Frontotemporal Dementia (FTD)
Frontotemporal dementia (FTD) is a rare, rapidly progressing neurodegenerative disease that is the most common form of dementia for people under the age of 60. Patients with FTD frequently develop symptoms such as behavioral changes, lapses in judgment, and diminished language skills when they are in their 40’s and 50’s with the disease running its course in 7-10 years. There are currently no FDA-approved treatment options available for any form of frontotemporal dementia.
Our efforts to advance the treatment of frontotemporal dementia
Alector’s pioneering work in the search for treatments that target the dysfunctional brain immune system is guided by an understanding of human genetics. By linking genetic information to immunology targets that may be losing functionality due to aging processes or mutations, we aim to restore healthy immune system function, slow the progression and prevent occurrence of neurodegenerative diseases like frontotemporal dementia.
There are multiple inherited forms of frontotemporal dementia, and research to understand the genetic causes or risk factors is ongoing, but we know that progranulin mutations play a direct role in FTD. Progranulin is a secreted protein with multiple functions including neuro-protection and regulating neuroinflammation. To date researchers have identified more than 120 inherited loss of function mutations in the progranulin gene that lead to frontotemporal dementia.
Our lead pipeline program, AL001, focuses on restoring diminished progranulin protein levels to normal ranges to help regulate the growth, division and survival of microglia cells. Microglia are the primary innate immune system cells of the central nervous system and play a crucial role in maintaining neuronal health.
Alector’s clinical trials in frontotemporal dementia
Today, there are no approved therapeutic treatments for FTD, though interventions and lifestyle modifications can help patients and their families better cope with the disease and find support. Alector is currently conducting the INFRONT-3 Phase 3 study, a randomized clinical trial of AL001. To test its efficacy in treating FTD-GRN INFRONT-3 is currently enrolling symptomatic and at-risk FTD patients with a progranulin mutation (FTD-GRN) in the U.S., Europe and Australia. A Phase 2 clinical trial in frontotemporal dementia patients with a C9orf72 genetic mutation is also ongoing.