{"id":312624,"date":"2025-11-25T11:16:17","date_gmt":"2025-11-25T11:16:17","guid":{"rendered":"https:\/\/www.newsbeep.com\/us\/312624\/"},"modified":"2025-11-25T11:16:17","modified_gmt":"2025-11-25T11:16:17","slug":"california-boy-3-receives-first-ever-gene-therapy-for-his-rare-disease-its-just-so-exciting","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/us\/312624\/","title":{"rendered":"California boy, 3, receives first-ever gene therapy for his rare disease: \u2018It\u2019s just so exciting\u2019"},"content":{"rendered":"

A California toddler is the first person in the world<\/a> to receive gene therapy to treat his devastating disease.<\/p>\n

Three-year-old Oliver Chu was born with a rare, genetic condition called Hunter syndrome.<\/p>\n

Also known as mucopolysaccharidosis type II (MPS II), Hunter syndrome is a disorder where the body cannot break down specific sugar molecules.<\/p>\n

As these molecules accumulate in organs and tissues, they can cause progressive damage that affects the child\u2019s physical and mental development.<\/p>\n

\"Three-year-old<\/p>\n

Three-year-old Oliver Chu was born with a rare, genetic condition called Hunter syndrome. Chu Family<\/p>\n

Researchers at the University of Manchester in the UK have spent more than 15 years developing a gene therapy for Hunter syndrome.<\/p>\n

Now, a team at Royal Manchester Children\u2019s Hospital has seemingly stalled the disease by altering Oliver\u2019s cells.<\/p>\n

Oliver is the first of five boys in the world to receive the experimental intervention, and a year after beginning treatment, he appears to be developing normally.<\/p>\n

\u201cEvery time we talk about it, I want to cry because it\u2019s just so amazing,\u201d his mother, Jingru Chu, told the\u00a0BBC<\/a>.<\/p>\n

Almost always exclusive to boys, Hunter syndrome affects 1 in 100,000 male births worldwide.<\/p>\n

Patients seem healthy at birth, but they begin to show symptoms around the age of 2.<\/p>\n

A genetic error prevents cells from producing the enzyme iduronate-2-sulfatase (IDS), which is essential for breaking down large sugar molecules.<\/p>\n

\"Oliver\u2019s<\/p>\n

Oliver\u2019s treatment began last December when his care team harvested stem cells from his blood. BBC<\/p>\n

In the most severe cases, children start to experience problems with basic functioning between 6 and 8.<\/p>\n

Patients with the severe form of the disease typically die in their late teens or early 20s.<\/p>\n

Oliver\u2019s treatment began last December when the team harvested stem cells from his blood. Scientists then inserted the missing IDS gene into a hollow virus shell designed to deliver it into the stem cells\u2019 nucleus.<\/p>\n

\u201cWe use the machinery from the virus to insert a working copy of the faulty gene into each of the stem cells,\u201d Dr. Karen Buckland, lead scientist for Great Ormond Street Hospital\u2019s Cell and Gene Therapy Service, explained to the BBC.<\/p>\n

\u201cWhen those go back to Oliver, they should repopulate his bone marrow and start to produce new white blood cells, and each of these will hopefully start to produce the missing protein [enzyme] in his body.\u201d<\/p>\n

As planned, these genetically engineered stem cells were delivered via infusion in February to repopulate Oliver\u2019s bone marrow. The team was hopeful that the stem cells would begin producing white blood cells, which would then make the missing enzyme and deliver it throughout his body.<\/p>\n

\"Follow-up<\/p>\n

Follow-up tests revealed that the gene therapy was working, and Oliver is producing the missing enzyme. BBC<\/p>\n

Follow-up tests in May revealed that the gene therapy was working, and Oliver was indeed producing the key enzyme.<\/p>\n

\u201cHe\u2019s doing really well. We have seen him progressing in his speech and mobility. In just three months, he has matured,\u201d his father, Ricky, told the BBC.<\/p>\n

Ricky and Jingru reported that Oliver is \u201cso different\u201d from before his treatment \u2014 now he is more verbal and engaged with other children.<\/p>\n

\u201cI\u2019ve been waiting 20 years to see a boy like Ollie doing as well as he is, and it\u2019s just so exciting,\u201d said Simon Jones, who is co-leading the trial.<\/p>\n

\u201cBefore the transplant, Ollie didn\u2019t make any enzyme at all, and now he\u2019s making hundreds of times the normal amount. But more importantly, we can see he\u2019s improving, he\u2019s learning, he\u2019s got new words and new skills, and he\u2019s moving around much more easily.\u201d<\/p>\n

Until now, the only medical treatment available for Hunter syndrome was Elaprase. The drug, which costs around $600,000<\/a> per patient per year, can slow the physical effects of the disease, but because it cannot cross the blood-brain barrier, it cannot stave off cognitive decline.<\/p>\n

Oliver was initially deemed too old for the trial, as the treatment cannot reverse existing damage. However, tests revealed that his development was largely unaffected.<\/p>\n

He, along with the four other boys who received the treatment, will be monitored for two years. The research team is hopeful that, if the trial is deemed successful, they can partner with a biotech firm to license the treatment.<\/p>\n

\u201cWe need to be careful and not get carried away in the excitement of all this, but things are as good as they could be at this point in time,\u201d said Jones.<\/p>\n","protected":false},"excerpt":{"rendered":"A California toddler is the first person in the world to receive gene therapy to treat his devastating…\n","protected":false},"author":2,"featured_media":312625,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[34],"tags":[2356,13056,95531,97,161215,161218,161216,15298,161219,161217],"class_list":["post-312624","post","type-post","status-publish","format-standard","has-post-thumbnail","category-health","tag-california","tag-gene-therapy","tag-genetic-condition","tag-health","tag-hunter-syndrome","tag-jingru-chu","tag-oliver-chu","tag-stem-cells","tag-sugar-molecules","tag-white-blood-cells"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts\/312624","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/comments?post=312624"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts\/312624\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/media\/312625"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/media?parent=312624"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/categories?post=312624"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/tags?post=312624"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}