Innovative gene therapy gives hope for sickle cell cure

Eighteen-year-old Razel Colon of Hoboken says he feels like he got a second chance at life.

“After the treatment they let me go home. And I came in about two weeks later and Dr. Nancy told me that I was four days cured. I was very, very happy. It was very emotional,” he said.

Razel is the first patient in New Jersey to receive an innovative sickle cell disease treatment being studied at the Joseph M. Sanzari Children’s Hospital in Hackensack.

Kyelia Colon, Razel’s mom, says the teen has been experiencing the pain since he was just two months old.

“When he was a baby he can’t say ‘I’m in pain, my side hurts, my leg hurts.’ It was mostly ear infections and fevers. And as he got older it started moving throughout the body,” said Kyelia.

“When you’re going through a crisis it gets so bad you start to shake. The next thing you know you’re calling your mom and telling her you’re at the hospital. I missed a lot of school events. I missed a lot of family events because of sickle cell,” Razel said.

Dr. Stacey Rifkin-Zenenberg is the principal investigator of the study that hand-picked for Razel due to his extreme and severe case of sickle cell.

“Sickle cell disease is an inherited form of anemia and it actually causes the cells to form a sickle cell shape. It actually impairs the carriage of oxygen throughout the body. It causes impairment the way that you fight infection, it causes painful crisis, it also causes end organ damage,” she said.

Rifkin-Zenenberg says the trial involved gene therapy which has the patient be their own donor.

“The way this works is we take the patient’s stem cells and we harvest them. And then we take their own gene and we alter their gene by using a type of LentiGlobin virus and we change the gene and alter it to make non-sickle hemoglobin — in essence curing them of their sickle cell,” she said.

The children’s hospital is the only pediatric site in New Jersey where the study is taking place.

“When the company put this trial together they came to the centers that had been doing bone marrow transplants and sickle cell. The on-site lab really makes this possible. You have to be able to thaw the cells and get them quickly,” said Dr. Alfred Gillio, the director of the Children’s Cancer Institute at the hospital.

“If this is what it feels like to hit the lottery, to win the mega-millions or Big Bucks, this is what it is,” said Kyelia.

Now that he’s cured, Razel says he wants to go on a vacation.

“A long plane ride out of the U.S. because I couldn’t do that before,” he said.

Rifkin-Zenenberg says she hopes the gene therapy will be approved by the FDA. In the meantime, they plan to enroll more patients in the trial between the ages of 12 and 50 who have severe sickle cell disease.