Melanoma Drug Nearly Doubles Survival « VICC News & Publications

In continuation of my update on Zelboraf (vemurafenib)

Melanoma Drug Nearly Doubles Survival « VICC News & Publications

FDA Approves Korlym for Patients with Endogenous Cushing's Syndrome..

Korlym (mifepristone) was approved by the U.S. Food and Drug Administration to control high blood sugar levels (hyperglycemia) in adults with endogenous Cushing’s syndrome. This drug was approved for use in patients with endogenous Cushing’s syndrome who have type 2 diabetes or glucose intolerance and are not candidates for surgery or who have not responded to prior surgery. Korlym should never be used (contraindicated) by pregnant women....

Ref : http://www.corcept.com/medicationguide.pdf

FDA Approves Zioptan (tafluprost ophthalmic solution), Merck's Once-Daily, Preservative-Free Ophthalmic Medication

U.S. Food and Drug Administration (FDA) has approved Zioptan  (tafluprost ophthalmic solution) 0.0015%, the first preservative-free prostaglandin analog ophthalmic solution. Zioptan (pronounced zye-OP-tan) is approved for reducing elevated intraocular pressure (IOP) in patients with open-angle glaucoma (OAG) or ocular hypertension. Open-angle glaucoma is the most common form of glaucoma, while ocular hypertension is a condition characterized by an increase in pressure inside the eye.

Ref : http://www.merck.com/newsroom/news-release-archive/prescription-medicine-news/2012_0213.html  

Compound makes imipenem 16 times more effective against antibiotic-resistant K. pneumoniae

North Carolina State University chemists have created a compound (see structure above - when used in conjunction with the antibiotic imipenem (below structure), increased the antibiotic's effectiveness against the antibiotic-resistant K. pneumoniae 16-fold. The researchers believe that these early results are very promising for future treatments.)  that makes existing antibiotics 16 times more effective against recently discovered antibiotic-resistant "superbugs." 

These so-called superbugs are actually bacterial strains that produce an enzyme known as New Delhi metallo-β-lactamase (NDM-1). Bacteria that produce this enzyme are practically impervious to antibiotics because NDM-1renders certain antibiotics unable to bind with their bacterial targets. Since NDM-1 is found in Gram-negative bacteria like K. pneumoniae, which causes pneumonia, urinary tract, and other common hospital-acquired infections, it is of particular concern. NC State chemist Dr. Christian Melander had found that a compound derived from a class of molecules known as 2-aminoimidazoles "recharged" existing antibiotics, making them effective against Gram-positive antibiotic-resistant bacteria like the Staphylococcus strain MRSA. So Melander, Worthington and graduate students Cynthia Bunders and Catherine Reed set to work on a variety of the compound that might prove similarly effective against their Gram-negative brethren.

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Prescription Shampoo (with Ivermectin) Approved to Treat Head Lice

In continuation of my update on ivermectin                                    

Sklice Lotion, a prescription-strength shampoo to treat head lice, has been approved by the U.S. Food and Drug Administration for people six months and older, the French product maker Sanofi said.

The shampoo contains ivermectin, which traditionally is prescribed in pill form to treat worm infections.   The product's safety and effectiveness were evaluated in clinical studies involving more than 780 people. After two weeks, most participants who had been lice infested did not require daily combing to remove lice eggs, the wire service reported.

The most common adverse reactions included eye infection and irritation, dandruff and dry skin.

Lice are small, blood-sucking insects that cause itching from the saliva they inject into the scalp and nearby areas to prevent premature clotting. Infestations are spread by direct contact or by shared use of brushes and other items that touch the scalp, such as pillows and hats.

Case Western Reserve University - One of the nation's top universities and the best college in Ohio

Neuroscientists at Case Western Reserve University School of Medicine have made a dramatic breakthrough in their efforts to find a cure for Alzheimer's disease. The researchers' findings, published in the journalScience, show that use of a drug in mice appears to quickly reverse the pathological, cognitive and memory deficits caused by the onset of Alzheimer's. The results point to the significant potential that the medication, bexarotene, has to help the roughly 5.4 million Americans suffering from the progressive brain disease. 

Bexarotene has been approved for the treatment of cancer by the U.S. Food and Drug Administration for more than a decade. These experiments explored whether the medication might also be used to help patients with Alzheimer's disease, and the results were more than promising. Landreth and his colleagues chose to explore the effectiveness of bexarotene for increasing ApoE expression. The elevation of brain ApoE levels, in turn, speeds the clearance of amyloid beta from the brain. Bexarotene acts by stimulating retinoid X receptors (RXR), which control how much ApoE is produced.

