Lysosomes News and Research

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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

Research offers new hope for rare metabolic disorder

A research team led by biochemist Scott Garman at the University of Massachusetts Amherst has for the first time determined the mechanism of one of the cell's "recycling" enzymes, human alpha-galactosidase or alpha-GAL, as it breaks down substances in the lysosome, the cell's recycling center.

2 Feb 2010

Results from Phase 2a clinical trial of Raptor Pharmaceutical's cysteamine bitartrate published

Raptor Pharmaceutical Corp. ("Raptor" or the "Company"), today announced the publication of results from a Phase 2a clinical trial of a prototype formulation of its proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis").

11 Jan 2010

IKK phosphorylate: A double-edged sword in Huntington's disease

The kinase IKK phosphorylates the protein mutated in Huntington's disease to promote its removal and neuron survival, but IKK may be a double-edged sword that increases neurotoxicity in later stages of the disease. The study, led by researchers from the University of California, Irvine, will be published online December 21 in the Journal of Cell Biology (www.jcb.org).

22 Dec 2009

Nanoprobes deliver drugs directly to the right tumor cellular compartments

Tiny nanoprobes have shown to be effective in delivering cancer drugs more directly to tumor cells - mitigating the damage to nearby healthy cells - and Purdue University research has shown that the nanoprobes are getting the drugs to right cellular compartments.

15 Dec 2009

Albert Einstein College of Medicine receives SU2C grant for cancer research

Matthew Levy, Ph.D., assistant professor of biochemistry at Albert Einstein College of Medicine of Yeshiva University, has been awarded more than $700,000 by Stand Up To Cancer (SU2C) for his high-risk/high-reward cancer research.

7 Dec 2009

Lipid that accumulates in brain cells drives cell death, says research

The lipid that accumulates in brain cells of individuals with an inherited enzyme disorder also drives the cell death that is a hallmark of the disease, according to new research led by St. Jude Children's Research Hospital investigators.

13 Nov 2009

Study suggests a new improved treatment option for children with Hurler's syndrome

Researchers have transplanted genetically modified hematopoietic stem cells into mice so that their developing red blood cells produce a critical lysosomal enzyme - preventing or reducing organ and central nervous system damage from the often-fatal genetic disorder Hurler's syndrome.

10 Nov 2009

Modified NAGA: New enzyme for enzyme replacement therapy

A new study uses a creative structure-based remodeling strategy to design a therapeutic protein that exhibits significant advantages over currently available treatments for a rare disease that often leads to cardiac and renal failure. The research, published by Cell Press on October 22nd in the American Journal of Human Genetics, describes a new and highly promising candidate for enzyme replacement therapy (ERT) for Fabry disease.

23 Oct 2009

Scientists find mechanism that protects neurons from calcium overload

Tsuruta et al. find that a lipid kinase directs a voltage-gated calcium channel's degradation to save neurons from a lethal dose of overexcitement. The study appears in the October 19, 2009 issue of the Journal of Cell Biology (www.jcb.org).

20 Oct 2009

FDA approves ZyStor Therapeutics' ZC-701 for Phase I human safety trial

ZyStor Therapeutics, Inc., a biotechnology company developing a new class of targeted protein therapeutics for the treatment of Lysosomal Storage Diseases using the Company's proprietary Glycosylation Independent Lysosomal Targeting technology, today announced that it has received clearance from the U. S. Food and Drug Administration to proceed into a clinical trial for its first drug candidate, ZC-701, an enzyme replacement therapy for the treatment of Pompe disease.

14 Oct 2009

Researchers find SNARE-like protein system to help bacteria avoid cell destruction

Infectious diseases currently cause about one-third of all human deaths worldwide, more than all forms of cancer combined. Advances in cell biology and microbial genetics have greatly enhanced understanding of the cause and mechanisms of infectious diseases.

12 Oct 2009

Mid-career scientists receive the 2009 Julie Martin Mid-Career Awards in Aging Research

Charleen T. Chu, MD, PhD, associate professor at the University of Pittsburgh and David M. Sabatini, MD, PhD, associate professor, Massachusetts Institute of Technology, received the 2009 Julie Martin Mid-Career Awards in Aging Research. Sponsored by The Ellison Medical Foundation, the grants provide funding of $550,000 to mid-career scientists whose research has great potential in advancing understanding of basic aging and its impact on age-related diseases.

26 Aug 2009

Estrogen-dependent switch tempers killing activity of immune cells

The sex hormone estrogen tempers the killing activity of a specific group of immune cells, the cytotoxic T cells (CTLs), which are known to attack tumor cells and cells infected by viruses. The key player in this process is a cytotoxic T cell molecule which has been known for a long time and which scientists have named EBAG9.

10 Aug 2009

Newly discovered gene regulates balance of 'bad' cholesterol

In an article in Science, Noam Zelcer from the LACDR describes a previously unknown mechanism for regulating the amount of LDL cholesterol. This offers opportunities for supplementing and improving the effect of so-called statins: medicines that remove 'bad' cholesterol from the bloodstream.

30 Jun 2009