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抗肿瘤药物尼罗替尼可能会逆转帕金森氏症

2015-10-24 MedSci MedSci原创

一种昂贵的抗癌药或能逆转晚期帕金森氏症,使一项小型临床试验的参与者多年来首次开口说话和重新行走。虽然对于帕金森氏症有不少治疗方法,但如果被证实,这将是一种药物首次对帕金森氏症的病因起作用。“我们看到,处于帕金森氏症晚期的病人恢复了健康。”来自美国华盛顿乔治敦大学医学中心的Charbel Moussa主导了此项临床试验。Moussa介绍说,这种名为尼罗替尼的药物,通过增强大脑自身的“垃圾处理系统”以

一种昂贵的抗癌药或能逆转晚期帕金森氏症,使一项小型临床试验的参与者多年来首次开口说话和重新行走。虽然对于帕金森氏症有不少治疗方法,但如果被证实,这将是一种药物首次对帕金森氏症的病因起作用。


“我们看到,处于帕金森氏症晚期的病人恢复了健康。”来自美国华盛顿乔治敦大学医学中心的Charbel Moussa主导了此项临床试验。

Moussa介绍说,这种名为尼罗替尼的药物,通过增强大脑自身的自噬系统以清除在帕金森氏症患者脑中累积的蛋白而发挥作用。这些蛋白被认为触发了使分子像多巴胺一样的大脑细胞的死亡,而多巴胺是运动和其他功能所必需的。

尼罗替尼已被批准用于治疗癌症。它会阻挡一种驱动慢性粒细胞白血病发展的蛋白,并且阻断破坏溶酶体的另一种蛋白。溶酶体是一种摧毁有害蛋白的细胞结构。Moussa认为,尼罗替尼能释放出更多溶酶体,使后者在清除同帕金森氏症相关的蛋白上做得更好。

在动物身上开展的试验显示出良好的前景,因此Moussa和团队成员招募了12名志愿者进行小型临床试验。这些志愿者患有帕金森氏症,或被称为路易体痴呆的类似症状。

尽管所有志愿者在试验开始时都处于晚期阶段,而且其中3人无法讲话,但在开始服用尼罗替尼时,所有人的症状开始改善。

“这看上去太过美好,以至于令人难以相信。我希望是自己错了。”来自英国桑德韦尔和西伯明翰医院的Carl Clarke表示。

“如果它真的能逆转帕金森氏症,我们将进入一个重大的转折点。但我对此持怀疑态度。”来自伦敦国王学院的Kallol Ray说,“我会说,‘等着听下面发表的消息吧’。”

小知识:

尼洛替尼(Nilotinib) 该药由诺华公司开发,是一种BCR ABL酪氨酸激酶抑制剂。该药被FDA授予“孤儿药”称号,并于2007年10月获批上市,用于治疗新诊断的慢性期、Ph+的成人CML患者,以及慢性期和加速期、Ph+且对包括伊马替尼在内的既往治疗耐药或不耐受的成人CML患者。该药2009年7月由诺华公司在我国进口上市,商品名达希纳。

原始出处:

Hebron ML, Lonskaya I, Olopade P, Selby ST, Pagan F, Moussa CE. Tyrosine Kinase Inhibition Regulates Early Systemic Immune Changes and Modulates the Neuroimmune Response in α-Synucleinopathy.J Clin Cell Immunol. 2014 Sep 30;5:259

Lonskaya I, Hebron ML, Desforges NM, Schachter JB, Moussa CE. Nilotinib-induced autophagic changes increase endogenous parkin level and ubiquitination, leading to amyloid clearance. J Mol Med (Berl). 2014 Apr;92(4):373-86.

Wenqiang C, Lonskaya I, Hebron ML, Ibrahim Z, Olszewski RT, Neale JH, Moussa CE. Parkin-mediated reduction of nuclear and soluble TDP-43 reverses behavioral decline in symptomatic mice. Hum Mol Genet. 2014 Sep 15;23(18):4960-9.

