Challenges and Opportunities in Developing Neurologic Therapies | Page 4

New Approaches to Neurologic Drug Development
Ongoing developments in basic science and in efficient capture and sharing of
data are creating opportunities for advancing the treatment landscape.

Targeted therapeutics. Increased use of new technologies such as genetics,
genomics, proteomics, and imaging will play an important role in helping
researchers to identify biomarkers to improve our understanding of the disease
process (Gomez-Mancilla, 2005). This will be the prerequisite to tailor drugs to
the particularities of the disease, serving both purposes, becoming more
efficacious as well as limiting unwanted side effects by unspecific non-targeted
pharmacological effects.
Having a greater understanding of genetic and molecular pathologies allows for
development of more targeted therapies. For example, several drugs in clinical
development target individual protein kinases, which are enzymes involved in
intracellular and extracellular signaling (Chico. 2009).

Onset of disease. Parties in industry and academia are developing biomarkers
for detecting the presence of mild disease (Pritchard, 2008). Detecting disease
before symptoms appear could lead to development of therapies that block
progression to severe disease.

Efficacy evaluations. In measuring subjective outcomes, trained independent
evaluators may be used in clinical trials to overcome concerns about inter-rater
and intra-rater reliability in measuring subjective outcomes.
Biomarkers are also a valuable tool in evaluating the effectiveness of a novel
therapy. As more are developed, the use of subjective measures in evaluating
progression of neurologic disease should decline. However, a hurdle to be
overcome is the predictive value of a given biomarker with regard to its
therapeutic effect to be established later in the drug development process.
Increasing emphasis is being placed on finding approaches for early assessment
of a drug’s potential for long-term success. Evaluations can be done at the stage
of screening potential compounds that can save time in refining the
composition of candidate drugs (Chico, 2009).

Use of technology. Digital collection and dissemination of data allows for rapid
communication of efficacy and safety data, which can be key to ensuring the
success of early phase clinical studies. Technology adoption can speed and
facilitate start-up of study sites, patient accrual, clinical monitoring, site
management, inventory monitoring, and study close-out.