Med-Chemist : "Quintessential Medicinal Chemistry"
Saturday, February 16, 2013
FDA Approves Pomalyst for Advanced Multiple Myeloma
We know that, Pomalidomide (INN, originally CC-4047 or 3-amino-thalidomide, marketed as Pomalyst by Celgene), is a derivative of thalidomide that is anti-angiogenic and also acts as an immunomodulator. Pomalidomide was approved on February 8, 2013 by the U.S. Food and Drug Administration (FDA) as a treatment for relapsed and refractory multiple myeloma....
Friday, February 15, 2013
FDA Approves Ravicti for the Chronic Management of Some Urea Cycle Disorders
The U.S. Food and Drug Administration today approved Ravicti (glycerol phenylbutyrate) for the chronic management of some urea cycle disorders (UCDs) in patients ages 2 years and older.
UCDs are genetic disorders that involve deficiencies of specific enzymes involved in the urea cycle, a series of biochemical steps normally required to remove ammonia from the blood. When protein is absorbed and broken down by the body, it produces nitrogen as a waste product. The urea cycle removes nitrogen from the blood and converts it to urea, which is removed from the body through urine. In people with UCDs, nitrogen accumulates and remains in the body as ammonia, which can travel to the brain and cause brain damage, coma or death.
Thursday, February 14, 2013
Wednesday, February 13, 2013
Top-line results from Vanda's tasimelteon Phase IIb/III study on major depressive disorder
We know that, Tasimelteon (BMS-214,778) is a drug which is under development for the treatment of insomnia and other sleep disorders. It is a selective agonist for the melatonin receptors MT1 and MT2 in the suprachiasmatic nucleus of the brain, similar to older drugs such as ramelteon. It has been through Phase III trials successfully and was shown to improve both onset and maintenance of sleep, with few side effects.
A year-long (2011-2012) study at Harvard is testing the use of tasimelteon in blind subjects with non-24-hour sleep-wake disorder.
Now Vanda Pharmaceuticals Inc. (NASDAQ: VNDA), announced top-line results of the Phase IIb/III clinical study (MAGELLAN) in Major Depressive Disorder (MDD), investigating the efficacy and safety of tasimelteon as a monotherapy in the treatment of patients with MDD. The clinical study did not meet the primary endpoint of change from baseline in the Hamilton Depression Scale (HAMD-17) after 8 weeks of treatment as compared to placebo. Both tasimelteon and placebo treated patients had an approximately 40% reduction of their MDD symptoms from baseline. Tasimelteon was shown to be safe and well-tolerated, consistent with observations in prior studies. Given these current proof of concept clinical study results, Vanda has decided to discontinue all activities in this indication.
Vanda has recently reported positive results in two phase III clinical studies of tasimelteon in Non-24-Hour Disorder (Non-24) and plans to submit a New Drug Application to the U.S. Food and Drug Administration in mid-2013......
"These results are disappointing, as there is still a significant unmet medical need for patients with Major Depression," said Mihael H. Polymeropoulos , M.D., President and CEO of Vanda. "Tasimelteon's application in the treatment of blind individuals with Non-24 remains our top priority as we pursue our planned NDA submission this year."
Vanda has recently reported positive results in two phase III clinical studies of tasimelteon in Non-24-Hour Disorder (Non-24) and plans to submit a New Drug Application to the U.S. Food and Drug Administration in mid-2013......
Tuesday, February 12, 2013
Hops, Key to Flavor in Beer, May Prove Useful in New Drugs - Drugs.com MedNews
We know that, Humulone (α-lupulic acid) is a bitter-tasting chemical compound found in the resin of mature hops (Humulus lupulus). Humulone is a prevalent member of the class of compounds known as alpha acids, which collectively give beer its characteristic bitter flavor.
-Humulone.svg.png)
In a new study, researchers determined the precise configuration of humulones, substances derived from hops that give beer its unique flavor.
"Now that we have the right results, what happens to the bitter hops in the beer-brewing process makes a lot more sense," study lead author Werner Kaminsky, a University of Washingto.
