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Learning English Broadcast, Gene Therapy Delivers Treatment Directly to Brain

Gene Therapy Delivers Treatment Directly to Brain

When Rylae-Ann Poulin was 1 year old, she did not crawl or make noises like other kids her age. A rare genetic disease prevented her from even lifting her head. Her parents took turns holding her all night to ease her breathing and help her sleep peacefully.

But months later, doctors in Taiwan treated the young patient's brain directly, attacking the disease with genetic medicine. Now the 4-year-old is walking, running, swimming, reading and riding horses.

Rylae-Ann lives with her family in Bangkok, Thailand. She was accepted into a medical trial for a new method of gene therapy. Experts say the treatment holds great promise for treating several brain diseases.

The method was approved in Europe and the United Kingdom for a condition called AADC deficiency. The disorder damages communication systems among nervous system cells.

American drug company PTC Therapeutics plans to seek U.S. approval for the treatment this year.

The National Institutes of Health (NIH) says there are about 30 studies underway in the United States right now to test gene therapy to the brain. The studies target a number of diseases.

Dr. Krystof Bankiewicz at Ohio State University leads one such study on AADC deficiency. Others test treatments for brain diseases like Alzheimer's, Parkinson's and Huntington's.

Difficulties remain, especially with diseases caused by more than a single gene. But scientists say evidence is growing in support of gene therapy to the brain.

“There's a lot of exciting times ahead of us,” said Bankiewicz, a brain surgeon. “We're seeing some breakthroughs.”

The most amazing of those breakthroughs involve Rylae-Ann's disease. It is caused by changes in a gene needed for an enzyme that helps make neurotransmitters, the body's chemical messengers. The one-time treatment provides a working version of the gene.

At around 3 months old, Rylae-Ann began having what her parents thought were seizures. Fluid sometimes got into her lungs after she ate, a condition that required hospital treatment. Doctors thought she might have epilepsy or cerebral palsy, more common brain diseases.

Later, her parents found a doctor who correctly identified Rylae-Ann's condition. Her parents entered her into a gene therapy trial in Taiwan. The treatment requires doctors to operate directly on the brain. The doctors injected the genetic material using an extremely narrow tube.

“It gets put into the brain cells and then the brain cells make the (neurotransmitter) dopamine,” said Stuart Peltz, leader of PTC Therapeutics.

Company officials said all patients in their trials showed movement and thinking improvements. Some of them, Peltz said, could stand and walk and continue getting better over time.

Bankiewicz of Ohio State University also reported success in his NIH-financed study. He said all 40 or so patients involved saw big improvements.

Scientists say there are difficulties to face before this treatment becomes common for more brain diseases.

For example, the timing of treatment is an issue. Generally, earlier in life is better because diseases can cause more problems over the years. Also, more complex disorders like Alzheimer's are harder to treat with gene therapy.

A more basic barrier is cost. The price of gene therapies, paid for mostly by insurers and governments, can be in the millions. The one-time PTC therapy costs more than $3 million in Europe, for example.

But drugmakers say they want people to get the treatments they need. And researchers are hopeful they can clear the remaining scientific barriers to this method.

“So I would say gene therapy can be leveraged for many sorts of brain diseases and disorders,” said Ryan Gilbert. He is a biomedical engineer at New York's Rensselaer Polytechnic Institute. "In the future, you're going to see more technology doing these kinds of things,” he added.

I'm Dan Novak.


Gene Therapy Delivers Treatment Directly to Brain يقدم العلاج الجيني العلاج مباشرة إلى الدماغ Gentherapie bringt Behandlung direkt ins Gehirn Gene Therapy Delivers Treatment Directly to Brain La terapia génica brinda tratamiento directamente al cerebro 遺伝子治療で脳に直接治療薬を届ける Terapia genowa dostarcza leczenie bezpośrednio do mózgu A Terapia Genética oferece o tratamento directamente ao cérebro Генная терапия доставляет лечение непосредственно в мозг Genska terapija zagotavlja zdravljenje neposredno v možgane Gen Terapisi Tedaviyi Doğrudan Beyne Ulaştırıyor Генна терапія доставляє лікування безпосередньо в мозок 基因疗法直接对大脑进行治疗 基因療法直接對大腦進行治療

