J006 Surgical delivery of advanced therapy medicinal products (ATMPs) to the brain

<h3>Background</h3> Advanced therapy medicinal products (ATMPs) for neurodegenerative diseases, such as drugs, genes or cells, often do not cross the blood brain barrier and require direct delivery to the brain. Despite efforts to increase the biodistribution of such therapeutics, this remains a challenging step that reduces the efficacy of many treatments that reach the clinic. The Surgical Task Force aims to address these challenges and issue recommendations to improve and standardise delivery parameters. <h3>Aims</h3> To identify opportunities to contour bottlenecks in surgical brain delivery to the brain and improve biodistribution of ATMPs. <h3>Methods/Results</h3> As part of the EHDN Advanced Therapies Working Group (ATWG) and Stem Cells for HD (SC4HD), the Surgical Task Force (STF) gathers expertise from preclinical and clinical surgeons from two continents and multiple countries. Over a series of virtual meetings and online surveys, the STF aims to produce a consensus white paper that identifies the challenges of ATMP delivery to the brain on the basis of state-of-the-art techniques and devices currently used in clinical practice and identifies paths to go forward. The results of a recent survey that set the basis for such consensus will be presented. Working together, the group aims to provide guidance and guidelines, and potentially undertake collaborative research projects, to improve the efficacy of the biodistribution of ATMPs and increase the clinical success of therapeutic strategies for brain disorders. <h3>Conclusions</h3> Here we highlight challenges associated with delivering advanced therapies into the brain in the context of HD and other neurodegenerative diseases and have set up a task force to address themes outlined above. Reaching international consensus constitutes a first step in going forward and suggesting solutions to current issues that hurdle the implementation and regulatory acceptance of new treatments.