Europe is on the cusp of approving a gene therapy for the first time, in what would be a landmark moment for the field.
Gene therapies alter a patient’s DNA to treat inherited diseases passed from parent to child.
The European Medicines Agency has recommended a therapy for a rare genetic disease which leaves people unable to properly digest fats.
The European Commission will now make the final decision.
The idea of gene therapy is simple: if there is a problem with part of a patient’s genetic code then replace that part of the code.
The reality has not been so easy. In one gene therapy trial a US teenager, Jesse Gelsinger, died, and other patients have developed leukaemia.
There are no gene therapies available outside of a research lab in Europe or the US.
Gene change
The European Medicines Agency’s Committee for Medicinal Products for Human Use has considered the use of Glybera to treat lipoprotein lipase deficiency.
One in a million people have the deficiency. They have damaged copies of a gene which is essential for breaking down fat.
It leads to fat building up in the blood, abdominal pain and life-threatening pancreatitis (inflammation of the pancreas).
The only way to manage the condition is by having a very low-fat diet.
The therapy uses a virus to infect muscle cells with a working copy of the gene.
It was recommended for patients with severe pancreatitis, who cannot control the disease through diet.
BBC has the full article
