Novel cell test developed for osteoarthritis drugs could help with other diseases
Osteoarthritis (OA) is a chronic degenerative joint disease that afflicts over 240 million people(opens in new window) around the world. It leads to pain and stiffness usually in the hips, hands and knee joints as the protective cushioning cartilage at the end of bones wears down. OA is the fastest growing cause of disability worldwide and is expected to worsen significantly with increasingly obese and ageing societies. Yet there are currently no drugs available to tackle the underlying causes. OA is a complex degenerative disease, and clinical trials for disease-modifying drugs consistently fail – mainly as clinical trials are run on unselected patient populations. As the underlying mechanism for OA is different among disparate groups of patients, the treatments required also vary – so selecting them at random will inevitably lead to high rates of failure. The EU-funded O-POINTED project set out to develop a way around this problem. “O-POINTED addresses the patient heterogeneity issue allowing you to specifically select the most promising target population for the candidate therapy before expensive and lengthy clinical trials are started,” says Tobias May, CSO at InSCREENeX(opens in new window) and O-POINTED project coordinator.
Reading between the lines
O-POINTED is a cell test that allows biomedical and pharmaceutical companies to use a personalised approach to drug development for the first time. It is a test based on cell lines generated from a cohort of over 50 OA patients, taken from different patient subpopulations through tissue exams (or from leftover material from a total joint replacement). The cell lines are genetically engineered so they can be easily handled in the lab and available at the necessary scale to test new drugs. “Thanks to our technology we can preserve the disease phenotype of the initial material," May points out. These cellular test systems can then be used in R&D departments of pharma companies and biotechs to test potential drug candidates for their efficacy in specific patient populations. “Based on this knowledge gained in the lab, only patients likely to respond to the candidate therapy can be enrolled in expensive and lengthy clinical trials, increasing the success chances of clinical trials,” May explains.
Expanding horizons
"Feedback from R&D departments in pharma companies and biotechs working on OA treatments was generally positive," says May. The team now needs to secure further validation data before entering the market with the test for OA drugs. Through the project, the team discovered demand for a similar test for other diseases and have now expanded the scope. “Most surprising for us, was that a lot of our collaborators and potential customers mentioned other diseases, which would make for a similarly interesting product but would require comparably less validation and characterisation work on our side,” May says. “Without the EU SME Phase 1 grant, we would have never had the capacity to thoroughly evaluate the market potential of O-POINTED and gather enough feedback to make a properly informed decision on how to go forward with the project. This means we either would have shelved the idea for now, meaning a loss of innovation, or would have gone forward with our previous plan, which would have meant significant additional investment of time and money that we as an SME could have used far more effectively and securely to grow,” concludes May.
