Early-Stage Challenges in Neurodegenerative Diseases and Optimal Drug Development Strategies
Neurodegenerative diseases are caused due to the progressive degeneration of brain function. These are age-dependent disorders which affect the neurons in the human brain and cause problems with body activities like breathing, heart function, ataxias or mental functioning (dementia). Many well-known neurodegenerative diseases that affect the functioning of the human brain are Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, frontotemporal dementia, and the spinocerebellar ataxias. Some of the potent biomarkers can detect the early onset of these commonly-prevalent neurodegenerative diseases. On the other hand, some rare diseases which mainly affect children are Batten disease, Duchenne muscular dystrophy, neuroblastoma, and Gaucher disease. Any of the neurodegenerative diseases of children is considered complicated, which often creates a great challenge for the researchers to diagnose and manage.
For patients of all age-groups, the therapies are extremely rare. The already available therapies only provide symptomatic relief. This is because of the challenges faced during the drug development process including identification and classification of patients at the early stage of the disease and the limited understanding of brain which further limits the development of relevant biomarkers.
Pharmaceutical companies are now progressing to understand the neurodegenerative disease mechanisms through the utilization of non-clinical in vivo models (ie, animals) by which drug therapies could now be tested safely, and new treatments could be designed. So, there is a need to improve human health by using in vivo models.
The dependence on single biomarkers should be avoided, and the collective effort of the pharmaceutical companies, academic researchers, and government bodies is required to make progress against the challenges presented by neurodegenerative diseases.