Scientist looking into microscope

Neurotherapeutic Innovation

Improving patient care and therapeutic trials in neurodegenerative diseases
Our team aims to bridge the gap between basic science research and clinical practice to advance the early identification, treatment and cure of the biological pathways underlying chronic neurological diseases.

About our research

The program targets innovation at the clinical stage for the design of disease outcome measures and patient stratification in prospective therapeutic trials, as well as enhancing clinical care and patient monitoring. 

The Neurotherapeutic Innovations team will pursue two translational clinical research streams:

Deep Clinical Phenotyping of Multi-modal Biomarkers

Industry Engagements:

  • Biomarker Repositories for Clinical Trials - The team formalises existing infrastructure development collaborations and provides a collective ‘identity’ through which to engage and attract prospective pharmaceutical partners. Forefront Neurology and Sydney Neurology have begun working closely with industry partner SNAC to develop a unified cloud-based framework on which to host, analyse, and generate automated quantitative reports for relevant disease-specific multi-modal biomarkers across chronic neurological disorders for the purpose of streamlining clinical trials.
  • Expanding Clinical Imaging Capacity - This endeavour is being driven in response to industry needs for surrogate markers targeting disease changes associated with the neurodegenerative process. The proposal will allow rapid translation, dissemination, and piloting of MR sequences across diverse neurology clinics and LHDs in a harmonized manner.
  • Precision Genotyping - For therapeutic trials in chronic neurodegenerative disorders, open-label extensions (OLE) are becoming the norm because data indicates that changes in disease trajectory have become increasingly apparent beyond the predetermined trial duration (e.g., 12 months), but also due to feedback received from patient advocacy groups. As all patients transition to the active drug, alternative comparison groups are required to replace placebo controls. Industry partners are turning towards precision genotyping to facilitate meaningful stratification of patient groups in the OLE phase of clinical trials.

Academic Engagements:

  • Multi-modal Biomarkers - The NTI provides the perfect avenue for transdiagnostic clinical validation of novel biomarkers and prospectively engaging industry partners. Collaborations between BMC neurology clinics and Computational Neuroscience researchers exist for neuroimaging research, but there is significant scope to expand. 
  • Neuropathological Phenotyping - The failure rate for translation of novel drug candidates from transgenic mouse models and induced pluripotent stem cells to effective disease-modifying therapies is remarkably high. Research has demonstrated that overlapping disease pathologies occur in >50% of patients with a neurodegenerative disorder. This highlights a need to capture the differential impact therapeutic drugs have based on underlying pathology to identify patient cohorts that would benefit from synergistic and/or repurposed drugs. The team provides a trans-diagnostic cohort of brain tissue, and importantly deep-phenotype clinical history, to refine clinical profiles for improving the precision of prospective clinical trial enrolment. The collection of patient tissue directly aligns with the new frontier of organoid stem cell research, led by Professor Matthew Kieran, as a novel pathway for precision drug screening.
  • Precision Drug Screening - Novel organoid technology seeks to improve the gap between drug development in animal models and trials in humans and has the potential to accelerate the translation of relevant drugs into the clinic. 

Digital Enhancement of Clinical Assessments 

  • Digitization of traditional pen-and-paper clinical assessments has been completed in the most basic sense through the direct translation of screening questionnaires into electronic forms. The team seeks to engage with cognitive design specialists and software engineers to form new collaborations to leverage existing technological capabilities to enhance participant engagement and provide greater ease of access to accommodate motor and sensory impairments.