Taiwanese scientists have created the first genetic treatment approved by the EU for L-amino acid decarboxylase (AADC) deficiency, an extremely rare and fatal disorder, they said yesterday.
The treatment, given the trade name Upstaza, was on Wednesday approved for market by the European Medicines Agency, the EU’s main drug regulator, the National Taiwan University Hospital-based research team told a news conference.
AADC deficiency inhibits the secretion of an enzyme that is key to making dopamine, said team leader Hu Wu-liang (胡務亮), who is also the director of the hospital’s pediatrics and genetic medicine departments.
Photo: Yang Yuan-ting, Taipei Times
The disorder primarily manifests in infants and severely impedes their ability to develop speech, walk, sleep or swallow food, with death typically occurring before the age of five, he said.
The condition affects about 700 children in Taiwan, comprising 30 percent of the world’s AADC cases, suggesting that the genetic trait causing the disorder has a strong regional prevalence, Hu said.
As cases are rare in North America and Europe, where most of the world’s drugs are made, there was a lack of impetus among Western drugmakers to develop a treatment for the condition, he said.
In response, Hu launched an initiative to find a cure, and received the university hospital’s approval in his effort, which led to the creation of Upstaza in 2010, he said.
Upstaza is a one-time treatment that introduces a functioning AADC gene to the patient by directly injecting it into the patient’s brain, using modified adeno-associated viruses as the vehicle mechanism, Hu said.
An affected person with the replaced AADC gene would gain the ability to produce dopamine, leading to a partial recovery of bodily functions and providing hope for a normal life, Hu said, adding that the therapy has been proven by a 12-year tracking study.
A two-year-old patient who had been bedridden and unable to speak gained the ability to run, use staircases unassisted and verbally communicate within four years of receiving the treatment, he said.
“Using the technology, the hospital successfully treated the 30 Taiwanese patients [with AADC] to bring hope to their families,” Hu said.
The rights to the drug have been transferred to US-based PTC Therapeutics, which is exploring the possibility of applying Upstaza’s underlying viral and surgical technology to treat other conditions affecting the brain, he said.
Upstaza cannot be used for Parkinson’s disease, as the treatment requires intact neurons to work, Hu added.
SEND A MESSAGE: Sinking the amphibious assault ship, the lead warship of its class, is meant to show China the US Navy is capable of sinking their ships, an analyst said The US and allied navies plan to sink a 40,000-tonne ship at the latest Rim of the Pacific (RIMPAC) exercise to simulate defeating a Chinese amphibious assault on Taiwan. This year’s RIMPAC — the 29th iteration of the world’s largest naval exercise — involves the US, 28 partners, more than 25,000 personnel, 40 warships, three submarines and more than 150 aircraft operating in and around Hawaii from yesterday to Aug. 1, the US Navy said in a press release. The major components of the event include multidomain warfare exercises in multiship surface engagements, anti-submarine warfare and multi-axis defense of a carrier strike
Taiwanese could risk being extradited to China when traveling in countries with close ties to Beijing, Taiwan Association of University Professors deputy chairman Chen Li-fu (陳俐甫) said on Friday. Chen’s comments came after China on Friday last week announced new judicial guidelines targeting Taiwanese independence advocates. Myanmar, Cambodia, Laos and Djibouti are among the countries where Taiwanese could risk being extradited to China, he said. The Mainland Affairs Council (MAC) on Thursday elevated the travel alert for China, Hong Kong and Macau to “orange” after Beijing announced its guidelines to “severely punish Taiwanese independence diehards for splitting the country and inciting secession.” Extradition treaties
The airspace around Taiwan Taoyuan International Airport and Taipei International Airport (Songshan airport) is to be closed for an hour on July 25 and July 23 respectively, due to the Han Kuang military exercises, the Ministry of National Defense said yesterday. The annual exercise is to be held on Taiwan proper and its outlying islands from July 22 to 26. During last year’s exercise, the military conducted anti-aircraft landing drills at the Taoyuan airport for the first time, for which a one-hour no-fly ban was issued. Based on a live-fire bulletin sent out by the Maritime and Port Bureau, the nation’s
Taiwan and Thailand have signed an agreement to promote and protect bilateral investment and trade, the Executive Yuan’s Office of Trade Negotiations (OTN) said on Friday. The agreement on “Promotion and Protection of Investments” was signed by Representative to Thailand Chang Chun-fu (張俊福) and Thailand Trade and Economic Office in Taipei executive director Narong Boonsatheanwong on Thursday, the OTN said in a news release. Thailand has become the fifth trading partner to sign an investment agreement with Taiwan since 2016, following earlier agreements with the Philippines, India, Vietnam and Canada, the OTN said. The deal marks a significant milestone in the development of