NEWS – Kendric Cromer, a 12-year-old with sickle cell disease from Washington, began the world’s first commercially approved gene therapy for the condition

HIGHLIGHTS

• Gene Therapy Authorization: The FDA authorized two companies to sell gene therapy for sickle cell disease, offering a promising alternative for those affected by the genetic disorder.
• Bluebird Bio’s Treatment: Kendric is the first commercial patient for Bluebird Bio, a company based in Somerville, Mass., offering gene therapy for sickle cell disease.
• Treatment Process:
  • Kendric’s treatment involves the removal of bone marrow stem cells, which will be genetically modified by Bluebird Bio in a specialized lab.
  • The entire treatment process will span several months, ensuring the sufficient collection of stem cells for genetic modification.

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SICKLE CELL DISEASE (SCD)

• Definition and Characteristics:
  • SCD is an inherited blood disorder characterized by flawed hemoglobin.
  • Hemoglobin is responsible for carrying oxygen in red blood cells (RBCs).
  • People with SCD have atypical hemoglobin molecules, known as hemoglobin S, leading to distorted, sickle-shaped RBCs.
• Impact on Blood Flow:
  • Normally, RBCs are disc-shaped and flexible, allowing easy movement through blood vessels.
  • In SCD, genetic mutations cause RBCs to become sickle-shaped, hindering their ability to bend and move easily.
  • These sickled RBCs can block blood flow, affecting oxygen delivery to tissues.
• Causes:
  • SCD is caused by a defective gene called the sickle cell gene.
  • A person develops SCD only if they inherit two sickle cell genes, one from each parent.
• Symptoms:
  • Early-stage symptoms include anemia-related fatigue, swollen hands/feet, and jaundice.
  • Later-stage symptoms include severe pain, anemia, organ damage, and increased susceptibility to infections.
• Treatment Options:
  • A bone marrow transplant (stem cell transplant) is a potential cure for SCD.
  • Other treatments focus on symptom relief, complication management, and improving life expectancy.
  • Gene therapy is being explored as a potential cure, with the UK being the first country to approve gene therapy treatment for SCD.