Introduction

(Howard Fillit, ADDF)

Neurodegenerative diseases currently affect 22 million people worldwide. Due to large projected increases over the next decade, neurodegenerative diseases are poised to become the major unmet medical need of the 21st century, surpassing cancer.

At the same time, there is a dearth of new molecular entities to serve as drug scaffolds.

  • There are currently less than 50 unique chemical scaffolds, upon which all the drugs on the market today are based.
  • Out of the millions of compounds synthesized every year, only a small percentage (10%) are disclosed in patents. An average of 9 new chemical entities are FDA approved per year.

In particular, the central nervous system poses many difficulties for drug discovery. For the major neurodenegerative diseases, the large majority of current treatments are symptomatic rather than disease-modifying.

  • Challenges in the development of new drugs include the lack of reliable biomarker assays and significant deficiencies in current animal disease models. (See ADDF animal model guidelines)

Many factors are involved in determining the success of a clinical candidate, including preclinical efficacy, drugability, safety and commercial viability.

  • Most CNS drugs fail due to a lack of efficacy, more so than other drug classes.

There is a great need for collaborative teams – across disciplines and between academia, industry and government – to meet this critical challenge of developing new, effective and safe drugs for neurodegenerative diseases.