As recently as two years ago, there was little evidence of any academic or biotech industry interest in pursuing clearance of AS as a therapeutic approach to PD, LBD, or other
synucleinopathies of aging: after a promising 2005 report, [31] there had been virtual silence.
«Our findings contribute important new evidence about the natural history and survival of people affected by
synucleinopathies of various types.
Rocha EM, Smith GA, Park E, Cao H, Brown E, Hayes MA, Beagan J, McLean JR, Izen SC, Perez - Torres E, Hallett PJ, Isacson O. Glucocerebrosidase gene therapy prevents α -
synucleinopathy of midbrain dopamine neurons.
Not exact matches
The population - based study by Rodolfo Savica, M.D., Ph.D., and coauthors
of the Mayo Clinic, Rochester Minn., included all the residents
of Minnesota's Olmsted County and identified 461 patients with
synucleinopathies and 452 patients without for comparison.
Of the 461 patients with
synucleinopathies, 316 (68.6 percent) died during follow - up, while among the 452 participants used for comparison, 220 (48.7 percent) died during follow - up.
«New clues on the base
of Parkinson's disease, other «
synucleinopathies».»
The test could enable earlier diagnosis
of a group
of degenerative brain diseases called
synucleinopathies, which include Parkinson's disease, Lewy Body dementia, and multiple system atrophy.
«This study shows that researchers working on problems in rare diseases, such as blood pressure in
synucleinopathies, can pool together data and ideas and develop a simple bedside test that can perhaps help spot these problems earlier,» says Dr. Kaufmann, the Felicia B. Axelrod Professor
of Dysautonomia Research in the Department
of Neurology, and professor
of medicine and pediatrics at NYU School
of Medicine.
Although several large studies have shown that α - synuclein levels are lower in the CSF
of PD patients and those with related
synucleinopathies compared to controls, its role in cognitive decline and dementia had been unexplored.
Yet if you look at people who develop the clinical syndrome
of dementia, especially later in life, yes, they have amyloid in the brain but they also have other pathologic entities — vascular disease;
synucleinopathies [insoluble fibrils
of the normally soluble protein, alpha - synuclein]; a tauopathy [which is marked by disease - inducing, insoluble tangles
of another protein, tau].
Roche and Prothena will collaborate on the development
of PRX002 for Parkinson's disease and potentially other
synucleinopathies.
Lewy bodies (LB) and other intracellular α - synuclein (AS) aggregates accumulate in the aging nervous system, and a high burden
of such aggregates are hallmark neuropathological signs
of Parkinson's disease (PD), Lewy body dementia (LBD), multiple systems atrophy (MSA), and other
synucleinopathies.
In the ensuing months, the two companies have published detailed, promising animal studies
of their immunotherapies, including preclinical efficacy studies in animal models
of human
synucleinopathies.
The clinical consortium is led by AFFiRiS itself, and in addition to the PD booster trial, it is now recruiting for a separate Phase I trial
of the α - synuclein vaccine against MSA, a
synucleinopathy differentiated from PD or LBD based on the cell populations affected, the regional concentration
of AS neuropathology, and the spectrum
of movement and other symptoms.
One might speculate that each
of these damage - repair therapeutics may exhibit differential targeting
of particular AS species or brain regions, as different clinical
synucleinopathies exhibit distinct regional localizations and conformations
of AS pathology.
AFF 1: Preclinical Efficacy Data AFF 1 was then tested for preclinical efficacy in two models
of human
synucleinopathy: mThy1 - AS mice, and also PDGF - AS mice expressing WT human AS under the PDGF - β promoter — a model with closer neuropathological and metabolic resemblance to LBD than PD.
The company's antibody - based product candidates target a number
of potential indications including AL and AA forms
of amyloidosis, Parkinson's disease and other related
synucleinopathies and novel cell adhesion targets involved in inflammatory diseases and cancers.
The therapies have a broad range
of indications including Parkinson's disease and related
synucleinopathies.