Study holds hope of a treatment for deadly genetic disease, MPS IIIB

Researchers find method for replacing missing enzyme in the brain

2014-09-29

(Press-News.org) LOS ANGELES – (Sept. 29, 2014) –MPS IIIB is a devastating and currently untreatable disease that causes progressive damage to the brain, leading to profound intellectual disability, dementia and death -- often before reaching adulthood.

Officially known as mucopolysaccharidosis type IIIB or Sanfilippo Syndrome type B, the disease causes the accumulation of waste products in the cells, leading to progressive damage to the brain. Patients with MPS IIIB lack a vital enzyme that is needed to break down long chains of sugars, known as mucopolysaccharides, leading these to accumulate in the cells.

Researchers reported today in the journal, Proceedings of the National Academy of Sciences of the United States of America, that they have found a way to replace the missing enzyme in the brains of disease models, opening the door to a potential treatment for MPS IIIB.

"Enzyme replacement therapy has been very successful in treating other forms of MPS but not MPS IIIB because the blood-brain barrier blocked delivery of the medication to the brain," said Patricia I. Dickson, MD, a Los Angeles Biomedical Research Institute (LA BioMed) lead researcher and one of the authors of the study. "By injecting a modified enzyme into the brain's left ventricle, we have found a way to bypass that barrier and deliver the needed enzyme to the brain. While more study is needed, this research holds great promise for the treatment of MPS IIIB."

Researchers at LA BioMed, the University of California, Los Angeles and BioMarin Pharmaceutical, Inc. administered a modified form of the missing enzyme, NAGLU, through the left ventricle of the brain. They found the modified enzyme was "taken up avidly" by cells in both the brain and the liver. The researchers reported that the modified enzyme reduced the pathological accumulation of heparan sulfate, a mucopolysaccharide, and other metabolites to normal or near-normal levels.

INFORMATION:

Dr. Dickson directs the MPS Research Laboratory at LA BioMed. Funding for this study was provided by BioMarin Pharmaceutical, Inc. and the National Institutes of Health Grant No. 1R21NS078314 and traineeship 5T32GM8243-28.

About LA BioMed

Founded in 1952, LA BioMed is one of the country's leading nonprofit independent biomedical research institutes. It has approximately 100 principal researchers conducting studies into improved treatments and cures for cancer, inherited diseases, infectious diseases, illnesses caused by environmental factors and more. It also educates young scientists and provides community services, including prenatal counseling and childhood nutrition programs. LA BioMed is academically affiliated with the David Geffen School of Medicine at UCLA and located on the campus of Harbor-UCLA Medical Center. For more information, please visit http://www.LABioMed.org

[Attachments] See images for this press release:

ELSE PRESS RELEASES FROM THIS DATE:

Feeling fatigued while driving? Don't reach for your iPod

2014-09-29

Research has shown that drinking caffeinated beverages and listening to music are two popular fatigue-fighting measures that drivers take, but very few studies have tested the usefulness of those measures. New research to be presented at the HFES 2014 Annual Meeting in Chicago evaluates which method, if either, can successfully combat driver fatigue. In their paper titled "Comparison of Caffeine and Music as Fatigue Countermeasures in Simulated Driving Tasks," human factors/ergonomics researchers ShiXu Liu, Shengji Yao, and Allan Spence designed a simulated driving study ...

New way to detox? 'Gold of Pleasure' oilseed boosts liver detoxification enzymes

2014-09-29

URBANA, Ill. – University of Illinois scientists have found compounds that boost liver detoxification enzymes nearly fivefold, and they've found them in a pretty unlikely place—the crushed seeds left after oil extraction from an oilseed crop used in jet fuel. "The bioactive compounds in Camelina sativa seed, also known as Gold of Pleasure, are a mixture of phytochemicals that work together synergistically far better than they do alone. The seed meal is a promising nutritional supplement because its bioactive ingredients increase the liver's ability to clear foreign chemicals ...

University of Alberta researchers explain 38-year-old mystery of the heart

2014-09-29

In a new study published in Proceedings of the National Academy of Sciences USA, researchers at the University of Alberta's Faculty of Medicine & Dentistry have explained how the function of a key protein in the heart changes in heart failure. Heart disease is the number-one killer in the developed world. The end stage of heart disease is heart failure, in which the heart cannot pump enough blood to satisfy the body's needs. Patients become progressively short of breath as the condition worsens, and they also begin to accumulate fluid in the legs and lungs, making it ...

