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.
An essay competition jointly organized by a local writing society and a publisher affiliated with the Chinese Communist Party (CCP) might have contravened the Act Governing Relations Between the People of the Taiwan Area and the Mainland Area (臺灣地區與大陸地區人民關係條例), the Mainland Affairs Council (MAC) said on Thursday. “In this case, the partner organization is clearly an agency under the CCP’s Fujian Provincial Committee,” MAC Deputy Minister and spokesperson Liang Wen-chieh (梁文傑) said at a news briefing in Taipei. “It also involves bringing Taiwanese students to China with all-expenses-paid arrangements to attend award ceremonies and camps,” Liang said. Those two “characteristics” are typically sufficient
A magnitude 5.9 earthquake that struck about 33km off the coast of Hualien City was the "main shock" in a series of quakes in the area, with aftershocks expected over the next three days, the Central Weather Administration (CWA) said yesterday. Prior to the magnitude 5.9 quake shaking most of Taiwan at 6:53pm yesterday, six other earthquakes stronger than a magnitude of 4, starting with a magnitude 5.5 quake at 6:09pm, occurred in the area. CWA Seismological Center Director Wu Chien-fu (吳健富) confirmed that the quakes were all part of the same series and that the magnitude 5.5 temblor was
The brilliant blue waters, thick foliage and bucolic atmosphere on this seemingly idyllic archipelago deep in the Pacific Ocean belie the key role it now plays in a titanic geopolitical struggle. Palau is again on the front line as China, and the US and its allies prepare their forces in an intensifying contest for control over the Asia-Pacific region. The democratic nation of just 17,000 people hosts US-controlled airstrips and soon-to-be-completed radar installations that the US military describes as “critical” to monitoring vast swathes of water and airspace. It is also a key piece of the second island chain, a string of
The Central Weather Administration has issued a heat alert for southeastern Taiwan, warning of temperatures as high as 36°C today, while alerting some coastal areas of strong winds later in the day. Kaohsiung’s Neimen District (內門) and Pingtung County’s Neipu Township (內埔) are under an orange heat alert, which warns of temperatures as high as 36°C for three consecutive days, the CWA said, citing southwest winds. The heat would also extend to Tainan’s Nansi (楠西) and Yujing (玉井) districts, as well as Pingtung’s Gaoshu (高樹), Yanpu (鹽埔) and Majia (瑪家) townships, it said, forecasting highs of up to 36°C in those areas
RSS