WIPO China: H3D: Africa’s First Integrated Drug Discovery and Development Platform
本文原载于2024年第1期《WIPO杂志》。
The article was originally published in the issue 1/2024 of WIPO Magazine.
图：H3D｜Photo: Courtesy of H3D
凯利·切贝莱是开普敦大学(UCT)的有机化学教授，担任以非洲为中心的药物发现与开发的内维尔·伊斯德尔教席，同时还是非洲首家综合性的药物发现与开发中心H3D的负责人。H3D于2010年4月在开普敦大学创办，重点关注转化医学，从实验室的早期医学发现到临床环境中的患者治疗均有涉猎。最近，《WIPO杂志》对切贝莱进行了访谈，以便进一步了解H3D以及知识产权在开创性工作中的作用。
Kelly Chibale, a Professor of Organic Chemistry at the University of Cape Town (UCT), holds the Neville Isdell Chair in African-centric Drug Discovery & Development and is also Director of H3D, Africaʼs first integrated drug discovery and development center. H3D was founded at UCT in April 2010 and focuses on translational medicine, which involves early-stage medicine discovery in the lab through to the treatment of patients in clinical settings. WIPO Magazine recently sat down with Chibale to learn more about H3D, and the role intellectual property plays in its groundbreaking work.
《WIPO杂志》:非洲的药物发现有什么潜力?
WM: What is the potential of drug discovery in Africa?
凯利·切贝莱:可以说，非洲是最具基因多样性的大陆。所有人都来自非洲，然后走向其他地方。这意味着疾病不是非洲特有的问题或者非洲人的疾病，它们是人类的疾病，人类的问题。因此，非洲的药物发现具有为人类作出贡献和创造当地就业的巨大潜力。
KC: Africa is arguably the most genetically diverse continent. Everybody came from Africa and went somewhere else. That means diseases are not African problems or African diseases, they are human diseases, human problems. So, drug discovery in Africa has huge potential to contribute to humanity and to create local jobs.
H3D如何影响非洲的卫生创新?
And how is H3D affecting health innovation in Africa?
H3D正在各个层面产生影响，特别是通过创建可进一步开发的并能推动全球创新产品线的药物发现基础设施和平台。
H3D is having an impact at various levels, particularly by creating drug discovery infrastructure and platforms capable of contributing to the global pipeline of innovative products that could be further developed.
图：hadynyah/E+｜Photo: hadynyah/E+
换言之，我们加强了自身将基础科学知识转化为潜在救命药物的能力。我们正在缩小实验室研发和患者治疗之间的差距。
In other words, we have strengthened our capacity to translate basic science knowledge into potential life-saving medicines. And we are bridging the gap between the lab and the patient.
您最初重点关注疟疾，为什么?
You focused initially on malaria. Why?
疟疾为我们建立转化医学所需要的基础设施提供了机会。归根结底，除了对人类疟疾寄生虫的生物特性进行了解，疟疾或癌症的药物发现原理是相同的。例如，不管什么疾病，除其他项外，共同的目标是要了解人体应对候选药物的方式。
Malaria was an opportunity for us to build the infrastructure required for translational medicine. At the end of the day, beyond understanding the biology of the human malaria parasite, the drug discovery principles are the same for malaria or cancer. For example, regardless of the disease, among other things, the common goal is to understand how the human body will handle the drug candidate.
图：H3D｜Photo: Courtesy of H3D
就药物发现而言，H3D正在关注的是行为研究，以确定生物靶点，并更好地了解这些靶标生物对药物的耐药性机制。
In terms of drug discovery, H3D is currently focusing on action studies to identify biological targets and better understand the mechanism of resistance of these targeted organisms to drugs.
疟疾项目是与疟疾新药研发公司(MMV)开展合作以及后续与默克公司和比尔及梅琳达·盖茨基金会等新合作伙伴接触的良机。我们一开发出项目所需要的基础设施，便开始将包括结核病在内的其他疾病以及抗微生物耐药性添加进来。2022年，我们有机会作为强生公司的三个全球健康发现卫星中心之一与之合作。总之，疟疾是一个支柱项目，它能够让我们获得我们希望发展的技能和经验，然后将这些技能和经验转移应用到其他疾病。
The malaria project was an opportunity to work with the Medicines for Malaria Venture (MMV) and to subsequently engage with new partners, such as Merck and the Bill and Melinda Gates Foundation. Once we developed the infrastructure we needed for that project, we began adding other diseases, including tuberculosis (TB), and antimicrobial resistance. In 2022, we had an opportunity to work with Johnson & Johnson as one of the company’s three satellite centers for global health discovery. In sum, malaria was an anchor program that enabled us to acquire the skills and experience we wanted to develop, and which we then transferred to other diseases.
