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Dr. José Luis Lanciego, Neurosciences Program of Cima University of Navarra, member of CIBERNED

Perspectives in Parkinson's disease

Ongoing research is aimed at goal to halt the progression of the disease.

Sun, 29 Sep 2019 14:14:00 +0000 Published in The Courier

Parkinson's disease is a progressive brain disease that affects approximately 1 in 100 people over the age of 60. It is considered sporadic in more than 99% of cases, that is, hereditary transmission has only been demonstrated in less than 1%. The main risk factor is advanced age, especially after 65 years of age, with the incidence increasing at higher ages. It has been estimated that by 2060 the European population will increase by about 3%. By then, the number of people over 65 years of age will double. This figure alone gives a clear and disturbing picture of the problem facing physicians, in particular, and society and national healthcare systems, in general. The reason for this is the high health and social cost of this disease, as well as the enormous impact it has on patients and their caregivers, who are often also family members of the patients.

In comparison with other neurodegenerative diseases, in the case of Parkinson's disease we fortunately have a complete pharmacological arsenal that allows us to treat this pathology very effectively. These treatments have achieved the milestone that the life expectancy of a Parkinson's patient is identical to that of the general population, i.e. no one dies of Parkinson's disease. However, it is also true that quality of life can be seriously compromised, especially in advanced stages of the disease.

From a medical point of view, the main limitation comes from the fact that existing treatments, although very effective, are merely symptomatic, relieving or eliminating symptoms, but do not prevent the natural progression of this neurodegenerative disease. As it is also incurable, since we do not know its causes, the more pragmatic approach focuses on designing new treatments that attenuate or even halt its progression. In this way, although it cannot be cured at present, we will at least try to convert it into a chronic disease that can be satisfactorily managed with the usual medical treatments.

The scientific advances achieved in the last decade place us in a privileged position. We know better than ever the biological, molecular and genetic processes underlying the disease, to the point that new therapeutic approaches that a few years ago would have been considered unattainable are already in the very advanced stages of research . In fact, some of these new treatments are being tested in the first patients. Among the current advances, possibly the most promising are the new gene treatments. Specifically, gene therapy is a "frontier" research based on the employment of different viruses, as a "Trojan Horse", to provide neurons with the information Genetics they need to prevent their progressive death during the course of the disease. Viruses are the simplest living organisms in existence, so much so that they lack the ability to reproduce by themselves. These viruses, composed of a genome enveloped in a capsid, need to infect a cell in order to reproduce. By infecting a cell, they inject its genome, giving it the information it needs to make more viruses. We take advantage of this natural characteristic of viruses to modify them at laboratory. The virus genome is cut and the genes we are interested in are added to it. These genes carry the information that a neuron needs to heal itself, giving it the capacity to produce a certain protein that prevents it from continuing to die progressively. The technique consists of injecting these modified viruses into the brain. This research, still in the preclinical phase, is showing promising results.

Engineering Genetics is one of the most rapidly advancing branches of science. New modified viruses are continually being perfected to achieve greater and better therapeutic effects. It is not unreasonable to think of a very hopeful mid-term future deadline for our patients, whom we should always thank for their enthusiasm and partnership every time we go to them to enroll them in a new clinical essay . It is equally necessary to recognize the spectacular work done by the Spanish Parkinson's Federation (and its regional affiliates). It is precisely the synergic partnership between researchers, physicians, patients and relatives, which allows rapid progress in incorporating new treatments to improve the status of our patients while minimizing the enormous burden borne by their caregivers.