At the Perelman School of Medicine and Children’s Hospital of Philadelphia, a gene therapy study for hemophilia A showed good long term results in 16 of 18 patients. By administering a vector-delivered agent, called SPK-8011, factor VIII levels in the hemophiliac patients promptly boosted.
The vector-delivered agent, SPK-8011, was an investigational adeno-associated viral (AAV) vector for the hepatocyte, (a cell part of the tissue of the liver), expression of factor VIII. The liver is the main cause of the body delivering the missing clotting factor.
Of the 18 patients, this phase 1/2 trial showed a 91.5% annualized bleeding rate reduction, no apparent decrease in FVIII activeness over the course of 2 years in 12 of the patients, and two of the patients losing expression of the therapy within a year following vector administration. This last case is due to a presumed immune response to the AAV vector which shows that a steroid does not universally prevent the potential loss of expression. Lindsey A. George, the lead study author, says that, “Future research will aim to further improve on this work to safely achieve sustained, stable, and predictable FVIII levels in all hemophilia A patients.” The study was sponsored by Spark Therapeutics.
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