d****f
发帖数: 313 | 1 6park上转来的:
http://site.6park.com/chan2/index.php?app=forum&act=threadview&
说实话我真是希望是真的,不过我目前所受的教育似乎尚不允许我相信之:
特大號外
美國公司宣佈人類已經攻克癌症
合成的雙鏈RNA進入肝細胞後，人體內的RNAi機制便會摧毀合成的RNA和任何與之匹配的
、與腫瘤生長相關的信使RNA，阻止蛋白質的繼續產生，從而使腫瘤停止生長....
據美國媒體報導，美國阿爾尼拉姆生物技術公司日前宣佈他們找到了一種能夠治癒所有
癌症的新型藥物，首批接受臨床試驗的19名晚期肝癌患者病情都有較大好轉。不僅如此
，該公司稱，假以時日，這種藥物甚至有可能治癒一切疾病。
首批患者反應良好
今年4月，19名接受化療但沒有好轉的肝癌病人開始服用這種名為ALN-VSP的新型藥物。
服用第一劑後的數周內，藥物就已經很明顯地開始阻止腫瘤產生自身生長需要的蛋白質。
到今年6月，阿爾尼拉姆公司稱，通過“喚醒”人體自身的一種很少使用的免疫防禦
系統，ALN-VSP成功切斷肝癌患者體內腫瘤62%的血流量。在治療肝癌時，傳統藥物一般
使用消除致病蛋白質的方法，而ALN-VSP則通過核糖核酸干擾(RNAi)療法直接阻止細胞
生成致病蛋白質。
喚醒人體自身防禦機制
科學家在研究中還發現核糖核酸(RNA)和去氧核糖核酸(DNA)之間一個奇妙的聯繫———
如果說DNA對蛋白質來說是一張圖紙，那麼RNA就是能夠下達指令的建築商。RNA把DNA上
的基因複製成單鏈的信使RNA，再由它向細胞傳遞資訊繼而產生蛋白質。
1998年，科學家發現了核糖核酸干擾(RNAi)機制，原始生物就利用這個系統來甄別
和摧毀病毒雙鏈RNA和病毒信使RNA。研究人員發現，將一小段雙鏈RNA引入細胞即能觸
發這一埋藏在人體內的古老機制，使RNAi再次發揮停止生產特定蛋白質的功效。從這一
角度看，可以說RNAi具有治癒包括癌症在內的許多疾病的能力，這些疾病的特點一般都
是由病變細胞產生過量的常見蛋白質所致。從理論上說，操控RNAi來殺死蛋白質並不難
。比方說，ALN-VSP內就含有合成的雙鏈RNA，它與肝臟腫瘤用於編碼兩種蛋白質的信使
RNA相匹配，那兩種蛋白質分別是促進腫瘤血管生長的血管內皮生長因數(VEGF)和加速
腫瘤細胞快速分裂的紡錘體驅動蛋白(KSP)。
合成的雙鏈RNA進入肝細胞後，人體內的RNAi機制便會摧毀合成的RNA和任何與之匹配的
、與腫瘤生長相關的信使RNA，阻止蛋白質的繼續產生，從而使腫瘤停止生長。
有望“包治百病”
除了在癌症領域的應用，這項能攻擊單個基因的技術還在其它醫學領域掀起一陣
RNAi療法旋風。目前，阿爾尼拉姆公司已經將這種療法用於亨廷頓氏舞蹈症、視網膜黃
斑變性、肌肉萎縮和愛滋病等疾病的研究。
核磁共振掃描顯示，使用ALN-VSP療法後，肝臟腫瘤中的血流量明顯減少
加利福尼亞州知名分子遺傳學家約翰・羅西稱，RNAi療法有望在兩年內成熟。由
於首批試驗效果相當好，ALN-VSP有望成為首批基於RNAi理論而推向市場的藥物。羅西
表示：“我認為RNAi療法對所有的病都有效。” | t****r
发帖数: 702 | 2 http://phx.corporate-ir.net/phoenix.zhtml?c=148005&p=irol-newsA
9293&highlight=
没那么夸张，只是phase I study 有效果而已。。。
Alnylam and Collaborators Publish Results from Phase I Clinical Trial and Ex
tension Study with ALN-VSP, an RNAi Therapeutic for the Treatment of Liver C
ancer
- Results Published in Cancer Discovery Document Most Comprehensive Human Ex
perience To Date for RNAi Therapeutics Delivered with Lipid Nanoparticles (L
NPs) -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jan. 30, 2013-- Alnylam Pharmaceuticals,
Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, and collaborators
announced today the publication of complete study results from a Phase I tri
al with ALN-VSP, a systemically delivered RNAi therapeutic for the treatment
of advanced solid tumors with liver involvement. The paper, titled 揊irst-i
n-Man Trial of an RNA Interference Therapeutic Targeting VEGF and KSP in Can
cer Patients with Liver Involvementappears as an OnlineFirst publication i
n the journal Cancer Discovery (Tabernero et al., Cancer Discovery CD-12-042
9; Published OnlineFirst January 2013). The study results document anti-tumo
r activity for ALN-VSP in a heavily pre-treated and advanced patient populat
