Validation & Biomarkers

In Vitro Validation

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. These include quantitative polymerase chain reaction (qPCR), enzyme linked immunosorbent assays (ELISA), and immunoblots using the fluorescent quantitation.

Biomarkers

The ALS TDI mission is to slow and stop ALS. The development of ALS specific biomarkers is complementary to this mission. 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. There are no validated biomarkers for ALS to date. Development of biomarkers for ALS could result in earlier disease interventions and additional end points to evaluation whether or not a treatment is effective.

In 2008, ALS TDI accelerated its ALS biomarker effort by expanding its network of clinical partnerships. To facilitate ALS patient participation, ALS TDI submitted and executed protocols to hospitals and clinics for the collection of blood, muscle, and adipose tissue. The program is designed to analyze samples from sporadic and familial ALS patients and compare them to samples from healthy individuals or individuals afflicted with other neurodegenerative conditions. The goal is the discovery of an ALS specific biological signal. The same genomics and proteomics technologies that are used by the Research Biology group to better understand the underlying pathology of ALS and identify the most promising ways to attack the disease are also used to identify biomarkers of disease.