Primate models for testing drug efficacy There is a high, unmet clinical need for drugs to cure neurodegenerative disorders. Towards this goal, European researchers translated the findings of their preclinical work in animal models. Health © Shutterstock Parkinson's disease is a progressive neuromuscular disorder that affects millions of people worldwide. Current therapies mainly treat the symptoms to reduce the impact of the diseases. However, they are far from ideal with many unwanted side effects and a lack of therapeutic efficacy. Despite large investment in the research and development drug pipeline for Parkinson’s disease, most lead compounds have failed to reach the market. Innovative candidates such as cell or molecular therapies are urgently needed as most neuroprotective therapies are years away from approval. Gene therapy is emerging as an attractive regenerative approach for treating Parkinson’s disease. Vectors that are delivered directly into the brain can supply the enzymes required for dopamine expression and show good safety and relative efficacy. The common denominator in drug development before proceeding to clinical trials is testing the efficacy of lead compounds in preclinical animal models. In this context, the EU-funded PRIMOMED (Use of primate models to support translational medicine and advance disease modifying therapies for unmet medical needs) project set out to develop innovative treatment options for Parkinson's disease. For this purpose, it brought together four small biotech companies owning innovative and proprietary drugs and two of the most renowned European primate research centres. PRIMOMED utilised well characterised non-human primate models to test the proprietary compounds of the participating SMEs and subsequently use this information towards clinical success. They discovered that the drug compound NRG-101 might be a potentially new clinical candidate for reduction of parkinsonism and disease progression. Overall, the translational approach of PRIMOMED ensured that solid preclinical evidence existed before proceeding further with specific compounds. Partners envisage their treatments could overcome the limitations of current therapies for Parkinson’s disease and address the unmet needs of patients and their families. Keywords Primate model, drug, Parkinson’s disease, gene therapy, NRG-101
