First results from the Genetic FTD Initiative, and the advance of an HDAC inhibitor drug into Phase 2, made big splashes at the ICFTD conference held last month in Vancouver. Five hundred and ninety scientists from 30 countries met to exchange the latest clinical and scientific news on the diseases that make up FTLD. Multi-center cohort studies in Europe and North America reinforced the sense that there is a community spirit that may allow a common approach to how to develop therapies. Frontotemporal lobar degeneration can start in myriad ways—with social disinhibition, overeating, halting speech, or even odd misperceptions of pain or cold—but it always ends in dementia. Attendees said that the FTD field is poised for rapid progress.
Scientific advances fire up researchers at FTD meeting in a dreary Vancouver.
Confirming hints from case reports, the Genetic FTD Initiative, a European-Canadian frontotemporal dementia network, shows which brain areas change long before tau, progranulin, and C9ORF72 mutation carriers become symptomatic.
You might not think it possible to assemble large, longitudinal cohorts of loyal study subjects for rare dementias marked by apathy and emotional blunting. Think again—at the International Conference for Frontotemporal Dementia, researchers showed that they just did it.
Not to be outdone by ongoing FTLD cohort studies underway mostly in Europe, U.S.-Canadian studies are now forging structured networks to track presymptomatic and symptomatic cases and to engage a panoply of funders and advocacy groups in a clinical trials initiative.
Using physiological measurements, researchers are tracing behavior changes in frontotemporal dementia back to crumbling neural circuitry.
FORUM Pharmaceuticals announced that its histone deacetylase inhibitor has passed a Phase 1 trial, and will advance to Phase 2 immediately. It was one of two trials presenting data on active investigational drugs at ICFTD.
In debating how C9ORF72 causes neurodegeneration, researchers struggle to connect cell-culture results with divergent patterns of human disease.
At ICFTD, researchers proposed new genes and explored genetic differences that contribute to the vast diversity of FTD clinical presentations.
Whether by themselves or in the company of mutations that cause frontotemporal dementia, microRNAs may play a hand in driving disease.