过去十年，医学界取得这四大突破

虽然面临过度炒作的问题，但在过去十年里，医疗保健行业也取得了不少实实在在的成绩。《财富》选出了过去十年内四项最值得关注的医学突破，包括创新型药物，针对罕见疾病的个性化基因疗法和针对传染性疾病和常见疾病（如心脏病和肺病）的普遍疗法。

1、特鲁瓦达（吉列德科学公司）

吉列德的艾滋病预防药物可能是生命科学领域最重要的进步之一。

HIV暴露前预防（Pre-Exposure Prophylaxis,以下简称PrEP），是指有感染艾滋病病毒风险的人，通过服用抗病毒治疗药物，来减少感染HIV风险的措施。

有研究表明，每日服用特鲁瓦达可以90%至99%地有效预防高危患者（如男男性行为者或其行为可能增加感染HIV/AIDS风险的男性）之间的艾滋病病毒传播。

这是基本的暴露前预防（PrEP）治疗，效果十分出色，甚至在一些国家，只需支付5美元，政府就可以提供三个月的用药。

在美国，因为人们难以承担高额的自付费用，PrEP推广进程缓慢。但有消息称，吉列德已承诺在11年内捐赠满足覆盖20万人的药物，免费为付不起药费的人提供该药，或为其他符合资格的患者提供保险共付额（co-pays）。

2、派姆单抗（默沙东公司）

派姆单抗是癌症免疫疗法蓬勃发展的一个例证。仅在2019年第三季度，该药物的销售额就达到了惊人的30亿美元，轻松超过了华尔街的预期，巩固了其“药王”地位。

派姆单抗首次获批是在2014年，而后它让美国食品与药品管理局大开绿灯、批准其用于多种癌症——其中既包括黑色素瘤（该药显然治疗了前总统吉米·卡特的癌症），又包括肺癌这种最致命的疾病之一。

派姆单抗对肺癌的治疗提高了肺癌患者的寿命，也推动了默沙东公司的成功。美国食品与药品管理局批准该药物可以作为“一线”治疗，与传统化疗一起用于某些肺癌患者（这是一种为从未接受过治疗的肺癌患者提供的组合疗法）。

默沙东面临来自百时美施贵宝和罗氏等公司的竞争，这两家公司都推出了自己的免疫治疗药物，但迄今为止，派姆单抗仍然是市场王者。

3、CAR-T、CRISPR和基因治疗（诺华、Spark Therapeutics公司等）

正在进行的基因革命覆盖生命科学的各个领域，其中包括基因编辑技术（比如CRISPR技术，可以将最基本的生物构成要素进行切割，或许可以被用来对抗从遗传性血液疾病到各类癌症在内的一切疾病）。

虽然仍处于实验阶段，但大量制药巨头和生物科技公司都在探索这项技术。

基因治疗之间也不是完全相同，存在细微差别。例如：诺华的“CAR-T”疗法Kymriah可用于治疗血癌，于2017年获得美国食品与药品管理局批准，将人体自身的免疫系统作为武器来抵御这种疾病，该过程包括将免疫细胞重组以瞄准癌细胞。Spark Therapeutics公司的罕见疾病疗法Luxturna也于2017年年底获得批准，而该疗法将一段健康基因拷贝插入细胞中以治疗遗传性失明。

此类疗法的创新之处在于，它们或许可以为治疗顽固性疾病（通常是致命疾病）提供一种永久解决方案。但是价格——以及复杂的制造过程——仍然是个问题。

4、人工心脏瓣膜和机器人治疗（爱德华生命科学、Intuitive Surgical）

日益发展的机器人和人造器官已成为用于治疗病人的主要手段。

领先的两家公司是爱德华生命科学公司和Intuitive Surgical，这两家公司都在最新的《财富》未来50强中榜上有名，说明其长远增长潜力被看好。

虽然精准医疗和基因治疗已经改变了药物的开发，但这两家公司都专注于影响更多美国人的医疗问题，比如心脏病和手术需求。

根据美国疾病控制中心的数据，心脏病占美国每年死因的四分之一。爱德华生命科学公司是人工心脏瓣膜领域的领导者，他们为那些不能冒险进行侵入性手术的病人提供人工心脏瓣膜。

Intuitive Surgical公司已经开发了微创手术技术，医生毋需把人切开就可以进行手术。外科医生可以通过患者腹部或其他部位的小孔引导各种机械臂和高清摄像机进入患者体内。该公司的增长说明了一切（仅在过去两年，其市值就飙升了57%）——但更令人印象深刻的可能是该技术被全球医生采用。根据该公司的数据，Intuitive Surgical的旗舰产品达·芬奇机器人平台已经被用于超过500万台手术了。（财富中文网）

译者：Agatha

Health care suffers from a hype problem. But drug makers have also produced some innovative medicines in the past decade—ranging from personalized gene therapies for rare diseases to more wide-spread treatments that tackle infectious disorders and more common conditions such as heart and lung disease.