When Rylae-Ann Poulin was 1 year old, she did not crawl or make noises like other kids her age. عندما كانت Rylae-Ann Poulin تبلغ من العمر عامًا واحدًا ، لم تزحف أو تصدر أصواتًا مثل الأطفال الآخرين في سنها. ライレー・アン・プーランが1歳のとき、彼女は他の同年齢の子供たちのようにハイハイもせず、声も出さなかった。 Quando Rylae-Ann Poulin tinha 1 ano de idade, ela não engatinhava nem fazia barulho como as outras crianças de sua idade. Ko je bila Rylae-Ann Poulin stara eno leto, se ni plazila in ni proizvajala zvokov kot drugi otroci njene starosti. 当 Rylae-Ann Poulin 一岁时,她不像其他同龄孩子那样爬行或发出声音。 A rare genetic disease prevented her from even lifting her head. منعها مرض وراثي نادر من رفع رأسها. 珍しい遺伝病で、彼女は頭を持ち上げることさえできなかった。 Uma doença genética rara a impedia de levantar a cabeça. Zaradi redke genetske bolezni ni mogla niti dvigniti glave. 一种罕见的遗传病让她连头都抬不起来。 Her parents took turns holding her all night to ease her breathing and help her sleep peacefully. تناوب والداها على إمساكها طوال الليل لتسهيل تنفسها ومساعدتها على النوم بسلام. 両親は一晩中交代で彼女を抱いて、呼吸を楽にし、安らかに眠れるようにした。 Seus pais se revezaram para segurá-la a noite toda para facilitar sua respiração e ajudá-la a dormir em paz. Starši so jo vso noč izmenično držali v naročju, da bi ji olajšali dihanje in ji pomagali mirno spati. 她的父母整夜轮流抱着她,以缓解她的呼吸,帮助她安然入睡。

But months later, doctors in Taiwan treated the young patient's brain directly, attacking the disease with genetic medicine. لكن بعد أشهر ، عالج الأطباء في تايوان دماغ المريض الشاب مباشرة ، وهاجموا المرض بالطب الوراثي. Mas meses depois, os médicos de Taiwan trataram diretamente o cérebro do jovem paciente, atacando a doença com a medicina genética. Nekaj mesecev pozneje pa so zdravniki na Tajvanu neposredno zdravili možgane mladega bolnika in bolezen napadli z genetsko medicino. 但几个月后,台湾的医生直接治疗了这位年轻患者的大脑,用基因药物来治疗这种疾病。 Now the 4-year-old is walking, running, swimming, reading and riding horses. Agora, a criança de 4 anos está andando, correndo, nadando, lendo e andando a cavalo. Zdaj štiriletnik hodi, teče, plava, bere in jaha konje. 现在,这个 4 岁的孩子可以散步、跑步、游泳、阅读和骑马。

Rylae-Ann lives with her family in Bangkok, Thailand. Rylae-Ann z družino živi v Bangkoku na Tajskem. Rylae-Ann 与家人住在泰国曼谷。 She was accepted into a medical trial for a new method of gene therapy. تم قبولها في تجربة طبية لطريقة جديدة للعلاج الجيني. Ela foi aceita em um teste médico para um novo método de terapia genética. Sprejeli so jo v medicinsko preskušanje nove metode genskega zdravljenja. 她被接受参加一项新的基因治疗方法的医学试验。 Experts say the treatment holds great promise for treating several brain diseases. يقول الخبراء إن العلاج يحمل وعدًا كبيرًا في علاج العديد من أمراض الدماغ. Especialistas dizem que o tratamento é uma grande promessa para o tratamento de várias doenças cerebrais. Strokovnjaki pravijo, da je zdravljenje zelo obetavno za zdravljenje več možganskih bolezni. 专家表示,这种疗法对于治疗多种脑部疾病具有广阔的前景。

The method was approved in Europe and the United Kingdom for a condition called AADC deficiency. تمت الموافقة على الطريقة في أوروبا والمملكة المتحدة لحالة تسمى نقص AADC. O método foi aprovado na Europa e no Reino Unido para uma condição chamada deficiência de AADC. Metoda je bila odobrena v Evropi in Združenem kraljestvu za bolezen, imenovano pomanjkanje AADC. 该方法在欧洲和英国获得批准用于治疗 AADC 缺乏症。 The disorder damages communication systems among nervous system cells. يضر هذا الاضطراب بجهاز الاتصال بين خلايا الجهاز العصبي. O distúrbio danifica os sistemas de comunicação entre as células do sistema nervoso. Motnja poškoduje komunikacijske sisteme med celicami živčnega sistema. 这种疾病会损害神经系统细胞之间的通讯系统。

American drug company PTC Therapeutics plans to seek U.S. تخطط شركة الأدوية الأمريكية PTC Therapeutics للبحث عن الولايات المتحدة A empresa farmacêutica americana PTC Therapeutics planeja buscar nos EUA Ameriško podjetje za zdravila PTC Therapeutics namerava v ZDA pridobiti 美国制药公司 PTC Therapeutics 计划寻求美国市场 approval for the treatment this year. الموافقة على العلاج هذا العام. aprovação para o tratamento neste ano. odobritev za zdravljenje v letošnjem letu.

