以干細胞為基礎的醫學領域一直讓人相當失望，還因夸大其詞遭到監管譴責。但Athersys的首席執行官吉爾·范·伯克倫表示，干細胞領域受到的待遇并不公平。

“說干細胞沒有真正發揮作用是一種誤解。”他在采訪中告訴《財富》雜志。 “現在干細胞是個非常熱門的領域，情況真的發生了變化。”

范·伯克倫介紹了公司的近期成果，包括一些中期、后期臨床試驗的細胞療法。就在兩周前，Athersys的實驗性干細胞療法MultiStem獲得了美國食品和藥物管理局（FDA）的快速審核資格，非常令人艷羨。該藥物可以用于治療名為急性呼吸窘迫綜合癥（ARDS）的嚴重肺衰竭疾病。獲得快速審核資格可以加速監管審批過程，通常是在治療某種疾病藥品缺乏的情況下。

極為嚴重的急性呼吸窘迫綜合癥顯然屬于此類疾病。美國肺病協會（American Lung Association）稱，罹患該病的患者通常因為某種嚴重傷害或疾病而引發肺部受創，液體滲入肺部，導致病情已然嚴重的患者出現呼吸困難（或完全無法呼吸）。

Athersys對MultiStem采取的研究發現，與安慰劑組相比，患者“確診患有急性呼吸窘迫綜合癥數天之內接受治療并使用呼吸機后，死亡率降低，無需呼吸機的天數增加，無需進入加護病房的天數增加”。初步治療結果推動該藥進入美國食品和藥物管理局的快速審批通道。

MultiStem療法的最吸引人之處是在名字里加入了“Multi”（意思是“多”——譯注）。范·伯克倫表示，Athersys正在努力避免許多干細胞領域新貴面臨的主要問題：可擴展性和多用途。Athersys不要求個人捐獻者完美配型，而是希望提供“現成”的再生干細胞治療，幫助病患修復組織。

“這種藥的功能并不單一，而是同時具備多項功能。”范·伯克倫說。例如，該藥正在針對缺血性中風患者進行后期臨床實驗（該項目也已經獲得美國食品和藥物管理局的快速審核資格）。“干細胞治療的市場機會比其他領域都大。”他補充道。

Athersys到底能否在眾多公司的慘敗之處獲得成功，應該很快就可以揭曉。（財富中文網）

譯者：馮豐

審校：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.