Three patients stopped treatment thanks to the gene therapy

Basic

  • It is related to a genetic mutation on the X chromosome that is responsible for the absence of the enzyme alpha-galactosidase A, which is necessary for the body to get rid of some fat cells that are toxic to the body.
  • The principle of this treatment method is to transplant the stem cells from the patient’s blood, then correct the gene in the laboratory.
  • At the same time, the cells carrying the mutated gene are destroyed by the chemotherapy drugs that are given to the patients.
  • Once “the method becomes clear,” doctors re-inject the modified stem cells.

It is a new form of treatment that could give Fabry patients a lot of hope. Progressive and hereditary Fabry disease most often manifests itself in childhood. It is associated with a genetic mutation on the X chromosome responsible for the absence of the enzyme Alpha-galactosidase A.It is necessary for the body to get rid of some fat cells that are toxic to the body.

Symptoms of Fabry disease are chronic and can be multiple: visual impairment, abdominal pain, kidney damage, Heart failureCurrently, the most common treatment to mitigate the effects of these chronic diseases is injections every two months. However, this gas therapy has been shown to the patient. Not to mention that these injections help with better tolerance of symptoms, but not to stop disease progression.

Based on Gene therapy Description of the new course of treatment In the review Nature Communications And it was developed by a team of Canadian doctors that could guarantee Fabry patients greater comfort in life. After successful experiments with mice, researchers administered the treatment in 2017 to the first 52-year-old patient, called Mister Darren Bedulka.

Three patients were able to stop their treatment

The principle of this treatment method is to transplant the stem cells from the patient’s blood, then correct the gene in the laboratory. At the same time, the cells carrying the mutated gene are destroyed by the chemotherapy drugs that are given to the patients. Once “the method becomes clear,” the doctors re-inject Stem Cells Modified.

Since then, the treatment has been prescribed to four other patients. “So far, we can say that gene therapy has partially or completely restored enzyme levels to the point that they are no longer considered deficient.Dr. Anil Khan of the University of Calgary (Canada), who led the research, explains in a press release.

According to the study, three of the five patients chose to stop injection therapy after three years of receiving the gene therapy. However, doctors remain cautious and will follow their patients until 2024, in order to ensure that the treatment’s effectiveness is prolonged over time.


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