Bridging the Gap Between Stem Cell Therapy Research and Clinical Trials
Dr. Bo Wiinberg discusses overcoming barriers preventing life-saving cell therapies from reaching clinical trials.
In recent years, stem cell-based cell therapies, which involve the transfer of stem cells or their derivatives into a patient for therapeutic purposes, have provided new hope for patients suffering from incurable diseases. However, despite major advances in stem cell research, many promising candidates never make their way to clinical trials.
Reasons for this gap between research and the clinic include strict regulatory requirements that vary between different countries and a lack of facilities that can manufacture large, consistent quantities of cell therapy products.
Technology Networks spoke with Dr. Bo Wiinberg, chief business development officer at the Novo Nordisk Foundation Cellerator, to learn more about the current obstacles halting advances in stem cell-based cell therapies and what can be done to overcome them. Wiinberg also discusses how the Novo Nordisk Foundation Cellerator is designed to help further develop cell therapies that have already been successfully tested in animals and manufacture these for early clinical trials.
Can you discuss the obstacles in translating successful stem cell research into clinical trials?
Bo Wiinberg, PhD (BW):
When we were developing a pipeline of stem cell-based cell therapies at Novo Nordisk, a lot of what we did was scout existing projects that we could help accelerate and bring to the clinic. We did this with a few projects at the academic stage and it was very apparent that technically there was a lot that had to be altered to get these projects into clinical trials. For example, the researchers might not have chosen a stem cell line that, from a regulatory point of view, would be suitable for a clinical trial. When you must change a factor like that it can take years and cost millions of dollars. Successful stem cell-based therapy development is about making the right choices early in the process development because it’s hard to make changes to these processes retrospectively.
It's also important to look beyond the first human dose, which is a classic way to approach small molecule and biologic development because you can tweak the process based on learnings from phase 1 trials. However, this is very difficult when developing stem cell therapies. It’s important from the beginning of your development process to consider the result, for example, is the process scalable? Is the technology used in development transferable to manufacture? If you get these initial steps wrong, changing things further along the process is almost prohibitively expensive.
Finally, licensing the cell lines and key technologies used to develop the therapy is paramount. If you are a startup and have never gone through these processes before, it can be extremely complex to navigate and get right the first time.
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What we are building is a physical facility, which was originally primarily designed for chemistry, manufacturing and controls development and good manufacturing practice manufacturing, because we needed that infrastructure to be available for startups and spinouts in Denmark. However, as the project developed, we realized that process development is one of the main obstacles to getting these therapies to market. We discovered that before you even get to manufacturing, taking a process that academic researchers have developed and may have tested in animals successfully, and transferring this technology to humans was a huge obstacle.
What we want to provide with the Cellerator is access to experts who can hands-on help developers choose the best technologies and identify any critical failures in existing processes that will need to be changed to get regulatory approval.
It’s not just about building the infrastructure but partnering with technology providers and setting up a team of experts who understand the regulatory landscape and what is needed to get to that first human dose and beyond.
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Everyone in the field hopes to see more cell therapies reaching patients. To achieve this, we need to generate more data from clinical trials to understand what is biologically feasible to inform the next generation of projects. We hope to see the acceleration of stem cell research into clinical trials and not just in the oncology CAR T space. We also hope to see this acceleration where it’s currently harder to get projects into clinical trials such as with regenerative medicine and allogeneic therapies. In addition, because we are a philanthropic organization we hope to bring together biotechnology companies, academics and pharma, in one big collaborative effort to push this field forward.
Driving innovation is a key goal for us. If you look at where the stem cell field is today compared to small molecules and other biological drugs, it's still in its infancy. The cost and risk associated with stem cell therapy development is a major contributor. Many things must happen on the technological front for the costs to come down significantly for producing these therapies. We're now considering how we can support with soft funding for academic groups. We are also considering what we can do to encourage venture capitalists to engage more in this therapy type and become more risk-willing. Being a trusted partner that can help de-risk the entire development process should help with this aim.
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