此类伙伴关系对H3D的工作以及对在非洲建立健全的卫生创新生态系统具有怎样的重要意义?
How important are such partnerships to H3Dʼs work, and to developing a robust health innovation ecosystem in Africa?
伙伴关系非常重要，即使对于有财务实力的创新制药公司也是如此。事实上，它们提供的一些产品组合包括从第三方获得许可的候选药物。这让它们能够降低药物开发早期阶段的风险。
Partnerships are extremely important, even for innovative pharmaceutical companies with financial muscle. Indeed, some of the product portfolios they offer include drug candidates licensed in from third parties. This enables them to de-risk the early stages of drug development.
对H3D而言，伙伴关系从一开始就很重要，原因有三个。首先，能够应对基础设施的挑战;其次，能够建立我们所需要的技术平台;第三，能够得到技术熟练的人。
For H3D, partnerships were important from the start, for three reasons. First, to tackle infrastructure challenges; second, to build the technology platforms we needed; and third, to access skilled people.
伙伴关系对于获得资助也至关重要。当你的项目受到全球支持时，你就会吸引目标相同的合作伙伴，资助就会增加，你就有机会进入一个卓越中心的网络。因为每个人都对项目成功感兴趣，所以伙伴关系能带来你所没有的东西。在共同利益的作用下，你可以大有作为。
Partnerships are also important to secure funding. When you have a project with global support, you attract partners who share the same goals, funding grows, and you gain access to a network of centers of excellence. Partnerships can bring to the table what you don’t have, because everyone is interested in the project’s success. When there is mutual interest, you can make a huge difference.
建立当地采购支持系统有什么重要性?
What about the importance of building a local procurement support system?
非洲科技创新的一个主要障碍是缺少广义上的基础设施。这包括一个当地的采购支持系统，其中配备有运转良好的实验室，在某些东西发生故障时获得你所需要的备件，方便和快速地获取试剂和化学制品的能力，等等。
One of the main barriers to scientific innovation in Africa has been a lack of infrastructure in the broad sense. This includes a local procurement support system with functioning laboratories, access to the spare parts you need when something breaks down, the ability to access reagents and chemicals readily and rapidly, and so on.
当然，从商业角度看，我们需要能证明商业合理性的规模。目前，参与者过少，商业机会有限。因此，例如，我们正在努力扩大社群，以创造需求，培育我们为研发提供必要化学品和试剂所需要的企业。
Of course, from a business perspective, we need scale that justifies the business. At present, there are too few players, so business opportunities are limited. That’s why we’re working to expand the community to create the demand that will foster the businesses we need to supply the chemicals and reagents required for research and development, for example.
知识产权在所有这些方面发挥了哪些作用?
What is the role of intellectual property in all this?
在医疗需求得不到满足时，你就必须要创新，而知识产权可以激励创新。知识产权是一个助推器，支撑着强大的创新生态系统。
When there’s an unmet medical need, you have to innovate, and IP incentivizes innovation. IP is an enabler and underpins robust innovation ecosystems.
例如，资金短缺的大学可以利用知识产权，通过大学衍生企业从其研究中创造新的收入来源。知识产权还可以吸引投资。人们愿意向尊重规则和法律(包括知识产权)的国家投资。
Cash-strapped universities can use IP to generate new sources of income from their research, through university spinouts, for example. IP is also a magnet for investment. People want to invest in a country where there is respect for rules and laws, including IP.
在非洲商业回报低的传染病领域还需要知识产权吗?
Do you still need IP in Africa for infectious diseases, where commercial returns are low?
绝对需要。因为知识产权是一种责任，即使对于人们认为商业回报低的传染病也是如此。如果没有知识产权，你就会陷入混乱。谈及健康公平，重要的是要记住，拥有知识产权的人可以决定是否自愿进行分享。
Absolutely. Because IP is also a responsibility, even for infectious diseases where commercial returns are perceived to be low. Without IP you would have a free-for-all. When it comes to health equity, itʼs important to remember that the person who owns the IP can decide whether to share it voluntarily or not.
