The Amyloidosis Foundation is a non profit 501(c) (3) corporation and is an approved Charitable
Trust. In addition to our mission statement, the foundation is dedicated to all who have passed on
from amyloid, amyloid sufferers, their caregivers and family members; and also to the researchers
that are looking for the cure.
2014 Guillermo A. Herrera, MD - LSU
"Intravenous stem cells to repair/heal damaged kidneys"
Kidney involvement and eventual kidney failure are common in amyloidosis. If the kidneys fail, the only options are dialysis and transplantation, both of which can cause significant complica-tions. Today, amyloidosis is diagnosed earlier resulting in prolonged survival. The need for repairing damaged organs is obvious.
This grant investigates the use of stem cells administered intravenously to repair/heal damaged kidneys in an animal model. Stem cells are undifferentiated and can transform into specialized cell types. Results from this study will pave the way to use stem cells in human patients with amyloidosis to improve survival and quality of life.
2014 Jennifer Kollmer, MD - University of Heidelberg
"earlier treatment after initial diagnosis and successful therapies"
A severe impairment of the peripheral nerves is one of the main manifestations in hereditary amyloidosis (TTR-FAP). Our grant will develop a new and highly sensitive diagnostic tool 1) for the detection of very early nerve damage in gene-carriers without symptoms (family members of patients with already symptomatic disease), and 2) for the monitoring of nerve lesions in patients with symptomatic amyloid polyneuropathy under treatment. Our study of participants will be done in different groups of disease severity. We will then perform a MRI of the lower limb peripheral nerves to find out which have the highest sensitivity in detecting peripheral nerve damage and determining a certain stage of polyneuropathy.
This will hopefully lead to earlier treatment after initial diagnosis and successful therapies for patients with advanced disease.
2014 Rockland L. Wiseman, PhD - Scripps Research Institute
"use a single drug to broadly treat these devastating disorders"
The systemic amyloid diseases are a group of diseases caused by the build-up of unstable proteins that form toxic tangles in the blood. These tangles accumulate on organs such as the heart, gut, and kidney, lead-ing to organ failure and ultimately death. Currently, no treatments besides invasive surgery exist to treat the majority of these diseases, making systemic amyloid diseases a large, unmet medical need. We are developing new strategies to reduce the lethal accumulation of unstable proteins associated with systemic amyloid diseases by enhancing the natural, protective pathways that regulate the levels of unstable proteins in the blood.
Our establishment of this strategy will demonstrate that a single therapeutic approach can be used to treat many different systemic amyloid diseases, and therefore that it may be possible to use a single drug to broadly treat these devastating disorders.