立即打开
许多罕见病治疗药物或早已存在,为什么没有投入使用?

许多罕见病治疗药物或早已存在,为什么没有投入使用?

Annette Bakker 2021年07月09日
有些药物在临床试验中最初前景良好,但制药公司由于战略或财务上的原因终止了试验。

已经终止研发的药物可以改变用途,用于治疗囊性纤维化和肌肉萎缩症等罕见病。图片来源:Thana Prasongsin—Getty Images

科学家们或许没有意识到,他们早已研发出治疗囊性纤维化、肌肉萎缩症和罕见癌症的药物。

大多数人会认为,试验性药物最终要么没有通过临床试验,要么没有获得美国食品与药品管理局(FDA)的批准。但绝大部分药品的最终命运在这两者之间。

例如,有些药物在临床试验中最初前景良好,但制药公司由于战略或财务上的原因终止了试验。这些药物被放弃之后,研究数据被束之高阁,研究项目也基本被大部分人遗忘。但它们只要经过较小的调整,就能够变成治疗目前一些罕见的不治之症的颠覆性药物。

针对一种疾病开发的药物可以有效治疗其他疾病的情况并不少见。比如,抗病毒药金刚烷胺最初被用于治疗流感,但后来被用于治疗帕金森症。一直被作为痛风药物的消炎药秋水仙素,目前正在进行新冠肺炎治疗试验。

当然,要确定哪些药物应该改变用途或调整定位并不容易。汇总相关数据可能面临挑战,因为原始研究团队通常早已被解散。药企往往不愿意花费数千万乃至数亿美元,调查哪一款已经终止研发的药物能够治疗罕见病,因为这些药物的市场通常较小,而且对数量较少的罕见病患者开展试验很有挑战性。

但这些被放弃的药物有可能拯救生命,因此克服这些障碍是值得的。许多公司和非营利组织正在尝试做这件事情。

例如生物科技初创公司SpringWorks Therapeutics。这是一家在2017年从辉瑞(Pfizer)剥离后成立的公司,其业务是继续开发早已被辉瑞搁置的药物。SpringWorks可以让辉瑞有财务上的动机来重新评估这些药物,比如给予辉瑞10%的股份。目前,SpringWorks正在研究可治疗多种不同罕见肿瘤的一系列药物。SpringWorks赞助了儿童肿瘤基金会(Children’s Tumor Foundation)的多场活动。

SpringWorks的例子给BRIDGE计划(BRIDGE Initiative)带来了启发。该计划致力于加快研发儿童肿瘤治疗药物。儿童癌症研究中心(CureSearch for Children’s Cancer)、FasterCures和我所在的儿童肿瘤基金会正在合作寻找激励措施和途径,鼓励制药公司和生物科技公司公开已经终止研发的药物,或者承担改变药物用途的风险。

我们正在研究的一种激励措施是为公司提供提高环境、社会和治理(ESG)评分的机会,交换条件是对于他们已经终止研发但有潜力治疗神经纤维瘤和小儿癌症的药物,允许我们改变药物的用途或回收再利用。环境、社会和治理用于衡量一家公司为改善人类社会和保护地球所做的贡献。许多公司都渴望提高在这方面的评分。

BRIDGE计划已经成功找到了投资者,他们愿意资助我们开发被药企放弃的治疗药物,并将其商业化。

另外一个例子是美国国立卫生研究院(National Institutes for Health)罕见病与被忽视疾病治疗项目(Therapeutics for Rare and Neglected Diseases)药物用途修改筛选小组(Repurposing Screening Group)。该项目与研究人员、非营利组织和制药公司合作,致力于发现有潜力治疗罕见病的药物,并安排进行临床试验。

将更多已经终止研发药物用于治疗罕见病,需要生物科技公司、投资者和非营利组织相互协作。让这些被放弃的药物焕发新生,能够拯救患者的生命。(财富中文网)

本文作者安妮特•巴克是儿童肿瘤基金会的总裁。

翻译:刘进龙

审校:汪皓

科学家们或许没有意识到,他们早已研发出治疗囊性纤维化、肌肉萎缩症和罕见癌症的药物。

大多数人会认为,试验性药物最终要么没有通过临床试验,要么没有获得美国食品与药品管理局(FDA)的批准。但绝大部分药品的最终命运在这两者之间。

例如,有些药物在临床试验中最初前景良好,但制药公司由于战略或财务上的原因终止了试验。这些药物被放弃之后,研究数据被束之高阁,研究项目也基本被大部分人遗忘。但它们只要经过较小的调整,就能够变成治疗目前一些罕见的不治之症的颠覆性药物。

针对一种疾病开发的药物可以有效治疗其他疾病的情况并不少见。比如,抗病毒药金刚烷胺最初被用于治疗流感,但后来被用于治疗帕金森症。一直被作为痛风药物的消炎药秋水仙素,目前正在进行新冠肺炎治疗试验。

当然,要确定哪些药物应该改变用途或调整定位并不容易。汇总相关数据可能面临挑战,因为原始研究团队通常早已被解散。药企往往不愿意花费数千万乃至数亿美元,调查哪一款已经终止研发的药物能够治疗罕见病,因为这些药物的市场通常较小,而且对数量较少的罕见病患者开展试验很有挑战性。

但这些被放弃的药物有可能拯救生命,因此克服这些障碍是值得的。许多公司和非营利组织正在尝试做这件事情。

例如生物科技初创公司SpringWorks Therapeutics。这是一家在2017年从辉瑞(Pfizer)剥离后成立的公司,其业务是继续开发早已被辉瑞搁置的药物。SpringWorks可以让辉瑞有财务上的动机来重新评估这些药物,比如给予辉瑞10%的股份。目前,SpringWorks正在研究可治疗多种不同罕见肿瘤的一系列药物。SpringWorks赞助了儿童肿瘤基金会(Children’s Tumor Foundation)的多场活动。

SpringWorks的例子给BRIDGE计划(BRIDGE Initiative)带来了启发。该计划致力于加快研发儿童肿瘤治疗药物。儿童癌症研究中心(CureSearch for Children’s Cancer)、FasterCures和我所在的儿童肿瘤基金会正在合作寻找激励措施和途径,鼓励制药公司和生物科技公司公开已经终止研发的药物,或者承担改变药物用途的风险。

我们正在研究的一种激励措施是为公司提供提高环境、社会和治理(ESG)评分的机会,交换条件是对于他们已经终止研发但有潜力治疗神经纤维瘤和小儿癌症的药物,允许我们改变药物的用途或回收再利用。环境、社会和治理用于衡量一家公司为改善人类社会和保护地球所做的贡献。许多公司都渴望提高在这方面的评分。

BRIDGE计划已经成功找到了投资者,他们愿意资助我们开发被药企放弃的治疗药物,并将其商业化。

另外一个例子是美国国立卫生研究院(National Institutes for Health)罕见病与被忽视疾病治疗项目(Therapeutics for Rare and Neglected Diseases)药物用途修改筛选小组(Repurposing Screening Group)。该项目与研究人员、非营利组织和制药公司合作,致力于发现有潜力治疗罕见病的药物,并安排进行临床试验。

将更多已经终止研发药物用于治疗罕见病,需要生物科技公司、投资者和非营利组织相互协作。让这些被放弃的药物焕发新生,能够拯救患者的生命。(财富中文网)

本文作者安妮特•巴克是儿童肿瘤基金会的总裁。

翻译:刘进龙

审校: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.

最新:
  • 热读文章
  • 热门视频
活动
扫码打开财富Plus App