ion, including a complete response in an endometrial cancer patient who had
multiple hepatic metastases. In addition, this study provided proof of RNAi
mechanism in man based on molecular analysis of biopsy samples from patients
. Finally, in this study the most comprehensive study of a systemically ad
ministered RNAi therapeutic to date chronic dosing of ALN-VSP for up to 26
months was found to be generally safe and well tolerated.
揙ur ALN-VSP Phase I clinical trial defines the most comprehensive human exp
erience for RNAi therapeutics delivered with lipid nanoparticle formulations
. Results from this study highlight safety and tolerability of multiple dose
s of ALN-VSP, proof of RNAi activity in man, and evidence for anti-tumor act
ivity in a very advanced, heavily pre-treated cancer patient population,sa
id Jared Gollob, M.D., Vice President, Clinical Research at Alnylam. 揥e are
encouraged by the anti-tumor activity observed in this study in multiple pa
tients who achieved stable disease or better; this includes a patient with e
ndometrial cancer metastatic to the liver who achieved a complete response.
Results from the extension study also give us increased confidence in long-t
erm chronic dosing for RNAi therapeutics delivered with lipid nanoparticle f
ormulations, as patients received an average of over 11 months of treatment
overall, including one patient who received treatment for over two full year
s.br />
ALN-VSP is a systemically delivered RNAi therapeutic using first-generation
lipid nanoparticle (LNP) or 揝NALPdelivery technology that comprises two s
iRNAs targeting two genes critical for the growth and development of cancer
cells: vascular endothelial growth factor (VEGF) and kinesin spindle protein
(KSP). The ALN-VSP Phase I trial was designed as a multi-center, open-label
, dose-escalation study in patients with advanced solid tumors with liver in
volvement who failed to respond to or had progressed after standard treatmen
t. A total of 41 patients were enrolled. The primary objective was to evalua
te the safety, tolerability, and pharmacokinetics of intravenously administe
red ALN-VSP given every two weeks. Other secondary and exploratory objective
s included: assessment of tumor response using Response Evaluation Criteria
for Solid Tumors (RECIST); quantitation of change in tumor blood flow and va
scular permeability as measured by dynamic contrast-enhanced magnetic resona
nce imaging (DCE-MRI); and, analysis of pharmacodynamic effects of ALN-VSP o
n tumors as measured in patients electing to proceed with voluntary pre- and
post-treatment biopsies.
