[Reading Science] "4.3 Billion per Dose"... The World's Most Expensive Drug Lives Up to Its Name

First Child Treated in the UK Recovers Health
Effectiveness Confirmed for Rare Genetic Disorder Treatment
Levemeldi for X-Linked Leukodystrophy
Ultra-High Cost of ?2.8 Million per Dose

The rare genetic disease treatment, which costs a staggering 4.37 billion KRW per dose, has proven its worth as the world's most expensive drug. A 19-month-old infant, whose condition was considered practically untreatable with limited life expectancy, is now maintaining a healthy state.

The UK's National Health Service (NHS) announced on the 13th that Teddy Shaw, a 19-month-old girl diagnosed with the fatal genetic disorder Metachromatic Leukodystrophy (MLD), has remained symptom-free and healthy six months after receiving gene therapy at the Royal Manchester Children's Hospital last August. Medical staff extracted hematopoietic stem cells from Teddy's spinal cord in April last year, corrected them, and reintroduced them into her body four months later, monitoring her progress since then.

The gene therapy prescribed to this girl is Libmeldy, an MLD treatment approved by the European Medicines Agency (EMA) in 2022 and developed by Orchard Therapeutics. It was created by inserting a modified gene that removes the factor causing MLD into the patient's hematopoietic stem cells using a lentivirus. The price per dose reaches approximately 4.37 billion KRW (2.8 million pounds), making it the most expensive drug in the world. The therapy helps the patient's cells break down sulfatides. When sulfatides accumulate, the patient's brain and nerve cells are destroyed, leading to cognitive impairment, motor control and sensory disabilities, and eventually seizures, paralysis, and blindness. Ultimately, it results in death. In Teddy's case, her remaining lifespan was predicted to be as short as 5 years and up to a maximum of 8 years. Until now, MLD treatment has been limited to symptom management. Techniques such as bone marrow transplantation or umbilical cord blood stem cell transplantation were only experimentally used to slow progression.

Libmeldy is a type of cell therapy that modifies genes using viral vectors or gene-editing technologies. Clinical trials showed clear therapeutic effects such as sulfatide breakdown and normal development when administered to infants or adolescents who had not yet exhibited MLD symptoms. However, due to high development costs, manufacturers claim that the expensive price of several billion KRW per treatment session is unavoidable. Nevertheless, the duration of the therapy's effectiveness remains uncertain. The EMA also states that it is unclear whether the effect will last a lifetime and that long-term follow-up is necessary. Meanwhile, Teddy's older sister Nyla, who was also diagnosed with MLD, was unable to receive treatment because her symptoms had already manifested.

Teddy and Nyla's mother, Ollie, expressed gratitude that "Teddy has been given the chance to live a normal life" and hopes that "one day treatment will be possible for all stages of MLD." Amanda Pichard, director of the NHS, also welcomed the news, saying, "This is a very hopeful outcome for children born with devastating genetic disorders and their parents. Teddy can now go to school and play with friends like other children. We are delighted to be able to provide such a miraculous treatment."

This content was produced with the assistance of AI translation services.