LA BioMed researchers report on promising new therapy for devastating genetic disorder

Investigational drug tested for treatment of Sanfilippo B syndrome

2014-02-12

(Press-News.org) LOS ANGELES – (Feb. 12, 2014) – A promising new therapy has – for the first time – reduced damage to the brain that can be caused by Sanfilippo B (MPS IIIB), a rare and devastating genetic disease, Los Angeles Biomedical Research Institute (LA BioMed) researchers will report today in a presentation at the Lysosomal Disease Network's 10th Annual WORLD Symposium™.

Sanfilippo B syndrome is a lysosomal storage disease belonging to the group of mucopolysaccharidoses (MPS) that is characterized by severe and rapid intellectual deterioration. LA BioMed pioneered the research that led to the first enzyme replacement therapy for MPS I, Aldurazyme©, which has saved hundreds of lives in the nearly 11 years since it was approved by the Food and Drug Administration.

LA BioMed's MPS Research Laboratory reported the results of its latest study for treating Sanfilippo B syndrome in a disease model that employed recombinant human "NAGLU-IGF2" (a novel fusion protein of alpha-N-acetyglucosaminidase, or NAGLU, coupled to a peptide derived from the insulin-like growth factor, IGF2). The fusion protein was produced by BioMarin Pharmaceutical Inc.

LA BioMed researcher Shih-hsin Kan reported that the laboratory's tests found NAGLU-IGF2 is taken up by neurons in the disease model, which then reduces brain damage by reversing heparan sulfate storage – one of the causes of damage to the brain in patients with Sanfilippo B syndrome. The researchers concluded that NAGLU-IGF2 can be the basis for enzyme replacement therapy for Sanfilippo B syndrome.

"This is an exciting new development that needs to be examined further to determine its effectiveness in patients with Sanfilippo B syndrome who currently have no therapies available to reduce the damage to their brains," said Patricia Dickson, MD, director of the MPS Research Laboratory at LA BioMed. "We look forward to continuing our partnership with BioMarin in developing therapies for MPS."

BioMarin announced Tuesday that it has developed BMN 250, a manufactured form of NAGLU-IGF2, for the treatment of Sanfilippo B syndrome and expects to initiate clinical studies with BMN 250 in mid-2015. INFORMATION: 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 diagnostics and treatments 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 END

ELSE PRESS RELEASES FROM THIS DATE:

From artificial to natural, the food industry makes a major shift

2014-02-12

Extracts from algae, rosemary and monk fruit could soon replace synthetic ingredients and food additives such as Blue No. 1, BHT and aspartame that label-conscious grocery shoppers are increasingly shunning. Research is enabling this shift from artificial colors, sweeteners and preservatives to naturally derived ones, and could soon yield many more natural options, reports Chemical & Engineering News, the weekly newsmagazine of the American Chemical Society. Melody M. Bomgardner, senior editor at C&EN, notes that the trend has built momentum as concern over negative ...

Help for a scarred heart: Scarring cells turned to beating muscle

2014-02-12

ANN ARBOR – Poets and physicians know that a scarred heart cannot beat the way it used to, but the science of reprogramming cells offers hope--for the physical heart, at least. A team of University of Michigan biomedical engineers has turned cells common in scar tissue into colonies of beating heart cells. Their findings could advance the path toward regenerating tissue that's been damaged in a heart attack. Previous work in direct reprogramming, jumping straight from a cell type involved in scarring to heart muscle cells, has a low success rate. But Andrew Putnam, ...

San Francisco's big 1906 quake was third of a series on San Andreas Fault

2014-02-12

EUGENE, Ore. -- (Feb. 12, 2014) -- Research led by a University of Oregon doctoral student in California's Santa Cruz Mountains has uncovered geologic evidence that supports historical narratives for two earthquakes in the 68 years prior to San Francisco's devastating 1906 disaster. The evidence places the two earthquakes, in 1838 and 1890, on the San Andreas Fault, as theorized by many researchers based on written accounts about damage to Spanish-built missions in the Monterey and San Francisco bay areas. These two quakes, as in 1906, were surface-rupturing events, the ...

