Jude, which helped fund the research along with the California Institute of Regenerative Medicine and the National Institutes of Health. Inspired by French researchers’ use of gene therapy to treat SCID (an experiment that ultimately led to serious complications like Leukemia), Sorrentino set out to find a way to initiate the growth of an immune system in a newborn without adding more risks.īuilding on the research out of France, his method involved the “use of a virus to transport and insert a correct copy of a gene into the genome of patients’ blood stem cells,” according to St. In recent years, researchers have used bone marrow transplants to treat the condition, but the procedure requires a sibling who is a match, which many do not have.Įnter the late Brian Sorrentino, MD, a physician, and survivor of childhood cancer who was determined to find a way to save kids with SCID. But the method made it difficult to live a normal life. SCID CURED MOVIEMore serious viruses such as pneumonia can quickly turn deadly, resulting in the death of many SCID patients before age one.Įarly treatment sometimes involved (as documented in the Travolta movie The Boy In The Plastic Bubble) putting individuals in NASA-level protective suits to insulate them from germs. As a result, those born with the condition (typically males) are unable to fight off infections - even those as minor as the common cold. SCID, which affects less than 20 infants a year, is caused by mutations in the genes that help develop and fuel the immune system. Jude Children's Research Hospital accepted a Smithsonian Ingenuity Award for what’s being referred to as a potential “cure.” This week, decades after it was captured on the big screen, researchers from St. It’s not often that a rare genetic immunodeficiency disorder captures the attention of the general public, but with an iconic John Travolta-led feature film centered on “bubble boy” disease, it seems the condition - formally known as severe combined immunodeficiency (SCID) - is an exception.įor those who have long been following the intricacies of the disease, there’s an important update. Pictured above is one of the 12 participants. Jude researchers successfully used a deactivated HIV virus to successfully treat 'bubble boy' disease in a groundbreaking study. The immune systems were restored for 48 of the 50 patients, as Kohn and his colleagues reported in a paper published May 11 in the New England Journal of Medicine, which generated self-congratulatory press releases from both Orchard and CIRM.St. SCID CURED TRIALThe success rate from donated stem cells was as low as 65% under Kohn’s therapy, it was 100% - that is, all the trial subjects were living trouble-free lives three years after the initial treatment. The new clinical trial was spectacularly successful. It lost more than $353 million in 20 and the first quarter of this year on revenues of a negligible $5 million. (Its stock closed Tuesday at $5.23.) Like most biotech startups, Orchard has never turned a profit. Orchard went public in 2018, ran as high as $20.25 a share in 2019, and has since traded in a range of about $5 to $8. SCID CURED LICENSEOrchard received an exclusive license to develop the treatment, which it designates internally as OTL-101. Kohn and Orchard collected grants from CIRM and other public institutions, including the National Institutes of Health and British medical foundations, and recruited 50 patients for a clinical trial to follow the trial that had included Evangeline. Since then, he says, he has been “trying to push them to move ahead.” Indeed, he says he was “gobsmacked” to hear of its decision to shelve the upcoming trial, which he learned from a press release the company issued last May. He was listed as a scientific co-founder upon the company’s launch in 2015, but he says he has no role or authority over its corporate decisions. Kohn partnered with Orchard Therapeutics to pursue further clinical trials. As a vivacious 4-year-old, she was featured on the cover of CIRM’s 2016 annual report astride a hobby horse and clad in a pink sweatshirt bearing a lightning bolt, under the legend “CURED.” Kohn’s discovery was, and still remains, perhaps the most advanced cure of any disease developed with CIRM funding.Įvangelina became the poster child for the potential of CIRM research. Luckily, Evangelina’s parents were referred to Kohn, who was launching a test of his stem cell-based therapy. That didn’t work in Evangelina’s case not even her fraternal twin sister was a match. Until now, the most effective treatment has involved bone marrow stem cell transplants from a matched donor such as a family member.
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