Results of the Phase I study in 41 patients were previously presented at the
American Society of Clinical Oncology (ASCO) Annual Meeting in 2011 and dem
onstrated proof of RNAi mechanism based on liver biopsy samples and disease
control (stable disease or better after first two months) in 13/31 (42%) pat
ients treated at doses greater than or equal to 0.4 mg/kg. ALN-VSP was gener
ally safe and well tolerated up to a dose of 1.0 mg/kg. The most common adve
rse events were grade 1-2 fatigue (24% of patients), nausea (17% of patients
) and fever (17% of patients), with no clear dose relationship. There were a
lso no dose-dependent changes in liver function tests. Grade 2 infusion-rela
ted reactions were observed in 15% of patients, or 3% of total doses adminis
tered; these reactions responded to slowing of the infusion of drug, and no
patients discontinued therapy because of an infusion reaction. Dose-limiting
toxicities included: liver failure and death in one patient with extensive
hepatic metastases involving greater than 70% of liver mass and prior splene
ctomy/partial hepatectomy at 0.7 mg/kg; transient grade 3 thrombocytopenia i
n two patients at 1.25 mg/kg; and grade 3 hypokalemia in one patient at 1.5
mg/kg.
The ALN-VSP extension study was designed to enable continued dosing with ALN
-VSP in patients who had achieved stable disease or better after completing
four months of treatment on the Phase I trial. Patients enrolled onto the ex
tension study were permitted to receive bi-weekly ALN-VSP at the same dose l
evel that they had been safely treated with in the Phase I study until disea
se progression or unacceptable toxicity; a total of seven patients were enro
lled. The primary objective was to collect long-term safety data. The second
ary objective was to assess tumor response.
Results from the extension study were previously presented at the American S
ociety of Clinical Oncology (ASCO) Annual Meeting in 2012 and demonstrated t
hat chronic dosing with ALN-VSP up to 1.0 mg/kg was generally safe and well
tolerated in this setting. On average, patients received bi-weekly treatment
s for 11.3 months. An endometrial cancer patient achieved a complete respons
e (CR) after 20 months of treatment at 0.7 mg/kg and remained in remission u
pon completion of 26 months of therapy. A patient with pancreatic neuroendoc
rine tumor (PNET) treated at 1.0 mg/kg remained on study with stable disease
(SD) for 18 months, and two patients with renal cell carcinoma (RCC) treate
d at 1.0 mg/kg remained on study with SD for approximately 8-12 months. No n
ew toxicities were reported among the patients enrolled onto the extension s
tudy. A PNET patient and an RCC patient who achieved SD at 1.0 mg/kg came of
f the study after 5.5 and 8.5 months, respectively, for adverse events that
included grade 3 elevated alkaline phosphatase or grade 2 fatigue deemed pos
sibly related to study drug. A decrease in spleen volume, likely an on-targe
t anti-KSP effect and not associated with any adverse events, occurred to a
greater degree on the extension study than in the Phase I trial and was most
pronounced in patients receiving 12 or more doses.
揃oth primary liver cancer and metastatic disease of the liver are associate
d with poor prognosis for patients, and new therapies are clearly needed,s
aid Josep Tabernero, M.D., Chairman of the Medical Oncology Department and P
hase I Program at Vall d扝ebron University Hospital in Barcelona, Spain. 揟h
is Phase I trial and extension study with ALN-VSP represents, to our knowled
ge, the most comprehensive clinical trial of a systemically delivered RNAi t
herapeutic and also the most extensive experience with RNAi therapeutics in
cancer. The safety data and anti-tumor activity with ALN-VSP, including a co
mplete response in a patient with multiple liver metastases who had failed m
ultiple prior therapies, are very encouraging and I look forward to the furt
her development of this promising agent.br />
In 2012, Alnylam and Ascletis Pharmaceuticals (Hangzhou) Co., Ltd., a privat
ely held U.S.-China joint venture pharmaceutical company, formed a strategic
collaboration for the development of ALN-VSP in China. Alnylam will retain
all rights in the rest of the world, and is eligible to receive milestones a
nd royalties from Ascletis based on product sales.
About Liver Cancers
Cancer affecting the liver, known as either primary or secondary liver cance
r, is associated with one of the poorest survival rates in oncology and repr
esents a major unmet medical need affecting a large number of patients world
wide. Primary liver cancer, or hepatocellular carcinoma (HCC), is one of the
most common cancers worldwide, with more than 630,000 people diagnosed each
year including approximately 350,000 in China. Secondary liver cancer, also
known as metastatic liver cancer, is cancer that spreads to the liver from
another part of the body due to other common cancers like colon, lung, or br
east cancer. Worldwide, more than 500,000 people are diagnosed with secondar
y liver cancer each year.
