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More than a year after starting a pandemic that incomparably ravaged communities of color, the predominantly African-American patients of sickle cell disease see a ray of hope with the release of another new treatment.
A new gene-editing therapy called CRISPR has been shown to eliminate all symptoms in at least one study among a small list of FDA-approved treatments for the rare and chronic disease.
The one-time treatment works by altering the DNA in the blood cells and reprogramming the body to stop producing the sickle-shaped cells that obstruct oxygen flow and cause extreme pain or severe fatigue to young and old patients. The body instead goes back to making the type of healthy blood cells produced in the womb.
It works by removing stem cells from the patient’s body and using CRISPR technology to alter the gene before putting them back in the body.
For Velvet Brown-Watts, more options mean more hope for the community of families affected by sickle cell disease and other related anemias. But she also acknowledged that the science behind the new curative treatment would be a tough sell for some black folks.
“I don’t ever tell people what to try or what not to try. We tell them to make sure you’re getting all the information,” Brown-Watts said. She’s the Executive Director of Supporters of Families with Sickle Cell Disease, an Oklahoma support group based in Tulsa. She also has a son who lives with the disease.
“Would the person still have the DNA of their family down the line? If my son did that, would he still be carrying the same bloodline,” Brown-Watts said, listing questions community members have asked her about the new CRISPR treatment.
Brown-Watts said it’s exciting whenever there’s a new treatment but that some in the black community, mostly faith leaders, may see the gene-editing therapy as playing God.
“I’m excited about all the new different therapies coming down the pipeline because now it does give patients living with sickle cell options,” Brown-Watts said.
Twenty years ago, it was a different story. Throughout the 80’s and 90’s the only FDA-approved treatment for sickle cell disease, which affects roughly 100,000 Americans and 1 in every 365 African American births, was a repurposed cancer drug called Hydroxyurea.
“When you look at diabetes and cancer, they had how many options,” Brown-Watts said. “And at the time, sickle cell only had one. And it wasn’t even a drug for sickle cell.”
Brown Watts said she feels that since sickle cell primarily affects African Americans, the FDA, historically, hasn’t spent much time investing in research or treatments toward it.
That has changed in recent years as more treatments have been approved and continue to be studied.
There’s now Envari, a powder, and Adakzeo, an infusion drug.
There’s also Oxbryta, a pill that Brown-Watts said has been working for her 16-year-old son, Jeremiah Jr.
“It’s been a rollercoaster,” Brown-Watts said. “For our family, the diagnosis changed the core of who we became. You learn to create a synergy for your family so you can build with the tools you have. For us, it was helping him learn how it impacted his body and what it is, what it does, and how he has to learn how to manage.”
Thanks to Oxbryta, Brown-Watts has noticed her son has more energy and better oxygen flow, though he still requires oxygen support.
At a time when the Center for Disease Control estimates that 7 percent of black sickle patients with Covid-19 have died compared to less than 1 percent of the general population, emotional and financial support means everything.
The recent study of the gene-editing therapy, published in the New England Journal of Medicine, revealed that months after the treatment, patients who had sickle cell disease remain pain-free and without a need for blood transfusions.
And as more treatments become available, Brown-Watts hopes the community will stand up to address these racial health disparities.
“It’s almost like when you’ve been living on an island all alone, and all you had was coconuts coconuts coconuts, and somebody else comes along, and maybe they bring an orange. And you get really excited because now you have options,” Brown-Watts said.