New Drug Formulations Are Potent Against ALS

Jun, 2021 - By SMI

Neurons and its degenerations are a very common cause of neurodegenerative disease, for the creation of motor neuron and its use for drug specific screening, required the involvement of the specific pluripotent stem cell, made in-vitro.

Neurons and its degenerations are a very common cause of neurodegenerative disease. Neurons exist in structural forms, sensory neurons and motor neurons. Sensory neurons are actually responsible for the perception of different types of sensations in the body, while motor neurons exhibit all the commands given by the sensory neuron system and the Central Nervous System (CNS).

One of the common neurodegenerative disease is Amylotrophic Lateral Sclerosis (ALS) that is caused due to the degeneration of different motor neurons. It is a fatal disease that is characterized by the loss of voluntary muscle function. Scientist’s at Boston Children’s Hospital, along with a collaboration with Pfizer, designed a new targeted therapy, using motor neurons procured from ALS patients. They used a Dopamine D2 receptor and particular drugs antagonistic to that particular receptor as a target and identified certain areas with which they can start the treatment. This approach is indeed a novel one.

For the creation of motor neuron and its use for drug specific screening, required the involvement of the specific pluripotent stem cell, made in-vitro. Live-Cell imaging technique has also been involved for the measurement of the hyper-excitable action just before and also after the incorporation of the drugs. It seen that the patients with ALS mutation in them appears to be more stimulated than that of the normal person’s motor neurons. This hyper-excitable nature of the motor neurons in specific make them very much susceptible towards degenerative pathway, resulting in to premature death.

On about analysing 2900 drugs in relation to this, round about 67 drug has an effect to reduce the effect of this hyper-excitability, with no such toxicity involved. out of that 67,13 drugs posed greater effect, of which some belonged to AMPA receptors and Kv7 potassium channels target groups. The study also depicts about the new group of drugs related to Dopamine D2 Receptor agonist. Although results were pretty accurate still in an aspect of patient derived action, these drugs and its mechanism is very much tough to understand.