New gene therapy Libmeldy is applied to a 19-month-old girl in the UK.
About six months into the treatment, Teddi is a happy and healthy baby with no signs of the monstrous illness she was born with,” the NHS statement read.
The genetic disorder MLD disrupts cells’ ability to break down sulfates, a fatty substance used to insulate the electrical wiring that runs through the brain’s white matter and much of the nervous system outside the brain. According to the Genetic and Rare Diseases Information Center, the accumulation of sulfates destroys the brain and nerve cells, leading to cognitive problems, loss of motor control and sensation, seizures, paralyzed and blind. Ultimately, this disorder leads to death.
Typically, treatment for MLD is aimed at controlling symptoms of the disease, although some experimental therapies, including bone marrow transplants and cord blood stem cell transplants, are sometimes used to slow progression of the disorder in infants, according to the Centers for Disease Control and Prevention.
The new gene therapy, called Libmeldy (generic name atidarsagene autotemcel), has only recently been approved by the NHS and works by introducing active copies of the gene that is faulty in MLD, so that restore the ability to degrade sulfates.
According to the European Medicines Agency (EMA), Libmeldy is created using stem cells taken from a patient’s blood or bone marrow and can produce different types of blood cells. These stem cells carry new functional genes into the body, where they make white blood cells that move in the blood.
In clinical trials, Libmeldy therapy provided clear benefits to infant and adolescent patients who had not yet developed MLD symptoms; For example, these patients are able to break down sulfates at a normal rate and show typical motor development patterns.
The EMA notes that the benefits of this therapy appear to last several years, but at this point it is unclear whether it is long-lasting and requires long-term follow-up.
Teddi and her sister Nala, were both diagnosed with MLD in April 2022, according to the NHS. Unfortunately, Nala was not eligible for Libmeldy because she developed symptoms of the disorder. Teddi had stem cells in June and received new, modified stem cells in August.
The gene therapy Libmeldy has been approved in the European Union and the UK, although its price is tightly controlled with a suggested price of £2.8 million ($3.4 million), according to BBC News in 2022.
This new gene therapy has not yet been approved by the US Food and Drug Administration (FDA).