Some of these drugs, and the diseases they tackle, don’t get a whole lot of attention. Here are a few worth highlighting.

1. Truvada (Gilead Sciences)

Gilead’s HIV/AIDS prevention medication may be one of the most important advances in the life science field.

Taking Truvada on a daily basis has proven to offer between 90% and 99% effectiveness in preventing HIV transmission in at-risk patients such as men who have sex with men or engage in behavior which may raise their risk for contracting HIV/AIDS.

It’s the foundational pre-exposure prophylaxis (PrEP) treatment, and it’s so effective that some nations have made a three-month-supply available for just five dollars.

There’s still a ways to go with PrEP adoption in the U.S. because of high out-of-pocket costs that can discourage people from taking it. But there is also some preliminary evidence that adoption of Truvada and other PrEP medication is linked with a dropoff in HIV infection rates.

2. Keytruda (Merck)

Keytruda is an example of the cancer immunotherapy boom. The drug brought in a stunning $3 billion in sales in the third quarter of 2019 alone, easily surpassing Wall Street expectations and cementing its status as “The Drug to Beat.”

That’s not just a sales story—there’s a reason that Keytruda, which was first approved way back in 2014, has won so many FDA green lights in cancers ranging from melanoma (the treatment apparently cleared former President Jimmy Carter of his own cancer) to lung cancer, one of the deadliest diseases out there.

The latter has fueled Merck’s success while boosting life expectancy among lung cancer patients. In fact, the FDA approved the drug as a “first-line” treatment, along with traditional chemotherapy for certain lung cancer patients (a combo that’s delivered for people who have never been treated before).

Merck faces competition from companies like Bristol-Myers Squibb and Roche, who have their own rival immunotherapy drugs, but to date, Keytruda remains the king.

3. CAR-T, CRISPR, and Gene Therapy (Novartis, Spark Therapeutics, and Many More)

The genomic revolution is underway—and it runs the gamut of the life sciences.

There’s gene-editing (such as the CRISPR technology that allows us to slice and dice our fundamental biological building blocks and which could potentially be used to combat everything from inherited blood disorders to various cancers). This is still experimental in nature, but a plethora of pharma giants and biotech startups alike are exploring the tech.

Gene therapy can be a different, and more nuanced, beast. For instance: Novartis’ “CAR-T” therapy Kymriah, a blood cancer treatment approved by the FDA in 2017, weaponizes the body’s own immune system in order to ward off the disease. This process involves re-engineering immune cells in order to target cancerous cells. Spark Therapeutics’ rare disease therapy Luxturna, also approved in late 2017, adds to the list—the treatment inserts a new copy of a healthy gene into cells for an inherited form of blindness.

What’s so innovative about these therapies is they have the potential to provide a permanent solution to intractable, often deadly disorders. But pricing—and a complicated manufacturing process—remains a problem.

4. Artificial Heart Valves and Robotic Therapy (Edwards Lifesciences, Intuitive Surgical)

The robots—and their counterparts—are on the rise. And they’re helping patients in a major way.

Two companies leading these efforts are Edwards Lifesciences and Intuitive Surgical, both featured in the latest Fortune Future 50, a list that identifies companies with the strongest long-term growth potential.

While precision medicine and gene therapy has transformed the drug development field, both of the firms are focused on medical problems which afflict a much wider swath of Americans, such as heart disease and the need for surgery.

For instance, Edwards Lifesciences is a leader in artificial heart valve development for patients who can’t necessarily risk a more invasive surgery. Heart disease causes one in four American deaths every single year, according to the Centers for Disease Control.

Intuitive Surgical has been building up minimally invasive surgical tech that allows doctors to perform operations without having to, well, slice people up. A surgeon can guide various robotic arms and a high-definition camera into patients’ bodies through tiny holes in the abdomen or other regions. The company’s growth speaks for itself (a 57% spike in market value in the past two years alone)—but what may be more impressive is the adoption by physicians across the globe. Intuitive’s flagship da Vinci robotic platform has been used in more than five million surgeries, according to the company.