CRISPR Study in Sickle-Cell Disease

First US patient treated with CRISPR for genetic disorder in ongoing clinical trial

This week NPR Shots reported on the progress of a US sickle-cell patient enrolled in a clinical study to look at CRISPR’s safety and efficacy in treating this disease. She’s the first US patient being treated with CRISPR for a genetic disorder.

Victoria Gray has been away from home for several months while receiving infusions of genetically modified bone marrow cells. Sickle-cell disease is an intensely painful genetic disease that turns normal red blood cells (usually round and pliable) into hard, sticky, sickle-shaped cells that don’t carry oxygen well, clog the bloodstream, damage organs, greatly increase stroke risk, and cause torturous bouts of pain. Until now the only treatment has been a bone-marrow transplant but due to high risk it was seldom performed in adults.

“The pain is excruciating. It’s like being in a car accident and having lightning in your chest. It’s a pain that makes a grown woman like me scream,” Gray says. “It’s an overwhelming pain.”

Like many sickle cell patients, Victoria had to drop out of school, quit work and spend weeks in the hospital away from her family. Since many sickle cell patients don’t survive past their 40s, Gray worries whether she’ll live to see her children grow up. She just turned 34.

Ms Gray’s treatment started in July, when doctors infused billions of her own bone marrow cells back into her body after editing them with CRISPR. The hope is that a gene in the cells will now make them produce fetal hemoglobin, a protein that babies usually stop making shortly after birth. The protein is intended to give sickle cell patients like Ms Gray healthy red blood cells.

CRISPR Therapeutics, one of the labs involved in Ms Gray’s treatment has already shown CRISPR’s efficacy in editing cells in beta thalassemia patients. Beta thalassemia is another genetic disorder affecting red blood cells: insufficient hemoglobin is produced, leading to anemia which can be life-threatening. The most serious form starts in children as young as two.

Ms Gray will be monitored with blood tests and a bone marrow biopsy once a month and keep a diary to symptoms and pain. The hope is that the treatment will reduce the severity of her disease’s symptoms. Because this treatment is so new, doctors aren’t able to estimate how soon she’ll see improvement. Her hope is just to be able to live a more normal, healthy life.

“Not just for me but for other people. This would be mind-blowing,” Gray says. “I can’t imagine the lives that could be saved if this thing actually works. Yeah, oh my God. Just to not have to deal with that pain anymore is enough.”

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