Cancer Cure?

When Emily Whitehead was 5 years old, bruises began to appear on her legs. Her gums bled when she brushed her teeth. Then she woke up one night in excruciating pain. Her parents rushed her to the emergency room. Doctors diagnosed Emily with a type of childhood blood cancer—a disease caused by abnormal cell growth. When two years of treatment with traditional medications failed to stop her cancer, Emily became the first kid to receive a groundbreaking new therapy. It would end up saving her life. “I was so happy because I knew I’d finally get to go home with my family,” says Emily, now 12.

Emily suffered from a type of cancer called acute lymphoblastic leukemia (ALL). “Cancer in kids is rare, but ALL is the most common,” says Dr. Stephan Grupp, a pediatric oncologist—a doctor who cares for children with cancer—who treated Emily at the Children’s Hospital of Philadelphia in Pennsylvania.

When Emily Whitehead was 5 years old, bruises appeared on her legs. Her gums bled when she brushed her teeth. Then she woke up one night in horrible pain. Her parents rushed her to the emergency room. Doctors found that Emily had a type of childhood blood cancer—a disease caused by abnormal cell growth. Emily went through two years of treatment with the usual medications, but that didn’t stop her cancer. So Emily became the first kid to receive a groundbreaking new therapy. It would save her life. “I was so happy because I knew I’d finally get to go home with my family,” says Emily, now 12.

Emily had a type of cancer called acute lymphoblastic leukemia (ALL). “Cancer in kids is rare, but ALL is the most common,” says Dr. Stephan Grupp. He’s a pediatric oncologist—a doctor who cares for children with cancer. He treated Emily at the Children’s Hospital of Philadelphia in Pennsylvania.

About 3,000 kids are diagnosed with ALL each year. Roughly 85 percent of them recover from the disease after undergoing chemotherapy. In Emily’s case, however, these medications didn’t kill the cancerous cells in her body. Her last hope was a clinical trial, or experimental study. It would test a treatment called CAR-T that uses a patient’s own cells to kill off cancer. For Emily, the treatment was a success. Five years later, she remains cancer-free. “I got to go back to the hospital to hang up my five years cancer-free photo,” says Emily.

Last year, the U.S. Food and Drug Administration approved the therapy that helped Emily. It can now be used to treat other kids with ALL. The agency also approved a similar therapy to combat a blood cancer in adults called non-Hodgkin’s lymphoma. Both therapies could help thousands of people suffering from previously untreatable forms of cancer.

About 3,000 kids learn they have ALL each year. About 85 percent of them get better after going through chemotherapy. But these medications didn’t kill the cancerous cells in Emily’s body. Her last hope was a clinical trial, or experimental study. It would test a treatment called CAR-T. This treatment uses a patient’s own cells to kill off cancer. For Emily, the treatment was a success. Five years later, she’s still cancer-free. “I got to go back to the hospital to hang up my five years cancer-free photo,” says Emily.

Last year, the U.S. Food and Drug Administration approved the therapy that helped Emily. Now it can be used to treat other kids with ALL. The agency also approved a similar therapy for adults. It fights a blood cancer called non-Hodgkin’s lymphoma. Both therapies could help thousands of people with forms of cancer that couldn’t be treated before.

Cancer can strike any part of the body. Emily’s leukemia originated within her bone marrow. This soft tissue within bones makes blood cells (see Blood Breakdown). “In leukemia patients, parts of their blood grow in ways that they shouldn’t,” explains Grupp.

In people with ALL, their bone marrow makes underdeveloped B-cells. This type of white blood cell normally helps the immune system fight infections. But Emily’s cancer cells multiplied out of control, crowding out normal B-cells. Without enough healthy immune cells, Emily became extremely tired and experienced fevers, infections, and bleeding.

When an initial round of chemotherapy failed to cure Emily’s cancer, she began a second, stronger round. Patients with ALL who need additional chemotherapy have only a 30 percent chance of being cured. Just four months after her second round of chemotherapy, Emily’s cancer returned. “We knew we had to try something different or Emily would likely die,” says Grupp.

Cancer can strike any part of the body. Emily’s leukemia started in her bone marrow. This soft tissue inside bones makes blood cells (see Blood Breakdown). “In leukemia patients, parts of their blood grow in ways that they shouldn’t,” explains Grupp.

Bone marrow makes B-cells, a type of white blood cell. B-cells normally help the immune system fight infections. But in people with ALL, B-cells don’t develop properly. Emily’s cancer cells multiplied out of control, crowding out normal B-cells. Emily didn’t have enough healthy immune cells, so she became extremely tired. She had fevers, infections, and bleeding.

The first round of chemotherapy didn’t cure Emily’s cancer. So she began a second, stronger round. When patients with ALL need more chemotherapy, they have only a 30 percent chance of being cured. Emily finished her second round of chemotherapy. Four months later, her cancer returned. “We knew we had to try something different or Emily would likely die,” says Grupp.

The only option was to enter Emily in a clinical trial, led by Grupp. It would test an experimental treatment on kids with ALL whose cancer didn’t respond to chemotherapy. Enrolling in a clinical trial—particularly one in an early phase—can be risky, because scientists and doctors haven’t yet determined if the treatment is safe or effective. For Emily and her parents, there was no other choice.

The clinical trial was studying the safety of chimeric antigen receptor T-cell therapy, or CAR-T for short. The treatment would use Emily’s own immune cells—another type of cell called T-cells—to kill the cancerous B-cells (see How CAR-T Works). T-cells naturally attack invaders in the body. But in ALL, they don’t recognize the harmful B-cells.

The only option was to enter Emily in a clinical trial. Grupp led this study. It would test an experimental treatment on kids like Emily. They had ALL, but their cancer wasn’t helped by chemotherapy. It can be risky to be in a clinical trial, especially one in an early stage. That’s because scientists and doctors don’t know yet if the treatment works or is safe. But Emily and her parents had no other choice.

The clinical trial studied the safety of chimeric antigen receptor T-cell therapy, or CAR-T for short. The treatment would kill the cancerous B-cells. It would use Emily’s own immune cells to do this. The treatment used another type of cell called T-cells (see How CAR-T Works). T-cells normally attack invaders in the body. But in ALL, they don’t spot the harmful B-cells.

During the trial, doctors collected T-cells from Emily’s blood. All cells contain DNA—a molecule that carries instructions for how a cell functions. In the lab, scientists inserted a new gene—a unit of hereditary material—into the DNA of the T-cells they’d gathered. The gene told the cells to produce a protein—a large, biological molecule—to recognize cancerous B-cells.

The reprogrammed T-cells were then injected back into Emily’s bloodstream, where doctors hoped they would seek out and destroy the malfunctioning B-cells. “The therapy teaches a person’s own cells how to do something new that they couldn’t do before,” explains Grupp.

Doctors took T-cells from Emily’s blood during the trial. All cells hold DNA. This molecule carries instructions for how a cell works. In the lab, scientists used a new gene—a unit of hereditary material. They placed it in the DNA of the T-cells they’d gathered. The gene told the cells to produce a protein—a large, biological molecule. This protein would spot cancerous B-cells.

Then the reprogrammed T-cells were injected back into Emily’s bloodstream. Doctors hoped they would find and destroy the cancerous B-cells. “The therapy teaches a person’s own cells how to do something new that they couldn’t do before,” explains Grupp.