The National Institutes of Health (NIH) says there are about 30 studies underway in the United States right now to test gene therapy to the brain. تقول المعاهد الوطنية للصحة (NIH) إن هناك حوالي 30 دراسة جارية في الولايات المتحدة في الوقت الحالي لاختبار العلاج الجيني للدماغ. Po podatkih Nacionalnega inštituta za zdravje (NIH) v ZDA trenutno poteka približno 30 študij za testiranje genskega zdravljenja možganov. 美国国立卫生研究院 (NIH) 表示,美国目前正在进行大约 30 项研究,以测试针对大脑的基因疗法。 The studies target a number of diseases. تستهدف الدراسات عددًا من الأمراض. Os estudos têm como alvo uma série de doenças. Študije so usmerjene v številne bolezni. 这些研究针对多种疾病。

Dr. Krystof Bankiewicz at Ohio State University leads one such study on AADC deficiency. يقود الدكتور Krystof Bankiewicz من جامعة ولاية أوهايو دراسة عن نقص AADC. O Dr. Krystof Bankiewicz, da Ohio State University, lidera um desses estudos sobre a deficiência de AADC. Dr. Krystof Bankiewicz na državni univerzi v Ohiu vodi eno od takšnih študij o pomanjkanju AADC. 俄亥俄州立大学的 Krystof Bankiewicz 博士领导了一项关于 AADC 缺乏症的研究。 Others test treatments for brain diseases like Alzheimer's, Parkinson's and Huntington's. يختبر آخرون علاجات لأمراض الدماغ مثل مرض الزهايمر وباركنسون وهنتنغتون. Outros testam tratamentos para doenças cerebrais como Alzheimer, Parkinson e Huntington. Drugi preizkušajo zdravljenje možganskih bolezni, kot so Alzheimerjeva, Parkinsonova in Huntingtonova bolezen. 其他人则测试阿尔茨海默氏症、帕金森氏症和亨廷顿氏症等脑部疾病的治疗方法。

Difficulties remain, especially with diseases caused by more than a single gene. لا تزال هناك صعوبات ، خاصة مع الأمراض التي تسببها أكثر من جين واحد. As dificuldades permanecem, especialmente com doenças causadas por mais de um único gene. Težave ostajajo, zlasti pri boleznih, ki jih povzroča več kot en gen. 困难依然存在,尤其是由多个基因引起的疾病。 But scientists say evidence is growing in support of gene therapy to the brain. لكن العلماء يقولون إن الأدلة تتزايد لدعم العلاج الجيني للدماغ. Mas os cientistas dizem que as evidências estão crescendo em apoio à terapia genética para o cérebro. Vendar znanstveniki pravijo, da je vse več dokazov v prid genskemu zdravljenju možganov. 但科学家表示,越来越多的证据支持对大脑进行基因治疗。

“There's a lot of exciting times ahead of us,” said Bankiewicz, a brain surgeon. قال بانكيويتز ، جراح المخ ، "هناك الكثير من الأوقات المثيرة التي تنتظرنا". “Há muitos momentos emocionantes pela frente”, disse Bankiewicz, um neurocirurgião. "Pred nami je veliko vznemirljivih časov," je dejal Bankiewicz, možganski kirurg. “我们面前还有很多激动人心的时刻,”脑外科医生班凯维奇说。 “We're seeing some breakthroughs.” "نحن نشهد بعض الاختراقات." "Estamos a ver alguns avanços". "Vidimo nekaj dosežkov." “我们看到了一些突破。”

