Author Archives: jpnd

New study from the USA aimed at modeling the course of Parkinson’s disease (PD) which describes the economic consequences of slower rates of progression

Multiple studies describe progression, dementia rates, direct and indirect costs, and health utility by Hoehn and Yahr (H&Y) stage, but research has not incorporated these data into a model to evaluate possible economic consequences of slowing progression.

A Markov model was developed by the authors to show the net monetary benefits of slower rates of progression. Four scenarios assuming hypothetical slower rates of progression were compared to a base case scenario.

Base case results indicate average excess direct costs of $303,754, life-years of 12.8 years and quality-adjusted life-years of 6.96. A scenario where PD progressed 20% slower than the base case resulted in net monetary benefits of $60,657 ($75,891 including lost income) per patient. The net monetary benefit comes from a $37,927 decrease in direct medical costs, 0.45 increase in quality-adjusted life-years, and $15,235 decrease in lost income.

The scenario where PD progression was arrested resulted in net monetary benefits of $442,429 per patient. Reducing progression rates could produce significant economic benefit. This benefit is strongly dependent on the degree to which progression is slowed.

Study details how brain enzyme interacts with drug-like lead compound for Huntington’s Disease

Researchers at the Manchester Institute of Biotechnology have detailed how an enzyme in the brain interacts with an exciting drug-like lead compound for Huntington’s Disease to inhibit its activity. The research is published in the journal Nature.

Working with colleagues at the University of Leicester and the University of Lisbon in Portugal, the researchers identified the molecular structure of the enzyme kynurenine 3-monooxygense (KMO), which is found in the human brain. It took five years for the team to establish the crystal structure of KMO – the first time it’s ever been done.

The scientists then studied how the compound UPF 648 binds incredibly tightly to the enzyme to act as an inhibitor. Previous studies with animal models of neurodegenerative disease have showed that switching off the enzyme activity through drug binding should be effective in the treatment of brain disorders.

Professor Nigel Scrutton who led the study said: "UPF 648 works very well as an inhibitor of enzyme activity. However, in its current form it does not pass into the brain from the blood. The search is now on for related compounds that can both inhibit the enzyme and pass into the brain."

The findings from this research will now be used in the search for more effective treatments for Huntington’s Disease.

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According to the latest US Alzheimer’s Association report, one in three seniors dies with Alzheimer’s or another dementia in the United States.

The new report shows that while deaths from other major diseases, such as heart disease, HIV/AIDS and stroke, continue to experience significant declines, Alzheimer’s deaths continue to rise — increasing 68 percent from 2000-2010.

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Promoting a shift to open science

It is generally accepted that outputs from publicly-funded research should be publicly available not only to researchers, but also to potential users in education, business, charitable and public sectors, and to the general public. 

The two major Open Access (OA) elements are OA for publications and OA for research data. These issues are under active consideration by a number of international initiatives. 

In this regard, aResearch Data Alliance  (EU-US-Australia) has been formed and held itsinaugural meeting on March 20th, 2013. The RDA alliance was formed initially by three research funding organisations:

• the Australian Commonwealth Government through the Australian National Data Service (www.ands.org.au)
• the European Commission through the iCordi project (www.icordi.eu)
• the United States of America through the RDA/US activity of the National Science Foundation (www.nsf.gov).

The goal of the alliance is to accelerate international data-driven innovation and discovery by facilitating research, data sharing and exchange, use and re-use, standards harmonisation for specific communities and across scientific disciplines.

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A systems biology & comparative genomics approach for studying human brain ageing and/or most common age-related diseases

A new European group of academic laboratories and industrial scientists from SMEs will combine an integrative systems biology & comparative genomics approach for studying human brain ageing with a special emphasis on late-onset Alzheimer Disease. The objective will be to identify and validate new molecular targets and biomarkers.

The project is calledAgedBrainSYSBIO – systems biology of synapse proteins & ageing. AgedBrainSYSBIO is a European collaborative research project funded by the European Commission under the Health Work Programme of the 7th Framework Programme.

A new study supports the concept that prolonged and intensive environmental stimulation may have beneficial effects in delaying one of the key negative factors in Alzheimer’s disease.

It is well known that staying mentally active throughout life lowers the risk of developing Alzheimer’s disease, but how this works is unknown.

In theMarch 6 edition of Neuron, researchers reported that beta2-adrenergic signaling plays a key role. Wild-type mice that explored new toys every day had more synaptic plasticity and better resisted the toxic effects of ABeta oligomers compared to mice housed in the standard, boring cages. When researchers blocked the beta2-adrenergic pathway, the protection vanished. Conversely, feeding a beta2-adrenergic agonist to mice in the bland cages mimicked the benefit of an enriched environment.

Intriguingly, beta-adrenergic signaling has been shown to decline in AD brains.

Moreover, the scientists found that exposing the brain to novel activities in particular provided greater protection against Alzheimer’s disease than did just aerobic exercise. According to the researchers, this observation may be due to stimulation that occurred not only physically, but also mentally, when the mice moved quickly from one novel object to another.

Diseases include ALS and a form of dementia called IBMPFD

Single mutations in one gene rarely cause different diseases. New research, published in the journal Neuron, gives insight into how single mutations in the VCP gene cause a range of neurological conditions including the motor neuron disease Amyotrophic Lateral Sclerosis (ALS) and a form of dementia called Inclusion Body Myopathy, Paget’s Disease of the Bone and Frontotemporal Dementia (IBMPFD) 

This study shows that these mutations disrupt energy production in cells shedding new light on the role of VCP in these multiple disorders.  

Genome-wide imaging study identifies a new gene called BCHE, found to be associated with Alzheimer’s plaques

The is the first genome-wide association study of plaque deposits using a specialized PET scan tracer that binds to amyloid – florbetapir.  This imaging tracer allows physicians to see the level of plaque buildup in a patient’s brain, something that previously could be determined only with an autopsy.
 
The researchers conducted PET scans of 555 participants in theAlzheimer’s Disease Neuroimaging Initiative, a long-term public-private research project that includes people at risk for Alzheimer’s disease and patients who have been diagnosed with the disease as well as participants with no symptoms.
  
The analysis found that a variant in BCHE was significantly associated with the levels of plaque deposits.

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UK’s leading dementia research charity has launched a new research strategy dedicated to driving forward development of treatments.

This strategic approach to research into dementia has four core components:

• Responsive and Targeted Funding
• New Drug Discovery
• Strategic Projects
• Partnerships

More information is available at the link below: