BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced the initiation of a Phase 1/2 clinical trial for BMN-110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. The company expects to report initial results in the first half of 2010. "We plan to leverage our clinical, manufacturing and regulatory expertise to bring a new therapeutic option to the significant number of untreated Morquio patients around the world, " said Henry Fuchs, M.D., Chief Medical Officer of BioMarin. "GALNS has been shown in mice to reach important tissues including cartilage and different zones of the bone such as bone marrow, calcified bone and importantly, the growth plate.
Many studies determine hormone levels in the blood as a marker of breast cancer risk. But it hasn't been known whether these blood tests reflect what is happening in the breast tissue, where certain hormones fuel cancer. Researchers at Georgetown University Medical Center's (GUMC) Lombardi Comprehensive Cancer Center found that measuring the levels of four hormones in blood known to be linked to breast cancer doesn't necessarily reflect the levels of these hormones in the breast tissue itself. In fact, the scientists say that blood tests used in research studies that measure these hormones could give a false impression of both the real breast cancer risk women face, and an imprecise picture of how these hormones affect breast cancer development.
In a large study, a national team of researchers led by Mayo Clinic scientists observed that self-reported use of hormone therapy was associated with a significantly lower colorectal cancer risk. However, the mechanisms for the apparent protective association are still unclear. The study, being presented at the AACR 100th Annual Meeting 2009, was designed to look at possible links between estrogen exposure and colon cancer molecular subtypes, to determine how these hormones might function as anti-cancer agents. "In our large, prospective study, use of hormone therapy seemed to be beneficial with respect to reducing colorectal cancer risk women who did use these drugs had a 28 percent lower incidence rate than women who did not use these drugs, " says the study's lead author, David Limsui, M.
In recent years, a series of highly publicized reports have warned of increases in breast cancer and other health problems in postmenopausal women who take hormone replacement therapy (HRT). But a closer look at the data suggests that those reports were often "distorted, oversimplified, or wrong, " according to a paper in the March/April issue of The Cancer Journal: The Journal of Principles & Practice of Oncology. The journal is published by Lippincott Williams & Wilkins, a part of Wolters Kluwer Health, a leading provider of information and business intelligence for students, professionals, and institutions in medicine, nursing, allied health, pharmacy and the pharmaceutical industry.
New guidelines designed to standardize and optimize the diagnosis, treatment, and monitoring of patients with Medullary Thyroid Carcinoma (MTC), an uncommon and challenging form of thyroid cancer, have been developed by the American Thyroid Association and published online ahead of print in Thyroid, a peer-reviewed journal published by Mary Ann Liebert, Inc. The guidelines are available free online at http://www.liebertpub.com/thy. "Following careful peer review, Thyroid has published the first comprehensive set of clinical guidelines for the treatment of all medullary thyroid carcinoma (MTC) disorders, " says Charles H. Emerson, MD, Editor-in-Chief of the Journal and Professor Emeritus at University of Massachusetts Medical School, in Worcester.
FDA Approves SAMSCA TM tolvaptan , The First And Only Oral Vasopressin Antagonist To Treat Patients With Clinically Significant Hypervolemic
Otsuka Pharmaceutical Co., Ltd. (OPC) and Otsuka Pharmaceutical Development and Commercialization, Inc. (OPDC) announced that the U.S. Food and Drug Administration (FDA) has approved SAMSCA(TM) (tolvaptan) as the only oral selective vasopressin antagonist for the treatment of patients with clinically significant hypervolemic and euvolemic hyponatremia (serum sodium less than 125 mEq/L, or less marked hyponatremia that is symptomatic and has resisted correction with fluid restriction) including patients with heart failure, cirrhosis, and the syndrome of inappropriate anti-diuretic hormone (SIADH). Patients requiring urgent treatment to raise serum sodium to prevent or to treat serious neurological deficits should not be treated with SAMSCA.