Addressing the technical, regulatory and ethical barriers to the safe and effective delivery of substances and cells directly into the central nervous system (CSF) or cerebrospinal fluid.
HD research is entering an exciting phase, with new approaches such as huntingtin lowering strategies and cell therapies on the horizon. Many of these potential therapeutic strategies require direct delivery of the therapeutic agents directly into the brain or CSF. However, it is clear that direct delivery into the brain presents multiple challenges, many of which are common to all these approaches, thus the mission of this new working group is to combine knowledge and expertise to develop solutions to these challenges.
- Identify and prioritise key challenge, which are likely to include: GMP production of cells or gene therapy products; differentiation protocols for cell products; safe, effective, and regulatory-compliant delivery of substances into the brain or CSF; the development of common protocols for clinical assessment; tracking of cells and substances following delivery; immunological reactions to the therapies; and trial design issues for interventional surgical studies.
- Determine working strategies, which may involve constituting several task and finish groups. These groups will identify specific barriers and will brainstorm ways forward. Part of this process may include review of the field, production of recommendations, identification of specific research requirements, planning of specific research projects and identification of funding pathways.
- Develop effective common protocols for specific processes for delivery of substances (including cell and gene therapies) into the brain.
In set up (June 2019). Please revisit this page for further updates.