Cellular therapies and treatments in Huntington’s

Josep Maria Canals, professor in the Department of Biomedicine, Faculty of Medicine and Health Sciences of the University of Barcelona.

One of the neurodegenerative diseases that does not yet have any type of curative or palliative treatment is that of Huntington. Patients with this pathology suffer a motor behavior disorder similar to parkinson’s, generating difficulties when controlling movements. In recent years, numerous essays with mice that have given good results, but when it has been tried to transfer to humans the conclusions have not been not at all satisfactory. The cell therapies have become a ray of hope for these patients, and a group of researchers from the August Pi i Sunyer Biomedical Research Institute (Idibaps) have prepared a white paper for the clinical translation of cell therapy in Huntington’s disease.

Bringing stem cell therapies closer to tackling neurodegenerative disease is the cornerstone of the report published in the journal Braina fact that has not yet been consummated effectively. Joseph Maria Canalsresearcher specializing in neurodegenerative diseases of Idibaps and professor of the Department of Biomedicine, Faculty of Medicine and Health Sciences of the University of Barcelonastates in Medical Writing that “currently there are two treatment approaches: One is to make cells serve so that they release factors within the brain that allow improve the functioning of neurons that still remain, so it is a protective treatment. On the other hand, another more ambitious formula that will take time to appear is replace tumor neurons. Stem cells have the ability to differentiate these neurons. In mouse models it works quite well, it gives us hope, but we must be cautious, there are a series of challenges that have to be resolved before moving on to humans”. Thus, the goal set by the researchers is go one step further to alleviate the symptoms and manage to find a healing for the disease.

“Stem cells have the ability to differentiate tumor neurons”

The report for the approach to Huntington’s disease highlights a total of four pillars to be able to start the right path and achieve an effective treatment. The first question to be resolved, according to the researchers, involves defining what is the best animal when carrying out a preclinical study: “We must know if we should go first with a small animal or a larger one, since they all end up having pros and cons. Once we know it, we will be able to pass it on to humans with the certainty that it will not suffer any harm, patient safety comes first”, recalls Canals.

The second puzzle to be solved is how these cells become produce in a pharmaceutical way. The researcher states that “at the moment we are producing them in the laboratory on a small scale. If we have to go to therapy with patients, we have to produce many more cells and we have to produce them under conditions that are compatible in clinical practice”. Designing instruments that allow these cells to be applied inside the brain without causing damage and defining what type of patients can be treated with this new therapy is the third leg highlighted in the report. “Getting to associate the cells to the clinical practice it would mean a great leap forward”, concludes Canals. Finally, the last unsolved piece of the puzzle is linked, as the researcher explains, to “follow-up what has to be done afterwards to confirm that the results of this treatment work, verify that the cells are not rejected and learn from each transplant performed”.

Learning, valid for other neurodegenerative diseases

Behind all these objectives, two working groups are working to achieve them, Stem Cells for Huntington’s Disease and the European Huntington Disease Network. “We are part of the management of both groups, which are focused on making specific working groups for each objective set. looking for one harmonization so that the analyzes carried out can draw comparable conclusions. Until now, all the laboratories act in a different way, making it difficult to move forward along the same line”, explains Canals.

The researcher goes so far as to state that all these guidelines for carrying out trials with patients suffering from Huntington’s disease can be used in other neurodegenerative pathologies such as parkinson’s or the Alzheimer’s. “There are many common goals, the affected neurons may be different but in the end the way to produce them is the same. There are diseases that do not have consortium or they are more minority, but we think that everything we learn from these work groups will be transferred to other neurodegenerative diseases”, says Canals.

“The affected neurons may be different but the way to produce them is the same”

The message that the researcher and the rest of the Idibaps team want to convey in this report is one of hope and faith that the research will end up bearing fruit: “There are a number of people worldwide who are working to develop novel cell therapy techniques for diseases that are currently incurable. We hope to find a cure or a palliative treatment soon to be able to remedy it”.

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