The most amazing of those breakthroughs involve Rylae-Ann's disease. ومن أكثر تلك الاكتشافات إثارة للإعجاب مرض ريلاي آن. Najbolj neverjetni od teh dosežkov so povezani z boleznijo Rylae-Ann. It is caused by changes in a gene needed for an enzyme that helps make neurotransmitters, the body's chemical messengers. وهو ناتج عن تغيرات في الجين اللازم لإنزيم يساعد في صنع الناقلات العصبية ، وهي الناقلات الكيميائية للجسم. É causada por alterações em um gene necessário para uma enzima que ajuda a produzir neurotransmissores, os mensageiros químicos do corpo. Povzroča jo sprememba gena, potrebnega za encim, ki pomaga proizvajati nevrotransmiterje, kemične prenašalce v telesu. 它是由一种酶所需的基因发生变化引起的,这种酶有助于制造神经递质(人体的化学信使)。 The one-time treatment provides a working version of the gene. يوفر العلاج لمرة واحدة نسخة عمل من الجين. O tratamento único fornece uma versão funcional do gene. Z enkratnim zdravljenjem se zagotovi delujoča različica gena. 一次性治疗提供了该基因的工作版本。

At around 3 months old, Rylae-Ann began having what her parents thought were seizures. في عمر 3 أشهر تقريبًا ، بدأت Rylae-Ann تعاني مما اعتقد والديها أنه نوبات صرع. Por volta dos 3 meses de idade, Rylae-Ann começou a ter o que seus pais pensavam ser convulsões. Pri približno treh mesecih je Rylae-Ann začela doživljati napade, za katere so njeni starši mislili, da so napadi. 大约 3 个月大时,Rylae-Ann 开始出现癫痫发作,她的父母认为是癫痫发作。 Fluid sometimes got into her lungs after she ate, a condition that required hospital treatment. كان السائل يدخل أحيانًا إلى رئتيها بعد أن أكلت ، وهي حالة تتطلب علاجًا في المستشفى. O líquido por vezes entrava nos pulmões depois de comer, uma condição que exigia tratamento hospitalar. Včasih ji je po jedi v pljuča prišla tekočina, kar je zahtevalo bolnišnično zdravljenje. 有时,进食后液体会进入肺部,这种情况需要住院治疗。 Doctors thought she might have epilepsy or cerebral palsy, more common brain diseases. يعتقد الأطباء أنها قد تكون مصابة بالصرع أو الشلل الدماغي ، وهي أمراض دماغية أكثر شيوعًا. Os médicos pensaram que ela poderia ter epilepsia ou paralisia cerebral, doenças cerebrais mais comuns. Zdravniki so menili, da ima morda epilepsijo ali cerebralno paralizo, ki sta pogostejši možganski bolezni. 医生认为她可能患有癫痫或脑瘫,这是更常见的脑部疾病。

Later, her parents found a doctor who correctly identified Rylae-Ann's condition. في وقت لاحق ، وجد والداها طبيبًا حدد بشكل صحيح حالة Rylae-Ann. Mais tarde, os seus pais encontraram um médico que identificou correctamente o estado de Rylae-Ann. Kasneje so njeni starši našli zdravnika, ki je pravilno ugotovil Rylae-Annino stanje. Her parents entered her into a gene therapy trial in Taiwan. Seus pais a inscreveram em um teste de terapia genética em Taiwan. Njeni starši so jo prijavili na poskus genskega zdravljenja na Tajvanu. 她的父母让她参加台湾的基因治疗试验。 The treatment requires doctors to operate directly on the brain. O tratamento requer que os médicos operem directamente sobre o cérebro. Zdravljenje zahteva, da zdravniki operirajo neposredno na možganih. 这种治疗需要医生直接对大脑进行手术。 The doctors injected the genetic material using an extremely narrow tube. Os médicos injetaram o material genético usando um tubo extremamente estreito. Zdravniki so genski material vbrizgali z izjemno ozko cevko. 医生使用一根极窄的管子注射遗传物质。

“It gets put into the brain cells and then the brain cells make the (neurotransmitter) dopamine,” said Stuart Peltz, leader of PTC Therapeutics. "É colocado nas células cerebrais e depois as células cerebrais fazem a dopamina (neurotransmissor)", disse Stuart Peltz, líder da PTC Therapeutics. "Vnese se v možganske celice in te nato proizvajajo (nevrotransmiter) dopamin," je povedal Stuart Peltz, vodja družbe PTC Therapeutics. PTC Therapeutics 的负责人斯图尔特·佩尔茨 (Stuart Peltz) 表示:“它被放入脑细胞中,然后脑细胞会产生(神经递质)多巴胺。”

Company officials said all patients in their trials showed movement and thinking improvements. Funcionários da empresa disseram que todos os pacientes em seus testes mostraram melhorias no movimento e no pensamento. Predstavniki podjetja so povedali, da so se pri vseh bolnikih v njihovih preskušanjih izboljšale gibalne sposobnosti in mišljenje. 公司官员表示,所有试验中的患者都表现出了运动和思维的改善。 Some of them, Peltz said, could stand and walk and continue getting better over time. Alguns deles, disse Peltz, podiam ficar de pé e andar e continuar melhorando com o tempo. Peltz pravi, da nekateri od njih lahko stojijo in hodijo ter se sčasoma izboljšujejo.

