在2016年，大量的ALS研究發(fā)現，臨床試驗，合作和戰略舉措的進(jìn)展都加快了發(fā)現治療和治愈ALS的速度。
我們總結了在A(yíng)LS研究中的10個(gè)最大進(jìn)展，這些進(jìn)展令到ALS病人在新的一年里充滿(mǎn)希望！
1。
所述的發(fā)現NEK-1基因，現在已知是有助於ALS的發(fā)展中最常見(jiàn)的基因中，取得世界各地的頭條新聞。80多名研究人員在11個(gè)國家進(jìn)行了這項規模最大的一次繼承ALS研究- 閱讀更多關(guān)於冰桶挑戰捐款如何幫助ALS協(xié)會(huì )投資於項目的MinE進(jìn)行這項重要的工作。
2。
神經(jīng)臨床研究所（NCRI）成像小組在麻省總醫院，由Nazem Atassi博士領(lǐng)導的，用於PET成像成功掃描與ALS生活的第一人測量大腦，在這種成像生物標記物研究有前途的第一步炎癥。從那時(shí)起，更多的人參加了這項研究。
3。
美國食品和藥物管理局（FDA）的認可，目前正在審查中，新藥申請（NDA）為依達拉奉 用於ALS治療。我們迫切地期待在2017年的FDA的消息！
4。
Aaron Gitler博士和他的研究者同事，由ALS協(xié)會(huì )支持，確定了C9orf72相關(guān)ALS的新治療靶點(diǎn)。我們採訪(fǎng)了Gitler博士在我們對治療潛力來(lái)治療ALS博客。
5。
在雪松-西奈調查獲得批準，從FDA測試相結合的幹細胞的基因治療的安全性在臨床試驗中。ALS協(xié)會(huì )先前認證Cedars-Sinai作為卓越治療中心，通過(guò)其全面，協(xié)作的方法來(lái)滿(mǎn)足協(xié)會(huì )的嚴格標準，提供患者護理和服務(wù)。
6。
頭腦風(fēng)暴宣布為NurOwn幹細胞的II期試驗陽(yáng)性結果在美國公佈再宣布更大的III期臨床試驗於2017年開(kāi)始。
7。
IBM的沃森超級計算機發(fā)現了5個(gè)新的ALS基因。IBM與Phoenix的Barrow神經(jīng)學(xué)研究所的合作展示了大數據的力量以及先進(jìn)計算加快治療進(jìn)程和治療的潛力。
8。
一個(gè)全國的最大的精密醫療課程開(kāi)始招收10月，在美國各地的大學(xué)和醫院9中心。ALS協(xié)會(huì )承諾350萬(wàn)美元的冰桶挑戰 - 籌集資金這個(gè)令人興奮的合作努力。
9。
在邁阿密ALS研究的領(lǐng)先大學(xué)國際ALS / MND研討會(huì )在都柏林，與製藥公司宣布Orphazyme的圓滿(mǎn)完成在SOD1 ALS arimoclomol的II期臨床試驗。
10。
全球合作的公告ALS ONE和NeuroLINCS，數以百萬(wàn)計來(lái)自ALS協(xié)會(huì )的資金支持。這兩個(gè)舉措將有助於產(chǎn)生研究人員需要繼續其重要工作的數據。
This Year’s 10 Biggest Advances in ALS Research
by The ALS AssociationJanuary 1, 2017Leave a comment
In 2016, a significant number of ALS research discoveries, advances in clinical trials, collaborations and strategic initiatives all accelerated the pace of discovery in finding treatments and a cure for ALS.
We’ve pulled together what we think are 10 of 2016’s biggest advances in ALS research that gave us, and people living with ALS, hope this year!
1.
The discovery of the NEK-1 gene, now known to be among the most common genes that contribute to the development of ALS, made headlines around the globe. More than 80 researchers in 11 countries conducted this largest-ever study of inherited ALS – read more about how Ice Bucket Challenge donations helped The ALS Association invest in this important work conducted by Project MinE.
2.
The Neurological Clinical Research Institute (NCRI) imaging team at MGH, led by Dr. Nazem Atassi, used PET imaging to successfully scan the first person living with ALS to measure inflammation in the brain, a promising first step in this imaging biomarker study. Since then, many more people have participated in the study.
3.
The U.S. Food and Drug Administration (FDA) accepted, and is currently reviewing, a New Drug Application (NDA) for Edaravone for the treatment of ALS. We’re eagerly anticipating news from the FDA in 2017!
4.
Dr. Aaron Gitler and his researcher colleagues, supported by The ALS Association, identified a new therapeutic target for C9orf72-associated ALS. We interviewed Dr. Gitler on our blog about the therapeutic potential to treat ALS.
5.
Investigators at Cedars-Sinai gained approval from the FDA to test the safety of a combination stem cell-gene therapy in a clinical trial. The ALS Association previously certified Cedars-Sinai as a Treatment Center of Excellence, meeting the Association’s rigorous standards with their comprehensive, collaborative approach to patient care and services.
6.
Brainstorm announces positive results for NurOwn stem cell phase II trial reported in the U.S. and then announces larger phase III trial to begin in 2017.
7.
IBM’s Watson supercomputer discovers 5 new ALS genes. IBM’s collaboration with the Barrow Neurological Institute in Phoenix shows the power of Big Data and the potential for advanced computing to speed up progress toward treatment and a cure.
8.
One of the nation’s largest precision medicine programs began enrolling in October, with 9 centers at universities and hospitals across the United States. The ALS Association committed $3.5 million in Ice Bucket Challenge-raised money to this exciting collaborative effort.
9.
At the International ALS/MND Symposium in Dublin, a leading University of Miami ALS researcher and the pharmaceutical company Orphazyme announced the successful completion of phase II trial of arimoclomol in SOD1 ALS.
10.
Announcements of global collaborations, ALS ONE and NeuroLINCS, supported by millions in funding from The ALS Association. These two initiatives will help generate the data researchers need to continue their important work.
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