In England, the National Health System NHS will offer Libmeldy, one of the most expensive drugs in the world, with a price of 3 million 800 thousand dollars, to patients free of charge.
Libmeldy, a gene therapy used in the very rare MLD disease that affects children’s nervous system and organs, is described as a ‘life-saving’ drug.
It is stated that about four babies are born with this disease each year in the UK.
It was announced that an agreement was reached on a discount in the negotiations conducted by NHS officials with the manufacturer and it was decided to offer the drug to patients.
NHS President Amanda Pritchard said: “This revolutionary drug is a life saver for babies and children suffering from this devastating hereditary disease. The drug will also ease the pain of families.”
Nicola Elson’s daughter Connie has MLD. Connie’s illness was diagnosed when she was a baby after she fell frequently and began to lose concentration.
Elson says he or his partner has never had a family history of MLD diagnosed.
It is stated that after Nicola Elson doctors mentioned a 25 percent probability for other children, Connie’s younger brother was also tested and his genes were found to be defective.
“The doctors said there was no hope”
“You never want to be in a situation like this. For a long time, doctors were saying there was no hope and there was nothing we could do,” Elson says.
According to her mother, Connie and her brother participated in early clinical trials of the drug in Italy.
However, during this time, Connie’s condition worsened. Experts say that this drug should be used after the appearance of symptoms in the initial stage of the disease.
MLD or Metachromatic Leukodystrophy is an extremely rare disease caused by enzyme deficiency. Over time, the nerves in the patient’s brain and other parts begin to not work as they should.
The disease is seen in babies younger than 30 months and can cause vision, hearing loss and speech impairment afterwards. Difficulty in movement and paralysis can be seen in the disease. The average life expectancy of patients is between five and eight.
Libmeldy is a form of gene therapy and replaces broken genes.
On December 5, 2014, Joe had an operation to remove stem cells from his bone marrow. As part of this experimental treatment, stem cells were repaired and reinjected into Joe’s body.
Joe Elson, now 11 years old, seven years after gene therapy, is no different from his peers, according to his family.
The long-term effects of this drug in children given it are not yet known.
It is stated that Joe’s condition will be monitored over the years. But both doctors and families hope the drug can provide a lasting cure.
Last year in England, the NHS did not give this medicine to patients because it was too expensive.