FDA reviews gene-editing cure for sickle cell disease

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In a significant step toward a new cure for sickle cell patients, the Federal Drug Administration (FDA) is reviewing a gene-editing treatment that has proven successful in attacking the disease at its roots.

Sickle cell disease is a painful, oxygen-depriving and life-threatening illness that primarily affects African Americans in the U.S. For years the only known cure has been a bone marrow transplant.

However, unlike a transplant, gene editing treatment works by altering the DNA in the blood cells. It reprograms the body to stop producing the sickle-shaped cells that obstruct oxygen flow and cause extreme pain or severe fatigue.

Velvet Brown-Watts is the executive director of Supporters of Families with Sickle Cell Disease, an Oklahoma-based organization.

“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 previously told The Black Wall Street Times.

The treatment “exa-cel” is based on CRISPR technology, which won a Nobel prize in 2020.

If the FDA approves the cure after its all-day meeting Tuesday, a new opportunity for relief will come for the approximately 100,000 sickle cell patients in the U.S.

The agency’s meeting is being live-streamed for public access:

FDA weighs approval for sickle cell cure

The advisory committee undergoing the review releases recommendations for the FDA to either approve or ignore. While the recommendations aren’t legally binding, the FDA usually follows them.

In sickle cell patients, a genetic mutation causes affects the red blood cells carrying oxygen throughout the body. The disease causes the cells to turn crescent-shaped, blocking the flow of oxygen and causing severe pain.

Ultimately, the FDA is expected to decide on whether to approve the treatment for sickle cell in early December, PBS News reported.

Nationally, nearly 1 in 365 African American births result in a baby with sickle cell disease. For decades, the medical community and federal government failed to adequately address the issue.

“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.”

In recent years, however, new treatment options have emerged.

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Treatments and cures

For the Guillory family in Georgia, a risky bone marrow transplant was the only option. When she learned of her son’s sickle cell diagnosis, there was no FDA review in the works.

“I literally felt someone had handed me a death sentence for a child,” Shea Guillory told The Black Wall Street Times after she discovered one of her twin boys was diagnosed with the most severe form of the disease.

With help from the Aflac Cancer and Blood Disorders Center at Children’s Healthcare of Atlanta, Guillory learned two-year-old Saxton was a perfect match for his twin brother Sawyer.

“I literally dropped to my knees, and I cried out. I thanked her, and I thanked God. Because I had a weight on me,” Guillory told The Black Wall St. Times.

Meanwhile, Victoria Gray of Forest, Mississippi, proved the success of the gene-editing technology on an international level. She’s shown no symptoms of the excruciatingly painful disease for nearly four years.

In March, Gray shared her experience at the Third International Summit on Human Genome Editing in London. It’s unclear whether her success story influenced the FDA review of the potential sickle cell cure.

“God did his part for what I prayed about for years,” Gray told NPR reporters in London. “And together, hand in hand, God and science worked for me.”

However, not everyone shares the enthusiasm for a technology that alters DNA in the human body.

Criticism

For Velvet Brown-Watts, leader of the Oklahoma support group for patients with sickle cell, the issue hits close to home. She left her job in the medical field to become a full-time advocate after her own son was diagnosed.

The ethics of gene-editing technology is an issue she recognizes may not be for everyone, even if the FDA approves it for sickle cell treatment.

“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.

The treatment by Vertex Pharmaceuticals and CRISPR Therapeutics permanently alters the patient’s DNA.

According to Vertex, 29 of 30 people were free of pain crises for at least a year after their study. All 30 avoided hospitalization during that time.

Ultimately, after years of few options, the FDA review for a potential sickle cell cure builds hope for a community long ignored.