In particular, the researchers were struck by the speed with which bexarotene improved memory deficits and behavior even as it also acted to reverse the pathology of Alzheimer's disease. The present view of the scientific community is that small soluble forms of amyloid beta cause the memory impairments seen in animal models and humans with the disease. Within six hours of administering bexarotene, however, soluble amyloid levels fell by 25 percent; even more impressive, the effect lasted as long as three days. Finally, this shift was correlated with rapid improvement in a broad range of behaviors in three different mouse models of Alzheimer's.

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Case Western Reserve University - One of the nation's top universities and the best college in Ohio

Mobius Therapeutics Receives Final FDA Approval for New Glaucoma Drug Mitosol

The U.S. Food and Drug Administration (FDA) has approved Mitosol (mitomycin for solution) for use in glaucoma surgery.

"The approval of Mitosol for use in glaucoma surgery represents the culmination of more than five years of work on the part of Mobius Therapeutics," said Ed Timm, President of Mobius Therapeutics.

It will provide surgeons, hospitals, and patients with enhanced convenience, safety, and consistency in the surgical treatment of glaucoma. 


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FDA Approves Kalydeco to Treat Rare Form of Cystic Fibrosis

The U.S. FDA approved Kalydeco (ivacaftor) for the treatment of a rare form of cystic fibrosis (CF) in patients ages 6 years and older who have the specific G551D mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene. 

“Kalydeco is an excellent example of the promise of personalized medicine – targeted drugs that treat patients with a specific genetic makeup,” said FDA Commissioner Margaret A. Hamburg, M.D. “

The unique and mutually beneficial partnership that led to the approval of Kalydeco serves as a great model for what companies and patient groups can achieve if they collaborate on drug development. 

“Kalydeco is the first available treatment that targets the defective CFTR protein, which is the underlying cause of cystic fibrosis,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “This is a breakthrough therapy for the cystic fibrosis community because current therapies only treat the symptoms of this genetic disease.”

Two 48-week, placebo-controlled clinical studies involving 213 patients, one in patients ages 12 years and older and another in patients ages 6 years to 11 years, were used to evaluate the safety and efficacy of Kalydeco in CF patients with the G551D mutation. In both studies, treatment with Kalydeco resulted in significant and sustained improvement in lung function.

Kalydeco is effective only in patients with CF who have the G551D mutation. It is not effective in CF patients with two copies of the F508 mutation in the CFTR gene, which is the most common mutation that results in CF. If a patient’s mutation status is not known, an FDA-cleared CF mutation test should be used to determine whether the G551D mutation is present.

Ref : http://investors.vrtx.com/releasedetail.cfm?ReleaseID=644158

Clazosentan reduces risk of blood vessel spasm in patients with brain aneurysm

Clazosentan reduces risk of blood vessel spasm in patients with brain aneurysm: An experimental drug, clazosentan, reduced the risk of blood vessel spasm in patients with a brain aneurysm, according to research presented at the American Stroke Association's International Stroke Conference 2012.

Genentech receives FDA approval for Vismodegib to treat skin cancer

In continuation of my update Vismdegib

Genentech receives FDA approval for Vismodegib to treat skin cancer: A new skin cancer drug tested for the first time in the world five years ago at the Virginia G. Piper Cancer Center at Scottsdale Healthcare just received expedited approval by the U.S. Food and Drug Administration, a remarkable accomplishment in new drug development.

Colchicine, another weapon against cancer

In continuation of my update on Colchine

 Colchicine, another weapon against cancer: Finding a drug that targets only the diseased cells in our body and is otherwise harmless to healthy tissue is a goal for cancer researchers. It's driven the work of Professor Laurence Patterson, Director of the Institute of Cancer Therapeutics at the University of Bradford, and his team of researchers.

Scientists discover new mechanisms by which RNA drugs can control gene activity

 In continuation of my update on RNAi

Short strands of nucleic acids, called small RNAs, can be used for targeted gene silencing, making them attractive drug candidates. These small RNAs block gene expression through multiple RNA interference (RNAi) pathways, including two newly discovered pathways in which small RNAs bind to Argonaute proteins or other forms of RNA present in the cell nucleus, such as long non-coding RNAs and pre-mRNA. 

