这家生物科技公司力挺干细胞疗法，它能成功吗？

以干细胞为基础的医学领域一直让人相当失望，还因夸大其词遭到监管谴责。但Athersys的首席执行官吉尔·范·伯克伦表示，干细胞领域受到的待遇并不公平。

“说干细胞没有真正发挥作用是一种误解。”他在采访中告诉《财富》杂志。 “现在干细胞是个非常热门的领域，情况真的发生了变化。”

范·伯克伦介绍了公司的近期成果，包括一些中期、后期临床试验的细胞疗法。就在两周前，Athersys的实验性干细胞疗法MultiStem获得了美国食品和药物管理局（FDA）的快速审核资格，非常令人艳羡。该药物可以用于治疗名为急性呼吸窘迫综合症（ARDS）的严重肺衰竭疾病。获得快速审核资格可以加速监管审批过程，通常是在治疗某种疾病药品缺乏的情况下。

极为严重的急性呼吸窘迫综合症显然属于此类疾病。美国肺病协会（American Lung Association）称，罹患该病的患者通常因为某种严重伤害或疾病而引发肺部受创，液体渗入肺部，导致病情已然严重的患者出现呼吸困难（或完全无法呼吸）。

Athersys对MultiStem采取的研究发现，与安慰剂组相比，患者“确诊患有急性呼吸窘迫综合症数天之内接受治疗并使用呼吸机后，死亡率降低，无需呼吸机的天数增加，无需进入加护病房的天数增加”。初步治疗结果推动该药进入美国食品和药物管理局的快速审批通道。

MultiStem疗法的最吸引人之处是在名字里加入了“Multi”（意思是“多”——译注）。范·伯克伦表示，Athersys正在努力避免许多干细胞领域新贵面临的主要问题：可扩展性和多用途。Athersys不要求个人捐献者完美配型，而是希望提供“现成”的再生干细胞治疗，帮助病患修复组织。

“这种药的功能并不单一，而是同时具备多项功能。”范·伯克伦说。例如，该药正在针对缺血性中风患者进行后期临床实验（该项目也已经获得美国食品和药物管理局的快速审核资格）。“干细胞治疗的市场机会比其他领域都大。”他补充道。

Athersys到底能否在众多公司的惨败之处获得成功，应该很快就可以揭晓。（财富中文网）

译者：冯丰

审校：夏林

The world of stem cell-based medicine has had its fair share of disappointments and regulatory recriminations over wildly inflated claims. But Athersys CEO Gil Van Bokkelen says the stem cell field isn’t getting a fair shake.

“It’s a misconception to say stem cells haven’t really delivered,” he tells Fortune in an interview. “This is a very hot field right now, and things have really changed.”

Van Bokkelen points to his own company’s recent achievements, including a number of cell therapies in mid- or late-stage clinical trials. Just two weeks ago, Athersys’ experimental MultiStem treatment received the Food and Drug Administration’s (FDA) coveted fast track designation in a devastating lung-wasting disease called acute respiratory distress syndrome (ARDS). These designations speed the regulatory process and is typically granted for products that treat disorders with a dearth of available treatments.

And devastating ARDS surely is. It’s a consequence of trauma to the lungs in patients who are typically already in the hospital for some kind of severe injury or illness, and it results in fluid leaking in to the lungs, making the very act of breathing even more difficult (or impossible) in patients who are already extremely sick, according to the American Lung Association.

Athersys’ studies of MultiStem found that patients who received the treatment “within several days after being diagnosed with ARDS and being placed on a ventilator experienced lower mortality, increased ventilator-free days, and increased ICU-free days” compared to a placebo group. Those preliminary results led to the FDA fast track.

What makes the MultiStem platform intriguing is, well, what puts the “Multi” in the name. Van Bokkelen says that Athersys is trying to avoid the main problem many stem cell upstarts face: Scaleability and multi-faceted uses. Rather than requiring an individual, perfect donor match, Athersys is attempting to create a product that’s an “off-the-shelf” version of regenerative stem cell treatment that can help repair tissue.

“The drug isn’t just doing one thing, it’s doing a bunch of different things,” says Van Bokkelen. For instance, it’s being tested out in late-stage trials for ischemic stroke patients (yet another program with FDA fast track designation). “Market opportunity is way bigger in this space than pretty much any other one,” he adds.

We should find out soon enough whether Athersys can succeed where so many others have failed.