Michael Pacanowski, PharmD, MPH; Shashi Amur, PhD; Issam Zineh, PharmD, MPH Author Affiliations: Genomics Group, Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland. Treatment of chronic hepatitis C (CHC) is a prototype for personalized medicine. Combination therapy with peginterferon alfa plus ribavirin was the standard of care for more than a decade. Greater understanding of the disease and determinants of treatment response have improved sustained virologic response (SVR) rates from less than 10% with interferon alfa in the 1990s to more than 80% with contemporary triple therapy regimens that include direct acting antivirals (DAAs) (Figure). Patient-specific factors such as viral genotype and early on-treatment responses are considered in therapeutic individualization. New approaches to search the human genome for predictors of drug response led to the discovery that single-nucleotide polymorphisms (SNPs) near the host IL28B gene are among the strongest predictors of response to peginterferon alfa and ribavirin. This Viewpoint discusses the evolution of CHC pharmacogenetics, its real-time incorporation into recent regulatory science evaluations, and its application in future drug development. cDNA indicates complementary Read the original here: New Genetic Discoveries and Treatment for Hepatitis C [Viewpoint] … Continue reading →

Public release date: 9-May-2012 [ | E-mail | Share ] Contact: Sandy Van sandy@prpacific.com 808-526-1708 Cedars-Sinai Medical Center LOS ANGELES (May 9, 2012) Scientists have confirmed that mutations of a gene are responsible for some cases of a rare, inherited disease that causes progressive muscle degeneration and weakness: spinal muscular atrophy with lower extremity predominance, also known as SMA-LED. "Typical spinal muscular atrophies begin in infancy or early childhood and are fatal, involving all motor neurons, but SMA-LED predominantly affects nerve cells controlling muscles of the legs. It is not fatal and the prognosis is good, although patients usually are moderately disabled and require assistive devices such as bracing and wheelchairs throughout their lives," said Robert H. Baloh, MD, PhD, director of Cedars-Sinai Medical Center's Neuromuscular Division and senior author of a Neurology article describing the new findings on DYNC1H1. It is a molecule inside cells that acts as a motor to transport cellular components. Using cells cultured from patients, Baloh's group showed that the mutation disrupts this motor's function. The researchers found that some subjects with mutations had global developmental delay in addition to weakness, indicating the brain also is involved. "Our observations suggest that a range of DYNC1H1-related … Continue reading →

SAN DIEGO, May 9, 2012 /PRNewswire/ -- Sequenom, Inc. (SQNM), a life sciences company providing innovative genetic analysis solutions, today announced that it has signed an agreement with U.S. health insurance provider Coventry Health Care National Network to provide coverage for the Sequenom Center for Molecular Medicine's (Sequenom CMM) MaterniT21 PLUS laboratory-developed test (LDT). The test detects certain fetal trisomies in women at increased risk of carrying a child with one of these chromosomal anomalies. The agreement ensures that the 2.2 million members of the Coventry First Health PPO network will have coverage for Sequenom CMM's MaterniT21 PLUS testing service, with access to more than 5,000 hospitals and 500,000 health professionals in all 50 states, including the District of Columbia and Puerto Rico. "The coverage of the Sequenom CMM MaterniT21 PLUS LDT by the Coventry network is an important milestone, as it will ensure that doctors who are Coventry members will have access to this important testing service to help provide expectant families with important information about their pregnancy through a noninvasive, highly accurate technology," said Harry F. Hixson, Jr., Ph.D., Chairman and CEO, Sequenom, Inc. The MaterniT21 PLUS LDT is intended for use in pregnant women at increased risk … Continue reading →

Public release date: 9-May-2012 [ | E-mail | Share ] Contact: Nicole Davis ndavis@broadinstitute.org 617-714-7152 Broad Institute of MIT and Harvard Melanoma the deadliest and most aggressive form of skin cancer has long been linked to time spent in the sun. Now a team led by scientists from the Broad Institute and Dana-Farber Cancer Institute has sequenced the whole genomes of 25 metastatic melanoma tumors, confirming the role of chronic sun exposure and revealing new genetic changes important in tumor formation. In an article published online May 9 in Nature, the authors provide the first high-resolution view of the genomic landscape of human melanoma tumors. Previous genetic analyses have focused on the exomes of many types of cancer tumors, concentrating on the tiny fraction of the genome that provides the genetic code for producing proteins. Whole genomes contain a wealth of genetic information, and by sequencing and analyzing 25 metastatic melanoma tumors a significant technical and computational feat scientists can learn vastly more about the variety of genetic alterations that matter in melanoma. "Sequencing the whole genome certainly adds a richness of discovery that can't be fully captured with a whole exome," said Levi A. Garraway, a senior associate member … Continue reading →