Keith T. Gagnon, PhD, and David R. Corey, PhD, University of Texas Southwestern Medical Center, in Dallas, review common features shared by RNAi pathways for controlling gene expression and focus in detail on the potential for Argonaute-RNA complexes in gene regulation and other exciting new options for targeting emerging forms of non-coding RNAs and pre-mRNAs in the article "Argonaute and the Nuclear RNAs: New Pathways for RNA Mediated Control of Gene Expression." 

"The field of RNA mediated control of gene expression is rapidly evolving and the article by Gagnon and Corey provides a highly informative and up to date review of this exciting and often surprising area of biomedical research. We are delighted to publish this important review for the field," says Co-Editor-in-Chief Bruce A. Sullenger, PhD, Duke Translational Research Institute, Duke University Medical Center, Durham, NC.

Ref : http://www.liebertpub.com/global/pressrelease/new-rna-based-therapeutic-strategies-for-controlling-gene-expression/987/

Researchers identify fexinidazole as potential new therapy for visceral leishmaniasis

Researchers at the University of Dundee have identified fexinidazole as a possible, much-needed, new treatment for the parasitic disease visceral leishmaniasis.

Fexinidazole is already in phase 1 clinical trials for a related disease - African sleeping sickness - but a research team at Dundee including Dr Susan Wyllie, Professor Alan Fairlamb and colleagues has identified it as having potential in treating leishmaniasis.

Their research has been published by the journal Science Translational Medicine, and was funded by the Wellcome Trust.

Tests in mice showed that the drug has a greater than 98% rate of suppressing infection of leishmaniasis, comparable to current treatments such as miltefosine and Pentostam.

These and other existing treatment options all suffer from disadvantages; they are not always safe, effective or easy to administer. The only oral drug miltefosine cannot be given to women of child-bearing age due to a substantial risk of birth defects; other drugs are costly and have to be given by injection. Thus there is a continuing need for safe and cost-effective drugs suitable for use in resource-poor settings.

Ref : http://www.dundee.ac.uk/pressreleases/2012/february12/leishmaniasis.htm

Erivedge Approved to Treat Basal Cell Carinoma

Erivedge(vismodegib) has been approved by the U.S. Food and Drug Administration to treat the most common form of skin cancer, basal cell carcinoma, the agency said Monday.

The drug was approved for people for whom surgery or radiation aren't options, and for people with basal cell that has spread to other parts of the body, according to an FDA news release. Erivedge was evaluated in clinical studies involving 96 people with basal cell carcinoma. The most common side effects included muscle spasms, hair loss, weight loss, nausea, diarrhea, fatigue, distorted taste, loss of appetite and constipation.

The drug was approved with an FDA's label warning that pregnant women who take Erivedge could have babies at greater risk of severe birth defects or death. "Pregnancy status must be verified prior to the start of Erivedge treatment," the agency release advised.

Ref : http://www.gene.com/gene/news/press-releases/display.do?method=detail&id=13827

Pertuzumab plus trastuzumab and docetaxel has competitive advantages in efficacy over our current proprietary clinical gold-standard treatment...

In continuation of my update docetaxel

"Pertuzumab plus trastuzumab and docetaxel has competitive advantages in efficacy over our current proprietary clinical gold-standard treatment, trastuzumab plus docetaxel," said Decision Resources Analyst Amy Duva"l. 

Decision Resources' analysis of the breast cancer drug market also finds that Roche/Genentech/Chugai's Trastuzumab-DM1 (T-DM1) is likely to initially enter later-lines of treatment before receiving approval for the first-line setting, which means it will gain use across all lines of therapy, fragmenting its patient share across lines of treatment and restricting its uptake in the first-line setting. 

Additionally, according to insights from interviewed thought-leaders, pertuzumab plus trastuzumab and docetaxel and T-DM1 plus pertuzumab have demonstrated the potential to increase patients' overall survival, which has not been improved by drug-treatment since the approval of trastuzumab over a decade ago. 

Findings also reveal that the overall breast cancer drug market declined from $10 billion in 2010 to $9.3 billion in 2011 in the United States, France, Germany, Italy, Spain, the United Kingdom and Japan. Decision Resources forecasts that the market will increase from $9.3 billion in 2011 to $10.8 billion in 2020. Significant declines in sales, due to generic and biosimilar price erosion and a substantial reduction in the prescribing of Roche/Genentech/Chugai's Avastin, particularly in the U.S., will be offset by the launch and uptake of premium-priced emerging therapies, particularly in the metastatic HER2-positive setting. 

Ref : http://decisionresources.com/News-and-Events/Press-Releases/Breast-Cancer-Drug-Market-020212