About LNP Technology
Alnylam has licenses to Tekmira LNP intellectual property for use in RNAi th
erapeutic products using LNP technology.
About RNA Interference (RNAi)
RNAi (RNA interference) is a revolution in biology, representing a breakthro
ugh in understanding how genes are turned on and off in cells, and a complet
ely new approach to drug discovery and development. Its discovery has been h
eralded as 揳 major scientific breakthrough that happens once every decade o
r so,and represents one of the most promising and rapidly advancing fronti
ers in biology and drug discovery today which was awarded the 2006 Nobel Pri
ze for Physiology or Medicine. RNAi is a natural process of gene silencing t
hat occurs in organisms ranging from plants to mammals. By harnessing the na
tural biological process of RNAi occurring in our cells, the creation of a m
ajor new class of medicines, known as RNAi therapeutics, is on the horizon.
Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise
Alnylam抯 RNAi therapeutic platform, target the cause of diseases by potentl
y silencing specific mRNAs, thereby preventing disease-causing proteins from
being made. RNAi therapeutics have the potential to treat disease and help
patients in a fundamentally new way.
About Alnylam Pharmaceuticals
Alnylam is a biopharmaceutical company developing novel therapeutics based o
n RNA interference, or RNAi. The company is leading the translation of RNAi
as a new class of innovative medicines with a core focus on RNAi therapeutic
s for the treatment of genetically defined diseases, including ALN-TTR for t
he treatment of transthyretin-mediated amyloidosis (ATTR), ALN-AT3 for the t
reatment of hemophilia and rare bleeding disorders (RBD), ALN-AS1 for the tr
eatment of acute intermittent porphyria, ALN-PCS for the treatment of hyperc
holesterolemia, and ALN-TMP for the treatment of hemoglobinopathies. As part
of its 揂lnylam 5x15TMstrategy, the company expects to have five RNAi the
rapeutic products for genetically defined diseases in clinical development,
including programs in advanced stages, on its own or with a partner by the e
nd of 2015. Alnylam has additional partnered programs in clinical or develop
ment stages, including ALN-RSV01 for the treatment of respiratory syncytial
virus (RSV) infection and ALN-VSP for the treatment of liver cancers. The co
mpany抯 leadership position on RNAi therapeutics and intellectual property h
ave enabled it to form major alliances with leading companies including Merc
k, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, Cubis
t, Ascletis, Monsanto, and Genzyme. In addition, Alnylam holds a significant
equity position in Regulus Therapeutics Inc., a company focused on discover
y, development, and commercialization of microRNA therapeutics. Alnylam has
also formed Alnylam Biotherapeutics, a division of the company focused on th
e development of RNAi technologies for applications in biologics manufacturi
ng, including recombinant proteins and monoclonal antibodies. Alnylam抯 Vaxi
RNAplatform applies RNAi technology to improve the manufacturing processes
for vaccines; GlaxoSmithKline is a collaborator in this effort. Alnylam sci
entists and collaborators have published their research on RNAi therapeutics
in over 100 peer-reviewed papers, including many in the world抯 top scienti
fic journals such as Nature, Nature Medicine, Nature Biotechnology, and Cell
. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusett
s. For more information, please visit www.alnylam.com.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam抯 future expectations,
plans and prospects, including without limitation, statements regarding Aln
ylam抯 views with respect to the potential for RNAi therapeutics, including
the potential for ALN-VSP, and Alnylam抯 expectations regarding its 揂lnylam
5x15product strategy, constitute forward-looking statements for the purpo
ses of the safe harbor provisions under The Private Securities Litigation Re
form Act of 1995. Actual results may differ materially from those indicated
by these forward-looking statements as a result of various important factors
, including, without limitation, Alnylam抯 ability to successfully demonstra
te the efficacy and safety of its drug candidates and the pre-clinical and c
linical results for these product candidates, including ALN-VSP, which may n
ot support further development of such product candidates, both our and Ascl
etisability to successfully advance ALN-VSP resulting in the potential pay
ment of milestones and royalties to us, actions of regulatory agencies, whic
h may affect the initiation, timing and progress of clinical trials for such
product candidates, obtaining, maintaining and protecting intellectual prop
erty, obtaining regulatory approval for products, competition from others us
ing technology similar to Alnylam抯 and others developing products for simil
ar uses, and Alnylam抯 ability to establish and maintain strategic business
alliances, including its collaboration with Ascletis, and new business initi
atives, as well as those risks more fully discussed in the 揜isk Factorsfi
led with Alnylam抯 current report on Form 8-K filed with the Securities and
Exchange Commission (SEC) on January 14, 2013 and in other filings that Alny
lam makes with the SEC. In addition, any forward-looking statements represen
t Alnylam抯 views only as of today and should not be relied upon as represen
ting its views as of any subsequent date. Alnylam does not assume any obliga
tion to update any forward-looking statements.
