Gene therapy cures infants with ‘bubble boy’ immune disease

By William Wan William Wan National correspondent covering health, science and news Email Bio Follow April 17 at 5:00 PM Ten newborns with the rare genetic disorder known as bubble boy disease were cured with gene therapy, researchers revealed Wednesday. The treatment appears to have completely rid the babies of their immune disorder with no side effects or complications – a result scientists have sought for decades through painstaking research and heartbreaking setbacks.

Infants with SCID are essentially born without a functioning immune system. Without treatment, they rarely survive past their first birthday and can be killed by infections as innocuous as the common cold. Such children were once kept isolated in sterile environments, giving rise to the term “bubble boy.” Their unusual predicament has caught national attention and been featured in movies and TV shows.

David Vetter, born with an inherited immune system disorder, is shown in this Sept. 11, 1982 photo in Texas. In interviews, the researchers said they first extracted blood stem cells from the infants’ bone marrow. They used a modified virus as a vehicle to deliver the correct copy of a defective gene into those patients’ stem cells. Those corrected cells were then reinfused back into the patient, where they proliferated and created healthy immune cells.

Sorrentino was diagnosed at a young age with Hodgkin’s lymphoma, said his widow, Suzanne Sorrentino. Because modern treatments did not yet exist, his doctors treated him using radiation that weakened his heart and produced the lung cancer that killed him last November. “He told me he thought he was saved as a kid from Hodgkin’s because there was something he thought he should be doing with his life,” said Suzanne, her voice cracking as she recalled one of her last conversations with her husband. “With the trial working, and the children doing so well, he felt it was a sign that he achieved what he was supposed to. That it was time for him to go.”

The disease drew national attention in the 1970s with the case of David Vetter. News outlets chronicled his life as he grew up cocooned in plastic. His story spawned the 1976 John Travolta movie “The Boy In the Plastic Bubble.” And even after Vetter’s death, something about the disease’s vulnerable isolating effects kept it in the national consciousness, making a famous cameo in a Seinfeld episode and becoming the panned premise of a Jake Gyllenhaal movie “Bubble Boy.

I wonder if GreysABC gave them the idea 🤔

jacklhasa

الشعب العراقي يطالب الولايات المتحدة من منع الدول المجاورة بالتدخل بالشأن العراقي وعليها حماية آمنة وسيادتة وإنقاذة من المليشيات المسلحة التابعة لدول الجوار ولو تطلب ذلك عبر قرار من مجلس الأمن الدولي .. مواطنين من العراق

andymarso Amazing medical news. Now if we only had a simple, proven method of preventing all of these new measles cases that was readily available to the public.

Wonderful news! Keep researching!!

This is amazing news. So many will have quality of life because of this. Bravo

Wow thanks

Medical scientists and researchers are the world’s real heroes!

That's awesome 👍

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Catalent to acquire Paragon Bioservices for $1.2 billion in cashCatalent Inc. said Monday it has agreed to acquire Paragon Bioservices Inc. for $1.2 billion in cash. Calent, a provider of delivery technologies for drugs, biologics and consumer health products, said it expects the deal to close in the second quarter. Privately held Paragon is a viral vector development and manufacturing partner for gene therapies. "Paragon brings specialized expertise in adeno-associated virus (AAV) vectors, the most commonly used delivery system for gene therapy, as well as unique capabilities in GMP plasmids and lentivirus vectors," Catalent said in a statement. Paragon is expected to generate more than $200 million in revenue in 2019 with nearly 90% of that sum already in signed contracts. The deal is expected to boost Catalent's adjusted net income per share in the second full fiscal year after closing. Catalent will finance the deal with the proceeds of a $650 million term loan and by issuing $650 million of convertible preferred stock to funds tied to Leonard Green and Partners LP. Shares were not yet active premarket, but have fallen 2.4% in the last 12 months, while the S&P 500 has gained 9.5%.
Source: MarketWatch - 🏆 3. / 97 Read more »

There's now a cure for the deadly genetic disorder known as 'bubble boy' diseaseDoctors at St. Jude's Children's Research Hospital used a cutting-edge medical technique called gene therapy to treat 10 babies born with the disease.
Source: BusinessInsider - 🏆 729. / 51 Read more »