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Jim and Joan

13th January 2020

Thirty years on from its first use in clinical trials, gene therapy is set to transform the way degenerative diseases are treated

Longer lives bring greater opportunities and greater happiness, and also a greater susceptibility to degenerative illness. There are upwards of 50 million dementia sufferers in the world today. Their stories and the stories of those who care for them may be rooted in sadness but they offer a reminder of what it means to be a loved one.

Last month the Alzheimers Society shared the story of Manchester couple Jim and Joan Pearson. Married for over sixty years, their final decade together was by far the hardest. Joan began to suffer from memory loss, which eventually led to a diagnosis of Alzheimers. When she was no longer able to recognise her children or grandchildren, Jim used photoshopped images of the family to make wooden figurines of each of them. When one came to visit, Jim prepared by using their figurine as the focus of a conversation reminding Joan of what the person meant to her and what she meant to them.

Along with the love and support of their families, Alzheimers patients can count on the ingenuity and vigilance of the Life Sciences sector. In the years to come they can realistically expect gene therapy to transform the way their ailment is treated. The addition of new genes to a patient's cells to replace missing or malfunctioning genes was first tested in human trials in 1990. Typically a virus will be used to carry these new genes, acting as a clinical courier service that helps to change the behaviour of existing cells or block the pathway of a disease.

Meira GTx, a cutting edge clinical-stage gene therapy company with hub offices in London, New York and Leiden, are leading the way in seeking improved quality of life for patients. With ongoing clinical trials in ocular and salivary gland treatments, they are making admirable progress in areas where patients are not currently well-served, and where gene therapy offers rich potential. They are also focused on neurodegenerative diseases, determined to tackle the cruelty of Alzheimers. A key process that causes this disease involves the movement of cellular protein under the control of endosomes, which function as a biological sorting office. When the sorting office makes a mistake, the protein movement is dysfunctional and the path is open for Alzheimers to strike. Meira GTx have set up a disease program that targets this critical area.

Their diligence and focus offers real hope that the 2020s will be a decade of major progress. Year on year, gene therapy clinical trials are growing in number, and it’s notable that in 2019 a majority of active global trials had reached phase two. The life sciences sector is leaping forward in our service, tackling the health issues that profoundly affect us. Leaders such as Meira GTx are also working closely with patient advocacy groups, always looking for ways to reassure and comfort trial participants. They see the people behind the statistics. They’re doing it for us. They’re doing it for Jim and Joan.


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