New Hope For Patients Using Animal Model ALS Genetics

Not so long ago, researchers reviewed the strengths and weaknesses of animal models currently in use for Amyotrophic Lateral Sclerosis (ALS) research. The study looked into the transition from animal models to human disease with a genetic approach for personalized medicine in ALS. It was published in Acta Neuropathologica Communications.

Through the study, the researchers suggested that promising therapies will soon be developed for ALS by making use of gene therapy discoveries derived from mouse models with the disease. In this respect, a human treatment model called personalized medicine that takes into consideration individual patient factors such as environment, genetics and laboratory findings will be designed.

It is approximated that 10 percent of ALS patients have a familial, inherited form of the condition. Multiple studies have singled out a number of genes involved in ALS and are researchers are now devising strategies to apply that information to come up with better clinical outcomes.

In laboratory studies animal models such as mice are used to replicate a variety of genetic contributors to ALS and then test potential treatments. This refers to gene therapy and biomarker monitoring in order to gain insight into the contributing mechanisms of ALS.
Researchers seek to make use of what is learned to develop different treatments for different patients among the various genetic subtypes of ALS. The only limitation to this strategic 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.
As an alternative to a generalized approach, ALS patients would be divided into subgroups to determine the best treatment approach based on their genetics, biomarker testing and any other existing relevant factors.

This will go a long way to predict groups that will respond better to specific treatments from those who will not respond or perhaps become worsen symptoms. The model goal is to enhance general patient response with minimal speculations.
The researchers conclude that there is an undying need for discovery of treatments to facilitate patient care in ALS. To add on, there is a need for optimizing current animal models that present human disease characteristics. Gene therapies have been shown to be effective in particular animal models that have at least four varied genetic mutations.
In humans, ALS is seen to be more complicated in its manifestation than the single genetic models tested in mice. This implies that different ALS patients can exhibit very different responses to the same treatment.

Approved treatments for ALS cannot reverse the devastating effects of the disease or provide a cure. However, they can slow the deterioration in function, prevent complications, and increase comfort and independence in patients.

However, the study inclined towards positivity in that the use of gene therapy will grow in clinical trials in future and more people with ALS will be included in the trials. Moreover, the extensive data reviewed in the current study proves that gene therapy with personalized medicine is a significant step forward for treatment of ALS.