As a scientist seeking funding to do rare disease research, Olivier Menzel confronted the lack of interest from funding sources. He eventually created the Blackswan Foundation to support research on any type of rare disease. Since then, the foundation has held scientific conferences, raised awareness about rare disease, and been involved in a large number of projects and collaborations around the world. The foundation also created the RE(ACT) Community, a crowdfunding and knowledge-sharing digital platform that connects researchers, patients, and other rare disease stakeholders. We spoke to Menzel, chairman and founder of the Blackswan Foundation, about the challenges of rare disease research, how it has worked to address common obstacles, and how it is serving as an accelerator to bridge the gap between basic scientific and commercial research.
Daniel Levine: Olivier, thanks for joining us.
Olivier Menzel: Thank you for having me.
Daniel Levine: We’re going to talk about the Blackswan foundation, the challenges of rare disease research, and how your organization is working to address some of those. Most of our listeners may not be familiar with Blackswan. Perhaps we can begin with you and your decision to create the organization. You were seeking funding to do gene therapy into a set of rare metabolic conditions. What happened and how did that lead to your decision to create black Swan?
Olivier Menzel: I’m a biologist by training and I used to do research in the academic settings. Because I was still interested in genetics and human genetics, very rapidly, I was confronted with rare diseases because most of them are of genetic origin. And because I also had the chance to start my academic career just before the Human Genome Project, the only way to discover new genes and to understand how they work was thanks to the families that were carriers of rare diseases. At that time, I was young and enthusiastic, and I was already surprised that there was no money to push a little bit forward the research once we identified the genes to try to find a therapeutic solution for those families. And then years passed on, I become less young, but always still enthusiastic, and unfortunately, the problem didn’t change. So, a lot of money for fundamental research, but very difficult to find money, at least from government, to support research in order to bring new therapeutic solution to patients. And once, when we had a very nice result on rare liver metabolic diseases when I used to run this laboratory, when we asked for a second round of funding, in fact, it was refused because our project was considered too ambitious. And so, to me, it was quite surprising because when you are too ambitious, it’s normally it’s something positive, not negative. So, this was really the moment that I decided to create the Blackswan Foundation, which mainly the aim is to support orphan disease research worldwide.
Daniel Levine: The foundation isn’t focused on any single rare disease. It’s involved in a number of initiatives. How would you describe the vision and mission of Blackswan?
Olivier Menzel: As you as said before, the mission is really to support research on rare diseases. And this is not only by giving money to laboratories, to PIs, to researchers, but it’s also by supporting researchers and put them in contact. So, our mission is also to create an international community of researchers, because when we are considering the numbers of patients living with diseases, they are not so rare those numbers, but researchers or scientists that invest their own career for rare disease research, those are really rare because it’s not the best choice of career that you can do. Of course, according to the “publish or perish” law in the laboratory, unfortunately, rare disease is not a subject that you publish a lot. And by consequence, you don’t find a lot of money to pursue your research.
Daniel Levine: Black Swan is working to address many of the obstacles rare disease researchers face. I’d like to have you walk through a few of those. What are the scientific obstacles to rare disease research?
Olivier Menzel: The scientific obstacle, besides the fact of a constant lack of money to do this research, can be missing some knowhow because there are some very specific diseases that you need to be an expert as a researcher in order to address them. And for that reason, working hand in hand with patients is the best solution, because in the end, the expert is the person that is living with the rare disease. Another obstacle can also be trying to find collaborators, other people that are working on the same diseases in order to share expertise, to share knowledge. Again, this is a matter that we address at the Blackswan Foundation by organizing the React Congress, which is an international congress on rare disease research, where the idea is to bring together all those researchers from around the world and put them in the same room and try to push as much as we can any kind of collaboration to maximize the lack of money that’s present in those laboratories. And just share the experience and know how to bring new solutions for patients.
Daniel Levine: You’ve talked about your own experience with the financial barriers to rare disease research, but how accessible are research funds for rare disease?
Olivier Menzel: It’s not so easy, I’m talking more about Europe. In the U.S., I must say that there are more opportunities. In Europe it’s quite difficult even though, the policies are changing, but it’s not so easy. We are still very concentrated on what we call common diseases, the big group of diseases that concern a lot of people for the same diseases, which to me is quite annoying and surprising because we know from years of field experience that by investigating rare diseases, most of the time the outcomes and the solutions that come out from this research are not only good for people that are living with these rare diseases, but they are very useful for the general public, for the common disease effort in the international scientific community.
