Your source for answers to the complex challenges of cell and gene therapy development.
We recently spoke with Ken Morrison, Global Lead of Operational Strategy & Planning Oncology at Labcorp Drug Development. He shared some of the advantages and challenges of gene therapy, key differences between autologous and allogeneic cell therapies and how to overcome common hurdles to enable effective patient access, recruitment and retention to advance these novel therapeutics.
Can you give us a high-level overview of what makes cell and gene therapy clinical development unique, especially for oncology?
Gene therapies require either individual dosing or multiple, different treatment regimes, focused on targeting the specific biology of each patient’s cancer, called personalized therapy. As a result, study sponsors must carefully navigate requirements around study execution, partnering with experienced cancer centers with familiarity to local requirements for study set-up and patient selection.
There are basically two types of cell therapies: allogeneic and autologous. While allogeneic treatments have more of an off-the-shelf donor approach and autologous therapies modify the cancer patient’s own immune cells, both treatments are personalized cell therapies. Therefore, it’s critical that sites have both the right experience and the right capabilities to help support patients. Sites must be incredibly organized in terms of patient orchestration, such as scheduling for cell collection and re-infusion, as well as have the appropriate facilities including apheresis capabilities, bed space and awareness and expertise in close assessment of CRS, neuro and cardiotoxicities, which are potential adverse events associated with this type of therapy.
We must also consider the need for long-term monitoring with these cell and gene therapies. After the initial, intense treatment period, there’s a very long-term follow up period of up to 15 years. At Labcorp, we partner with cancer centers and the cancer patient and their families to maintain these vital connections.
In thinking about the difference between autologous or allogeneic cell therapy, what are some of the key considerations?
The allogeneic platform involves a standard path of therapy initiation. A donor provides their T-cells or another type of functional immune cells, which are then genetically manipulated. The patient obtains the requisite pre-treatment either to boost their immune system or reduce their number of T-cells, and then receives the allogenic treatment.
In contrast, with autologous therapies, the patient orchestration process is a lot more complex. We estimate that it takes more than 1000 emails or other points of communications between the cancer center, the sponsor, the manufacturer and the clinical partner for just one patient to go through the process of collecting an autologous cell product, manufacturing and then re-infusion.
To help streamline this process, we partnered with OCELLOS by TrakCel, a technology that follows all the stakeholders to connect with one central data source. This platform provides a robust chain of custody and chain of identity. We can now see each cell product’s location for every patient, at any point in the manufacturing and shipment process. At the end of the day, we provide the most efficient solutions to allow all stakeholders to effectively communicate, ensuring that every single patient receives the right treatment at the right time, maintaining the highest quality of cell product.
What are the main challenges in transitioning a cell or gene therapy development into the clinical phase?
In the transition from the pre-clinical space into the clinical space, Labcorp has the unique ability to provide insights on characterization of the cell products and cell functionality. This also helps when it comes to patient recruitment. By the very nature of cell and gene therapy products, we are tasked with identifying a rare cancer population. Here at Labcorp, we have a network of institutions with the capability to identify those patients quickly and engage with the treating oncologist to support the patient and their family, while raising awareness of possible clinical trials that are available for the patient.
As developers transition from the hematological space into solid tumor disease, they can benefit from the integration of unique diagnostics capabilities. At Labcorp, we can identify and characterize these patients from a very broad number of different immuno-oncology based targets, then recruit them into the right study with urgency.
The final challenge involves the requisite long-term patient follow up. Before we start the clinical program, it’s important to consider the types of data points that need to be collected, as well as the overall strategy and approach for data collection. This ensures a seamless transition into long-term follow up for the patient, with a pre-agreed mechanism for data collection.
What are some approaches you would recommend for overcoming these challenges to de-risk your cell and gene therapy development?
It’s important to come into the pre-clinical space with the patient profile, clinical endpoints and safety profile of the asset in mind.
Labcorp maintains complex and diverse diagnostics capabilities as well as preclinical end-to-end solutions for cell and gene therapy. We have the unique ability through our clinical expertise to also support from the pre-clinical stage onward to define the patient population, help identify possible patient challenges and understand each patient’s unique journey.
When entering the clinical space, we focus on preserving and valuing patients’ time. Labcorp’s Decentralized Clinical Trial solutions accommodate patients’ needs—from physician tele-visits to electronic collection of adverse event data to home visits via our network of nurses, all of which helps reduce the patient burden of participating in trials.
Finally, when it comes to long-term follow up, Labcorp is heavily invested in finding more efficient solutions that meet the needs of the customer. We look at real-world evidence to determine how to best support ongoing data collection for patients as they hopefully lead long lives. Our focus on the patient journey around both recruitment and retention is critical for the success of any client participating in clinical programs in this space.
What are some of the hurdles that must be overcome for effective patient access to cell and gene therapy studies?
There are three main challenges.
The first issue involves the patient’s knowledge and awareness of clinical trials in general and cell therapy studies in particular. For instance, what are the risks and expected side effects? What will the patient go through for each visit? Are there long-term follow up needs and expectations? This patient knowledge is critical due to the unique nature of these therapies.
Second, patients need help to understand where these trials are running and how to gain access to them. Labcorp has invested in a web portal called Global Trial Source, which allows patients to gain study referrals (with assistance from their physician), access study information and learn about possible future studies.
Finally, we work to help patients appreciate the overall importance of trial participation at a global level. Labcorp has a global network of cancer center partners for cell and gene therapy. Together with our partners and patients, we are creating an infrastructure that will help other cancer patients in the future.
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