Gene & Cell Therapy + Cell therapy (ASGCT) annual meeting

[Tlexander]

With Precision Neuroscience we aim to maximize patient benefit by recognizing the heterogeneity within a given disease driven by its underlying biology and genetic etiology. The recent breakthroughs in the treatment of Alzheimer’s Disease (AD) are founded in the detailed understanding of the disease pathology (“A-T-N research framework”) enabled by biomarkers. On Wednesday, I had the pleasure of participating in a panel discussion at the American Society of Gene & Cell Therapy + Cell therapy (ASGCT) annual meeting, organized by the California Institute for Regenerative Medicine (CIRM), chaired by Abla Creasey and Shyam Patel, who have provided the questions below.

1. What are new impactful scientific discoveries that you think are key in advancing neurological diseases diagnosis and therapeutics development?
New tracers will allow us to visualize disease pathology in individual patients, much like what Amyloid and Tau PET have done in AD. Examples are imaging tracers for TDP43 (e.g. Amyotrophic Lateral Sclerosis - ALS, Limbic-predominant age-related TDP-43 encephalopathy - LATE), synaptic density (e.g. SV2A – Schizophrenia, epilepsy), neuroinflammation, a-Synuclein (Parkinson’s Disease - PD). Other discoveries are blood-based diagnostics that can revolutionize precision medicine because of their broad reach (e.g. plasma assays for identifying patients with likely AD pathology, biosignatures for determining neurotransmitter & neuropeptide dysregulation).

2. What data and resource sharing mechanisms need to be in place?
Pre-competitive consortia play a big role here, since there is a of common ground in developing diagnostics across the different organizations. Examples are the Foundation for the National Institutes of Health, the Critical Path Institute (C-Path), including the CPAD effort, managed by Sudhir Sivakumaran, Ph.D., which is working on Tau PET standardization and surrogate endpoint topics. Other drivers are large cohort studies, such as the Alzheimer’s Disease Neuroimaging Initiative (ADNI).

3. How does the field ensure conscious development of technology platforms so that their benefits are accessible to diverse patient populations?
Imaging is clearly limited to select centers with the required expensive technology. To democratize precision medicine, we need to develop less costly, scalable tools, such as blood tests, that can be deployed everywhere, including underserved communities. The key is to validate these diagnostic tools in diverse patient populations, so sourcing samples and patient data across diverse populations is an active focus for the field.

Precision neuroscience will ultimately benefit patients through better routine screening (digital, blood tests), biomarker/diagnostics driven disease prevention or interception and better outcomes. I am proud of #mycompany for contributing to this effort.