Daniel Levine: Alas, there are commercial barriers. How significant are these two in enabling the development of potential therapies for rare diseases?
Olivier Menzel: It’s the same problem. The industry wants to have a return on investment. So, if they invest in a project, they want to be sure that they have enough patients to sell the treatment, the drug, or the molecule that they produce. This is the first barrier, although we see some new companies out there that the business model is really based on rare diseases and it works. Of course, I will not enter this debate about the price of the therapies, but it’s a model that can exist. The problem is that there is a death valley between the academic research and the industry. Academic research is really too early to be interesting for the industry for that reason. We also invest and create an institute that wants to fulfill this death valley, which means we want to valorize those academic projects to enter in a drug discovery and development process in order to be interesting for the industry and see if there are any opportunities to give them to the industry in order to develop new treatments and put it in on the market for patients.
Daniel Levine: Rare disease research still faces a number of challenges. But do you think the landscape for doing this research has changed, or do you still think the types of obstacles you were trying to address when you created Blackswan are still firmly in place?
Olivier Menzel: The landscape is changing. As I said, there are plenty of international and national organizations and agencies that address more and more rare disease research. I must say that there is also more implication from the patient side, which in the end, they are the expert. They are also the ones that normally start a new research project because they are able to put in a network of different researchers from around the world, but they are also able to fundraise the money that’s necessary to start those projects. So, the landscape is changing, unfortunately, not for all diseases because rare diseases are spanning between one out of 3000 persons up to one out of one million. Of course, the less cases out there, the more difficulties to support research. But there is, at least from my experience, a more sensitivity also from the public about those diseases. And so, it’s easier a little bit to fundraise, to support research from that side.
Daniel Levine: What’s the case you’d make for investing in research into rare disease and its potential beyond improving the understanding of a specific condition?
Olivier Menzel: As I said before, investing in some particular diseases, most of the time gives insight and outcomes that are very useful for other common diseases. I know about projects on narcolepsy, for instance, which is a sleep disorder, the outcome can be very useful for Alzheimer’s or Parkinson’s. So, I think this is the angle that we need to approach these diseases. We always have to defend why we want to push further these diseases, but again I think 95 percent of the drugs that are out there can be used also for common diseases. So again, supporting rare disease research is beneficial for everybody.
Daniel Levine: I think if there are themes to what Blackswan has done in its efforts, it’s been an emphasis on fostering both information sharing and collaboration within the world of rare disease. Why is information sharing and collaboration so critical?
Olivier Menzel: It’s fundamental for a few different reasons. The first one, as I said, the researchers that are committed to rare disease research, there are not so many out there and it’s not a choice of a career that it’s very smart if you want to evolve in an academic field. But there are people that are very committed and the collaboration is quite outstanding within those kind of researchers. In fact, it’s the opposite of the scientific world, where normally collaboration is only if there is an interest or a win-win situation between researchers. In the case of rare diseases, most of the researchers are seeking collaboration because it’s the only way to survive to bring the project further in a way that can really be at a certain point on the bedside of the patients. The other approach to getting together all these researchers, it’s so they can really benefit from the patient experience directly. We are talking more and more in Europe, especially about PPI, which is patients and public involvement. And it was demonstrated that the [sic. participation] of patients from the beginning of a research project will increase the permanency and the validity of the research.
Daniel Levine: Have you found any cultural or institutional barriers to information sharing or collaboration?
Olivier Menzel: Not really. I don’t see any barrier of trying to collaborate on rare diseases. Everybody, all the stakeholders from the patient perspective to the physician researcher, are really keen to explore more, to know more about the different topics on a particular disease. Because in fact, in the end, they all have an interest in knowing more because they can help in a better way a patient, they can try a new strategy in the research process. They can maybe benefit from a physician that on the other side of the world because they try a new flavor or compound that can be used to increase the quality of life of patients. So it’s a highly collaborative field and I’m very happy about that.
