Among the medicines that have been used for certain uses for a long time, it is sometimes discovered that one of them is also used to treat a disease against which no one suspected that they would be useful. Since it has already been shown that the use of the drug does not carry an unacceptably high level of risk, a lot of time is saved in clinical trials and paperwork, so that the new use of the drug can be authorized quite quickly.
This is very useful for any disease, but there are cases where it is extremely important to find an already approved drug that works against a disease. These cases are those of rare diseases, so called because a tiny percentage of the population suffers from them. The problem with each rare disease is that, since the recipients of a drug developed against it will be very few, it is not profitable for pharmaceutical companies to invest time and money in creating that drug.
Here it may be providential to be able to use an existing drug to treat a different disease for which it was originally developed. This strategy is known as drug repositioning.
This is the path taken by researchers at the Center for Biomedical Technology (CTB) of the Polytechnic University of Madrid (UPM) in Spain, who have proposed a series of repositioning hypotheses for more than ten rare diseases. Thanks to the development of computational methods that consider biological characteristics related to these diseases, they obtained a list of potential drugs that in 75% of the cases have turned out to be therapeutic for these rare diseases. This can bring great social benefits.
There are more than 7,000 rare diseases worldwide and most of them do not have a treatment. Since there are so many of these diseases, the total number of people affected is quite high. It is estimated that in Europe and the United States alone there are already more than 55 million people suffering from a disease of this type.
Thanks to drug repositioning, it is feasible to find treatments for rare diseases in a shorter period of time and at a lower cost, compared to having to develop drugs from scratch.
It is feasible to reuse existing drugs to treat some rare diseases. (Photo: Amazings/NCYT)
In the work carried out by the MEDAL (Medical Data Analytics) research group of the UPM, a series of repositioning hypotheses have been proposed for 13 rare diseases, such as “diffuse cutaneous mastocytosis” or “muscular dystrophy”. X-linked Emery-Dreifuss syndrome. Specifically, four computational methods have been developed that involve biological characteristics related to rare diseases, including genes, protein interactions, biological pathways, symptoms, and therapeutic drug targets. The information on the biological characteristics used in this study has been extracted from the DISNET project database, directed by Professor Alejandro Rodríguez González, which aims to provide a better understanding of diseases and reposition drugs by integrating large-scale biomedical data. scale.
The proposed computational methods are based on similarities between the studied rare disease and other diseases taking into account different biological elements. These methods are based on the fact that those diseases that are most similar to each other (due to a certain biological characteristic, such as sharing many symptoms) can be treated with the same drug. In this way, the drugs that are being used to treat the non-rare disease become potential treatments for the rare diseases with which they share that equality.
Applying these methods, a set of potential drugs was obtained to treat each of the 13 diseases considered. These computationally obtained drugs were verified in the scientific literature including different data sources such as Pubmed and Clinicaltrials.org, and the results showed that in 75% of the cases, the drugs obtained were potential treatments for these diseases. A representative example of the results obtained is that of diffuse cutaneous mastocytosis, a skin rash caused by the formation of abnormal collections of cells (mast cells) in the skin. Thanks to the computer strategy and the large amount of information contained in the medical literature, it was possible to find four drugs that could be very useful to combat diffuse cutaneous mastocytosis.
In the opinion of the UPM researcher Belén Otero Carrasco: “The results of the work show that the development of this type of study can have great social benefits, since drugs can be found for diseases for which there are not so many resources, which would help much to the people who suffer from these pathologies”.
The study is titled “Repositioning Drugs for Rare Diseases Based on Biological Features and Computational Approaches”. And it has been published in the academic journal Healthcare. (Source: UPM)