research
Strategy
The ALS TDI's unique and comprehensive approach leverages over a decade of treatment focused ALS research and applies an industrial
scale to efficiently advance therapeutics to patients. With a focus always on the shortest path to treating today's ALS patients, the
ALS TDI research team evaluates the most promising therapeutic hypotheses through the lenses of the richest ALS research databases in the
world. Treatments of all kinds are tested as rigorously and rapidly as possible.
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TECHNIQUES
Research Biology
The ALS Therapy Development Institute's scientists aim to discover and prioritize the most promising strategies to slow,
stop, and reverse amyotrophic lateral sclerosis (ALS) disease progression. The Research Biology team dissects ALS pathology
using broad genome-wide profiling technologies (trillions of data-points) integrated with high resolution studies of single
proteins within single cells.
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Gene Therapy
Gene Therapy is a highly targeted therapeutic approach designed to insert modified
genes into cells in order to produce a potentially therapeutic protein or reduce production
of a potentially toxic target. In a neurodegenerative disease like ALS there are multiple
cell types potentially involved in disease including motor neurons, glial cells in the
central nervous system, skeletal muscle, and peripheral immune cells. In order to reduce
potential toxicity it is often necessary to target the gene therapy construct to specific
tissues or even specific cell types within a given tissue.
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Validation & Biomarkers
In vitro validation uses living cells grown in nutrient solutions for experiments that
confirm the activity of promising therapeutics prior to initiation of longer and more
costly in vivo (animal) studies. Many of the same technologies used to evaluate in vivo
studies are also employed to interpret in vitro results. Biomarkers are biological signals
that can be used to definitively diagnose a condition or disease and can also correlate
with disease progression. Biomarkers can come from blood sampling, tissue biopsy, or less
invasive techniques like skin elasticity measurement.
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Testing Therapeutics
In vivo validation involves testing therapeutics in an animal model of disease. ALS TDI
tests small molecules, gene therapies, biologics and stem cell products in its
state-of-the-art research facility. Established in 1995, the SOD1 mouse model is
internationally accepted as a robust model for ALS research. In 2008, ALS TDI
published guidelines for the model's use and interpretation of experimental
results.
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