For the first time, cell and gene therapy, a treatment particularly promising for poorly and unmet diseases, can be said to have a bright future.
Cell therapy is the administration of healthy, living cells into a patient, with the origin of these cells being either autologous—from the same individual—or allogeneic—from another individual. Gene therapy is the modification of gene expression to repair abnormal genes. (Source: American Society of Gene and Cell Therapy)
While these therapies are gaining traction and attention, short delivery windows, changing regulations, and storage and temperature requirements are just a few challenges that plague the cell and gene therapy supply chain.
World Courier, a leading designer of supply chain programs, is no stranger to these challenges. For more than 45 years, the company has provided specialty logistics for the transport and storage of time- and temperature-sensitive products, and now they’re taking on cell and gene therapy.
Pharmaceutical Processing recently contacted Simon Ellison, cell and gene therapy service director at World Courier, to discuss the importance of a personalized supply chain in cell and gene therapy and the logistics platform World Courier is developing to address some of its most pressing hurdles.
Q: Can you speak to the merit and promise of these therapies for a moment? Why is this something to pay attention to?
Simon Ellison: The beauty of these things is their ability to meet poorly or unmet medical needs. There are patients out there that have no other options, whether that’s because its stage four cancer or some genetic disease. I think the beauty of the therapy is that they’re using the body as the therapy. The immune system is an awesome tool, but sometimes it just needs to be taught which cells to target.
Q: What is important to know about the personalized supply chain in cell and gene therapy?
Ellison: It depends on the therapy you’re talking about because at the moment, they’re autologous therapy. So they’re your cells going through a clever manufacturing and modification process. That’s personalized. But if it’s allogenic, that’s much closer to your traditional pharma system where you’re making up a master and working cell bank and you’re distributing your therapy.
However, you have a similar challenge in that they probably have defined patients. You can’t put the wrong therapy into the wrong person. You can’t really do that with pharmaceutical drugs either, but I think that the impact is twice as big with cell therapy. If you put your cells into me, it could have catastrophic impacts on me and if I receive your therapy, it means you didn’t, and so that’s twice the downside.
You’ve got to get your cells back because they’re toxic to everyone else is what it boils down to.
What’s going to be important moving forward with this supply chain is traceability and control. Traditionally, clinical trials are managed by experts and then it goes into the standard pharmaceutical supply chain, and patients and pharmacies have their drugs delivered to them. With cell and gene, the focus on the supply chain will be very similar.
World Courier is creating a logistics platform. I like to equate it to a car manufacturing plant. Different makes and models go down the production system, one after the other. The robots know which doors, windows, and so on, to put on each car. We’ve created a platform where you can put any therapy at any temperature through the production line, and it will be delivered to the correct patient or clinic, standardizing it and putting control into it.
Q: What do you foresee the future of cell & gene therapy looking like? What can people in the industry expect?
Ellison: I think it’s set to grow. And this is probably first time people in the industry can say that on solid ground because until things started commercializing at the end of last year, we had no proof.
I can see more growth in the autologous market, but long term, people are probably going to be looking for allogeneic, just because the cost of goods and the practicality of delivering to the quantity of patients that you need to makes it more and more attractive. I can’t see autologous ever going away because it’s fantastic at the indications that it’s fantastic at, but I think the balance will switch over time.
Q: What are some of the biggest challenges manufacturers face when bringing these products to market?
Ellison: The biggest challenge people are going to face is just the scale up or scale out system, just the sheer volume of things that have to happen. In a very small clinical trial, you can have an ad hoc team in house to manage the logistics between them.
As you move up and into more patients and centers, more countries, times zone, regulations, more everything, you can’t do that in house any more. So you have to be working with a tech expert who understands how to do that. There is an ever increasing complexity and complexity leads to risk, and one of the best ways to handle that is to throw more people at it, and that gets expensive.
I think that the way to address that is to pull you logistics expert in early. You may not need them in phase one, but you need to be optimizing your process as you’re going through the trial, so when you come out of the trial, you have a commercial ready logistics platform that’s good to go. You need to have a vision for what that platform is going to look like and you need to have that at phase one, so you can roll it out and test it as you go through the clinical process.
With autologous therapies, there’s five years to first-in-man commercial launch. You haven’t got the traditional decade for thinking about stuff that you used to. You’ve got five years. And if you don’t have it ready, you can’t delivery your therapy to your patient, and if your patient doesn’t receive it, then everything else is irrelevant.
Q: What makes a disease a good candidate for this type of treatment?
Ellison: From the practical sense, diseases that haven’t been treated before.
I also think you need to think about what the supply chain is, not just what the biological indication is. Biologically it might be possible, and from a manufacturing point of view, you might be able to draw on a piece of paper that it’s possible, but how are you going to delivery it?
For example, we have a therapy that has an under 10-hour shipping window and that’s defined by the clinical indication. How do you do that if you have one manufacturing center in North America, and you have a patient in Europe? If you can’t write on a piece of paper what the logistics system would look like, why are going for that indication?
Q: What type of diseases and illness are currently being treated with these therapies?
Ellison: It’s mostly oncology at the moment.
Q: Do you expect therapies to be developed for more common diseases?
Ellison: The Holy Grail is something around diabetes or something around those larger indications, and there are plenty of people working on those. But it’s obviously very difficult to do.
As people overcome the challenges, it will happen. The issue is people are thinking in the box, the box being the manufacturing center, so they’re trying to drive down cost of goods and all that. They haven’t thought about how to move it, or about temperature or shelf life, and these things need to be thought about earlier in the process.
Q: How would the expansion of these therapies impact the pharmaceutical industry and drug development?
Ellison: Well, they’ll never replace your traditional pharma. Why would you use a therapy that cost half a million a treatment when there’s something already available?
Combination therapies where you see a more traditional pharma in conjunction with a cell therapy might be more common, but I think the short term is more around infrastructure and how the industry thinks.
If you take the UK for example, they’re sort of punching well above their weight in the cell and gene therapy space. They’ve just created three advanced therapy treatment centers with are coordinated by the cell and gene therapy catapult. That’s part of the industrial strategies, but the concept is to get clinical and tech experts and therapy development and the NHS all working together to define how we create this seamless vein to vein clinical pathway, so therapies can get to patients quicker.
You have to think about how the courier delivers it to the right place in the hospital, what the storage situation is, and so on. That’s a three year project, and I think that will really define the way these therapies are rolled out and we’re very excited to be part of those projects.
Q: What are the different steps of your logistics platform?
Ellison: What we’ve identified is that a therapy at the moment of development is an effective therapy, that’s what clinical trials have proven. What we’ve got to do is build that into a commercially viable business proposition and then roll that out globally.
There’s two sides of that: 1) all of the expertise that we can bring as a partner, such as an understanding of airline shipping things, temperature information, customer brokerage, all those things that we do; and 2) we need to enable our customers to use the other technologies and resources around, so as part of the platform, we develop relationships with shippers, and so on, and build these resources directly into our portfolio, so clients can use the best in class tech, and further, can just use it directly through us.
Our goal is to make the lives of therapy developers easier, so that these therapies get to the patients who need them.