In CNS research, the use of intrathecal drug administration and cell population analysis as a biomarker for studying immune responses in the brain are tools to watch in 2019
Intrathecal Drug Delivery
The continued increase in demand for treatment of central nervous system diseases and the bypass of the blood-brain barrier with intrathecal drug administration is a hot topic right now. Beyond the last century, the blood-brain barrier (BBB) was conceived to be a passive, impermeable barrier that shielded the brain from the blood’s trajectories. However, revolutionary work over the years has given way to the idea that the BBB is an active, selectively permeable channel for transport between the blood and the brain of proteins, cells, and substrates that have access to transport systems localized within the BBB membranes. With these innovations in motion, intrathecal delivery, which allows you to deliver the drug to the central nervous system by circumventing the BBB, offers, offers some unique opportunities for developers of CNS drugs. It allows you, among other things, to administer genetically-engineered vectors into the central nervous system to express targeted proteins or oligonucleotides that modulate the expression of genes in order to address unmet medical needs for neurodegenerative and often rare diseases; this potentially overcomes challenges with intravenous administration that are not always able to deliver sufficient therapeutic levels across the BBB. Prospects for the intrathecal delivery are on the rise with the increasing need to treat patients and genetic diseases, including orphan diseases affecting pediatric and juvenile populations.
—Ria Falvo, Principal Research Scientist, Toxicology Reporting, Charles River Laboratories
Immunophenotyping in studying CNS diseases
Flow cytometric technology has been around for 50 years. As we learn more about the immune system’s role in incurable diseases, the tool is suddenly popular again, particularly in the study of CNS diseases like Alzheimer’s or Parkinson’s.As immunophenotyping may reveal not only inflammatory processes, but also the activation status of immune cell subsets, it can guide selecting individual immunomodulatory therapies. In CNS animal models this provides insight in the dynamics of peripheral and resident cells. Understanding the innate and adaptive immune cell status within the CNS and in the periphery during disease progression can be applied to monitor biomarker expression profiles as well possible pharmacodynamic changes related to treatment.
—Tuulia Huhtala, Head of Biomarkers and Molecular Imaging, CNS Discovery, Charles River Laboratories