A new
National Institutes of Health grant merges two ongoing frontotemporal
lobar degeneration (FTLD) studies to form a new, integrated consortium.
FTLD is a rare disease that can affect parts of the brain
responsible for personality, behavior, language, and motor function.

The funding,
from NIH’s National Institute on Aging (NIA) and National Institute of Neurological
Disorders and Stroke (NINDS), is expected to total more than $63 million over
five years to advance the development of treatments for FTLD.

The existing
NIH-supported studies — Advancing Research and Treatment in Frontotemporal
Lobar Degeneration (ARTFL) and Longitudinal Evaluation of Familial
Frontotemporal Dementia Subjects (LEFFTDS) — together form the new
ARTFL–LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD) research
consortium. The overarching goal for the consortium is to prepare for clinical
trials by helping researchers better understand the FTLD disease process by
finding improved methods for accurately identifying participants with FTLD and
for measuring disease progression. Specifically, the consortium aims to find the
best biomarkers, such as MRI neuroimaging, to track disease burden and develop
ways to calculate, and predict risk of worsening symptoms.

“This
unified approach is another example of collaborative NIH efforts meant to speed
up discovery in a very challenging research area and make measurable progress
against a devastating group of diseases,” said NIA Director Richard J. Hodes,
M.D. “The discoveries made in FTLD could also help with finding treatments for
other dementias, such as Alzheimer’s disease.”

While
Alzheimer’s disease is the most common dementia, scientists think FTLD is the most
common cause of dementia in people younger than age 60. Roughly 60% of
people with FTLD are 45 to 64 years old, and people as young as
in their 30s can be affected. There are no current treatments for FTLD, which
is the neuropathological definition for frontotemporal dementia (FTD).

FTLD is
associated with shrinking of the frontal and temporal lobes of the brain and
shares some characteristics of Alzheimer’s disease, including misfolded
proteins and loss of brain cells. However, the diseases that make up FTLD are
distinct. For example, while Alzheimer’s is primarily associated with amyloid
and tau proteins, FTLD can be caused by another form of tau or the protein
TDP43. While Alzheimer’s is characterized by problems in memory, the range of
symptoms of FTLD can include unusual behaviors, emotional problems, trouble
communicating, difficulty with work or difficulty with walking.

“We are
deeply aware of the large burden of these brain diseases on people as well as
their families and caregivers, and recognize the urgent need for therapies,”
said NINDS Director Walter Koroshetz, M.D. “Key to this research is measuring
and understanding how FTLD progresses so we can slow or stop it.”

Now combined
within the ALLFTD consortium, the LEFFTDS study enrolls and follows individuals
from families with a known genetic mutation causing FTD, and the ARTFL study
focuses on those without known mutations, although some have strong family
histories without one of the known FTD mutations.

Led by
researchers at the Mayo Clinic, Rochester, Minnesota, and the University of
California, San Francisco, ALLFTD is a single infrastructure using the same
clinical data platform with collaborative decision-making among 18 sites.

National Institute on Aging (NIA): NIA leads the U.S. federal government effort to conduct and support research on aging and the health and well-being of older people.

National Institute of Neurological Disorders and Stroke (NINDS): NINDS is the USA’s leading funder of research on the brain and nervous system.

National Institutes of Health (NIH): NIH, the USA’s medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services.