2020-09-06 14:42:40
When a person, generally over 60 years of age, begins to feel an uncontrolled tremor in the hands that, over time, worsens and progressively spreads to the arms, legs, face…, the movements become slow and the balance unstable, the most You probably have Parkinson’s disease. The name is due to the first physician who described it, Dr. James Parkinson, who disclosed the symptoms in a monograph published in 1817 entitled An essay on the shaking palsy.
A lot of time has passed since then and, as he comments today Analia Bortolozzi, our guest in Speaking with Scientists, “Today, despite having passed more than 200 years, we know many things, but we still cannot know exactly what the causes are.” It is known that some of the people who suffer from the disease have their origin in genetic causes but these patients are only 10% of the total, the origin of the rest is unknown.
Among the many things that are known is the fact that the disease is caused by a disorder of the nervous system that affects certain neurons in a region of the brain known as the “substantia nigra.” These neurons are responsible for generating dopamine, an essential molecule for the transmission of information, a neurotransmitter, which makes it possible for us to carry out movements normally. If these dopaminergic neurons die or deteriorate, transmission is increasingly difficult and, as a consequence, motor coordination problems appear, which translate into tremors, rigidity and other mentioned symptoms.
Analia Bortolozzi He comments that there are two very important facts in Parkinson’s disease, one is the loss of dopaminergic neurons in the substantia nigra and another is the presence of aggregates of a protein known as “alpha-synuclein”. Alpha-synuclein has a very important role not only in the brain but throughout the human body. Its role in the brain is essential because the molecule is bound to synaptic vesicles that, when communication between neurons occurs, release dopamine content. Thus, alpha-synuclein, by controlling the transmission between neurons, must be in the right balance because, whether it exists in excess or lack, it results in impaired function that can play an essential role during Parkinson’s disease.
Alpha-synuclein is produced in greater quantity in the elderly and, as in Parkinson’s patients, the excess produces insoluble aggregates that impede its function. Thus, controlling the amount of alpha-synuclein in neurons is one of the objectives pursued by researchers to find drugs that help control the progress of this disease. In this sense, Bortolozzi’s team, in collaboration with scientists from the biotechnological company n-Life Therapeutics, has synthesized a new molecule aimed at controlling the amount of alpha-synuclein that exists in the neurons of the substantia nigra. The molecule is associated with another that has a great affinity for dopaminergic neurons and serves as a guide to act in the appropriate place in the brain if it is given to a patient orally or intranasally.
As with this type of research, the road to a safe and effective drug that can help Parkinson’s patients in the future is long and expensive. At the present time of the investigation, Analia Bortolozzi and his team have carried out a series of experiments with mice genetically modified so that they express human alpha-synuclein in their neurons. This model has been developed by the same researchers, with support from the Michael J. Fox Foundation.
The results with these modified mice were very positive and encouraged the team to take a new leap in research and test the drug in an animal model closer to humans. The researchers established a collaboration with Dr. Jeffrey Kordower of the Rush University Medical Center at the University of Chicago to test the molecule in elderly rhesus monkeys, which, unlike mice, do suffer from alpha-synuclein buildup, as occurs in mice. the humans.
The results have been encouraging and now, declares Analia Bortolozzi, “Having a molecule is an important step, but now another type of research is coming that will take time, for now they are preclinical tests, with laboratory animals” The next steps will consist of optimizing the molecule to increase the potency and duration of effect after intranasal administration. And, of course, the participation of the pharmaceutical industry will be necessary to develop the appropriate steps that will make it possible in the future to make the leap to clinical trials with patients. It is a long road that will have to be traveled and, if all goes well, perhaps in the future, in a few years, we will be able to count on a new drug that will help patients with this disease, which between 1990 and 2015 doubled the number of people affected worldwide to more than 6 million and, due to the aging of the population, it is expected that it may affect more than 12 million in 2040.
I invite you to listen to Analia Bortolozzi, a scientist at the CSIC and researcher of Center for Biomedical Research in Mental Health Network in it Institute of Biomedical Research of Barcelona (IIBB–CSIC) of the Superior Council of Scientific Investigationsassociated to Institute of Biomedical Research August Pi i Sunyer
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