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Cell and gene therapy based approaches for treatment of Parkinson's disease: from models to clinics

Final Report Summary - TREATPD (Cell and gene therapy based approaches for treatment of Parkinson's disease: from models to clinics)

Dr Kirik is an internationally leading expert in animal models of Parkinson’s disease and pre-clinical development of new therapeutic strategies focusing on cell and gene-based approaches. Hypothesis-driven studies distinguish his contribution to this field and are widely cited not only among the scientific community but also by clinical experts that implement new therapies to patients – a key outcome of Dr Kirik’s program. His ERC StG project had four main aims. In the initial period of the program, he used a combination of advanced genetic tools to over express or knock down proteins in brain cells lost in Parkinson’s disease and tackled a key question on why these neurons are vulnerable and are lost as the disease progresses, while other brain cells might be resistant to the disease-related stress in the brain. In a recently published study, his team illustrated how mishandling dopamine – a key neurotransmitter lost in the parkinsonian brain – could trigger an abnormal reaction leading to increased toxicity mediated by a protein called alpha-synuclein, which is a pathological hallmark of Parkinson’s disease. Next, his team implemented refined methods for regulation of genes in the brain that can be used for therapeutic purposes. This part of his work represented a novel step with innovation potential and not only lead to scientific publications but gave him the opportunity to seek patent protection of these results. Through support from ERC PoC program awarded to Prof Kirik in 2012, an initial market evaluation of the idea has been made. Another key area of Dr Kirik’s work related to understanding how to use cell transplantation better so that potential side effects of grafts can be prevented. In this part of the project, his team worked closely with experts in PET imaging and using an array of in vivo and post mortem data showed that the abnormal responses after grafting might be associated with low rather than high levels of dopamine synthesis and poor storage and re-uptake capabilities in some of the cells. Finally, Dr Kirik used a very recently described MR spectroscopic method with enhanced sensitivity to carry out proof-of-concept studies in how neurotransmitter synthesis in the brain can be followed. Although these efforts represent initial studies where the development need to continue beyond the project period funded by the ERC, they mark the first pioneering steps of future imaging technologies that are expected to change how we see the brain function.