Representatives of NORD and government agencies including the National Institutes of Health and the Food and Drug Administration will speak at the 2nd Annual Rare Disease Leadership Summit of the Center for Business Intelligence in Washington, DC, next week (December 5-6). For details, go to www.cbinet.com.
Do you experience neuropathic pain from Dejerine-Sottas?
November 29, 2007
A new Miami Institute for Human Genomics, which will search for genetic origins of common diseases such as autism and Alzheimer’s, opened its doors Tuesday to great expectations.
Its purpose: changing the way medicine works.
”The future of medicine depends entirely on projects from the field of genomics,” or the study of all the genes in humans, medical school dean Dr. Pascal Goldschmidt said at Tuesday’s opening.
The University of Miami institute is only the second of its kind in the United States. The Broad Institute of Harvard University and Massachusetts Institute of Technology was founded in 2003. Genomic research is part of the focus at Scripps Institute at Florida Atlantic University in Boca Raton, where 230 researchers are looking at discovering new drugs.
The UM genomics institute will focus on the genetic origins of multiple sclerosis, age-related macular degeneration, amyotrophic lateral sclerosis (Lou Gehrig’s disease), tuberculosis and Charcot-Marie-Tooth disease, as well as cardiovascular disease, neurodevelopmental disorders and cancer.
Read the rest of University of Miami center seeks diseases’ origins
November 27, 2007
Newly launched nerve cells in a growing embryo must chart their course to distant destinations, and many of the means they use to navigate have yet to surface. In a study published in the current issue of the journal Neuron, scientists at the Salk Institute for Biological Studies have recovered a key signal that guides motor neurons — the nascent cells that extend from the spinal cord and must find their way down the length of limbs such as arms, wings and legs.
The Salk study, led by Samuel Pfaff, Ph.D, a professor in the Gene Expression Laboratory, identifies a mutation they christened Magellan, after the Portuguese mariner whose ship Victoria was first to circumnavigate the globe. The Magellan mutation occurs in a gene that normally pilots motor neurons on the correct course employing a newly discovered mechanism, their results demonstrate.
Read the rest of Key Nerve Navigation Pathway Identified
November 21, 2007
Mol Pharm. 2007 Nov 14
Anand P, Kunnumakkara AB, Newman RA, Aggarwal BB.
Cytokine Research Laboratory and Pharmaceutical Development Center, Department of Experimental Therapeutics, The University of Texas M. D. Anderson Cancer Center, Houston, Texas 77030
Curcumin, a polyphenolic compound derived from dietary spice turmeric, possesses diverse pharmacologic effects including anti-inflammatory, antioxidant, antiproliferative and antiangiogenic activities. Phase I clinical trials have shown that curcumin is safe even at high doses (12 g/day) in humans but exhibit poor bioavailability. Major reasons contributing to the low plasma and tissue levels of curcumin appear to be due to poor absorption, rapid metabolism, and rapid systemic elimination.
To improve the bioavailability of curcumin, numerous approaches have been undertaken. These approaches involve, first, the use of adjuvant like piperine that interferes with glucuronidation; second, the use of liposomal curcumin; third, curcumin nanoparticles; fourth, the use of curcumin phospholipid complex; and fifth, the use of structural analogues of curcumin (e.g., EF-24). The latter has been reported to have a rapid absorption with a peak plasma half-life.
Despite the lower bioavailability, therapeutic efficacy of curcumin against various human diseases, including cancer, cardiovascular diseases, diabetes, arthritis, neurological diseases and Crohn’s disease, has been documented. Enhanced bioavailability of curcumin in the near future is likely to bring this promising natural product to the forefront of therapeutic agents for treatment of human disease.
November 8, 2007
Uncover the neural communication links involved in myelination, the process of protecting a nerve’s axon, and it may become possible to reverse the breakdown of the nervous system’s electrical transmissions in such disorders as multiple sclerosis, spinal cord injuries, diabetes and cancers of the nervous system.
With $697,065 in grants from the New Jersey Commission on Spinal Cord Injury and the New Jersey Commission on Brain Injury Research, Haesun Kim of Teaneck, NJ, assistant professor of biological sciences at Rutgers University in Newark, is working on gaining a better understanding of those links.
Specifically, her work focuses on Schwann cells within the peripheral nervous system and their communication links with the axons they myelinate by enwrapping them in myelin. Axons are the long fibrous part of neurons that carry the nerve’s electrical signals. A fatty substance, myelin covers those axons both to protect them and to provide a conduit for the fast conduction of electrical signals within the nervous system. Once that myelin is lost,the electrical signal breaks down and eventually the neuron dies — like a cell phone that loses its signal.
November 3, 2007
Curr Gene Ther. 2007 Aug;7(4):239-48.
Federici T, Boulis N.
Cleveland Clinic, Department of Neurosciences and Center for Neurological Restoration, 9500 Euclid Avenue, Cleveland, Ohio 44195, USA.
Peripheral nerve diseases, also known as peripheral neuropathies, affect 15-20 million of Americans and diabetic neuropathy is the most common condition. Currently, the treatment of peripheral neuropathies is more focused on managing pain rather than providing permissive conditions for regeneration. Despite advances in microsurgical techniques, including nerve grafting and reanastomosis, axonal regeneration after peripheral nerve injury remains suboptimal. Also, no satisfactory treatments are available at this time for peripheral neurodegeneration occurring in motor neuron diseases (MND), including amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA).
Peripheral nerves have the inherent capacity of regeneration. Gene therapy strategies focused on neuroprotection may help optimizing axonal regrowth. A better understanding of the cellular and molecular events involved in axonal degeneration and regeneration have helped researchers to identify targets for intervention. This review summarizes the current state on the clinical experience as well as gene therapy strategies for peripheral neuropathies, including MND, peripheral nerve injury, neuropathic pain, and diabetic neuropathy.