Metachromatic leukodystrophy (MDL) represents a huge medical challenge, affecting the central nervous system through progressive degeneration. This rare disease, with an incidence of one in 45,000 births in France (15 to 20 cases per year), leads to severe neurological disorders.Loss of motor skills, as well as deterioration of vision, hearing and mental functions.
Lenmeldi: A costly but promising step forward
Until recently, treatment options were nonexistent, limited to palliative management of symptoms. The outlook for diagnosed patients was bleak, as well Life expectancy after diagnosis of the disease is only five years.
The therapeutic landscape for patients with MLD is set to change dramatically with the introduction of Lanmeldi by Orchard Therapeutics. The gene therapy treatment, marketed at $4.25 million per injection, uses a harmless retrovirus to carry functional copies of the defective gene directly into the patient’s cells.
Once administered, these genetically modified cells can potentially Correct the root cause of the disease By differentiating into several cell types, some of which are able to cross the blood-brain barrier to restore essential enzyme function in the central nervous system.
A promising future for gene therapies
This innovative approach, although incredibly expensive, offers Glimpses of hope for stopping or at least slowing the progression of the disease with a single intervention. Before Lenmeldi is administered, patients must undergo high-dose chemotherapy to remove cells from the bone marrow, allowing replacement with repaired cells. Although unavailable in France, this treatment has been approved by the FDA in the United States, an important step toward global availability.
The introduction of Lenmeldi continues the remarkable progress made in the field of gene therapy. Like Zolgensma, treating spinal muscular atrophy costs $2 million per injection, Lenmeldi points out. The transformative potential of this treatment Despite their prohibitive cost.
These advances usher in an era of tailored treatment for genetic diseases, which not only improves quality of life but also provides significant extension of life expectancy for patients with previously incurable diseases.
