A Cretan scientist in the fight against Parkinson and Alzheimer’s disease

Professor Nicholas D. Mazarakis holds the Lucas-Lee chair of Molecular BioMedicine and is head of Gene Therapy in Brain Sciences, Faculty of Medicine, Imperial College London. He is a molecular neuroscientist with an international reputation in gene therapy of neurological diseases.

Nicholas Mazarakis is from Crete.

His research focuses on investigating molecular pathways of neurodegeneration and developing translational gene therapies for neurological diseases. He received his Ph.D. from King’s College University of London and is an elected fellow of the Society of Biology. He has lectured in conferences worldwide and published in top science journals such as Nature, Science and PNAS. His research is supported by several grants including an Advanced Investigators award in 2008 and a Proof of Concept grant in 2013 from the European Research Council.

In 2016 researchers, led by Professor Nicholas Mazarakis and Dr Magdalena Sastre from the School of Medicine, Imperial College London, published a research in the journal of the National Academy of Sciences (PNAS) showing that Alzheimer’s disease could be stopped in its tracks with an injection into the memory centres of the brain to boost a gene which clears out destructive sticky plaques.

He has served as Vice President of Neurobiology at Oxford BioMedica plc, where he pioneered the first ever lentiviral gene therapy to the clinic for Parkinson’s disease (ProSavin®).

The treatment, called ProSavin, which was discovered by Oxford Biomedica, is importing in the brain, by using a «disarmed» virus -three genes involved in dopamine production which is the basic chemical neurotransmitter that sends chemical messages, the lack of which describes the brain disease called Parknson. Thus, genes turn some brain cells to dopamine producers.

Dr. N. Mazarakis explains that this is a gene therapy that uses viruses, which are constructed so as to carry therapeutic genes and in this case three genes produce dopamine in the cell when it’s missing in Parkinson’s disease. «In that disease some neurons, which produce a substance, called dopamine, are degenerated, so there is a shortage of this substance, which is generated and transmit messages from neuron to neuron. The concept behind this treatment, which has entered clinical trials in France, was to be able with genetic way to reintroduce the cells in a specific part of the brain that had dopamine deficiency. We succeeded and we created this innovative virus and demonstrated a first cultivated neurons after making experiments in the brain in young and then older test animals that not only can we build the dopamine in a specific part of the brain that is lacking from but it was also shocking that this can be effective in young and older models which have all the cardinal symptoms of the disease,» as he says.

«The treatment is a painless and somewhat specialized introduction of the virus in this part of the brain. The patient is being operated for four hours with very thin needles which put the virus in this part of the brain, he remains in the hospital all night, coming out the next day and within a few weeks he shows a large difference in mobility» as he describes. It is noted that patients are at very advanced stages when dyskinesia appears. «This treatment has begun to show even to people that it is effective, this will be shown in phase two of the clinical trials that will come later after seeing the dose and ensure that we have no negative side effects. In the second stage we will be able to demonstrate how effective the treatment is, through statistical way» he says.

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