Project Summary
The ALD Connect consortium empowers patients, caregivers, and their affinity groups to move beyond conventional research participation, advocacy, and fundraising efforts to improve care for and ultimately eradicate the debilitating single-gene disorder, X-linked Adrenoleukodystrophy (ALD). The ALD Connect collaborative network will introduce a novel, all-inclusive model to improve care and drug discovery for well-defined single-gene disorders.
The first goal of the network is to inventory, and collect information from, existing resources worldwide (patient registries, advocacy groups, electronic health records, academic databases, brain and tissue banks) and design common data elements. Data will be captured, harmonized, and aggregated in the NeuroBANKTM platform through a partnership of the ALD/AMN Global Alliance and the Neurological Clinical Research Institute at Massachusetts General Hospital. By using NeuroBANK™ and Global Unique Patient ID (GUID) technology, the network will aggregate patient-reported information along with date captured by clinicians and researchers. This approach will validate and enhance patient-reported outcomes within the overall repository.
The network’s second goal is to create a social network platform that allows for communication between patients and researchers. Examples include ALD Knowledge Portal, where patients vote on research projects, track their disease progression, and report outcomes, as well as ALD Patient Learning Academies, where patients learn about clinical research and therapy development to become ambassadors for ALD clinical research and champions of the latest recommendations in clinical care.
Three innovative, patient-centered pilot projects will supplement the two main goals. These projects will evaluate new drugs through the ALD Treatment Pipeline, assess devices to provide mobility training for ALD Patients, and test remote data collection in ALD patients.
Collaboration between an educated and engaged patient community and academic centers will allow for data comparison and validation, patient feedback on research directions, and rapid trial development. Social network maps and analytics will assess activity in the network and measure and improve its success.