Clever NIST/JPL technology decodes more information from single photons

2014-02-12

It's not quite Star Trek communications—yet. But long-distance communications in space may be easier now that researchers at the National Institute of Standards and Technology (NIST) and Jet Propulsion Laboratory (JPL) have designed a clever detector array that can extract more information than usual from single particles of light. Described in a new paper,* the NIST/JPL array-on-a-chip easily identifies the position of the exact detector in a multi-detector system that absorbs an incoming infrared light particle, or photon. That's the norm for digital photography cameras, ...

New NIST method evaluates response to oxidation in live cells

2014-02-12

Researchers at the National Institute of Standards and Technology (NIST) have developed a new method for accurately measuring a key process governing a wide variety of cellular functions that may become the basis for a "health checkup" for living cells. The NIST technique measures changes in a living cell's internal redox (reduction-oxidation) potential, a chemistry concept that expresses the favorability of reactions in which molecules or atoms either gain or lose electrons. Redox reactions are important to cell chemistry because they regulate many genes and the proteins ...

Popular disinfectants do not kill HPV

2014-02-12

Commonly used disinfectants do not kill human papillomavirus (HPV) that makes possible non-sexual transmission of the virus, thus creating a need for hospital policy changes, according to researchers from Penn State College of Medicine and Brigham Young University. "Because it is difficult to produce infectious HPV particles for research, little has been known about HPV susceptibility to disinfection," said Craig Meyers, Distinguished Professor of Microbiology and Immunology, Penn State College of Medicine. Use of disinfectants on HPV in health care settings has been ...

Hollywood failing to keep up with rapidly increasing diversity, UCLA study warns

2014-02-12

When it comes to influential positions in the entertainment industry, minorities and women are represented at rates far below what would be expected given their percentage of the general population, according to a new study done at UCLA's Ralph J. Bunche Center for African American Studies. In fact, the report shows, the proportion of female and minority actors, writers, directors and producers in films and TV ranges from just one-twelfth to one-half of their actual population percentage. "The report paints a picture of an industry that is woefully out of ...

Meeting the eye-witnesses of ocean change

2014-02-12

Members of the German research network BIOACID (Biological Impacts of Ocean Acidification) are developing a model that links ecosystem changes triggered by ocean acidification and climate change with their economic and societal consequences. Workshops and interviews with stakeholders from the Norwegian fishing industry and tourism sector, the government and environmental organisations help them to identify key aspects for their assessment. During the past ten years, scientists have learned a lot about the effects of ocean acidification on marine ecosystems. It has become ...

Cities support more native biodiversity than previously thought

2014-02-12

(Santa Barbara, Calif.) — The rapid conversion of natural lands to cement-dominated urban centers is causing great losses in biodiversity. Yet, according to a new study involving 147 cities worldwide, surprisingly high numbers of plant and animal species persist and even flourish in urban environments — to the tune of hundreds of bird species and thousands of plant species in a single city. Contrary to conventional wisdom that cities are a wasteland for biodiversity, the study found that while a few species — such as pigeons and annual meadow grass — are shared across ...

4 years on, ICU Patients with kidney injury show high mortality & elevated urinary protein

2014-02-12

In 4 years of follow up of 1464 participants in the randomized controlled trial Randomised Evaluation of Normal vs. Augmented Levels of RRT (RENAL) study, Martin Gallagher (The George Institute for Global Health, Sydney, Australia) and colleagues found that patients with acute kidney injury (AKI) in an intensive care unit (ICU) who require renal replacement therapy (RRT; hemodialysis combined with hemofiltration) do not benefit from higher intensity RRT. At a median of 43.9 months follow up, mortality (63% in the low intensity and 63% in the high intensity group), as well ...

Walking further and faster is linked to a reduced risk of heart attacks, heart failure and stroke in people with high blood pressure

Adolescent girls who have weight concerns despite not being obese are more likely to also experience depression and suicidality, per Korean survey of more than 50,000 middle and high school students

LA BioMed researchers report on promising new therapy for devastating genetic disorder

ELSE PRESS RELEASES FROM THIS DATE:

LAST 30 PRESS RELEASES: