干细胞研究人员对未经证实的干细胞疗法感到担忧（附原文）

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5 h( w# N% [3 w1 g% L1 Z- w干细胞研究人员对未经证实的干细胞疗法感到担忧
6 X! g5 I3 ~9 g) a+ unaturalkillerce注：意大利议会最近更改一项法规以便允许公共卫生体系使用未经验证的干细胞疗法。这在欧洲乃至全世界引发激烈的争论。
  ^2 w+ w  E& ?0 F* p% P一个国际的顶尖干细胞研究人员小组发布一项声明：它详细地说明了对未经证实的干细胞疗法的开发和使用感到担忧。这项评论性声明发表在EMBO J期刊上。该研究小组主张对干细胞疗法进行严格的临床验证和监管是为病人引入安全的有效的医学介入所必需的。/ i, E6 s6 A: v6 ^& b, p8 \
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Researchers Outline Concerns About Unproven Stem Cell Therapies. J3 q  U. f0 ]8 ^3 s$ U
"Stem cells may offer unprecedented opportunities to develop treatments for many diseases with unmet medical needs. This will take time. However, only rigorous science and responsible regulation can ensure the safe and effective translation of science into effective therapies," remarked Paolo Bianco, Pathologist, Stem Cell Biologist, Professor of Pathology at the University of Roma "La Sapienza" and one of the 13 authors of the commentary who come from Italy, Germany, the United Kingdom, The Netherlands and the United States.: V- D! h9 A+ }
The concerns of the scientific community have been heightened by pending legislative action that may allow routine administration of unproven stem cell therapies to patients in Italy. Despite a lack of rigorous clinical trials to test safety or efficacy, a ban of the treatment by health authorities, and a lack of peer review by the scientific community, the privately funded Stamina Foundation has been using cultured mesenchymal stem cells that have been exposed to putative conditions that favour neuronal differentiation to treat different diseases in severely or terminally ill patients.! j; }* ~( o- a. g: B3 Z) v$ c
The Italian Chamber decides shortly whether they will proceed with controversial legislation passed in the Senate on 21 March that allows the unproven stem cell treatment developed by the Stamina Foundation to be used for severely or terminally ill new patients for 18 months. "The adoption of this law may set a dangerous precedent for patients looking to be treated with other unproven stem cell therapies in Europe and other countries," remarked Hans Clevers, Professor of Molecular Genetics and President of the Royal Netherlands Academy of Arts and Sciences.
- R1 K  G) z+ v) z; Z! b"Irrational and unverified stem cell treatments based on methods that are not validated or scientifically documented should not reach patients. Preventing this from happening is a specific responsibility of health authorities and governments worldwide to make sure that the hope and trust of patients are not misused," remarked Elena Cattaneo, Director of the Centre for Stem Cell Research at the University of Milan, Italy, and one of the scientists who contributed to the commentary. "Patients can be harmed and killed by medicines that have not been proven to be safe and effective via rigorously controlled clinical trials. The use of medicines that have not been manufactured to the highest possible standards is irresponsible."
% W: @. i. @: q2 J* k8 H/ w3 BPreclinical and clinical tests have been used successfully in the past for the introduction of therapies for bone marrow transplantation and the regeneration of skin and cornea in patients. The authors of the commentary emphasize that cell therapies must be approved by international and national regulatory agencies and remain under the strict vigilance of health authorities. Regulations already in place in the European Union insist that stem cell therapies follow the same safety and efficacy rules as pharmaceuticals. They need to be prepared and manufactured in highly controlled environments with precise protocols, traceability and accountability.% G) K( A8 k; ]. ~* H
"It is disconcerting that the Italian Senate has passed amendments that permit the use of unproven stem cell therapies without proper vigilance or proper experiment, reclassifying them as transplants," commented Bianco. "Infusions of mesenchymal stem cells are not transplants in any way. In Europe and the United States, all kinds of cell preparations that are administered to patients following ex vivo culturing are classified as medicines, and monitored by drug agencies such as the US Food and Drug Administration, the European Medicines Agency and, in Italy, the Agenzia Italiana del Farmaco or AIFA. A host of proper and improper commercial interests might benefit from these new rules that abrogate both safety and proper ways of experimentation. Patients may be harmed," concluded Bianco.
" h6 Y7 v: n# J' U. R) dSean Morrison, Professor and Director of the Children's Medical Center Research Institute at UT Southwestern Medical Center in the United States, who is not an author on the paper, added: "Patients are ultimately not helped by therapies that are not based on sound science and that are not tested in systematic clinical trials. Efforts to water down regulation in this area may create opportunities for some individuals to prey on the hopes of desperate patients."8 R4 }: i9 E' E

4 {) w5 U5 l& r& M0 @7 L0 u& kRegulation of stem cell therapies under attack in Europe: For whom the bell tolls. The EMBO Journal, 2013 DOI: 10.1038/emboj.2013.114& d8 @5 D* m9 u
http://www.nature.com/emboj/jour ... /emboj2013114a.html

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医学新希望还是医学炼金术4 @& o7 N) C# D6 U9 e

% t+ B# l4 R# Z! s2013年05月04日 09:50:43 $ f+ t. _  i7 |- e  D' l
来源： 光明网 4 E; y2 C2 y' i$ u+ Z

- q9 J) e7 Q$ t# t1 q- ]3月21日，意大利卫生部部长雷纳多・巴尔杜奇（Renato Balduzzi）颁布了一项新法令，批准对约30名已经开始接受干细胞治疗的儿童继续采用这一疗法。有评论认为，这一新法令等于间接承认了意大利干细胞临床治疗的合法性，并为公共医疗机构采用这一疗法提供了法律保障。新法令明确了干细胞治疗的具体实施细则，同时强调干细胞疗法在临床应用过程中具有不可重复性，需要根据病人的具体情况进行有针对性的个案治疗。法令还规定，在采取干细胞疗法进行临床治疗前，需要由主治医师提出申请，在经过医疗、伦理委员会同意后，方可进行操作。法令还特别规定，对于罕见疾病的治疗可以使用干细胞疗法，但需要对治疗效果进行跟踪监测。虽然条件严苛，但这一新法令仍然引发了医学界的广泛争议，世界著名的科学期刊《自然》杂志也就此发表文章，反对在干细胞研究尚未成熟阶段就将其临床应用合法化。
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$ a8 P& |. J, A9 Y: @4 V2 ?干细胞是一类具有自我复制能力的多潜能细胞。在一定条件下，它可以分化成多种功能细胞。干细胞的这一特性使其在医学领域内的应用潜能十分巨大，为许多目前尚不能有效治愈的疾病提供了新的可能和希望。人类如能完全掌握干细胞的秘密并加以合理利用，将会产生一种全新的医疗技术：通过再造人体正常的组织器官，使人体能够用上自己的或他人的干细胞或由干细胞所衍生出的新的组织器官，替换自身病变的或衰老的组织器官。在世界范围内，利用人体干细胞治疗罕见疾病的方法仍处于起步阶段，医学界对此也一直持有谨慎怀疑的态度。因此，巴尔杜奇的这一决定在意大利医学界，甚至在世界医学界引起不小争议也就不足为奇了。 + E) U" W, p$ v! x) L6 ~4 l+ T
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许多申请尝试这一新疗法的患者表示，自己所患疾病已经没有其他可能的治疗方法，迫不得已才决定尝试尚未成熟的干细胞疗法。意大利同意对约30位儿童进行这一治疗也是因为孩子们各自的病情已经不具备其他治愈的可能性，是基于“同情”的治疗。有上述接受治疗的儿童家长在接受意大利媒体采访时表示，虽然对于这一治疗的效果并不具有十足的把握，但为了孩子的生命，他们愿意冒险一试。然而，在2012年5月，意大利医药协会曾明确表示不应向患者采取“任何尚未证实的医疗手段，诸如干细胞治疗等”。
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《自然》杂志在文章中指出，“在世界范围内，确实有一些医疗机构向患者提供尚未验证效果的干细胞疗法，但一般情况下，这些医疗机构都是在私下里进行此类治疗。然而在意大利，类似的治疗手段却得到了政府的官方批准。”《自然》杂志认为，意大利官方允许对约30名患病儿童继续已经开始的干细胞疗法令科学家们“感到恐惧”。文章回顾了目前将干细胞疗法应用于临床治疗的混乱现状，认为巴尔杜奇颁布的法令向公共医疗机构打开了一扇合法应用干细胞进行临床治疗的大门，但却有许多安全隐患隐藏在大门背后。
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在米兰大学干细胞实验室主任埃莱娜・卡塔内奥（Elena Cattaneo）看来，干细胞疗法在目前的研究阶段就像是医学界的“炼金术”。罗马大学生化学家、林琴学院院士毛里奇奥・布鲁诺利（Maurizio Brunori）则评论认为，“我还从没听说过一位卫生部部长的决定竟然与医药协会的规定背道而驰”。巴尔杜奇随后强调这一法令并不意味着承认干细胞治疗一定会产生积极的效果。《自然》杂志援引帕维亚大学一位教授的话：“干细胞疗法从未被证实对于患者具有积极效果。因此，基于同情的治疗也不能印证其合法性。”《自然》杂志还介绍了一个于2012年12月发表在《神经肌肉疾病》学术期刊上的一次利用干细胞疗法进行治疗的临床记录。该记录分析了2010年12月至2011年12月间针对5位3个月到20个月大的患有脊髓性肌肉萎缩症的儿童采用干细胞治疗的过程。在经过治疗后，5位儿童中的2位不幸死亡，剩下的3位儿童也没有任何好转的迹象。
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在科学技术日益发达的今天，新的医疗技术手段和方法无疑会为成千上万的患者带来生活的新希望，我们也坚信会有越来越多的曾经无法医治的疾病最终会被科学家们攻克。但无论哪种新的医疗技术和手段的临床应用前提，都必须以缜密的科学分析和大量的科学实验为基础，将未经确认的治疗手段盲目地加以临床应用，都是对患者，甚至是对生命的不负责任。急功近利的心态如果在科学界，尤其在医学界蔓延，带来的后果是不堪设想的，也是无法弥补的。白衣天使这一神圣的职业，不能因为急于寻求一时的“突破”而违背了基本的职业操守。因为，人的生命无可争议地高于一切。(本报驻罗马记者陈晓晨) 3 M; F2 k, `& B