Source: Alnylam Pharmaceuticals, Inc.
Alnylam Pharmaceuticals, Inc.
Cynthia Clayton, 617-551-8207
Vice President, Investor Relations and Corporate Communications
or
Spectrum
Amanda Sellers, 202-955-6222 x2597 (Media)
Home About Alnylam Leadership in RNAi Programs & Pipeline
Intellectual Property Business Development News and Invest
ors Capella
Legal Site Map Copyright 2008 Alnylam Pharmaceuticals, Inc. All
Rights Reserved
【在 d****f 的大作中提到】
: 6park上转来的:
: http://site.6park.com/chan2/index.php?app=forum&act=threadview&
: 说实话我真是希望是真的,不过我目前所受的教育似乎尚不允许我相信之:
: 特大號外
: 美國公司宣佈人類已經攻克癌症
: 合成的雙鏈RNA進入肝細胞後，人體內的RNAi機制便會摧毀合成的RNA和任何與之匹配的
: 、與腫瘤生長相關的信使RNA，阻止蛋白質的繼續產生，從而使腫瘤停止生長....
: 據美國媒體報導，美國阿爾尼拉姆生物技術公司日前宣佈他們找到了一種能夠治癒所有
: 癌症的新型藥物，首批接受臨床試驗的19名晚期肝癌患者病情都有較大好轉。不僅如此
: ，該公司稱，假以時日，這種藥物甚至有可能治癒一切疾病。
| d****f
发帖数: 313 | 3 不好意思我实在是外行,什么是phase I study
Ex
C
Ex
(L
,
【在 t****r 的大作中提到】
: http://phx.corporate-ir.net/phoenix.zhtml?c=148005&p=irol-newsA
: 9293&highlight=
: 没那么夸张，只是phase I study 有效果而已。。。
: Alnylam and Collaborators Publish Results from Phase I Clinical Trial and Ex
: tension Study with ALN-VSP, an RNAi Therapeutic for the Treatment of Liver C
: ancer
: - Results Published in Cancer Discovery Document Most Comprehensive Human Ex
: perience To Date for RNAi Therapeutics Delivered with Lipid Nanoparticles (L
: NPs) -
: CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jan. 30, 2013-- Alnylam Pharmaceuticals,
| t****r
发帖数: 702 | 4 就是刚完成第一期的临床人体试验，检验药物的剂量和安全性。 还有第二期，第三期的
临床试验，全部通过了才能上市。
还是处在比较初级的阶段，是否真正有效还要经过很多年的检验。
【在 d****f 的大作中提到】
: 不好意思我实在是外行,什么是phase I study
:
: Ex
: C
: Ex
: (L
: ,
| v**o
发帖数: 4956 | 5 活跃经济，总是要有点激动人心的新闻，也总是不缺为买希望不惜重金的
来钱不易的在边上看看就行了 |
|
