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Charles H. Hood Foundation | Novalia Pishesha, Ph.D. – July 2024
By identifying innovative pediatric advancements and providing funding in the critical phases of development, we are able to expedite high-impact breakthroughs that improve the health and lives of millions.
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Novalia Pishesha, Ph.D.

Assistant Professor of Pediatrics

Boston Children’s Hospital

Nanobody-based Immunotherapy for Modulating Inflammation in Juvenile Idiopathic Arthritis

 

Key Words: Nanobody, Targeted Drug Delivery, Corticosteroid, Juvenile Idiopathic Arthritis

Systemic Juvenile Idiopathic Arthritis (sJIA) is a serious condition that not only causes joint pain in children but can also lead to widespread inflammation and severe complications, like macrophage activation syndrome (MAS), which can be life-threatening. In this research project, we’re taking a novel approach to managing sJIA by harnessing the power of specially crafted nanobodies. These small proteins, sourced from alpacas, are being engineered to transport specific anti-inflammatory agents directly to the immune cells responsible for sJIA in a controlled mouse model of the disease. Our early findings are encouraging, showing a significant reduction in inflammation similar to what we’ve seen in models of rheumatoid arthritis (RA), another arthritic disease. This study is divided into two main objectives. The first aim is to dive deep into how these nanobody treatments affect a mouse model of sJIA by closely examining their distribution and action within the body and their overall impact on the immune system. The second aim seeks to enhance this new treatment strategy. We plan to do this by integrating additional anti-inflammatory drugs into our approach, aiming for a synergistic effect that offers longer-lasting and more comprehensive disease control. Additionally, we’re looking to assess the effectiveness of these engineered nanobodies on human immune cells to pave the way for potential human applications. The goal of this research is to develop a precise, targeted treatment for sJIA that minimizes side effects, offering a significant step forward in how we manage this challenging disease. By focusing on innovative solutions like nanobody technology, we hope to improve the quality of life for children with sJIA and potentially offer new treatment avenues for other pediatric conditions that require steroid-based therapies.