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許多罕見病治療藥物或早已存在,為什么沒有投入使用?

許多罕見病治療藥物或早已存在,為什么沒有投入使用?

Annette Bakker 2021-07-09
有些藥物在臨床試驗中最初前景良好,但制藥公司由于戰略或財務上的原因終止了試驗。

已經終止研發的藥物可以改變用途,用于治療囊性纖維化和肌肉萎縮癥等罕見病。圖片來源:Thana Prasongsin—Getty Images

科學家們或許沒有意識到,他們早已研發出治療囊性纖維化、肌肉萎縮癥和罕見癌癥的藥物。

大多數人會認為,試驗性藥物最終要么沒有通過臨床試驗,要么沒有獲得美國食品與藥品管理局(FDA)的批準。但絕大部分藥品的最終命運在這兩者之間。

例如,有些藥物在臨床試驗中最初前景良好,但制藥公司由于戰略或財務上的原因終止了試驗。這些藥物被放棄之后,研究數據被束之高閣,研究項目也基本被大部分人遺忘。但它們只要經過較小的調整,就能夠變成治療目前一些罕見的不治之癥的顛覆性藥物。

針對一種疾病開發的藥物可以有效治療其他疾病的情況并不少見。比如,抗病毒藥金剛烷胺最初被用于治療流感,但后來被用于治療帕金森癥。一直被作為痛風藥物的消炎藥秋水仙素,目前正在進行新冠肺炎治療試驗。

當然,要確定哪些藥物應該改變用途或調整定位并不容易。匯總相關數據可能面臨挑戰,因為原始研究團隊通常早已被解散。藥企往往不愿意花費數千萬乃至數億美元,調查哪一款已經終止研發的藥物能夠治療罕見病,因為這些藥物的市場通常較小,而且對數量較少的罕見病患者開展試驗很有挑戰性。

但這些被放棄的藥物有可能拯救生命,因此克服這些障礙是值得的。許多公司和非營利組織正在嘗試做這件事情。

例如生物科技初創公司SpringWorks Therapeutics。這是一家在2017年從輝瑞(Pfizer)剝離后成立的公司,其業務是繼續開發早已被輝瑞擱置的藥物。SpringWorks可以讓輝瑞有財務上的動機來重新評估這些藥物,比如給予輝瑞10%的股份。目前,SpringWorks正在研究可治療多種不同罕見腫瘤的一系列藥物。SpringWorks贊助了兒童腫瘤基金會(Children’s Tumor Foundation)的多場活動。

SpringWorks的例子給BRIDGE計劃(BRIDGE Initiative)帶來了啟發。該計劃致力于加快研發兒童腫瘤治療藥物。兒童癌癥研究中心(CureSearch for Children’s Cancer)、FasterCures和我所在的兒童腫瘤基金會正在合作尋找激勵措施和途徑,鼓勵制藥公司和生物科技公司公開已經終止研發的藥物,或者承擔改變藥物用途的風險。

我們正在研究的一種激勵措施是為公司提供提高環境、社會和治理(ESG)評分的機會,交換條件是對于他們已經終止研發但有潛力治療神經纖維瘤和小兒癌癥的藥物,允許我們改變藥物的用途或回收再利用。環境、社會和治理用于衡量一家公司為改善人類社會和保護地球所做的貢獻。許多公司都渴望提高在這方面的評分。

BRIDGE計劃已經成功找到了投資者,他們愿意資助我們開發被藥企放棄的治療藥物,并將其商業化。

另外一個例子是美國國立衛生研究院(National Institutes for Health)罕見病與被忽視疾病治療項目(Therapeutics for Rare and Neglected Diseases)藥物用途修改篩選小組(Repurposing Screening Group)。該項目與研究人員、非營利組織和制藥公司合作,致力于發現有潛力治療罕見病的藥物,并安排進行臨床試驗。

將更多已經終止研發藥物用于治療罕見病,需要生物科技公司、投資者和非營利組織相互協作。讓這些被放棄的藥物煥發新生,能夠拯救患者的生命。(財富中文網)

本文作者安妮特?巴克是兒童腫瘤基金會的總裁。

翻譯:劉進龍

審校:汪皓

科學家們或許沒有意識到,他們早已研發出治療囊性纖維化、肌肉萎縮癥和罕見癌癥的藥物。

大多數人會認為,試驗性藥物最終要么沒有通過臨床試驗,要么沒有獲得美國食品與藥品管理局(FDA)的批準。但絕大部分藥品的最終命運在這兩者之間。

例如,有些藥物在臨床試驗中最初前景良好,但制藥公司由于戰略或財務上的原因終止了試驗。這些藥物被放棄之后,研究數據被束之高閣,研究項目也基本被大部分人遺忘。但它們只要經過較小的調整,就能夠變成治療目前一些罕見的不治之癥的顛覆性藥物。

針對一種疾病開發的藥物可以有效治療其他疾病的情況并不少見。比如,抗病毒藥金剛烷胺最初被用于治療流感,但后來被用于治療帕金森癥。一直被作為痛風藥物的消炎藥秋水仙素,目前正在進行新冠肺炎治療試驗。

當然,要確定哪些藥物應該改變用途或調整定位并不容易。匯總相關數據可能面臨挑戰,因為原始研究團隊通常早已被解散。藥企往往不愿意花費數千萬乃至數億美元,調查哪一款已經終止研發的藥物能夠治療罕見病,因為這些藥物的市場通常較小,而且對數量較少的罕見病患者開展試驗很有挑戰性。