Good working relationships between clients, bankers can reduce defaults

2014-09-29

You've probably seen advertising campaigns in which banks describe how much their customer relationships matter to them. While such messaging might have been cooked up at an ad agency, it turns out there is some truth underlying these slogans. As a newly published study co-authored by an MIT professor shows, strong working relationships between bankers and clients reduce the likelihood of loan delinquencies and defaults, at least in the context of an emerging economy. Using propriety data from a large bank in Chile, the study finds that when loan officers go on leave, ...

Causes of California drought linked to climate change

2014-09-29

The atmospheric conditions associated with the unprecedented drought currently afflicting California are "very likely" linked to human-caused climate change, Stanford scientists say. In a new study, a team led by Stanford climate scientist Noah Diffenbaugh used a novel combination of computer simulations and statistical techniques to show that a persistent region of high atmospheric pressure hovering over the Pacific Ocean that diverted storms away from California was much more likely to form in the presence of modern greenhouse gas concentrations. The research, published ...

Decision to reintroduce aprotinin in cardiac surgery may put patients at risk

2014-09-29

Cardiac surgery patients may be at risk because of the decision by Health Canada and the European Medicines Agency to reintroduce the use of aprotinin after its withdrawal from the worldwide market in 2007, assert the authors of a previous major trial that found a substantially increased risk of death associated with the drug. In an analysis in CMAJ (Canadian Medical Association Journal), the authors refute three major criticisms of the trial made by the regulatory bodies. Aprotinin, used to control bleeding in cardiac surgery, was withdrawn worldwide in 2007 after the ...

Revolutionary hamstring tester will keep more players on the field

2014-09-29

Elite sporting stars can assess and reduce their risk of a hamstring injury thanks to a breakthrough made by QUT researchers. The discovery could be worth a fortune to football codes, with hamstring strain injuries accounting for most non-contact injuries in Australian rules football, football and rugby union, as well as track events like sprinting. Using an innovative field device, a research team led by Dr Anthony Shield, from QUT's School - Exercise and Nutrition Sciences, and former QUT PhD student, Dr David Opar, now at the Australian Catholic University, measured ...

Drug for kidney injury after cardiac surgery does not reduce need for dialysis

2014-09-29

Among patients with acute kidney injury after cardiac surgery, infusion with the antihypertensive agent fenoldopam, compared with placebo, did not reduce the need for renal replacement therapy (dialysis) or risk of death at 30 days, but was associated with an increased rate of abnormally low blood pressure, according to a study published in JAMA. The study is being posted early online to coincide with its presentation at the European Society of Intensive Care Medicine annual congress. More than 1 million patients undergo cardiac surgery every year in the United States ...

Climate detectives reveal handprint of human caused climate change in Australia

2014-09-29

Australia's hottest year on record in 2013 along with the accompanying droughts, heat waves and record-breaking seasons of that year was virtually impossible without the influence of human-caused global warming. New research from ARC Centre of Excellence for Climate System Science (ARCCSS) researchers and colleagues, over five different Australian papers in a special edition of the Bulletin of the American Meteorological Society (BAMS), has highlighted the powerful influence of global warming on Australia's climate. "We often talk about the fingerprint of human-caused ...

Rising prevalence of sleep apnea in US threatens public health

2014-09-29

DARIEN, IL – Public health and safety are threatened by the increasing prevalence of obstructive sleep apnea, which now afflicts at least 25 million adults in the U.S., according to the National Healthy Sleep Awareness Project. Several new studies highlight the destructive nature of obstructive sleep apnea, a chronic disease that increases the risk of high blood pressure, heart disease, Type 2 diabetes, stroke and depression. "Obstructive sleep apnea is destroying the health of millions of Americans, and the problem has only gotten worse over the last two decades," said ...

The Lancet: Parent-focused programs insufficient to prevent obesity in toddlers, finds meta-analysis; authors call for a re-think of childhood obesity prevention approaches

Tiny metal figurines from Sardinia's Nuragic civilization in around 1,000 BC reveal extensive ancient Mediterranean metal trading networks

Natural microfibers may degrade differently to synthetic materials under simulated sunlight exposure in freshwater and seawater conditions, with implications for how such pollutants affect aquatic lif

Indian new mums report better postpartum wellbeing when their own mum acts as their primary support - while women whose mother-in-law is the primary caregiver instead report significantly lower overal

Young adult intelligence and education are correlated with socioeconomic status in midlife

[Press-News.org] Study holds hope of a treatment for deadly genetic disease, MPS IIIB
Researchers find method for replacing missing enzyme in the brain

Study holds hope of a treatment for deadly genetic disease, MPS IIIB

ELSE PRESS RELEASES FROM THIS DATE:

LAST 30 PRESS RELEASES: