Gene Therapy Ushers In a New Era in Cancer Treatment

Oct 23, 2017

CAR t-cells attack a cancer cell in this image from researchers at the National Institutes of Health. The CAR t-cell therapy for Acute Lymphoblastic Leukemia was the first to receive FDA approval.
Credit NIH

A new treatment for a type of childhood cancer is the first FDA-approved use of gene-therapy.  It provides an alternative for patients who have exhausted all other options.

In this week’s Exploradio, WKSU’s Jeff St.Clair sits down with two cancer physicians who are hoping to bring the therapy to Akron.

It’s called CAR T-cell therapy.  The CAR means Chimeric Antigen ReceptorsT-cells are cells in our bodies that fight infections.

The therapy is a ‘living drug’ that uses the body’s own immune system to kill cancer cells.

It made headlines in 2012 when the then-experimental therapy was first used on 7-year-old Emily Whitehead.

Emily Whitehead was the first patient to receive CAR t-cell therapy for ALL. After overcoming serious side-effects, she has continued to thrive and is the namesake of the Emily Whitehead Foundation.
Credit EMILY WHITEHEAD FOUNDATION

She had been fighting a form of childhood leukemia called Acute Lymphoblastic Leukemia or ALL for two years and nothing was working.

Emily became the first patient to receive CAR T-cell therapy, which uses a disabled form of the HIV virus to train killer T-cells to attack the leukemia cells.

Dr. Stephen Hunger is head of oncology and director of research at Children’s Hospital of Philadelphia where the treatment was developed. He was invited to Akron recently to talk to doctors about the breakthrough.

A Scary Start to CAR T-cell Therapy
Hunger says the therapy, at first, came close to killing Emily and sent doctors in the ICU into high alert. The team worked around the clock,  he says, "to measure everything they could think of measuring.”

They quickly identified a drug that would stop the potentially fatal side-effect known as cytokine release syndrome, “and as soon as she got the drug, she got better," says Hunger.

Hunger says it was a scary start to a therapy that otherwise has worked remarkably well for Emily Whitehead, who five years later remains cancer free.

“If you had told me 5 ½ years ago, when this first girl was treated, how good it could be, I would have said that you were nuts," says Hunger. 

"I don’t think anybody involved had any realistic assessment that it could be this effective.”

The drug company Novartis purchased the technology from the Children’s Hospital of Philadelphia and sells it under the name Kymriah.

Considering the Cost
Hunger says the therapy begins when a hospital isolates the T-cells from the patient’s blood and then the cells are shipped to the production facility "to be manipulated and manufactured, and when they’re done they’re sent back to the treating facility to infuse the patient.”

It’s not cheap.

Novartis operates a production facility in New Jersey that manufactures the CAR t-cells.
Credit NOVARTIS

Without insurance, each treatment runs around $475,000.

But, with the cost of a bone marrow transplant running up to a $1 million in some cases, Hunger says the new gene-therapy treatment provides another option.

“And part of the question going forward is: When should you apply this in the course of a patient’s disease ... the chance to improve outcome and what is the most cost-effective approach?”

Dr. Jeffrey Hord is head of the cancer and blood disorders unit at Akron Children’s Hospital.

He says it will take time before the therapy comes to Akron.

“We don’t know yet what the requirements will be of institutions to use the drug.  I’m sure it will be a limited number of institutions initially.”

Dr. Stephen Hunger, left, is chief of oncology and director of the Center for Childhood Cancer Research at Children's Hospital of Philadelphia. Dr. Jeffrey Hord, right, is director of hematology-oncology at Akron Children's Hospital.
Credit JEFF ST.CLAIR / WKSU

He says the drug maker still needs to determine which hospitals can handle the treatment.

“One of the things no doubt that will be considered is can you handle the complications of those infusions,” says Hord.

Akron Children’s sees about 25 cases of the type of childhood leukemia the therapy was designed for.  According to Hord, around five of them might be candidates for it when standard treatments don’t work.

He says for now he’ll still send those patients to places like Children’s Hospital of Philadelphia that offer CAR T-cell therapy.

Gene Therapy Comes of Age
It’s the first in a wave of gene-therapy innovations in the pipeline for FDA approval. Just months after the childhood leukemia treatment became the first, the same technology has now been approved to treat lymphoma in adults. 

It’s one of 75 new uses of the killer CAR T-cells pending approval, according to the FDA, with hundreds of other gene-therapy procedures waiting in the wings.

“Still not every patient will be cured,” says Hunger, something he knows all too well. But he says the decades of basic research in labs around the world are beginning to pay off for families that have run out of options.