Neuroinflammation is a hallmark of serious neurodegenerative diseases, such as frontotemporal lobar degeneration (FTLD). The underlying mechanism driving disease progression is characterized by abnormal function of the immune system. In a healthy person, regulatory T cells (Tregs), a type of blood white cells, maintain immune balance by arresting the cells producing inflammation and lowering the release of proinflammatory substances in the body. In FTLD, dysfunctional Tregs are unable to maintain immune balance, resulting in increased and persistent inflammation. This growing inflammatory environment prevents cell repair, leading to neuronal damage.

Published scientific data strongly support the role of Tregs dysfunction and increase of inflammatory mechanisms in FTLD.  Following the strong scientific evidence, we evaluated Treg cells ability to suppress inflammation in patients with FTLD. Results of our study demonstrate that Treg suppressive function is significantly decreased in FTLD compared to healthy controls.

In addition, available data using the combination of low-dose interleukin-2 and CTLA4-Ig (the components of COYA 302) to enhance Tregs function in animals and in preliminary human studies in amyotrophic lateral sclerosis (ALS) support the development of COYA 302 as a treatment strategy to effectively modify inflammation in FTLD patients to slow down the disease progression. Overall, our data supports the scientific rationale that harnessing the immune system to enhance Treg suppressive function and reduce inflammation responses is a logical therapeutic strategy to ameliorate neurodegeneration in patients with FTLD.

The goal of this proposal is to conduct a clinical study for the treatment of FTLD. Our goal is to assess the safety and efficacy of this combination therapy in individuals with a subtype of FTLD. The knowledge obtained from this study and this innovative disease-modifying treatment have the potential to be extended to other neurodegenerative conditions.