The Guardian view on gene therapy: money well spent | Editorial

A rare and fatal disease will be now treated on the NHS. But the real problems come from common and unglamorous complaints

The NHS is to fund a very expensive treatment for a very rare but terrible childhood disorder that leaves babies condemned to life in a sterile bubble. This is a triumph for medical science but it should also provoke some deep and careful thought. The treatment in question, strimvelis, qualifies as the second most expensive drug ever put on the market (the only one more expensive was withdrawn due to lack of demand). A single dose costs nearly £500,000 plus VAT, and can only be administered in Milan, where the preparation is made. On the other hand, that one dose is literally life-saving, and as far as we now know, is the only treatment the disorder will ever need. At the rate that the National Institute for Health and Care Excellence (Nice), is prepared to pay for treatments of rare diseases – £100,000 a year of good life – this represents good value. The calculation may seem heartless, but it is unavoidable. Money spent on one patient is unavailable for all others. Someone, somehow, must decide who benefits.

Looked at purely as a medical advance, this is great. The babies born with this syndrome have two defective copies of a gene essential to the functioning of white blood cells. They are therefore unable to defend themselves against infection and unless kept in wholly sterile surroundings will die of a variety of horrible diseases and developmental disorders before they reach school age. Until now the only treatment was with a stem cell transplant, which was only effective in about three quarters of the cases, and relied on finding matched donors, who are rare. The new treatment removes defective stem cells and replaces their genes with properly functioning versions before returning these to the patients. Once these are incorporated into the bone marrow, they produce healthy white blood cells and the immune system functions as it should. This is how genetic medicine is supposed to work, but has not done reliably until now.

Continue reading... October 23, 2017 at 11:15PM