雖然面臨過度炒作的問題，但在過去十年里，醫(yī)療保健行業(yè)也取得了不少實(shí)實(shí)在在的成績。《財(cái)富》選出了過去十年內(nèi)四項(xiàng)最值得關(guān)注的醫(yī)學(xué)突破，包括創(chuàng)新型藥物，針對(duì)罕見疾病的個(gè)性化基因療法和針對(duì)傳染性疾病和常見疾病（如心臟病和肺病）的普遍療法。

1、特魯瓦達(dá)（吉列德科學(xué)公司）

吉列德的艾滋病預(yù)防藥物可能是生命科學(xué)領(lǐng)域最重要的進(jìn)步之一。

HIV暴露前預(yù)防（Pre-Exposure Prophylaxis,以下簡稱PrEP），是指有感染艾滋病病毒風(fēng)險(xiǎn)的人，通過服用抗病毒治療藥物，來減少感染HIV風(fēng)險(xiǎn)的措施。

有研究表明，每日服用特魯瓦達(dá)可以90%至99%地有效預(yù)防高危患者（如男男性行為者或其行為可能增加感染HIV/AIDS風(fēng)險(xiǎn)的男性）之間的艾滋病病毒傳播。

這是基本的暴露前預(yù)防（PrEP）治療，效果十分出色，甚至在一些國家，只需支付5美元，政府就可以提供三個(gè)月的用藥。

在美國，因?yàn)槿藗冸y以承擔(dān)高額的自付費(fèi)用，PrEP推廣進(jìn)程緩慢。但有消息稱，吉列德已承諾在11年內(nèi)捐贈(zèng)滿足覆蓋20萬人的藥物，免費(fèi)為付不起藥費(fèi)的人提供該藥，或?yàn)槠渌腺Y格的患者提供保險(xiǎn)共付額（co-pays）。

2、派姆單抗（默沙東公司）

派姆單抗是癌癥免疫療法蓬勃發(fā)展的一個(gè)例證。僅在2019年第三季度，該藥物的銷售額就達(dá)到了驚人的30億美元，輕松超過了華爾街的預(yù)期，鞏固了其“藥王”地位。

派姆單抗首次獲批是在2014年，而后它讓美國食品與藥品管理局大開綠燈、批準(zhǔn)其用于多種癌癥——其中既包括黑色素瘤（該藥顯然治療了前總統(tǒng)吉米·卡特的癌癥），又包括肺癌這種最致命的疾病之一。

派姆單抗對(duì)肺癌的治療提高了肺癌患者的壽命，也推動(dòng)了默沙東公司的成功。美國食品與藥品管理局批準(zhǔn)該藥物可以作為“一線”治療，與傳統(tǒng)化療一起用于某些肺癌患者（這是一種為從未接受過治療的肺癌患者提供的組合療法）。

默沙東面臨來自百時(shí)美施貴寶和羅氏等公司的競爭，這兩家公司都推出了自己的免疫治療藥物，但迄今為止，派姆單抗仍然是市場王者。

3、CAR-T、CRISPR和基因治療（諾華、Spark Therapeutics公司等）

正在進(jìn)行的基因革命覆蓋生命科學(xué)的各個(gè)領(lǐng)域，其中包括基因編輯技術(shù)（比如CRISPR技術(shù)，可以將最基本的生物構(gòu)成要素進(jìn)行切割，或許可以被用來對(duì)抗從遺傳性血液疾病到各類癌癥在內(nèi)的一切疾病）。

雖然仍處于實(shí)驗(yàn)階段，但大量制藥巨頭和生物科技公司都在探索這項(xiàng)技術(shù)。

基因治療之間也不是完全相同，存在細(xì)微差別。例如：諾華的“CAR-T”療法Kymriah可用于治療血癌，于2017年獲得美國食品與藥品管理局批準(zhǔn)，將人體自身的免疫系統(tǒng)作為武器來抵御這種疾病，該過程包括將免疫細(xì)胞重組以瞄準(zhǔn)癌細(xì)胞。Spark Therapeutics公司的罕見疾病療法Luxturna也于2017年年底獲得批準(zhǔn)，而該療法將一段健康基因拷貝插入細(xì)胞中以治療遺傳性失明。

此類療法的創(chuàng)新之處在于，它們或許可以為治療頑固性疾病（通常是致命疾病）提供一種永久解決方案。但是價(jià)格——以及復(fù)雜的制造過程——仍然是個(gè)問題。

4、人工心臟瓣膜和機(jī)器人治療（愛德華生命科學(xué)、Intuitive Surgical）

日益發(fā)展的機(jī)器人和人造器官已成為用于治療病人的主要手段。

領(lǐng)先的兩家公司是愛德華生命科學(xué)公司和Intuitive Surgical，這兩家公司都在最新的《財(cái)富》未來50強(qiáng)中榜上有名，說明其長遠(yuǎn)增長潛力被看好。

雖然精準(zhǔn)醫(yī)療和基因治療已經(jīng)改變了藥物的開發(fā)，但這兩家公司都專注于影響更多美國人的醫(yī)療問題，比如心臟病和手術(shù)需求。

根據(jù)美國疾病控制中心的數(shù)據(jù)，心臟病占美國每年死因的四分之一。愛德華生命科學(xué)公司是人工心臟瓣膜領(lǐng)域的領(lǐng)導(dǎo)者，他們?yōu)槟切┎荒苊半U(xiǎn)進(jìn)行侵入性手術(shù)的病人提供人工心臟瓣膜。

Intuitive Surgical公司已經(jīng)開發(fā)了微創(chuàng)手術(shù)技術(shù)，醫(yī)生毋需把人切開就可以進(jìn)行手術(shù)。外科醫(yī)生可以通過患者腹部或其他部位的小孔引導(dǎo)各種機(jī)械臂和高清攝像機(jī)進(jìn)入患者體內(nèi)。該公司的增長說明了一切（僅在過去兩年，其市值就飆升了57%）——但更令人印象深刻的可能是該技術(shù)被全球醫(yī)生采用。根據(jù)該公司的數(shù)據(jù)，Intuitive Surgical的旗艦產(chǎn)品達(dá)·芬奇機(jī)器人平臺(tái)已經(jīng)被用于超過500萬臺(tái)手術(shù)了。（財(cái)富中文網(wǎng)）

譯者：Agatha

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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.