Space is often described as the final frontier for exploration, but medical researchers know that the challenges to understanding and treating diseases of the brain are similarly daunting. Despite steady progress in brain science, effectively penetrating the blood-brain barrier (BBB) to deliver medicines for many neurodegenerative and neuropsychiatric disorders remains a major hurdle.
In September, Janssen Neuroscience co-chaired a workshop at the U.S. National Academies of Science, Engineering and Medicine in Washington, D.C. to discuss current efforts to pioneer methods for penetrating the BBB to deliver therapeutics for nervous system disorders. I was honored to serve on the planning committee for this forum, which brought together nearly 100 leading experts from across the private and public sectors to evaluate a range of novel approaches to delivering medicines across the BBB. The workshop focused on identifying novel research, regulatory and partnering strategies that could lead to important breakthroughs in this critical field.
Husseini Manji, Janssen Neuroscience Global Head, and Danica Stanimirovic of the National Research Council of Canada kicked off the discussion by underscoring the challenges that researchers around the world have sought to overcome. “Getting drugs into the brain is a tall order,” said Dr. Manji. “While industry chemists have learned how to make small molecule drugs that cross the blood-brain barrier, this is a greater hurdle for large molecules like antibodies. We know that antibodies have had some success, but new technologies have the potential to get even more of these drugs into the brain, thereby further improving their efficacy.”
The BBB is no ordinary obstacle. It is composed of several cell types, passive and active transport mechanisms and highly adaptive functions capable of controlling the exchange of substances between a person’s blood and central nervous system. Today, few therapies can breach the BBB to reach the brain or spinal cord. However, promising innovative technologies are on the horizon, including nanoparticles, ultrasound and genetically engineered Trojan Horses that can deliver a therapeutic payload into the brain by binding to a BBB transporter protein.
The workshop also covered some of the current clinical trials that are taking place to evaluate synthetic molecules, biologics and gene therapies, as well as the regulatory considerations and best practices that should be applied when evaluating novel BBB therapies. The discussion yielded valuable insights about the importance of designing and funding trials that may lead to incremental, but significant, results that are the critical building blocks for subsequent research.
The potential for public-private partnerships to accelerate research and clinical translation was underscored in the workshop’s final panel, which included a cross-section of industry and government voices, as well as interesting insights from representatives of the Chan-Zuckerberg Initiative and the X-Prize Foundation. As panelists and participants noted, it is not enough to uncover promising new BBB therapies: the neuroscience community needs greater collaboration, and unconventional partnerships, that support long-term research and elevate the importance of treating nervous system disorders through more effective advocacy.
Bringing all of these elements together – innovative research, collaboration, and advocacy – is essential to yield important breakthroughs in our efforts to better understand diseases of the brain and to accelerate the development of transformative treatments for patients in need.