New Humanized Mouse Study Assists Cytokines Therapy

A new study has recently appeared in Annals of Neurology, showing how neuro-protective results were obtained in a Friedreich’s Ataxia (FA) model. The mouse model in question was treated with clinically approved cytokines used for other diseases, and the results showed that future research using the humanized mouse models could be promising for the effective treatment of FA in humans.

New Hope for FA Patients

The study known as “Cytokine therapy-mediated neuroprotection in a Friedreich’s ataxia mouse model” was aimed toward finding new treatments for severe neurological disorders such as FA. This specific disease affects muscle strength, coordination and speech, with patients progressively losing control over various areas of their bodies and coordination and a significant percentage of them also known to develop diabetes.

Although no approved treatment exists for FA, growth factors and a specific group of cytokines may hold the key to helping protect against the damages that FA causes in patients’ nervous systems. Cytokines are present in particular cells of the immune system, and can exercise a significant effect on cell/cell interaction and individual cell behavior.

Their use in humanized mouse models for the specific purpose of containing the effects of Friedreich’s ataxia is unprecedented, and the results obtained were more than surprising.

A Key Study in the Use of Cytokine Treatments

The study shows that cytokines administered to FA-affected humanized mice on a monthly basis through subcutaneously injected infusions of G-CSF (granulocyte-colony stimulating factor) and SCF (stem cell factor) can induce neuro-protective effects that were primarily showcased through various behavioral and motor performance tests.

The FA mouse model carried functioning human genes, tissues and cells that faithfully reflected the impact that FA would have on the human body. Aside from the monthly cytokines infusions, they were also treated with subcutaneous injections of SCF and G-CSF for five consecutive days during a single month. At the same time, a group of mice was simply treated with a saline solution during the period as a control group.

Analysis and Results

The results of the tests were thoroughly analyzed and combined with results of neurophysiciological analysis performed after 6 months of treatment. Significant emphasis was placed on assessing the electrical conduction of motor and sensory nerves, and the final analysis consisted of the study of various nervous system cells – from the spinal cord, cerebellum and dorsal root ganglion – to account for mRNA and protein expression. A histological analysis was subsequently also performed later on.

Frataxin expression was increased, and sensory nerves became more conducive as a result of the treatment. The humanized mouse has also displayed reduced inflammation as well as a greater number of stem cells in areas where the tissue was severely affected by the disease.

The conclusive observations made during and after the treatment, as well as during the various tests and analysis performed, showed how the treatment has led to a reverse in neuropathological, neurophysiological and negative behavioral effects that were normally assigned to FA. The neuro-protective effects of the cytokines have also promoted a pronounced reversal of biochemical parameters, and clinical symptoms have subsided, while motor coordination was greatly improved.

While these specific cytokines were already approved as a valid treatment for other diseases, this is the first evidence that they can be used to treat FA. The promising results may lead to doctors being able to effectively help their patients recover from Friedreich’s Ataxia.