The treatment is called Strimvelis, and it is the first outright cure for the autoimmune disease ADA-SCID, that leaves newborn babies with almost no defense against viruses, bacteria, or fungi, and is often fatal. This same disease was documented in the 1976 movie Bubble Boy about an American child who lived his life inside a plastic bubble.
Strimvelis uses a virus to deploy a missing gene into the bone marrow of afflicted children, allowing the stem cells within the marrow to naturally produce the proper immune defenses for life. It is considered a “repair and replace” strategy because doctors extract stem cells from bone marrow, then soak the cells with viruses to transfer a correct copy of the ADA gene.
Strimvelis was initially developed in Milan, Itlay at the San Raffaele Telethon Institute for Gene Therapy, but pharmaceutical giant GlaxoSmithKline purchased the rights to the procedure in 2010. The price tag for the one-time treatment will be $665,000. This makes it one of the most expensive single procedures in all of medicine, but is considered pennies compared to the cost of the established method of treating the disease.
GSK sells $30 billion is drug each year, so revenue from the treatment is not the bottom line. Rather, the British company hopes to master gene therapy technology, including virus manufacturing. Sven Kili, the executive of gene therapy development at GSK stated, "If we can first make products that change lives, then we can develop them into things that affect more people. We believe gene therapy is an area of important future growth; we don’t want to rush or cut corners."
Read more: https://www.technologyreview.com/s/602113/gene-therapy-cure-has-money-back-guarantee/
https://www.technologyreview.com/s/601390/gene-therapys-first-out-and-out-cure-is-here/
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