The Promise of Individualized Therapies

Sefton Eisenhart
Sefton Eisenhart

Individualized drug development is becoming more feasible on a larger scale as a result of new technologies, which could mean great things for rare disease communities. According to Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, “Academic clinician–investigators now have the capacity to rapidly uncover specific mutations and pinpoint the putative mechanisms leading to certain rare disease phenotypes.” This kind of specificity might mean researchers will have the ability to drill down further when curing specific illnesses.

Small populations have always posed problems in development processes. When it comes to rare diseases, it can be challenging to find enough patients to enlist in a clinical trial. By shrinking the number of patients needed for initial studies, individualized therapies might make it easier to build evidence that will result in bigger initiatives with more resources.

Recent news of a breakthrough in Tay-Sachs disease illustrates how individualized therapies can grow into full-blown clinical trials. Just two patients received gene therapy, and the initial success of the treatment has resulted in the licensing of the program by Axovant and the planning of a phase 2 clinical trial.

A small victory in the lives of two infants might have a positive impact on the lives of many more.