Validation & Biomarkers

In Vivo Validation

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. Combined, the nine person in vivo validation team has 100 years of experience. The staff has more experience with the SOD1 mouse model than any other, having studied more than 35,000 through more than 1000 different studies.

Testing therapeutics in vivo, especially the variety of therapeutics tested at ALS TDI, is a complex endeavor involving formulations, pharmacokinetics, and pharmacodynamics.

Formulations

Generally speaking, formulation is the process by which different chemical substances are combined to produce a final, potential medicinal product which can be tested. For our purposes, "Formulation" is used more broadly as an umbrella term to characterize the stage of therapeutic development where the treatment is being manufactured, purified, and prepared for experimentation in cell culture and/or in vivo. There are several different types of medicinal products which can be created in order to deliver and affect the intended pathway; the formulation process for the four being used at the ALS Therapy Development Institute are described here. Each has both advantages and disadvantages and it is not uncommon for more than one type of product to be created for a target pathway: