Strimvelis

Strimvelis is a gene therapy product used for the treatment of adenosine deaminase deficiency (ADA-SCID), a rare genetic disorder that affects the immune system. It is the first ex vivo stem cell gene therapy to be approved by the European Medicines Agency (EMA).

Overview

Strimvelis is a one-time treatment that uses the patient's own stem cells to correct the genetic defect causing ADA-SCID. The therapy involves extracting bone marrow from the patient, isolating the stem cells, and introducing a normal copy of the ADA gene into these cells. The genetically modified cells are then returned to the patient's body, where they can produce the ADA enzyme that is missing in people with ADA-SCID.

Mechanism of Action

Strimvelis works by introducing a normal copy of the ADA gene into the patient's stem cells. This is done using a virus that has been modified to carry the ADA gene but cannot cause disease. Once the gene is inside the stem cells, it provides instructions for making the ADA enzyme. This enzyme is crucial for the function of the immune system, as it breaks down toxic substances that can harm immune cells.

Efficacy and Safety

Clinical trials have shown that Strimvelis is effective in restoring immune function in patients with ADA-SCID. The therapy has also been found to be safe, with few side effects reported. However, as with any gene therapy, there are potential risks, including the possibility of insertional oncogenesis (the development of cancer due to the insertion of the gene into the patient's DNA).

Regulatory Status

Strimvelis was approved by the EMA in 2016 for the treatment of ADA-SCID in patients for whom no suitable human leukocyte antigen (HLA)-matched stem cell donor is available. It is currently the only gene therapy approved for this indication.

See Also

• Gene therapy
• Adenosine deaminase deficiency
• Stem cell therapy
• European Medicines Agency