The SENS Foundation, a charity dedicated to accelerating the development of rejuvenation biotechnology was given the opportunity recently to fund a research project towards therapies in the Genetic Amyloidosis field.
More specifically, the project will focus on therapies for ‘Senile’ or ‘Age-Related’ Systemic Amyloidosis (SSA) caused by aggregated wild-type ‘transthyretin’ (TTR), and isolated atrial amyloidosis (IAA), caused by aggregated atrial natriureptide (ANP). These cardiac amyloidoses are poised to become a widespread medical problem for the global aging population. The deposits of abnormal protein or transthyretin affect the heart tissue, resulting in decreased heart function and deterioration.
The prevalence of cardiac amyloidoses is shocking among the elderly and instances of this disorder are rising rapidly. Cases are now beginning to increase among the “Baby Boomer” generation in the United States and the incidence and severity of the disease will certainly rise further as this group ages.
Thanks to the important research and collaborative efforts of Dr. Stan Primmer, Dr. Alan Solomon and Dr. O’Nuallain, there has been significant progress towards a promising novel approach to catalytic Aβ-targeting antibody fragments that cleave, rather than sequester, the pathological aggregates of wild-type protein underlying SSA.
Stan Primmer is the President of the Supercentenarian Research Foundation (SRF). Because of his involvement with SRF, Primmer had long been very conscious of the clinical burden of SSA, and had surveyed the landscape of possible therapeutic approaches. While only a limited number of pathological studies have yet to be performed on humans in this age category, results to date have been highly suggestive that the most significant factor in the death of these individuals was senile systemic amyloidosis.
Primmer, Solomon and O’Nuallain had proposed the following four-phase research and development project:
Phase 1 will consist of in vitro generation of two separate sets of monoclonal antibodies: (a) antibodies that bind to TTR amyloid for subsequent diagnostic use, and (b) catalytic antibodies that can directly destroy TTR amyloid.
Phase 2 will consist of studies in an animal model of TTR amyloidosis to determine the safety and efficacy of the potential diagnostic and therapeutic antibodies discovered in Phase 1.
Phase 3 will involve clinical trials on the diagnostic and therapeutic potential of the novel antibodies for comparatively young human subjects who develop amyloidosis due to mutation(s) in the TTR molecule. Initial testing of the methodology in the younger cohort is designed to avoid risk to the uniquely fragile group consisting of supercentenarians.
Phase 4 will then serve to apply the results of previous research to volunteer supercentenarians in order to improve their health and extend their lives beyond what they would otherwise be expected to live.
With regards to funding this important project, the SENS Foundation’s Research Committee approved the plan for Phase I as well as a $150 000 research investment that has been disbursed to the researchers and work is already underway.
More about Senile Systemic Amyloidoses Research.