Chronic traumatic encephalopathy - a potential orphan indication for Alzheimer's drugs


In July, researchers at Boston University reported that 110 out of 111 brains donated by families of former NFL players showed signs of chronic traumatic encephalopathy (CTE), a rare condition marked by tau tangles in the brain.

The prevalence rate of the condition is unknown. While the research team is not predicting any rates for the future, the study shows a powerful link between CTE and American football, making it as an unexplored market with strong future potential for drug development.

No treatment has ever been approved for CTE. It belongs to a group of neurological conditions marked by aggregated forms of tau protein. Like many tauopathies, CTE presents symptoms that span from depression and suicidal thoughts to language and motor deficiencies.

Although much about CTE is still unknown, it has a potential for being identified in many more patients, particularly in American footballers, rugby players, boxers, and military veterans.

A number of products are already being developed for the most common tauopathy, progressive supranuclear palsy (PSP). Companies developing products in this market include Otsuka, TauRx, Abbvie, Merck & Co, and Bristol-Myers Squibb.

Many manufacturers are investigating their tau-targeting products in PSP patients, given that PSP is the best understood of all tauopathies and it has the largest patient population. However, once approved for use in PSP patients, these PSP-indicated products are likely to be used off-label in other closely related indications, making tauopathies an attractive indication group.

In addition, tau-targeting drugs are likely candidates for treating Alzheimer’s disease, given both conditions share tau-based pathology. This means that the product will receive incentives for targeting a niche tauopathy indication through orphan designation, and will have the potential to become a blockbuster if it is then shown to be effective in patients with Alzheimer’s disease.

The first tauopathy drug to market is expected to see a sharp uptake in patients, given the vast unmet needs in this area. As researchers’ understanding of CTE improves and its diagnosis during patients’ lifetimes becomes possible, the potentials for these tauopathy drugs will continue to grow.