Louisiana Man Becomes First In Region to Be Functionally Cured of Sickle Cell Disease

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Louisiana Man Becomes First In Region to Be Functionally Cured of Sickle Cell Disease

The short version

  • Image Credit: Marti Sans/Stocksy Daniel Cressy of Louisiana has become the first person in the Gulf South to be functionally cured of sickle cell disease.
  • The milestone highlights the growing promise of gene-editing therapies for the blood disorder, which disproportionately affects Black people.
  • Cost, insurance approval, and limited access remain barriers for many eligible patients.
  • A Louisiana man has become the first person in the state to be declared functionally cured of sickle cell disease after receiving the gene-editing therapy Casgevy…
  • For Daniel Cressy, 23, the milestone marks the end of a more than two-year treatment journey.

The story

A Louisiana man has become the first in the Gulf South to be functionally cured of sickle cell disease using gene therapy. Image Credit: Marti Sans/Stocksy
  • Daniel Cressy of Louisiana has become the first person in the Gulf South to be functionally cured of sickle cell disease.
  • The milestone highlights the growing promise of gene-editing therapies for the blood disorder, which disproportionately affects Black people.
  • Cost, insurance approval, and limited access remain barriers for many eligible patients.

A Louisiana man has become the first person in the state to be declared functionally cured of sickle cell disease after receiving the gene-editing therapy Casgevy, Manning Family Children’s Hospital announced on June 22.

For Daniel Cressy, 23, the milestone marks the end of a more than two-year treatment journey. It also highlights the growing promise of CRISPR-based gene-editing treatments for people living with the blood disorder.

Sickle cell disease (sickle cell anemia) affects more than 100,000 people in the United States, with Louisiana having the highest per capita rate of any state.

The inherited disorder causes red blood cells to become rigid and crescent-shaped, restricting blood flow and leading to painful vaso-occlusive crises, organ damage, stroke, infection, and other serious complications.

It also disproportionately affects Black Americans.

Casgevy is among the first CRISPR-based gene-editing therapies approved by the Food and Drug Administration (FDA) to treat sickle cell disease by targeting the underlying cause of the condition rather than simply managing its symptoms.

Daniel Cressy’s journey to a functional cure

Diagnosed with sickle cell disease as an infant, Cressy spent much of his childhood in and out of hospitals because of painful sickle cell crises. At times, he was hospitalized between six and 12 times each year.

Although his symptoms became more manageable during high school, the disease continued to shape his future.

Cressy had long dreamed of becoming a commercial airline pilot, but he later learned that the Federal Aviation Administration would not medically certify him due to his diagnosis.

Hoping there might be another path forward, he appealed the decision.

“The answer came back,” Cressy said in the statement released by Manning Family Children’s Hospital. “If I could cure my sickle cell disease either through a bone marrow transplant or through gene therapy, then I could become a pilot.”

When Casgevy became available, Cressy decided to pursue treatment.

The process ultimately took more than two years and included collecting his stem cells, chemotherapy to prepare his bone marrow, an infusion of the gene-edited cells, and months of recovery and follow-up care.

On June 22, Cressy rang the ceremonial bell at Manning Family Children’s Hospital to celebrate the end of his treatment journey and being declared functionally cured.

What it means to be functionally cured

Doctors use the term “functionally cured” because treatments like Casgevy are designed to prevent sickle cell disease from causing the painful complications that have historically defined patients’ lives.

Casgevy works by collecting a patient’s blood-forming stem cells and using CRISPR gene-editing technology to modify them so they produce higher levels of fetal hemoglobin.

After patients undergo chemotherapy, the edited cells are returned to the body, where they can produce healthier red blood cells that are less likely to sickle and block blood flow.

Although patients continue to receive long-term medical follow-up, many no longer experience the repeated vaso-occlusive crises that frequently require emergency care and hospitalization.

In clinical trials supporting the therapy’s approval, most participants remained free of severe vaso-occlusive crises for at least 12 consecutive months after treatment.

Why access to Casgevy remains limited

Getting access to the treatment can also be challenging, and proved to be an obstacle that Cressy needed to overcome.

“I wasn’t denied gene therapy … I just had to go through extra hoops because it was new and expensive,” Cressy told People. “Trying to navigate approval took a lot out of me, but my friends who are flying jets and fighting sickle cell all kept me going. The team at Manning Family is amazing too.”

Patients must undergo chemotherapy, receive care at specialized medical centers, and spend months recovering after treatment. Cost, insurance approval, and strict eligibility requirements continue to limit access for many people who could potentially benefit from the therapy.

Cressy hopes his experience encourages others living with sickle cell disease to learn about emerging treatment options and advocate for the care they need.

He has also founded the Privileged Pilots Project, a nonprofit dedicated to helping aspiring pilots facing health and life challenges, raising awareness about sickle cell disease, and expanding access to opportunities.

“While many spend their lives searching for purpose, mine found me,” Cressy said in a statement released by Manning Family Children’s Hospital. “Now, instead of looking for meaning, I can spend my life fulfilling it.”

For Cressy, that purpose now includes pursuing the medical clearance he hopes will finally allow him to realize the dream that sickle cell disease once put out of reach.

Read the full story at Healthline NewsOriginal

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