Gene Therapy Guarantee

The ultimate goal of gene therapy is to permanently cure genetic disorders by making a one-time correction to the faulty portions of a patient’s DNA. Recently, a new treatment called Strimvelis was cleared for marketing in Europe that may actually live up to that challenge. In fact, the company is so confident that the drug comes with a money-back guarantee.

Strimvelis was developed to treat severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), a rare disease that severely compromises the immune system in children and frequently proves fatal within the first year of life. In a study involving 18 children, 15 were cured without further intervention, while three required additional treatment, including enzyme replacement therapy and/or stem cell transplantation. However, all 18 of the original subjects are alive today, the first having received Strimvelis more than 13 years ago.

At a price of 594,000 euros (about $665,000), the cost of the treatment seems high, and indeed, Strimvelis is one of the most expensive one-time treatments to ever be sold. But considering that the cost of a bone marrow transplant can easily reach $1 million, and other treatments such as enzyme injections can cost upwards of $250,000 per year, not to mention the costs associated with caring for a sick child, the price tag isn’t outrageous.

Strimvelis will only be available at Ospedale San Raffaele in Milan, Italy, a clinical research hospital that provides advanced specialty care for some of the most complex health issues.

For information: GlaxoSmithKline PLC, GSK House, 980 Great West Road, Brentford, Middlesex TW8 9GS, United Kingdom; phone: +44-(0)20-8047-5000; website: http://www.gsk.com/en-gb/