Clinical trials for cell and gene therapies (CGT) present unique challenges as they require special resources and training.

Martin Lachs

Vice President, Project Management

Brandon Fletcher

Cell and Gene Therapy Principal

Olivier Saulin

Cell and Gene Therapy Principal

Tamie Joeckel

Cell and Gene Therapy Business Lead

In a recent podcast interview, our experts Brandon Fletcher (Cell and Gene Therapy Principal), Tamie Joeckel (Cell and Gene Therapy Business Lead), Martin Lachs (Vice President, Project Management) and Olivier Saulin (Cell and Gene Therapy Principal) discuss the intricacies of CGT development, the solutions that will enable CGT development on a global basis, and the future of CGT clinical trials.

Q: What are the unique challenges posed by manufacturing and developing CGTs?

ML: There’s a whole raft of challenges in the development space – from logistics to manufacturing to patient safety to the limitation on facilities that can actually administer these drugs. One challenge is that no two studies are necessarily alike. For example, you can’t assume that autologous studies are the same as allogeneic studies, or that the needs within them are the same. There are lots of commonalities, of course. You’re still talking about a living therapy. But when it comes to processes, such as apheresis, there are different nuances that have to be taken into account.

Q: What patient recruitment challenges have you faced, in particular for oncology and the rare diseases?

ML: When we started doing these trials about five years ago, it was very easy to find willing patients because the results were very hopeful, and they continue to be in terms of response rates and durability. But now, the space has become quite congested. At the same time, there are alternative and cheaper treatments that are more accessible to a broader number of physicians and hospital institutions. This competition, not just from the CGT space, but also from other therapies, such as immunotherapies and so on, is putting pressure on enrolment capabilities and capacities.

TJ: The implications of rare disease patient recruitment are obvious: we’re looking at an extremely small patient population. Not only rare, but in many cases ultra-rare. It’s really important to understand where these patients are located. For example, when we were starting a study on severe combined immune-deficiency, we worked very closely with patient advocacy groups. These groups are already providing support services to these patients and know where these families are and they can help us build awareness within the community about the availability of the trial.

Q: How is trial design evolving to meet the needs of the CGT space, and what are the biggest innovations you are seeing in this area? 

OS: One of the most innovative trials we recently worked on involved evaluating patients based on the presence of cancer-specific antigens in solid tumors, regardless of the tumor type. The study tested the effect of drugs in a variety of cancer histologies, which allowed us to test a lot of different tumor types. This, in turn, helped us to quickly determine which specific tumor type to concentrate on. 

ML: In terms of oncology development and beyond, we’re seeing that trials are becoming more registration-focused at an earlier phase. There’s a move towards more adaptive designs, such as basket type trials, umbrella trials and platform trials. We’re trying to combine as many treatment groups within the framework of a single trial as possible.

Q: What would you consider to be the hot topics in CGT development, and how can CROs, in particular, be involved in addressing them?

ML: Cost is probably the largest tangible challenge, if you look into the commercialisation of what are otherwise extraordinarily exciting technologies. Not just the cost of doing trials, which is not trivial, but also the cost of the product once you get close to commercialisation. When you factor in the additional costs of hospitals administering treatment and general care for the patient, they have an even higher price. A lot of development is targeted towards managing that cost downwards, whether it’s through allogeneic therapies, which arguably should be a lot cheaper, or in other ways. 

Realistically, we can work to support companies as we do because we have our own pricing, market access, and commercialisation groups who are experts at looking at things such as net present value and doing full evaluations on potential. 

Q: Looking to the future, what do you predict as the key challenges and opportunities for the CGT sector within the next 5 to 10 years?

BF: A strong focus will be on streamlining logistics and reducing cost to truly support the opportunity of access. In oncology, the field still awaits a clear demonstration of clinical efficacy of CGTs in solid tumors. This challenge is becoming a defining issue in cellular immunology as the new decade begins. Solid tumors make up almost 80 percent of all cancers, and the key challenge is creating and managing therapies that can manipulate and/or withstand the inhospitable environments of these tumors, known as the tumor microenvironment. In the future we’ll see a focus on bringing these therapies to solid tumors in a meaningful and effective way.

Our knowledge of disease, the genome and tissues still far outweighs our ability to respond from a medical technology perspective. In the CGT world, the emergence and elevation of gene editing technologies is key. As gene therapies and genetically modified cell therapies leverage these emerging gene editing technologies, coupled with ever more optimal material resources, the future of CGTs is more than just promising – it’s tangible.

Learn more about our offerings for CGT trials, or contact us to speak to one of our experts.

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