Pre-Conference Workshop Day

Workshop A: Investigating BBB Disruption Devices - Focused Ultrasound to Temporarily Disrupt the BBB

9:00 – 12:00

There is an ever-growing interest in leveraging devices modulating the BBB such as focused ultrasound and microbubbles to temporarily open the BBB. This idea has led to much controversy in the field but increasing evidence shows that these techniques could have the potential to add significant and much needed value to help get more therapeutics to the brain.

This workshop aims to provide an unbiased overview of the role of devices to increase the permeability of the BBB looking at the pros and cons of different platform technologies to enable BBB penetration.

We will explore these technical questions unfolding:

  • Insight into how the oscillation of the microbubbles causes mechanical effects on the capillaries in the target area and how this results in a transient loosening of the blood-brain barrier
  • Exploring different methods to deliver ultrasound, what makes one technology better than another? How are we going to rank them?
  • How long does it take for the BBB to fully close?
  • Is there a safety window? Evaluating the safety and effectiveness of the technique. If you open the BBB, what else enters the brain? Is the risk outweighed by the benefit?
  • Investigating Preclinical work with various pathologies and different therapeutics:
    • How can it be useful for neurodegenerative diseases?
      Alone or in combination with treatments?
    • How does the ultrasound affect the amyloid? To what extent?
    • Are repeated treatments necessary and feasible?
  • What is feasibility, acceptability, clinical-, and cost-effectiveness?
  • How can we implement the technology on a large scale? What is the route towards more clinically validated technology?
  • What is the immune response to the ultrasound?
  • How translatable from mice to non-human primates is this?
  • Are we inching closer to understanding the mechanism behind the therapeutic effects of ultrasound alone?

This workshop will equip you with the knowledge to better understand the strengths and limitations of BBB modulation using focused ultrasound and highlight where we see opportunity with this technology and what combination of drug / device would best
help patients suffering with neurodegenerative diseases.

Workshop Leaders:

Elisa Konofagou

Elisa E. Konofagou
Robert & Margaret Hariri Professor of Biomedical Engineering, Professor of Radiology
Columbia University

Rikke H Kofoed

Rikke Hahn Kofoed
Post-doctoral Fellow
Sunnybrook Research Institute

Workshop B: Bypassing the BBB for More Effective Delivery to the Brain

1:00 – 4:00

This workshop provides an evaluation of intranasal and intrathecal delivery routes of drug administration for brain targeting.

Honing into the following topics:

  • Exploring the main pathways of intranasal and intrathecal drug absorption
  • Insights into how these intranasal / intrathecal drugs can bypass the BBB and blood-CSF barrier
  • Evaluating the in vitro, ex vivo and in vivo models for testing drug penetration by these routes
  • What is the drug penetration rate and how can we use this in conjunction with other devices and therapeutics?
  • What are the different pumps for intrathecal delivery?
  • How can transporter modulation aid the delivery of the drug?
  • What is the effectiveness of nasal delivery or intrathecal delivery and how safe they are?
  • How would the molecule have to be different? Would you have to utilize different receptors? Would the delivery depend on transcytosis at all?

Leave this session having gained valuable insight into different techniques and approaches towards bypassing the BBB.

Workshop Leaders:

Larry Brown

Larry Brown
Executive Vice President & Chief Scientific Officer
Noveome Biotherapeutics

Ben Bleier

Benjamin S. Bleier
Post-doctoral Fellow, Associate Professor, Director of Translational Research & Endoscopic Skull Base Surgery, Massachusetts Eye and Ear
Harvard Medical School

Mansoor

Mansoor M. Amiji
University Distinguished Professor, Professor of Pharmaceutical Sciences & Chemical Engineering
Northeastern University