Hope for Shayla
On a sunny summer morning, 6-year-old Shayla arrives at CHOP for her monthly chemotherapy treatment with her favorite doll, Jiquan, in her arms.
Shayla proudly shows off the contents of her colorful striped backpack, which she's packed with a few things the two will need today: some potato chips and a Barbie doll for her, a soft pink blanket and a plastic sippy cup for Jiquan.
"We're just so grateful we have this," says Shayla's mom, Jennifer, glancing up at CHOP's Main Building as her daughter plays in the grass nearby.
It's a beautiful moment for a mother and daughter who, just a few months ago, thought they were about to lose everything.
Shayla has been fighting neuroblastoma, one of the most devastating childhood cancers, for more than three years. At a hospital in her home state of North Carolina, she endured harsh chemotherapy treatments, radiation and a bone marrow transplant. She suffered from severe nausea and diarrhea and received blood transfusions at least twice a week. "She was in the hospital all the time," says her mom.
After those initial treatments, Shayla's cancer was in remission for almost a year. But in November 2010, a routine CT scan showed that the cancer had returned. "It was a shock," Jennifer says. "It was devastating."
And when Shayla's parents brought her to CHOP to explore new treatment options, it seemed like time was running out for the usually energetic kindergartner. "She had a hard time walking," her mom says. "She was in pain."
Still, Yael Mosse, M.D., co-director of CHOP's Neuroblastoma Developmental Therapeutics program, had hope for Shayla. And the next day, on the 640-mile drive home, Jennifer says, "I finally smiled a little bit."
Shayla began an experimental treatment at CHOP in December. Six weeks later, her cancer was nearly gone.
Shayla is one of the many patients who have benefited from CHOP's world-renowned neuroblastoma program, which focuses on translating lab discoveries into new treatments and new hope for patients.
"In the lab, we try to identify the molecular abnormalities that will allow us to be much smarter about how we find drugs to target those abnormalities," says Mosse.
The team has celebrated many significant successes in just the past few years. In 2008, they discovered the gene that can cause neuroblastoma to occur — a finding that has already led to a clinical trial of a targeted therapy. And in the last few years, the team has further identified most of the genetic factors causing the disease. In 2010, they were part of a large research group that found that a type of immunotherapy improved two-year survival rates in patients with high-risk neuroblastoma by 20 percent the first significant increase in neuroblastoma cure rates in more than a decade. They also recently identified several proteins that are promising treatment targets for high-risk neuroblastoma, and have made major inroads toward translating these findings into new drug treatments.
"We have developed a philosophy that relapsed neuroblastoma can be a chronic disease," says John Maris, M.D., chief of the Division of Oncology and director of the Center for Childhood Cancer Research at CHOP. "Even though there is not a known cure for relapsed neuroblastoma at this point, there are ways to treat it that can get the disease under control and give the child an outstanding quality of life."
The neuroblastoma team has already made an incredible difference in Shayla's life. She, her mom and her older sister Savannah recently moved to New Jersey, in large part to be closer to CHOP. And although Shayla's condition is still very serious, the frequent blood transfusions, debilitating side effects and long hospital stays are a thing of the past.
"She's like a regular 6-year-old," Jennifer says. "She's able to go out and play and go to concerts and go to school. She's a rambunctious little girl, full of energy. And every day that I have her in my life, I have CHOP to thank."