case, it almost killed her.
Within days, her fever had spiked to 105 and her body was swollen almost beyond recognition. Within the week, she was put on a ventilator, unable to breathe on her own.
“They took us out in the hall and said… she is as sick as she can get. You should call your family in. There’s a good chance she won’t be here in the morning,” her father told CBS.
Doctors noticed a spike in her blood levels they knew could be treated with a rheumatoid arthritis drug.
Within hours, Emma began to recover. Less than six weeks later she was well enough to go home, and now, eight months later, she is a happy, active second grade student who remains in remission with no sign of cancer.
Researchers are presenting their findings at the American Society of Hematology meeting in the US state of Georgia this week, where some colleagues say it’s the breakthrough they’ve all been waiting for.
“Most scientists, researchers, clinicians spend the entirety of their careers trying to develop something and not succeeding… but it’s the dream of all of us that do this. To actually be part of something that succeeded at the level that this succeeded is extraordinary,” said Kalos.
The next steps are larger trials and then eventually applying for FDA approval.
Researchers say the treatment could be used widely to treat leukemia and other blood cancers, and may eventually be used against other kinds of cancers.