Health Check: A cure for Sickle Cell Disease?

Gene therapy might provide a cure for children living with sickle cell disease.

Gene therapy might provide a cure for children living with sickle cell disease.

“Right now, I know kids with sickle cell disease who are eventually going to get worse,” said Dr. Anjulika Chawla, who is the medical director of the sickle cell program at Hasbro Children’s Hospital.

Babies who are born with the inherited disease, Chawla said, have abnormally shaped cells that become stiff and rip up blood vessels, causing excruciating pain during flare-ups. Antibiotics can help, as do medications and/or monthly blood transfusions, depending on the severity.

But these are only band-aid solutions.

That's why Chawla is so excited about a recent case study published in the New England Journal of Medicine.

"The first patient with sickle cell disease that was done with gene therapy was announced about a year ago," she said.

It involved a 13-year-old boy from France who had the gene therapy two and a half years ago, said Chawla. That teenager is reported to be disease-free. This, Chawla said, is huge.

"Huge breakthrough," she said.

Chawla explained how the therapy works using animation produced by Bluebird Bio, the company that came up with this gene therapy. It involves using a virus.

"So, what this virus does is it's created in the lab and the virus has been given the normal gene and it goes and it infects the cell that has the bad gene and it could do one of two things,” said Chawla. “It can add to the gene so it dilutes out the bad gene or it can replace the bad gene."

Chawla said clinical trials in this country are in the works.

Meanwhile, Rhode Island Hospital will be hosting what it calls a “CELL-abration,” during the weekend. It is open to anyone interested in sickle cell and other blood disorder.

There is also a sickle cell symposium scheduled for Oct. 5 in Worcester.

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