While it may not have been common a few short years ago, cell and gene therapy’s popularity has grown exponentially in the past year, as researchers are rapidly expanding our understanding and potential uses of these innovative therapies.
The possibilities with this new technology are exciting. However, it’s important to understand how cell and gene therapy research is different from traditional drug development.
Cell and Gene Therapy vs Small Molecule Research: Understanding the Difference
From an institutional review board (IRB) perspective, the ethical and regulatory framework to review cell and gene therapy clinical trials and traditional drug trials are the same.
However, in many gene therapy trials, there’s an extra layer of review from an institutional biosafety committee (IBC). This isn’t always required, as it depends on federal support, but it’s considered best practice to include IBC review. This extra review is required because some gene therapies may make permanent, unchangeable impacts on the body, where traditional drug therapies eventually metabolizing out of the system. Since gene therapy has the ability to alter the human body at a cellular level, research staff preparing the products need to be educated and have appropriate procedures for safe handling.
As research and development of cell and gene therapies for cancer, Alzheimer’s, Huntington’s, a multitude of rare diseases, and other conditions move beyond academic medical centers (AMCs) and make their way into broader communities via Phase II and III trials, specific infrastructure is critical to ensure research sites are prepared to run a cell and gene therapy trial.
Building an Infrastructure
Many sites need guidance for conducting cell and gene therapy research safely and successfully. The extra work involved in these trials is sometimes surprising to stakeholders. These studies involve more regulatory logistics and preparation for executing the protocols, mainly through product handling. It’s important to account for shipping logistics, reviews, and other necessary startup requirements. Working with participants in different parts of the US may necessitate shipping biological materials with specific storage requirements, and not all sites are prepared for the specialized containment necessary. For more information on how to prepare your site, see our blog How Sites Can Win in the New Age of Genetically Engineered Treatments.
A Focus on Rare Diseases
Much of the promise of cell and gene therapies surrounds cures for genetically based rare diseases. When working in rare disease research, startup speed and first-patient-in timelines are of utmost importance. Oftentimes, when researchers are working with rare diseases, they are working with a very specific patient population – there may only be 1000 people in the world affected by this disease. Recruitment efforts need to be very precise to find the appropriate participants for these studies.
Because these studies can be very niche and selective, oftentimes, research staff and participants form a bond as they get to know one another. As the therapy is personalized, relationships become personal as well. Since the relationship is personal, the race to find a solution is more urgent. The mission of these studies has the ability to become very personalized since everyone is banded together.
Since this technology is still relatively new to the industry, study cadence and best practices are not yet identified or built into standard study designs. However, patient advocacy organizations may be willing to help and offer their expertise and support as researchers work to find a cure.
Current Cell and Gene Therapy Volume
Over the past year, Operation Warp Speed – the race to create a vaccine to combat the COVID-19 virus – elevated cell and gene therapy research into the spotlight via notable vaccines developed using mRNA and viral vector technology. While cell and gene therapy has been in the works for the past decade, the pandemic exposed the world to the promise of using genetically engineered materials in treatments. Recent FDA approval of a gene therapy with the ability to cure childhood blindness provided additional excitement for the cell and gene therapy space.