Bankiewicz of Ohio State University also reported success in his NIH-financed study. Bankiewicz da Universidade Estatal de Ohio também relatou sucesso no seu estudo financiado pelo NIH. Tudi Bankiewicz z državne univerze v Ohiu je v svoji študiji, ki jo je financiral NIH, poročal o uspehu. 俄亥俄州立大学的 Bankiewicz 也报告了他的 NIH 资助的研究取得了成功。 He said all 40 or so patients involved saw big improvements. Ele disse que todos os cerca de 40 pacientes envolvidos viram grandes melhorias. Povedal je, da so pri vseh približno 40 bolnikih, ki so bili vključeni v zdravljenje, opazili velike izboljšave. 他说所有 40 名左右的患者都看到了很大的改善。

Scientists say there are difficulties to face before this treatment becomes common for more brain diseases. Os cientistas dizem que há dificuldades a enfrentar antes que esse tratamento se torne comum para mais doenças cerebrais. Znanstveniki pravijo, da se je treba soočiti s težavami, preden bo to zdravljenje postalo običajno za več možganskih bolezni. 科学家表示,在这种治疗方法广泛应用于更多脑部疾病之前,还需要面对一些困难。

For example, the timing of treatment is an issue. Por exemplo, o momento do tratamento é um problema. Vprašanje je na primer čas zdravljenja. Generally, earlier in life is better because diseases can cause more problems over the years. Geralmente, mais cedo na vida é melhor porque as doenças podem causar mais problemas ao longo dos anos. Na splošno je bolje, da se zdravite prej, saj lahko bolezni z leti povzročijo več težav. 一般来说,越早越好,因为随着时间的推移,疾病会导致更多的问题。 Also, more complex disorders like Alzheimer's are harder to treat with gene therapy. Prav tako je z genskim zdravljenjem težje zdraviti bolj zapletene bolezni, kot je Alzheimerjeva bolezen. 此外,像阿尔茨海默病这样更复杂的疾病更难用基因疗法治疗。

A more basic barrier is cost. Uma barreira mais básica é o custo. Osnovna ovira so stroški. 一个更基本的障碍是成本。 The price of gene therapies, paid for mostly by insurers and governments, can be in the millions. Cena genskih terapij, ki jih večinoma plačujejo zavarovalnice in vlade, je lahko milijonska. The one-time PTC therapy costs more than $3 million in Europe, for example. A terapia única de PTC custa mais de 3 milhões de dólares na Europa, por exemplo. V Evropi na primer enkratna terapija PTC stane več kot 3 milijone dolarjev.

But drugmakers say they want people to get the treatments they need. Mas os fabricantes de medicamentos dizem que querem que as pessoas recebam os tratamentos de que necessitam. Proizvajalci zdravil pa pravijo, da želijo, da ljudje dobijo zdravljenje, ki ga potrebujejo. And researchers are hopeful they can clear the remaining scientific barriers to this method. E os pesquisadores esperam poder eliminar as barreiras científicas remanescentes a esse método. Raziskovalci upajo, da jim bo uspelo odpraviti preostale znanstvene ovire za to metodo. 研究人员希望他们能够清除这种方法剩余的科学障碍。

“So I would say gene therapy can be leveraged for many sorts of brain diseases and disorders,” said Ryan Gilbert. "Zato bi rekel, da je gensko zdravljenje mogoče uporabiti za številne vrste možganskih bolezni in motenj," je dejal Ryan Gilbert. “所以我想说基因疗法可以用于治疗多种脑部疾病和紊乱,”瑞安吉尔伯特说。 He is a biomedical engineer at New York's Rensselaer Polytechnic Institute. É engenheiro biomédico no Rensselaer Polytechnic Institute de Nova Iorque. Je biomedicinski inženir na newyorškem politehničnem inštitutu Rensselaer. "In the future, you're going to see more technology doing these kinds of things,” he added. "No futuro, verás mais tecnologia a fazer este tipo de coisas", acrescentou ele. "V prihodnosti bomo videli več tehnologije, ki bo opravljala tovrstne stvari," je dodal.

I'm Dan Novak.