当你拥有一种药物的知识产权权利时，你就可以在某种程度上控制其使用。这正是我们在非洲需要拥有知识产权的原因。当我们拥有它并找到合适的合作伙伴向前推动知识产权时，我们就会得到回报。我宁愿拥有十亿的百分之一，也不愿拥有零的99.99%。
When you hold IP rights in a medicine, you can control its use to some extent. Thatʼs why, in Africa, we need to be owning IP. When we do, and we find an appropriate partner to take the IP forward, we get a return. I would rather own 1% of one billion than 99.99% of zero.
✦
知识产权也是一种责任，即使对于人们认为商业回报低的传染病也是如此。
IP is also a responsibility, even for infectious diseases where commercial returns are perceived to be low.
✦
H3D当前的工作重点是什么?
What is the current focus of H3Dʼs work?
就药物发现而言，我们正在关注的是行为研究，以确定生物靶点，更好地了解这些靶标生物对药物的耐药性机制。这些生物非常聪明。我们要做的工作是通过计谋打败它们。
In terms of drug discovery, weʼre focusing on action studies to identify biological targets and to better understand the mechanism of resistance of these targeted organisms to drugs. These organisms are very clever. Our job is to outsmart them.
您仍认为需要采用新办法吗?
Do you still see the need for new approaches?
是的。在科学层面上，我支持以非洲为中心的药物发现。你需要找到一个打击目标——一种酶或一种蛋白质——由于遗传原因，该目标在不同的人群中可能会作出不同的反应。
Yes. At a scientific level, Iʼm an advocate for Afro-centric drug discovery. You need to find a target to hit – an enzyme or a protein – which may respond differently in different populations for genetic reasons.
✦
药物开发需要将关注点从一刀切转向以人群为中心的方法。
Drug development needs to move from a one-size-fits-all focus towards a population- centric approach.
✦
药物代谢酶在表达和活性方面的遗传差异可导致对各种疗法的不同反应。例如，与其他人群相比，由于基因突变，负责抗逆转录病毒药物依非韦伦代谢的酶在非洲裔人群中起效较慢，而且如果剂量调整不当，可能会因药物过量而导致毒性，甚至死亡。所以，药物开发需要将关注点从一刀切转向以人群为中心的方法。
Genetic differences in the expression and activity of drug-metabolizing enzymes can lead to variable responses to therapeutics. For example, in people of African descent, due to genetic mutations, enzymes responsible for metabolizing the antiretroviral drug Efavirenz work more slowly than in other populations and this can result in toxicity, even death, due to drug overdose if dosages aren’t adjusted appropriately. So, drug development needs to move from a one-size-fits-all focus toward a population-centric approach.
就我们追求的生物药物靶点和负责特定药物代谢的酶而言，我们确实需要投入资源以便了解非洲人口的基因特征。
We really need to invest in understanding the genetics of the African population with respect to biological drug targets we go after and the enzymes responsible for metabolizing specific drugs.
此外，我们还需要解决转化医学的资金缺口问题，许多投资者认为这方面风险太大。这将需要进行政策变革，以鼓励投资者将药物开发视为在价值链的每个阶段均需要投资的一个连续过程。这将会创造分享风险和收益的机会，并且最终惠及每个人。
Also, we need to address the funding gap in translational medicine, which many investors find too risky. This will require policy changes to encourage investors to see drug development as a continuum that requires investment at each stage of the value chain. This would create opportunities to share both risks and benefits, and ultimately will benefit everyone.
在线阅读访谈全文，进一步了解切贝莱对非洲发展有力的卫生创新体系的建议。
Read the full interview online and learn more about Chibaleʼs recommendations for developing a robust health innovation system in Africa.
欢迎扫描下列二维码进行WIPO杂志订阅！
Scan the QR code below to subscribe to WIPO Magazine!
订阅步骤：
Simply follow these steps:
1. 在第一步中选择"WIPO杂志“；
Select "WIPO Magazine" in the step 1；
2. 在第二步中填写必填项信息；
Fill in the required information in the step;
3. 根据个人邮箱收到的验证邮件进行确认；
Confirm your subscription by clicking the verification link in the email sent to your inbox.
订阅成功，阅读愉快！
That's it! You're now subscribed to WIPO Magazine. Enjoy your reading!
点击阅读原文查看关于WIPO中国办事处的更多信息。
Click on Read more for more information about the WIPO Office in China.
