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First UK child to receive gene therapy for fatal genetic disorder is now healthy (livescience.com)

791 points by ryzvonusef · 1293 days ago · 187 comments on HN

Article summary

A 19-month-old girl in the UK has become the first child to receive a new gene therapy for metachromatic leukodystrophy (MLD), a fatal genetic disorder. The therapy, called Libmeldy, works by inserting a working copy of the faulty gene into the patient's stem cells, which are then used to produce healthy cells. The treatment has shown promise in clinical trials, with patients able to break down sulfatides at normal rates and showing typical patterns of motor development. The long-term effectiveness of the therapy is still being studied.

Main themes

  • gene therapy
  • MLD treatment
  • stem cell transplants
  • cost-effectiveness
  • healthcare ethics
  • regulatory policy

What commenters say

  • Gene therapy has the potential to be a more effective and longer-lasting treatment for genetic disorders like MLD compared to traditional treatments like bone marrow transplants.
  • The use of lentiviral vectors to insert genes into stem cells is a promising approach for treating diseases that affect the bone marrow and blood cells.
  • The high cost of gene therapy is a significant barrier to its adoption, and some argue that it is not cost-effective compared to other treatments.
  • Others argue that the value of a human life cannot be measured solely in economic terms, and that gene therapy should be made available to those who need it regardless of cost.
  • The effectiveness of gene therapy in treating genetic disorders like MLD may depend on the specific disease and the individual patient's circumstances.
  • Some commenters expressed concern about the potential risks and side effects of gene therapy, including the possibility of unintended consequences or long-term effects.
  • The use of gene therapy to treat genetic disorders raises ethical questions about the value of human life and the allocation of resources in the healthcare system.
  • Regulatory pressure may be necessary to make gene therapy more affordable and accessible to those who need it.