Neurodegenerative diseases can affect many of the body’s functions and the causes can be as diverse as genetic factors, toxins absorption, chemicals, viruses, alcoholism and in some cases, the explanation has not been yet identified. The most common neurodegenerative conditions are Alzheimer’s and Parkinson’s diseases but there are also many rare neurodegenerative diseases with currently no available treatment.
Neurodegenerative diseases and clinical trials
It is anticipated that diseases like Alzheimer’s will become more prevalent in society as the global population ages according to this patient organisation describing the elderly population of Japan.
Although more clinical trials for neurodegenerative diseases are needed, organising clinical trials for this fragile population of patients can be challenging. Indeed asking those patients to participate in trials often means travelling to clinical sites, and that may not be feasible due to mobility issues, fatigue, balance, and altered cognitive functions.
For neurodegenerative diseases, patient-centric trials and remote patient monitoring with adapted measuring tools appears to be as crucial as the development of novel digital endpoints.
In the case of orphan neurological conditions, the difficulties lie within the limited number of patients and lack of knowledge about the course of the disease. There is hope that personalised medicines will modify the future by focusing on the mechanisms and underlying causes of those rare diseases.
For Aparito, rare neurological diseases remain a high priority and we participated in several research projects including patients suffering from late-onset gm2 gangliosidosis (Gm2 gangliosidosis), and patients affected by the Gaucher disease (Gaucher).
Aparito’s technology enabled the collection of real-world evidence and the development of disease-specific digital endpoints (Gaucher trial) using our Atom5TM platform. High adherence and engagement from the patients showed the benefits of the patient-centric trials (Late GM2 trial) and how Atom5TM can support a range of neurodegenerative clinical trials.