In 2009 I was a 3rd-year Medical Biology student, following different courses across the biology spectrum, and trying to figure out which Master I wanted to do. It was also the year I first heard about engineering immune cells to treat cancer, and about ‘tiny organ-like structures in a dish’, now commonly known as organoids. The common denominator between those inventions is that they can lead to treatments and diagnostic tools that are based on a patients’ own cells, and that they are highly complex. I still vividly remember thinking: “This sounds amazing. Where will these developments take us ten years from now?”
At the time, I was unaware that 2009 was the year in which the first cell-based therapy was approved in the EU. This registration marked the entry of complex medicinal products (also known as ATMPs) on the market. Since then, ten other cell-based therapies have been approved for commercial use within the EU. These include the engineered immune cells I heard about when I was in college. Research on organoids also took off after 2009. Currently their added value is explored across a number of conditions.
Treatments and tools based on patients’ own cells provide tailor-made solutions for patients with diseases that might otherwise go untreated. On the other hand, they are vastly complex. This complexity creates hurdles that make it notoriously difficult to get these treatments and tools available for patients.
To spark new ideas and discussion on this topic, I invited Dr. Marwin Velders (chairman HollandBio board and former Vice President Operations & Managing Director of Kite EU B.V., a Gilead Company) and Prof. Kors van der Ent (Professor in Paediatric Pulmonology and chair of the Child Health program at UMC Utrecht) to our last Lygature Partnerships Meetup. I asked them to share their views on and experiences with cell-based therapies and diagnostics.
During the session, it became clear once again that the road of bringing a novel pharmaceutical therapy to the market can be bumpy. There are several hurdles to overcome before a market authorisation is granted, and also post-authorisation there are challenges for potential new therapies. For example, the cell manufacturing process is patient specific, which means that the quality control tests need to fit this specificity. The manufacturing process is also very complex. A high level of communication between parties along the manufacturing pipeline is required, and solid chains of identity and custody are needed in order to deliver the final product.
Prof. van der Ent shared his experiences in using organoids for Cystic Fibrosis (CF) drug screening. This disease is caused by mutations in the CFTR gene, of which over 2.000 mutations are currently known. Finding the right medicine for a patient can be a frustrating process of trial and error, and organoids carry the potential to improve this. Prof. van der Ent and his group have developed an organoid model for CF. These organoids offer an improved personalised approach for patients with rare variants of the disease and can potentially improve the screening pipeline of pharmaceutical companies. One of the big hurdles in this process is that it can be difficult to find the right organisation or government body to discuss the further implementation of organoids.
In biomedical research, the active engagement of patients in research and drug development is a relatively new. Dr. Velders and Prof. van der Ent explained that they engage patients and patient organisations in their research activities. They shared examples of how creating an active dialogue between researchers and patients brought value to their work. Again, ten years ago I would have never imagined the shift that took place. In the past, researchers and physicians were seen as the authorities in disease conditions. Nowadays, it is increasingly acknowledged that patients carry valuable expert knowledge because they experience a disease on a daily basis. Creating a dialogue between researchers and patients is therefore highly relevant if we want to develop meaningful therapies.
One of my professional dreams is to build a partnership that supports bringing cell-based therapies and tools from the pioneering field to practice. An independent partner, such as Lygature, can help to safeguard the different interests of the different stakeholders. We have a similar role in Oncode Institute, a public private partnership that aims to boost the development of cancer therapies in the Netherlands. We can also help organise the patient voice, as we do for example in collaboration with Alzheimer Europe in RADAR-AD.
By doing so, I hope that ten years from now we will have access to therapies we cannot even imagine today. Feel free to contact me if you would like to discuss this dream further.