Animal Model ALS Genetics Leading to Personalized Medicine for Humans

Researchers reviewed the strengths and weaknesses of animal models currently used for ALS research, in the study “From animal models to human disease: a genetic approach for personalized medicine in ALS,” recently published in Acta Neuropathologica Communications.

Through the study, authors suggest that promising therapies are on the horizon for ALS by using gene therapy discoveries gleaned from mouse models with the disease, and a human treatment model called personalized medicine that takes individual patient factors such as environment, genetics and lab results into account.

At least 10% of patients with ALS have a familial, inherited form of the disease. Scientists have identified several genes involved in ALS and are now looking for ways to use that information for discovering better clinical outcomes. In laboratory studies, mice and other animals are used to replicate different genetic contributors to ALS and then test potential treatments, such as gene therapy and biomarker monitoring, to better understand the contributing mechanisms of ALS.

The hope is to apply what is learned to create different treatments for different patients among the various genetic subtypes of ALS. The downside of the plan, according to the study, is that it does not provide any one-size-fits-all treatments and could lead to patient exclusion based on genetic status.

Instead of a generalized approach, patients would be divided into subgroups to determine the best treatment approach based on their genetics, biomarker testing and any other relevant factors . This will help to predict groups that will respond better to certain treatments from those who will not respond or perhaps become worse. The model goal is to improve overall patient response, with less guesswork.

The researchers conclude that there is a great need for discovery of treatments to help patient care. Moreover, there is a need for optimizing current animal models that exhibit human disease characteristics. Gene therapies have been shown to be effective in certain animal models, representing at least four different genetic mutations.

In humans, ALS is nearly always more complicated than the single genetic models tested in mice, which means different people can show very different responses to the same treatment. However, the study revealed optimism that the use of gene therapy will grow in clinical trials during the next few years, more people with ALS will be included in the trials, and that the extensive data reviewed in the current study proves that gene therapy with personalized medicine is a solid step forward for ALS treatment.