Lysosomes News and Research

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CytRx sells global rights of molecular chaperone assets to Orphazyme

CytRx Corporation, a biopharmaceutical company specializing in oncology, today announced that it has sold the worldwide rights to its molecular chaperone assets to privately-held, Copenhagen, Denmark-based Orphazyme ApS.

17 May 2011

Genetic mutation linked to Kufs disease

According to a new study, a genetic mutation thought to be responsible for the rare hereditary brain disorder Kufs disease is finally identified. The research was carried out by Dr Melanie Bahlo and her team at the Walter and Eliza Hall Institute's bioinformatics department in Parkville, Australia, in partnership with neurologist and epilepsy specialists Professor Sam Berkovic and Dr Todor Arsov from the University of Melbourne, Australia.

10 May 2011

Scientists identify biochemical abnormality behind Machado-Joseph disease

Scientists in Portugal have identified a biochemical abnormality behind the potentially fatal neurodegenerative Machado-Joseph disease and using several models of the disease were able to reverse the problem in what can be a crucial step towards a cure for humans .

2 May 2011

New insight into multiple sclerosis

Scientists have blocked harmful immune cells from entering the brain in mice with a condition similar to multiple sclerosis.

8 Mar 2011

Novel gene therapy can promote removal of amyloid plaques and prevent Alzheimer's disease

Gene therapy that boosts the ability of brain cells to gobble up toxic proteins prevents development of Alzheimer's disease in mice that are predestined to develop it, report researchers at Georgetown University Medical Center. They say the treatment - which is given just once - could potentially do the same in people at the beginning stages of the disease.

5 Mar 2011

Biomarin commences BMN 701 Phase 1/2 trial for treatment of Pompe disease

BioMarin Pharmaceutical Inc. announced today that it has initiated a Phase 1/2 trial for BMN 701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase in development for the treatment of Pompe disease.

20 Jan 2011

Researchers discover recycling mechanism of signaling receptors

An international team of researchers led by Carnegie Mellon University's Manojkumar Puthenveedu has discovered the mechanism by which signaling receptors recycle, a critical piece in understanding signaling receptor function. Writing in the journal Cell, the team for the first time describes how a signaling receptor travels back to the cell membrane after it has been activated and internalized.

23 Dec 2010

New findings identify Sortilin as a potential therapeutic site to alter PGRN pathology in dementia

Frontotemporal lobar degeneration (FTLD) refers to a group of disorders associated with degeneration of the frontal and temporal lobes of the brain. Symptoms include dementia, aphasia, and semantic disorders.

18 Nov 2010

New simple blood test may help diagnose Niemann-Pick type C disease

A fatal genetic disorder that frequently takes years to diagnose may soon be detectable with a simple blood test, researchers at Washington University School of Medicine in St. Louis and the National Institutes of Health (NIH) report this week in Simple blood test may diagnose deadly Niemann-Pick type C disease.

4 Nov 2010

Alpha-synuclein protein in Parkinson's disease inhibits autophagy: Research

A protein linked to Parkinson's disease may cause neurodegeneration by inhibiting autophagy-the process in which cells digest some of their contents-according to a study in the September 20 issue of the Journal of Cell Biology

21 Sep 2010

BioMarin's BMN-701 receives FDA orphan drug designation for treatment of Pompe disease

BioMarin Pharmaceutical Inc. announced today that it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for BMN-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for the treatment of Pompe disease. An investigational new drug application (IND) for BMN-701 has been submitted, investigational material has been manufactured and a Phase I/II study is expected to start in the first quarter of 2011.

30 Aug 2010

BioMarin Pharmaceutical acquires ZyStor Therapeutics, furthers development of ZC-701 for Pompe disease

BioMarin Pharmaceutical Inc. announced today that it has acquired ZyStor Therapeutics, Inc. (ZyStor), a privately-held biotechnology company developing enzyme replacement therapies (ERT) for the treatment of lysosomal storage disorders. ZyStor's lead product candidate is ZC-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for Pompe disease.

18 Aug 2010

Scientists develop novel chemotherapeutic method for anticancer drug delivery

Combining nature and Greek mythology, researchers at the Technion-Israel Institute of Technology have developed a tiny "Trojan Horse" system for delivering cancer-fighting drugs. With this system, the drugs are delivered only once they are inside the cancerous cells, so there is no damage to the healthy cells in the surrounding area.

28 Jul 2010

Researchers discover potential method for neurodegenerative disease detection

New investigations, initiated by research workers at CIC bioGUNE and led by Dr. Aitor Hierro, have opened possibilities for making progress in the knowledge and prediction of neurodegenerative diseases such as Amyotrophic Lateral Sclerosis (ALS), according to the journal of The Proceedings of the National Academy of Sciences of the United States of America.This study opens the possibility of research in humans to detect this pathology before the symptoms arise.

26 Jul 2010

Enzyme mTOR regulates autophagy: Study

Cells that consume parts of themselves can stop this process autonomously as well. This was discovered by NWO researcher Judith Klumperman's group, in collaboration with colleagues from the National Institute of Health in Washington. The self-cannibalism is part of the normal digestive process of the cell, but also a survival mechanism in times of famine. This is what makes it difficult for doctors to 'starve out' cancer cells, for instance. The research results were published on 6 June in Nature.

15 Jul 2010

New research focuses on calcium channels in lysosome membrane

In cells, as in cities, disposing of garbage and recycling anything that can be reused is an essential service. In both city and cell, health problems can arise when the process breaks down.

15 Jul 2010

Elevation of Smurf1 may play a role in molecular basis of PAH: Study

Pulmonary arterial hypertension (PAH) is a progressive disease, marked by shortness of breath and fatigue which can be fatal if untreated. Increased pressure in the pulmonary artery and its branches is associated with dysfunctional growth control of endothelial and smooth muscle cells leading to excessive thickening of the blood vessel wall, obliteration of the lumen and right heart failure.

22 Jun 2010

Disorder in degradation of proteins and other macromolecules in cells leads to serious diseases

The degradation of proteins and other macromolecules in cells is vital to survival. Disruption of this process can result in serious disease. The research group of Professor Thomas Jentsch (Leibniz Institute for Molecular Pharmacology, FMP/ Max Delbr-ck Center for Molecular Medicine, MDC, Berlin-Buch) has now succeeded in identifying an essential cellular process necessary for the transport and degradation of macromolecules in endosomes and lysosomes, respectively.

16 Jun 2010

Accumulation of mutated protein may explain damaging cellular behavior in Huntington's disease

In a step towards a possible treatment for Huntington's disease, scientists at Albert Einstein College of Medicine of Yeshiva University have shown for the first time that the accumulation of a mutated protein may explain damaging cellular behavior in Huntington's disease. Their research is described in the April 11 online edition of Nature Neuroscience.

12 Apr 2010

Raptor Pharmaceutical to present DR Cysteamine trial data at Lysosomal Disease Network Symposium

Raptor Pharmaceutical Corp., today announced the presentation of cystinosis data at the Annual Lysosomal Disease Network WORLD Symposium 2010, being held February 10-12 in Miami, Florida. Biomarker data from a Phase IIb pilot study of Raptor's proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") will be the subject of a poster titled, "Correlation of Plasma Cysteamine and WBC Cystine Levels at Steady State in Patients Treated with Cysteamine Bitartrate." The results will also be published in an upcoming issue of the journal Molecular Genetics and Metabolism.

10 Feb 2010