Imagine pitting one fatal disease against another and staging the warfare inside the body of a 6-year-old girl who was 48 hours away from total organ failure.
Now imagine you’re the parent of that child.
For one Pennsylvania family this was the reality they were facing when their doctors had no other option but to treat their daughter Emily, who was suffering from acute lymphoblastic leukemia (ALL), with genetically altered HIV.
A research team at the University of Pennsylvania and Children’s Hospital of Philadelphia, led by Dr. Carl June, had genetically engineered T cells and used them to help several adult patients in a clinical trial with chronic lymphocytic leukemia (CLL) and ALL. The T cells needed a partner to bond with to fight the cancerous B cells that plagued Emily.
Enter HIV — the virus that causes AIDS. The idea was to use the genetically engineered HIV cells to bond with the patient’s cultured T cells to seek out the cancerous B cells and destroy them.
“HIV is a scary thing,” acknowledged Dr. Stephan Grupp, director of Translational Research at the Center for Childhood Cancer Research at The Children’s Hospital of Philadelphia and professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania. Grupp, and his team, treated Emily with the new procedure in April 2012, funded, in part, by a $16 million research grant from the Leukemia & Lymphoma Society.
Grupp explains the process: “We’re taking the property of HIV that is valuable and isolating that property. We use the virus to basically genetically engineer T cells from kids that are good at fighting viruses and potentially good at fighting cancer. The trouble is they don’t seek cancer. We try to force the T cells to seek cancer so we put the gene in the cell. Turns out the HIV-type virus is very good at inserting into genes and cells. That’s the function we want.
“The type we use has been genetically engineered and all the characteristics of the HIV that can cause disease are out of it,” Grupp said. “It can’t grow. It can’t make copies of itself. The crippled form of HIV only retains the function of putting a gene into a cell.”
Emily Whitehead, a plucky, bright girl — call her Emma, she asks — was no longer a candidate for chemotherapy or a bone marrow transplant, a standard treatment for her type of leukemia.
In early May of 2010, at age 5, she had had a clean bill of health from her pediatrician, aside from a few swollen glands everyone chalked up to a recent cold. But as the month progressed there were signs something was amiss: blood crusting at the bottom of her nose that couldn’t be explained, blood from her gums while brushing her teeth, bruising — Kari, her mother, called her husband at work when she counted 21 bruises on Emma’s body while she was in the bathtub. She developed debilitating pain in her knees, too. A family member recognized the signs.
“My sister-in-law, a nurse, called me, crying, and said, ‘You realize, Em’s symptoms can be leukemia,’ ” her father Tom Whitehead recalled. “I said, ‘You need to calm down. We’ll take her to the doctor in the morning.’ ”
Emma woke at 1 a.m. crying in pain. Her knees hurt like never before.