多发性硬化病(MS)影响全球250万余人。与许多疾病不同，对于再发-缓解型MS(RRMS)目前还没有可明确区分的一线治疗黄金标准。历史上，出现过4个主要减慢RRMS进展的病情改善药物(DMD)。其中3个配方都包括干扰素：2个包括干扰素β-1a、1个包括干扰素β-1b。   

     最近，每月给药1次的新药人源化单克隆抗体那他珠单抗(natalizumab)改变了传统的DMD常用疗效评价终点，即对MS治疗的应答以无病情活动而不是简单的复发率降低来衡量。那他珠单抗据信能降低炎性免疫细胞附着和通过肠道细胞衬层及血脑屏障的能力。最近的临床试验数据显示，与安慰剂相比，它可使两年期间保持无病情活动的患者数多4倍，因而可考虑作为新的疗效评价标准。   

     目前那他珠单抗作为二线治疗用药，用于对上述4大DMD无良好应答的患者。尽管如此，它不大可能广泛应用，因为它具有严重的安全性问题。刚上市不久，使用该药的3例患者就出现了一种罕见的、不可治愈的神经疾病——进行性多病灶性脑白质病（PML)。该药因此于2004年撤出市场，2006年重新上市，但受专门的处方计划所限制。不过，此后又有10例PML报告出现。因此，研究人员正在努力寻找MS新的治疗途径。   

      口服药物正在研制中   

     所有现在治疗MS药物的一个主要缺点是必须通过静脉或皮下注射给药。因此，需要能以更好的方式给药的安全有效药物，口服药物由此进入了研究人员的视线。目前，有5个口服MS治疗药物在经过Ⅲ期临床试验后准备上市：芬戈莫德（fingolimod）、克拉屈滨（cladribine)、BG12、特立氟胺(teriflunomide)和拉喹莫德（laquinimod）。有关克拉屈滨的Ⅲ期临床试验数据显示，与安慰剂比较它能将MS再发率降低60%。此外，对这项口服克拉屈滨片剂治疗MS（CLARITY试验)的另一项分析表明，短期口服该药治疗MS在临床和MRI造影预后方面都有迅速和持续的改善，它伴有对参与MS发病机理的血细胞亚型有快速和持续的作用。不良反应方面，在今年公布的数据中，用克拉屈滨治疗的1,326例患者中有4例在不同部位出现癌症。同样，迄今有关芬戈莫德的临床试验也报告了两例致命性感染和7例被成功治疗的皮肤癌。因此，对发生癌症和机会性感染的担心是它们可能成为一线治疗的真正障碍。   

      虽然老的注射用药物存在很多方面的不足，但从没有1例患者因此而患癌症或死亡。所以研究人员正在探索新的注射药物输送方法，以便于患者用药。今年6月，一项随机双盲安慰剂对照的Ⅲ期临床试验ADVANCE研究启动。这项临床试验将对RRMS患者评估聚乙二醇化干扰素β-1a的疗效和安全性。由于聚乙二醇化可防止干扰素β-1a降解，所以有可能2～4周给药1次。   

     抗癌药或可用于二线治疗   

     新适应证的开发是一种明显价廉的战略。有研究者正在试验将治疗B细胞淋巴瘤药物——阿仑单抗（alemtuzumab）开发用于MS，目前正在Ⅱ期临床试验中，期望2011年能获批准。它可1年输注1次的潜能最终可能超过1天给药1次口服片剂的吸引力。但这种人源化抗CD-52单克隆抗体的免疫抑制性质可能会使患者受到机会性感染的威胁。此外，有1/3经它治疗的患者出现了甲状腺方面的问题，还有一个亚群(6%)会发展成免疫血小板减少性紫癜(ITP)。   

     虽然这两个问题都是可发现和治愈的，但这意味着需要在血液检验及相关监视方面能提供必须支持的专门医疗机构内才可出具处方。它很可能作为一个二线治疗药物，用于MS病情进展的患者。   

     干细胞治疗研究刚刚起步   

     以美国西北大学R  Burt博士为首的国际团队称，他们已经发现对MS有逆转作用的治疗方法。这种治疗方法是收集MS患者的骨髓干细胞并将其冷冻，与此同时给予常规药物除去引起损伤的免疫或淋巴细胞，然后将骨髓细胞置入以使患者免疫系统得以补充。值得注意的是，研究发现，接受干细胞移植的21例成人患者在3年研究期间没有1例的症状出现任何恶化。   

     采用干细胞移植试验治疗MS，这并不是第一次，但这是第一次获得成功。其主要原因是先前的试验对象是MS更晚期的继发性进展型患者，而对不很晚期的患者可以改善许多MS参数。需要提到的是，在这项研究中给予的环磷酰胺就是一种已用于MS的强化疗药。目前，有关具有感染可能和对发生肿瘤的担忧使这一疗法不能成为切实可行的疗法。

Natalizumab review

Natalizumab is a prescribed drug that belongs to the category of medicines called immune modulators. It is used to treat and prevent symptoms of multiple sclerosis and Chrohn’s disease. Nazalizumab only prevents the symptoms but cannot be used to cure MS and Chrohn’s disease. This drug prevents the spreading of a cell to the immune system which causes damage to the brain and spinal cord.

Natalizumab is a drug injected to patients suffering from multiple sclerosis or MS. Natalizumab is being distributed under the brand name Tysabri. This drug prevents or slows down the effect associated with having an MS. Multiple sclerosis is a disease that causes disability to a person such as loss of bladder control, loss of muscle coordination, weakness of the body, and also affects normal vision.

This drug is in a form of concentrated solution which is diluted and being inject to the veins. Natalizumab is usually given once in every four weeks in an infusion center or hospital. It usually takes an hour to complete the infusion. In most cases, you need to stay at the hospital or clinic for an hour after infusion so that the doctor can monitor any signs of side effects.

If you have missed a scheduled infusion, you must immediately inform your doctor.

Using Natalizumab may cause several side effects. Some of the side effects are less serious like heartburn, stomach pain, headache, diarrhea, constipation, depression, night sweats, tiredness, white vaginal discharge, sudden pain in the arms or legs, weight loss or weight gain, cold sores, difficulty in urinating, and irregular menstruation.

There are also serious side effects that may occur. Some of these include chest pain, sore throat, dizziness, flushing, chills, vomiting, nausea, yellowing of the skin or eyes, difficulty in breathing, and darkening of the urine.

There might be other side effects that may occur that is not included in this article. If you experience any of these side effects, contact or visit your doctor the soonest time possible.

Certain precautions should be observed before undergoing medication with Natalizumab. Inform your doctor if you are allergic to the drug and other medicines. This includes vitamins, herbal products and nutritional products. This will help your doctor to decide on the proper dosage and also to monitor any unusual reactions or side effects it may cause.

It is also important to inform your doctor if you are pregnant or are breast feeding prior to medication. In case you become pregnant while under medication with Natalizumab, inform your doctor immediately.

Do not take any kind of vaccinations without consulting your doctor. You must always visit your doctor as scheduled and undergo any medical tests required for your treatment. This is to get the best benefit from your medication.

If you need to undergo surgery, inform your surgeon that you are using Natalizumab. This drug may affect your alertness so it is best to avoid driving or doing other extreme activities that needs normal alertness.


Brand name(s): Tysabri

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