但這些被放棄的藥物有可能拯救生命,因此克服這些障礙是值得的。許多公司和非營利組織正在嘗試做這件事情。

例如生物科技初創公司SpringWorks Therapeutics。這是一家在2017年從輝瑞(Pfizer)剝離后成立的公司,其業務是繼續開發早已被輝瑞擱置的藥物。SpringWorks可以讓輝瑞有財務上的動機來重新評估這些藥物,比如給予輝瑞10%的股份。目前,SpringWorks正在研究可治療多種不同罕見腫瘤的一系列藥物。SpringWorks贊助了兒童腫瘤基金會(Children’s Tumor Foundation)的多場活動。

SpringWorks的例子給BRIDGE計劃(BRIDGE Initiative)帶來了啟發。該計劃致力于加快研發兒童腫瘤治療藥物。兒童癌癥研究中心(CureSearch for Children’s Cancer)、FasterCures和我所在的兒童腫瘤基金會正在合作尋找激勵措施和途徑,鼓勵制藥公司和生物科技公司公開已經終止研發的藥物,或者承擔改變藥物用途的風險。

我們正在研究的一種激勵措施是為公司提供提高環境、社會和治理(ESG)評分的機會,交換條件是對于他們已經終止研發但有潛力治療神經纖維瘤和小兒癌癥的藥物,允許我們改變藥物的用途或回收再利用。環境、社會和治理用于衡量一家公司為改善人類社會和保護地球所做的貢獻。許多公司都渴望提高在這方面的評分。

BRIDGE計劃已經成功找到了投資者,他們愿意資助我們開發被藥企放棄的治療藥物,并將其商業化。

另外一個例子是美國國立衛生研究院(National Institutes for Health)罕見病與被忽視疾病治療項目(Therapeutics for Rare and Neglected Diseases)藥物用途修改篩選小組(Repurposing Screening Group)。該項目與研究人員、非營利組織和制藥公司合作,致力于發現有潛力治療罕見病的藥物,并安排進行臨床試驗。

將更多已經終止研發藥物用于治療罕見病,需要生物科技公司、投資者和非營利組織相互協作。讓這些被放棄的藥物煥發新生,能夠拯救患者的生命。(財富中文網)

本文作者安妮特?巴克是兒童腫瘤基金會的總裁。

翻譯:劉進龍

審校:汪皓

Scientists may have already developed cures for cystic fibrosis, muscular dystrophy, and rare cancers—they just don't realize it yet.

Most people assume that experimental treatments either conclusively fail clinical trials or earn FDA approval. But there are a vast number of medicines whose fate lies somewhere in between.

Some experimental medicines, for instance, show initial promise in clinical trials but are discontinued by drug companies for strategic or financial reasons. Once these drugs are abandoned, the research data is locked away, and the projects are largely forgotten. But with minor tweaks, some of those discarded drugs could be turned into game-changing therapies for currently untreatable rare diseases.

It's not uncommon for drugs developed for one condition to effectively treat a different illness. The antiviral drug amantadine, for instance, was originally developed for influenza, but was repurposed as a Parkinson's therapy. Right now, the anti-inflammatory colchicine—long given to patients with gout—is being investigated as a COVID-19 treatment.

Deciphering which discontinued medicines ought to be repurposed or repositioned isn't easy, of course. Piecing together the relevant data can be challenging, since the original research teams have usually long since disbanded. Pharmaceutical firms are also generally reluctant to spend tens, or even hundreds, of millions of dollars investigating whether a discontinued medicine could treat rare diseases, since the market for these treatments is typically small and running trials on a low number of available rare diseases patients can be challenging.

But given the potential for these abandoned drugs to save lives, the obstacles are well worth overcoming. A number of companies and nonprofits are trying to do just that.

Take the biotech startup SpringWorks Therapeutics. The company spun off from Pfizer in 2017 for the specific purpose of developing compounds that were gathering dust on Pfizer's shelves. SpringWorks was able to create a financial incentive for Pfizer to revisit these compounds, in part by granting the firm a 10% stake in the venture. And right now, SpringWorks is at work on an array of drugs for a number of different rare tumors. (SpringWorks sponsors some Children’s Tumor Foundation events.)

The learnings from the SpringWorks example sparked the BRIDGE Initiative, aimed at speeding up the development of treatments for children with tumors. A collaboration between CureSearch for Children’s Cancer, FasterCures, and my organization, Children’s Tumor Foundation, seeks to find incentives and pathways for pharmaceutical and biotech firms to release their discontinued drugs or take a risk on repurposing them.

One incentive we are exploring is the opportunity for firms to raise their environmental, social, and governance (ESG) scores by offering us the opportunity to repurpose or recycle their discontinued but potentially valuable medicines for neurofibromatosis (NF) and pediatric cancer. ESG is a metric for gauging a firm's contribution to improving our society and protecting our planet. And it's something that many firms are eager to boost.

The BRIDGE Initiative has also had real success finding investors who are willing to finance the development and commercialization of abandoned treatments.

Another example is the National Institutes for Health's Therapeutics for Rare and Neglected Diseases (TRND) Repurposing Screening Group. The program partners with researchers, nonprofits, and pharmaceutical companies to identify drugs with the potential to treat rare diseases, and shepherd those medicines into clinical trials.

It's up to biotech companies, investors, and nonprofits to collaborate and transform more discontinued medicines into rare disease treatments. Giving new life to these discarded drugs could save patients' lives.

Annette Bakker is president of the Children's Tumor Foundation.

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