Ann: PAA completes Phase 1 and files for Orphan Drug Designation, page-379

Bruce Willis has frontotemporal degeneration (FTD).

FTD represents a group of brain disorders caused by degeneration of the frontal and/or temporallobes of the brain. It is also frequently referred to as frontotemporal dementia, frontotemporal lobardegeneration (FTLD), or Pick’s disease.

Currently, no treatments are available to cure or slow the progression of FTD, but healthcare providers may prescribe medicine to treat symptoms. Antidepressants may help treat anxiety and control obsessive-compulsive behaviours and other symptoms. People with FTD typically live six to eight years with their condition, sometimes longer, sometimes less. Most people die of problems related to advanced disease

Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are diseases that affect the neurological system. Research has found that the genes and genetic components that play a role in both conditions are similar. ALS and FTD are considered neurodegenerative diseases. That means that the neurological system and neurons (nerve cells) are affected. Both conditions develop because motor neurons, the cells that aid in movement, become damaged or destroyed.In the case of ALS, motor neurons found in the frontal cortex, brainstem, and spinal cord are affected. In FTD, the motor neurons in the frontal and temporal lobes of the brain are damaged.

Though it’s unclear whether having one makes it more likely to have the other, 30–50% of people with ALS or FTD will develop both conditions. Researchers have found that the connection between the two, besides the neurological link, comes down to genetics. FTD's estimated U.S. prevalence is around 60,000 cases, and many in the medical community remain unfamiliar with it. FTD is frequently misdiagnosed as Alzheimer's, depression, Parkinson's disease, or a psychiatric condition.

Pathomechanism Heterogeneity in the Amyotrophic Lateral Sclerosis and Frontotemporal Dementia Disease Spectrum: Providing Focus Through the Lens of Autophagy

Abstract

The autophagy-lysosome system has been identified as a main intersection for the onset and progression of neurodegeneration in ALS/FTD. Genetic evidence has revealed that several genes with a mechanistic role at different stages of the autophagy process are mutated in patients with ALS/FTD. Moreover, the three main proteins aggregating in ALS/FTD, including in sporadic cases, are also targeted by autophagy and affect this process. Here, we examine the varied dysfunctions and degrees of involvement of the autophagy-lysosome system that have been discovered in ALS/FTD. We argue that these findings shed light on the pathological mechanisms in the ALS/FTD spectrum and conclude that they have important consequences both for treatment options and for the basic biomolecular understanding of how this process intersects with RNA metabolism, the other major cellular process reported to be dysfunctional in part of the ALS/FTD spectrum.

Is amyotrophic lateral sclerosis/frontotemporal dementia an autophagy disease?

Abstract

Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are neurodegenerative disorders that share genetic risk factors and pathological hallmarks. Intriguingly, these shared factors result in a high rate of comorbidity of these diseases in patients. Intracellular protein aggregates are a common pathological hallmark of both diseases. Emerging evidence suggests that impaired RNA processing and disrupted protein homeostasis are two major pathogenic pathways for these diseases. Indeed, recent evidence from genetic and cellular studies of the etiology and pathogenesis of ALS-FTD has suggested that defects in autophagy may underlie various aspects of these diseases. In this review, we discuss the link between genetic mutations, autophagy dysfunction, and the pathogenesis of ALS-FTD. Although dysfunction in a variety of cellular pathways can lead to these diseases, we provide evidence that ALS-FTD is, in many cases, an autophagy disease.

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Why the sudden interest in FTD, what does it man for PAA and our development of treatment for further indications and multiplying value.

I was led here by our CEO via LInkedIn







Seems we may be lining up to offer hope for another rare disease with NO treatment at all.

Treatment, Management and What to Expect

Today, there is no cure for FTD, and unfortunately, no current treatments slow or stop the
progression of the disease. However, a growing number of interventions can help manage FTD’s
symptoms and maximize quality of life.