Daniel Levine: Well, let’s talk about some of the specific projects at Blackswan. You have two specific initiatives I’d like you to talk about and how they’ve advanced your greater goals. The first is the React Congress. What is this?
Olivier Menzel: The React Congress was an idea that I had back in 2011, because I was a researcher and I used to work liver metabolic diseases. And I participated once, one full week in a huge congress on liver diseases in general and I had the chance to have an oral presentation, which means that the project that I presented was recognized as a little bit innovative and good by my colleagues and when I had the chance to present it, even though there were more than 6,000 participant in the room, there were less than 100 people. In fact, I came back from this full week of science immersion in the liver world, without creating any contacts, any knowledge in the liver world, because was rare diseases of the liver were not a subject that were of interest for this kind of congress. It was a pity because as a laboratory, we spend money to be there, I took time to go there. And the benefits of being there for my research project was zero. So, the idea of the React Congress, it’s really to bring together researchers working on rare disease in general, which they have the same problems—lack of patient, lack of money, but they also have an enormous knowhow that can be beneficial for other rare diseases. And I remember having breakfast where one researcher was complaining because he spent three years trying to find some cells to test a new compound. And the guy that could give him the cells was always saying no. And in fact, on the next table, there was a patient that said, oh, look, I know another researcher that probably has cells, that can be interesting for you. And I know that they start a huge collaboration on that project. So, I think that putting together people that are not used to meeting because they are going to other scientific congresses, from an intellectual perspective in order to give more chance for research projects—it’s a huge idea. And in the end, we had six editions with average of 300 participants from all around the world. And I know that we have an average of between 15 and 20 new collaborative projects that started just because people met at the Congress.
Daniel Levine: You’ve also launched the React Discovery Institute. What is the Discovery Institute and how does it work?
Olivier Menzel: The React Discovery Institute is a virtual institute, which means that we don’t have wet labs, but we are just managing and finding projects. The idea is to find an academic project or from small startups that arrive at a certain point that they are missing some industrial skills in order to enter in a drug development or drug discovery phase. And the idea is that we create an international effort in having three different boards that are able to bring counseling advice and also networking in order to valorize this project. Of course, we put money on those projects. We do fundraising, specifically for those projects that have been internalized in the Institute. And the idea is to valorize those projects until [they reach] a level that can be become interesting for the industry side, to find an industry partner that will be able to put the treatment or the solutions or the compound on the market in order that can be used by the patients.
Daniel Levine: You talk about the Institute being an accelerator to fill the gap between public funded, basic science research, and commercially supported, applied research and development, bridging that valley of death you referred to earlier. How does the Institute determine what it works on?
Olivier Menzel: So normally, we are open, again, we are not focused on a particular disease. In fact, it should be of course, a physical project, but as I said, we have three advisory boards. One is composed by international scientists that can review the scientific part, but we also have two other boards that are very important. One’s composed by patients because patient perspective’s very important for those kind of projects. And the third one is an industry advisory board that help us in shaping the project to maximize the chance to arrive at a level of the project that can be interesting for the private sector.
Daniel Levine: How do you leverage relationships with partners to accelerate research through the Institute?
Olivier Menzel: So, by using the huge expertise within our boards, but also because we are creating a network of external partners that can share their services in accelerating the projects, like CROs or other academics that have the expertise, which is necessary to advance the project that we are interested in. So, it’s a really huge, collaborative, and international effort in order to push further the project that we internalized.
Daniel Levine: Is the goal to de-risk investment in potential therapies where common commercial entities will step in, or is there a different vision for bringing these therapies to market?
Olivier Menzel: We are open to all kind of settings. Ideally, we would like to start with funds that are coming from donations just to keep our independence and the transparency of the project, but we are aware that at a certain point, the amount of money that is necessary can only come from the private sector. So for that reason, for each project, we are open to an investor that can be interested then in taking over the project and bringing it to the market. Even though we are a not-for-profit organization, the idea is that thanks to this step, the money that we could obtain from these kind of agreements will be shared by the researchers and the inventor of the project and the Institute that will reinvest it in other projects,
Daniel Levine: Olivier Menzel, chairman and founder of the Blackswan Foundation. Olivier, thanks so much for your time today.
Olivier Menzel: Thank you, Dan. It was a pleasure to be with you.
This transcript has been edited for clarity and readability.
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