Hope for high-risk cancer patients where there was none before
SALT LAKE CITY — The first gene therapy approved in the U.S. is now healing patients in Utah. Scientists genetically modify a patient’s own immune cells to target and kill the cancer. It’s revolutionizing medicine.
All Zachary Johansen, 12, wanted when he was fighting a rare form of leukemia was a Dobermann. The two are inseparable now. And you’d never know how sick Zachary was just a couple of years ago.
“They said, ‘He probably will live four or five more months, if you want to live out your days, you can. Or do you want to fight?’ And he said he wanted to fight,” Monica Jansen, Zachary’s mother.
He entered clinical trials for CAR-T cell therapy, a type of gene therapy his parents knew little about. But Zachary was out of options.
“So many months of being in the hospital and watching your child suffer and not being able to take the pain away. And as a parent, you would do anything, right? Do anything to make it better,” Jansen said.
CAR-T cell therapy is an acronym for a complex scientific name, but let’s make it simple.
Scientists take a patient’s blood and genetically engineer their own immune cells, called T-cells, to make new cells, called a chimeric antigen receptor, or CAR. The new CAR-T cells then attack a protein on the cancer that the T-cells alone normally wouldn’t.
“They’ve inserted a gene to make it make a specific key for the lock that you have on the leukemia cells,” said Dr. Andy Harris of Primary Children’s Hospital.
CAR-T cell treatment works even in those patients who are resistant to chemotherapy, while bone marrow transplants require a patient be put into remission first; patients like Jesus Adame, 8, who just received his infusion.
“It was a tablespoon’s worth of cells only,” Harris said.
That small amount is a river of hope for his mother, Maria Villagomez. Her son’s Down syndrome makes him prone to leukemia. Jesus relapsed last September after fighting it for the first time four years ago. Villagomez is hopeful her son will make a full recovery.
“There are literally billions of these cells that are no targeted to the patient’s own cancer cells,” said Dr. Michael Boyer.
The CAR-T cells are selective in their toxicity and will only attack the cancer. CAR-T cell therapy typically requires a shorter recovery than treatments like bone marrow transplants, which carry a greater risk of second cancers, and long-term side effects like infertility.
It was a lifesaver for Irving Suchite, 26,of Logan, one of the first patients after the study at Primary Children’s Hospital. He’s still more susceptible to infection, but only a month after his infusion, there are no signs of cancer.
Diagnosed last May, Suchite said it was a shocker. He couldn’t tolerate radiation because doctors feared it would destroy his liver.
“I’m feeling really great. I’m feeling more energetic, like I can do anything,” Suchite said.
CAR-T cell therapy came along just in time. “He’s doing so good, I think we can start working toward a future,” said Ann Suchite, Irving’s wife.
The future is bright for Zachary, too.
“And it’s really hard to get mad at him. It’s really hard. Cause I’m like, ‘You need to clean your room,’ and he looks at me and I’m like, ‘Never mind,'” Johansen said.
Zachary and his mom are proud to be pioneers in a therapy many consider the future of treatment for children with high-risk cancers. Giving hope where there was none before.
“Every day we give prayers of gratitude to be at this place,” Johansen said.
In clinical trials, patients 5 years later did not have a recurrence of leukemia.
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