In recent years, cell and gene therapies have shown promising results in everything from cancer to neurological diseases. However, the path from academic breakthrough to becoming established in healthcare is a long one – and many projects are lost in the translational gap.
“As academic researchers it is totally reasonable to devote all your energy to getting through to the first clinical study. But this is not enough if the objective is for the therapy to actually reach patients. Somewhere along the line, the development must also be attractive for healthcare, investors and industry – and we must think about this much earlier than we do today,” says Anna Falk, professor of neuroscience at Lund University.
In a new research article in the journal Molecular Therapy, Methods and Clinical Development she presents, along with researchers at Lund University and Skåne University Hospital, a new working method. The goal is to break down the silos between academia, healthcare and innovation systems.
“At present, crucial technical, regulatory or commercial obstacles are often detected too late. And then it’s an expensive and slow process to make corrections – or the therapy never reaches the patients. We present a model and a tool that can provide support and reminders about which parts you need to have in place when you develop new advanced cell and gene therapies. It’s development support that can be used by all those working in the field,” says Anna Falk.
Bridging the translational gap through early collaboration
The core of the model is that the university, hospital and innovation stakeholders work in parallel and in an integrated way right from the start, instead of step by step. An important change is that the hospital is involved at an early stage in development and production – something that is necessary for cell and gene therapies, where healthcare is not only the user but also a part of the production chain. Here, it is clear that the conventional structures for drug development do not meet the needs of advanced therapies.
“It’s a paradigm shift in which the hospital not only receives or tests a finished product, but also all those involved – academia, hospital and innovation stakeholders – need in different ways to participate throughout the development process, manufacturing and quality control. There is a need here for a new working methods and tools. Together they constitute a model that has been developed in Lund,” says Stefan Jovinge, research director at Skåne University Hospital.
A practical tool: the cell and gene therapy navigator
As a practical tool, the researchers have developed the Cell and Gene Therapy Navigator, which follows a project’s technical, clinical and commercial maturity simultaneously. Using this approach, imbalances and future bottlenecks can be identified in time.
“It’s not enough to be the first to test the therapy on humans. If we want the therapies to reach many patients we must think about production, regulations and reimbursement right from the start. The Navigator works as a joint checklist and mirror for the project,” says Gisela Helenius, head of the ATMP Centre at Skåne University Hospital.
The researchers consider that the model could act as a template for other regions in Sweden and internationally – but that each region must build their own cooperation set-up locally.
“Our hope is that fewer promising therapies will get stuck or fall by the wayside – and that more patients actually get access to treatments that today remain in the lab.”
