"Gene Therapy Will Cure You, or Your Money Back!"

For the first time ever, doctors in Italy are offering commercial gene therapy to people inflicted with rare diseases. The procedures come with a hefty price tag, but are guaranteed to work - or your money back!

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/

Nobel Laureates Take a Stand for GMOs

110 of the world's 300 living Nobel Laureates recently signed a letter directed at the global NGO Greenpeace demanding that the group backdown on their stance against genetically modified organisms (GMO). The letter was prompted because Greenpeace has continually blocked the introduction of a genetically engineered (GE) strain of rice called "Golden Rice" that could vastly reduce the number of Vitamin-A deficiencies (VAD) in the developing world.

According to the World Health Organization, VAD impacts an estimated 250 million people worldwide by compromising a person's immune system, resulting in blindness or death. Its' greatest impact can be seen on the poorest people of Africa and Southeast Asia. UNICEF statistics suggest that one to two million preventable deaths occur globally each year as a result of VAD, especially among infants and children. Golden Rice contains higher levels of Vitamin-A than normal strains of rice, and could therefore help combat the deficiencies, as those most affected consume rice as the mainstay of their diet.

Nobel laureate Randy Schekman, a cell biologist at UC Berkeley stated:

“I find it surprising that groups that are very supportive of science when it comes to global climate change, or even, for the most part, in the appreciation of the value of vaccination in preventing human disease, yet can be so dismissive of the general views of scientists when it comes to something as important as the world’s agricultural future.”

Most scientists believe that gene editing in a laboratory is no different and poses no greater threat to humans than selective breeding that has gone on for centuries. Additionally, GE plants can reduce the need for pesticides on crops (among other advantages), providing health and ecological benefits.

Greenpeace responded to the letter through their Southeast Asia campaigners, countering with the argument that:

“Corporations are overhyping ‘Golden’ Rice to pave the way for global approval of other more profitable genetically engineered crops. This costly experiment has failed to produce results for the last 20 years and diverted attention from methods that already work. Rather than invest in this overpriced public relations exercise, we need to address malnutrition through a more diverse diet, equitable access to food and eco-agriculture.”

Obviously, the debate between scientists and activists over GMOs is not a new phenomenon, nor does it appear that this letter will persuade GMO opponents to end their fight.

https://www.washingtonpost.com/news/speaking-of-science/wp/2016/06/29/more-than-100-nobel-laureates-take-on-greenpeace-over-gmo-stance/