A stem cell breakthrough at UCLA could mark a big step for a biopharmaceutical company to use its proprietary technology to forge partnerships with pharmaceutical companies and other research institutions. Fibrocell Sciences technology isolates, purifies and multiplies a patients fibroblast cells, connective skin cells that make collagen. In a research collaboration with the company, UCLA used the technology to isolate, identify and increase the number of different skin cell types, which lead to two rare adult stem cell-like subpopulations being identified in adult human skin SSEA3-expressing regeneration-associated cells associated with skin regeneration after injuries and mesenchymal adult stem cells. The findings could have broad applications for personalized medicine. Currently, adult stem cells are derived from adipose tissue and bone marrow. Using mesenchymal stem cells would be less invasive and could be more efficient. Mesenchymal stem cells are being used in research to develop osteoblasts, or bone cells; chondrocytes, or cartilage cells; and adipocytes, or fat cells. David Pernock, the chairman and CEO of Fibrocell, said the move could mark a significant step in the companys growth. Continue reading here: Stem cell collaboration could set stage for company’s growth … Continue reading →

Public release date: 8-May-2012 [ | E-mail | Share ] Contact: David Eve celltransplantation@gmail.com Cell Transplantation Center of Excellence for Aging and Brain Repair Tampa, Fla. (May. 8, 2012) Two studies appearing in a recent issue of Cell Transplantation (20:11-12), now freely available on-line at http://www.ingentaconnect.com/content/cog/ct/, evaluate stem cells derived from dental tissues for characteristics that may make them therapeutically useful and appropriate for transplantation purposes. Induced pluripotent stem cells from immature dental pulp stem cells A Brazilian and American team of researchers used human immature dental pulp stem cells (IDPSCs) as an alternative source for creating induced pluripotent stem cells (iPSCs), stem cells that can be derived from several kinds of adult tissues. According to the study authors, production of iPSCs "opens new opportunities for increased understanding of human genetic diseases and embryogenesis" and will likely have a "great impact on future drug screening and toxicology tests." The authors note, however, that the reprogramming methodology for making iPSCs is relatively new and "needs refining" in terms of technique, efficiency and cell type choice. The researchers report that they easily, and in a short time frame, programmed human immature dental pulp stem cells into iPSCs with the hallmarks of pluripotent … Continue reading →

Wed May 9, 2012 3:35pm BST (Reuters) - Pluristem Therapeutics Inc said a 7-year old girl suffering from a bone marrow disease experienced a reversal of her condition after receiving its experimental stem cell therapy, sending the Israeli company's shares up 32 percent. The girl, suffering from aplastic bone marrow in which the patient has no blood-forming stem cells, had a significant rise in her red cells, white cells and platelets following an injection of Pluristem's therapy -- PLacental eXpanded cells. "The results of this unique case indicate that PLX cells may be effective in treating other diseases that affect the bone marrow," Reuven Or, the child's physician at Hadassah Medical Center, was quoted in a statement by Pluristem. Last September, the company said animal studies showed that the therapy had the potential to treat blood tissue complications related with acute radiation syndrome, commonly called radiation sickness. Last month, the U.S. health regulators gave a go ahead to the company to start a mid-stage trial of the therapy for treating Intermittent Claudication -- a subset of peripheral artery disease. Pluristem shares, which have gained 5 percent since receiving the FDA nod for the mid-stage trial, were up 15 percent at … Continue reading →

PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Research Corporation (NASDAQ: ARWR - News), a nanomedicine company with development programs in RNA therapeutics and obesity, today announced financial results for its fiscal 2012 second quarter ended March 31, 2012. Fiscal 2012 Second Quarter and Recent Company Highlights Recent acquisitions of Roches siRNA business and Alvos Therapeutics, Inc., coupled with our own assets, have given Arrowhead a platform from which we can rapidly generate and optimize therapeutic candidates, said Dr. Christopher Anzalone, President and Chief Executive Officer. Our ability and experience in combining the right targeting agent with the right delivery vehicle and the right payload is unique in the industry and is generating increasing collaboration interest from potential partners. During the second quarter, we continued to enroll patients in our Phase 1b trial for CALAA-01 and we expect to wrap up this trial during the summer. Additionally, we received clearance to commence a Phase I trial for Adipotide, our anti-obesity candidate, and anticipate dosing the first patient shortly. Selected Financial Results For the quarter ended March 31, 2012, Arrowhead reported revenues of $31,250. There was no revenue in the comparable prior period. Total operating expenses for the quarter ended March 31, 2012 were $4.9 … Continue reading →