In recent years, neurodegenerative diseases have gained prominence not just in the scientific community, but in pop culture. Films like Concussion and Still Alice investigated the subject from the perspectives of researchers and patients; actor Seth Rogen has become a high-profile advocate for Alzheimer’s research, even taking the cause before Congress in 20141. This attention is not unwarranted. Neurodegenerative diseases can be debilitating and often fatal. They can develop unexpectedly and be elusive to treatment2. And they affect millions upon millions of people worldwide.
There are currently two Phase I clinical trials underway for treatments targeting progressive supranuclear palsy (PSP), a neurodegenerative disease caused by an accumulation in the brain of a protein called tau. Like most Phase I trials, they aim to test the treatments’ safety and tolerability with a secondary focus on efficacy; however, they are atypical in their use of patients diagnosed with PSP as opposed to healthy volunteers. The treatments are designed to treat PSP by clearing tau from the brain, and if the trials yield positive results, the implications could be far-reaching: numerous other neurodegenerative diseases, including Alzheimer’s and chronic traumatic encephalopathy (CTE), are caused by tau buildup, and researchers hope that if the medications prove successful against PSP, they could be applied to those diseases as well3.
The chart below shows the approximate number of Americans who are directly affected by four tau-related diseases and who could therefore benefit from the findings of the Phase I trials currently in progress.
The ability to clear tau from the brain could unlock treatments and cures for a host of neurodegenerative diseases. Though cases of PSP are relatively rare, the Phase I PSP trials currently underway could lead to a breakthrough for millions.
1 Begley, Sarah; Seth Rogen Says Government Needs to Step Up On Alzheimer’s; Time; 4/23/2015
2 Robertson, Sally; What is Neurodegeneration?; News-Medical.net; 1/21/2015
3 Woods, Alisa; Anti-Tau Clinical Trials for PSP May Shed Light on Alzheimer’s, Other Diseases; Alzheimer’s News Today; 3/22/2016
4 2016 Alzheimer’s Disease Facts and Figures; The Alzheimer’s Association
5 Statistics on Parkinson’s; Parkinson’s Disease Foundation
6 Progressive Supranuclear Palsy Fact Sheet; National Institute of Neurological Disorders and Stroke; Sept. 2015
7 Breslow, Jason M.; New: 87 Deceased NFL Players Test Positive for Brain Disease; Frontline; 9/18/2015