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attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=aa5e18156549, createdName=zhu_jun9843, createdTime=Mon Oct 26 13:13:00 CST 2015, time=2015-10-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1446754, encodeId=ff631446e54dc, content=<a href='/topic/show?id=94bd830865e' target=_blank style='color:#2F92EE;'>#肿瘤药物#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=70, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=83086, encryptionId=94bd830865e, topicName=肿瘤药物)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/PiajxSqBRaEKneTxOtx0KRAPibib3sfmib4P2269UG6op6ZN5ZKddPvUibR7RfFob8X6FicRugoeY8Dicy4cibArjj168Q/132, createdBy=c17d5287383, createdName=smartjoy, createdTime=Mon Oct 26 13:13:00 CST 2015, time=2015-10-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=41288, encodeId=9af14128819, content=难道自噬的真的这么强大?, beContent=null, 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    2017-06-22 海棠胜雪

    学习了

    0

  2. 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    2017-04-04 jin321

    这么神奇吗

    0

  3. 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  4. 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    2015-11-26 老段

    希望不大

    0

  5. 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  6. 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  7. 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  8. 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objectType=article, channel=null, level=null, likeNumber=138, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8c171373773, createdName=gzhjjx, createdTime=Mon Oct 26 08:22:00 CST 2015, time=2015-10-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=41270, encodeId=011e412e050, content=自噬在帕金森作用巨大啊!, beContent=null, objectType=article, channel=null, level=null, likeNumber=114, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=387c1637569, createdName=liusanxin, createdTime=Mon Oct 26 00:29:00 CST 2015, time=2015-10-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=41124, encodeId=c6634112457, content=喔, beContent=null, objectType=article, channel=null, level=null, likeNumber=150, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://cacheapi.medsci.cn/resource/upload/20150904/IMG55E8DB68F05883098.jpg, createdBy=e00a1631985, createdName=fengzhigu, createdTime=Sun Oct 25 17:16:00 CST 2015, time=2015-10-25, status=1, ipAttribution=)]
    2015-10-26 gzhjjx

    难道自噬的真的这么强大?

    0

  9. 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20:48:00 CST 2015, time=2015-11-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1537672, encodeId=57a4153e67279, content=<a href='/topic/show?id=1f9983082fa' target=_blank style='color:#2F92EE;'>#肿瘤药#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=71, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=83082, encryptionId=1f9983082fa, topicName=肿瘤药)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ed1012894470, createdName=30397608, createdTime=Mon Oct 26 13:13:00 CST 2015, time=2015-10-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1593670, encodeId=b68315936e0a5, content=<a href='/topic/show?id=ff895565586' target=_blank style='color:#2F92EE;'>#抗肿瘤药#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=51, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=55655, encryptionId=ff895565586, topicName=抗肿瘤药)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=aa5e18156549, createdName=zhu_jun9843, createdTime=Mon Oct 26 13:13:00 CST 2015, time=2015-10-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1446754, encodeId=ff631446e54dc, content=<a href='/topic/show?id=94bd830865e' target=_blank style='color:#2F92EE;'>#肿瘤药物#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=70, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=83086, encryptionId=94bd830865e, topicName=肿瘤药物)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/PiajxSqBRaEKneTxOtx0KRAPibib3sfmib4P2269UG6op6ZN5ZKddPvUibR7RfFob8X6FicRugoeY8Dicy4cibArjj168Q/132, createdBy=c17d5287383, createdName=smartjoy, createdTime=Mon Oct 26 13:13:00 CST 2015, time=2015-10-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=41288, encodeId=9af14128819, content=难道自噬的真的这么强大?, beContent=null, objectType=article, channel=null, level=null, likeNumber=138, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8c171373773, createdName=gzhjjx, createdTime=Mon Oct 26 08:22:00 CST 2015, time=2015-10-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=41270, encodeId=011e412e050, content=自噬在帕金森作用巨大啊!, beContent=null, objectType=article, channel=null, level=null, likeNumber=114, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=387c1637569, createdName=liusanxin, createdTime=Mon Oct 26 00:29:00 CST 2015, time=2015-10-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=41124, encodeId=c6634112457, content=喔, beContent=null, objectType=article, channel=null, level=null, likeNumber=150, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://cacheapi.medsci.cn/resource/upload/20150904/IMG55E8DB68F05883098.jpg, createdBy=e00a1631985, createdName=fengzhigu, createdTime=Sun Oct 25 17:16:00 CST 2015, time=2015-10-25, status=1, ipAttribution=)]
    2015-10-26 liusanxin

    自噬在帕金森作用巨大啊!

    0

  10. 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createdAvatar=http://cacheapi.medsci.cn/resource/upload/20150904/IMG55E8DB68F05883098.jpg, createdBy=e00a1631985, createdName=fengzhigu, createdTime=Sun Oct 25 17:16:00 CST 2015, time=2015-10-25, status=1, ipAttribution=)]
    2015-10-25 fengzhigu

    0

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