Sickle Cell: One Last Chance?
HACKENSACK, N.J. (Ivanhoe Newswire)— Imagine having an inherited blood condition that causes extreme fatigue and sudden pain during everyday activities. Sickle cell disease causes red blood cells to form the shape of a sickle, making it difficult for blood to flow. Now, doctors are testing an experimental therapy that could be lifesaving for patients running out of treatment options.
Nineteen-year-old Razel Colon lived his entire childhood knowing the slightest wrong move could cause him terrible pain—or put him in danger.
“It was scary when I used to play outside with my friends and a football would hit me in the chest, knowing that I had sickle cell that could transfer over to a heart attack or stroke,” stated Razel.
“It’s really hard for a parent because there’s nothing you can do to help your child,” shared Kyelia Colon,
Razel was diagnosed with sickle cell disease at three months. As he grew older, pain crises meant living in the hospital for days and weeks at a time. Doctors suggested Razel undergo a bone marrow transplant, but no one in his family was a match. That’s when Stacey Rifkin-Zenenberg, DO, pediatric hematologist, Joseph Sanzari Children’s Hospital suggested a new, experimental gene therapy.
“So gene therapy was engineered, so that the patient could be their own donor,” explained Dr. Rifkin.
During the gene therapy procedure, doctors remove a patient’s bone marrow with chemotherapy. The patient’s harvested stem cells are sent to a lab, where a gene is added.
“Then we give them back their own stem cells with the added gene,” continued Dr. Rifkin.
The body begins to produce healthy red blood cells. The process takes about a month.
For Razel, the gene therapy all but wiped out the disease. He is still a carrier of sickle cell, but no longer has pain crises.
“It’s amazing because he gets to live a normal life, which he didn’t live before,” said Kyelia.
“Don’t give up and there’s hope,” exclaimed Razel.
The developers of the gene therapy, Bluebird Bio temporarily halted clinical trials earlier this year after a patient treated in 2016 developed blood cancer. Scientists have since determined the cancer was not linked to the sickle cell treatment and have asked the FDA for permission to resume the trial. The researchers are hoping to apply to the FDA for approval next year.
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