Previous research has suggested that moderate consumption of beer and therefore its bitter compounds might have positive effects on diseases such as diabetes and some types of cancer, as well as aiding weight loss and decreasing inflammation.
The new findings could help scientists determine which humulones might prove useful in efforts to develop new drugs, Kaminsky said.
The authors wrote in their report that while "excessive beer consumption cannot be recommended to propagate good health isolated humulones and their derivatives can be prescribed with documented health benefits."
Ref : http://onlinelibrary.wiley.com/doi/10.1002/anie.201208450/abstract
Ref : http://onlinelibrary.wiley.com/doi/10.1002/anie.201208450/abstract
Monday, February 11, 2013
Sunday, February 10, 2013
Messenger RNA–Based Vaccines for Cancer | Articles | Drug Discovery and Development Magazine
Nucleic acids are being extensively investigated for use in gene therapy and in genetic vaccinations in which foreign nucleic acid is translated into proteins by the host cells. Vaccines based on DNA and messenger RNA (mRNA) are able to stimulate all effectors of the adaptive immune response: B lymphocytes, cytotoxic T cells, and T helper cells. This makes them a useful tool in the creation of prophylactic vaccines for infectious diseases and for cancer immunotherapy.
Saturday, February 9, 2013
Phenformin decreases size of lung tumors and increases survival in mice
In continuation of my update on metformin and phenformin
In a new study in the journal Cancer Cell, Shaw and a team of scientists at the Salk Institute for Biological Studies found that phenformin, a derivative of the widely-used diabetes drug metformin, decreased the size of lung tumors in mice and increased the animals' survival. The findings may give hope to the nearly 30 percent of patients with non-small cell lung cancer (NSCLC) whose tumors lack LKB1 (also called STK11).
In a new study in the journal Cancer Cell, Shaw and a team of scientists at the Salk Institute for Biological Studies found that phenformin, a derivative of the widely-used diabetes drug metformin, decreased the size of lung tumors in mice and increased the animals' survival. The findings may give hope to the nearly 30 percent of patients with non-small cell lung cancer (NSCLC) whose tumors lack LKB1 (also called STK11).
The LKB1 gene turns on a metabolic enzyme called AMPK when energy levels of ATP, molecules that store the energy we need for just about everything we do, run low in cells. In a previous study, Shaw, an associate professor in Salk's Molecular and Cell Biology Laboratory and researcher in the Institute's new Helmsley Center for Genomic Medicine, demonstrated that cells lacking a normal copy of the LKB1 gene fail to activate AMPK in response to low energy levels. LKB1-dependent activation of AMPK serves as a low-energy checkpoint in the cell. Cells that lack LKB1 are unable to sense such metabolic stress and initiate the process to restore their ATP levels following a metabolic change. As a result, these LKB1-mutant cells run out of cellular energy and undergo apoptosis, or programmed cell death, whereas cells with intact LKB1 are alerted to the crisis and re-correct their metabolism.
"The driving idea behind the research is knowing that AMPK serves as a sensor for low energy loss in cells and that LKB1-deficient cells lack the ability to activate AMPK and sense energy loss," says David Shackelford, a postdoctoral researcher at Salk who spearheaded the study in Shaw's lab and is now an assistant professor at UCLA's David Geffen School of Medicine.
That led Shaw and his team to a class of drugs called biguanides, which lower cellular energy levels by attacking the power stations of the cell, called mitochondria. Metformin and phenformin both inhibit mitochondria; however, phenformin is nearly 50 times as potent as metformin. In the study, the researchers tested phenformin as a chemotherapy agent in genetically-engineered mice lacking LKB1 and which had advanced stage lung tumors. After three weeks of treatment, Shaw and his team saw a modest reduction in tumor burden in the mice.
Ref : https://www.cell.com/cancer-cell/abstract/S1535-6108%2812%2900518-1
Ref : https://www.cell.com/cancer-cell/abstract/S1535-6108%2812%2900518-1
Subscribe to:
Posts (Atom)