Gene therapy is a rapidly evolving field that has the potential to offer life-changing treatments for a variety of genetic diseases. The recent approval of Libmeldy, a gene therapy for MLD (metachromatic leukodystrophy) in Europe and the U.K., is an exciting development for those suffering from this rare, progressive neurological disorder. Libmeldy works by inserting working copies of the genes that are faulty in MLD into the body, thus restoring the ability to break down sulfatides. The therapy has been able to provide long term benefits to patients with infantile and juvenile MLD who have not yet developed symptoms, though it remains unclear whether it will persist lifelong or if further follow-up may be necessary.
At this point gene therapy remains an expensive option - Libmeldy’s list price was initially £2.8 million ($3.4 million at today’s exchange rates) - but its efficacy in treating certain genetic disorders may offer hope for those affected by diseases which are otherwise difficult or impossible to cure through other methods such as standard bone marrow transplants or immunosuppressive drugs used post-transplantation maintenance therapy .
The process used by Libmeldy involves extracting stem cells from either bone marrow or mobilised peripheral blood then transducing them with clinical grade lentiviral vector encoding human ARSA cDNA under control of the human phosphoglycerate kinase gene promoter before putting them back into the patient’s body where they can give rise to new white blood cells that travel through bloodstream carrying functional genes into body parts where they are needed most. This treatment essentially fixes disease at its source as any new cells produced using treated stem cells will inherit this fix rather than being susceptible to disease mutation and progression as they were previously .
Gene therapy is still a relatively new field so there remain many questions about how best it can be applied safely and effectively over time; however advances like these show promise for treating many more genetic conditions in future - particularly those related to bone marrow such as Von Hippel Landau Syndrome which could benefit from similar approaches such as CAR-T and cell / gene therapies . It will still take time before we fully understand how safe these treatments are over long periods but, given their potential impact on quality of life , I’m sure research efforts will continue until then!
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Author Eliza Ng