Sickle Cell Disease Pediatric Gene Therapy Program Offers Children Hope for a Cure

As America recognizes World Sickle Cell Disease (SCD) Day on June 19, a gene therapy treatment program soon to be offered through a partnership between Downey Medical Center and City of Hope will provide a cure and hope for many Kaiser Permanente pediatric patients from across Southern California, enabling them to live a more normal life without pain and other health implications.

Dr. David Simon, director of the pediatric regional Sickle Cell Center at Kaiser Permanente Downey Medical Center, said Kaiser Permanente Southern California currently treats about 220 children aged 12 and above with SCD. Many of them are expected to benefit from the gene therapy program, with the first child slated to undergo the procedure this summer.

The Federal Food and Drug Administration-approved gene therapy process starts with extracting stem cells from the child’s blood, then modifying them in a laboratory before reinfusing them into the child’s blood stream for a cure, according to Dr. Simon.

“Before this procedure, the only potential cure of Sickle Cell Disease involved a bone marrow transplant that can have many complications, as well as a lack of suitable donors,” Dr. Simon said.” It may also not even be an option for many patients who need curative therapy.”

Sickle cell disease is an inherited blood disorder where red blood cells are abnormally shaped, leading to reduced oxygen delivery and various health complications. These abnormal red blood cells, shaped like a sickle, become rigid and sticky, blocking small blood vessels and causing pain and damage to organs.

Consider the following facts related to SCD:

• Total affected in the U.S.: Approximately 100,000 people live with SCD, according to the Centers for Disease Control and Prevention.
• Racial distribution: More than 90% of individuals with SCD in the U.S. are non-Hispanic Black or African American.
• Birth incidence: About 1 in every 365 Black or African American babies is born with SCD.
• Carrier rate: Roughly 1 in 13 Black or African American babies is born with sickle cell trait, meaning they carry one copy of the sickle cell gene.

Offering children a cure from SCD is a great medical achievement, considering the following:

• Life expectancy: Individuals with SCD have a life expectancy more than 20 years shorter than the national average.
• Complications: SCD can lead to severe health issues including:
  • Chronic pain
  • Stroke
  • Acute chest syndrome
  • Organ damage (kidney, liver, heart)
  • Increased risk of infections

“As you can imagine, there’s a huge demand for this gene therapy treatment,” Dr. Simon said. “It’s very exciting. The only other curative option has been a bone marrow transplant, and there are a lot of barriers to that. You need a donor and there’s a lack of bone marrow donors. Gene therapy takes that problem away from the patient. It’s very exciting because it opens the door to the possibility of being cured to a lot of patients who otherwise wouldn’t have other options.”

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