Research Breaks through to the Other Side of the Blood-Brain Barrier

A quiet revolution is occurring at Lifespan’s Center of Biomedical Research Excellence (COBRE) for Skeletal Health and Repair that may change the way patients with specific cancers, brain diseases, and musculoskeletal disorders are treated.

After 12 years of research, Qian Chen, PhD, director of the center, and a team of scientists, clinicians, and engineers have created Nanopieces, a miniscule tube-like vehicle that packs a powerful delivery system to specifically target a disease gene with a sustained, efficient, and nontoxic therapy.

Beyond the Blood-Brain Barrier

The research, initially intended to discover new treatment for osteoarthritis, has also increased the potential for treatment of diseases including bone cancers, dementia, and autoimmune diseases. Nanopieces provide a unique, non-harmful way to penetrate the blood-brain barrier, a network of blood vessels and tissue that is made up of closely spaced cells and prevents harmful substances from reaching the brain.

The blood-brain barrier allows some substances, such as water, oxygen, carbon dioxide, and general anesthetics, to pass into the brain, while preventing entry to bacteria and other substances, including many anticancer drugs. Nanopieces allows nucleic acid therapeutics to inhibit diseases at the genetic source rather than targeting symptoms or by-products of the disease. 

Funding Supports Lifespan Research

The breakthrough technology is a product of NanoDe Therapeutics, Inc., a development-stage company dedicated to developing a new generation of nucleic acid therapeutics and founded around technology developed at Lifespan and Brown University. A stream of federal funding from the National Institutes of Health through a Small Business Innovation Research (SBIR) grant, contract and other sources, including Rhode Island Commerce Corporation, has funded the research. 

“The blood-brain barrier has been a stumbling block to most drugs that are intended to reach the brain,” Dr. Chen said. He is also vice chair for research at the department of orthopedics at The Warren Alpert Medical School of Brown University. “Our research into treatment for debilitating conditions such as osteoarthritis has led to a novel discovery that we hope can be further utilized to develop treatments for cancer and dementia patients.” 

Research and education are major components of Lifespan’s strategic plan as it deepens its role in the development of new medical treatments and cures through collaboration with leading academic institutions and healthcare systems.