New Drug, Ruxolitinib May Help Fight Rare Bone Marrow Disorder

In continuation of my update on ruxolitinib

New Drug May Help Fight Rare Bone Marrow Disorder: WEDNESDAY, Feb. 29 -- Two new studies confirm that the new drug ruxolitinib can help people with the rare bone marrow disorder called myelofibrosis.

While the drug, marketed in the United States under the brand name Jakafi, won't cure...

New drug improves glycaemic control with minimum risk of hypoglycemia in type 2 diabetics..

TAK-875, a new treatment for type 2 diabetes, improves blood sugar control and is equally effective as glimepiride, but has a significantly lower risk of creating a dangerous drop in blood sugar, called hypoglycemia, according to a new study. TAK-875 is a novel oral medication designed to enhance insulin secretion in a glucose-dependant manner, which means that it has no effect on insulin secretion when glucose levels are normal, and as such has the potential to improve the control of blood sugar levels without the risk of hypoglycemia.

In the study, Charles Burant, M.D., Ph.D., professor of internal medicine at the University of Michigan Health System, and colleagues randomly assigned 426 patients with type 2 diabetes who were not achieving adequate glucose control through diet, exercise or metformin treatment to one of five doses of TAK-875, a placebo, or glimepiride, a conventional diabetes treatment. The primary outcome was change in hemogloblin A1c from the start of the study.

At 12 weeks, all doses of TAK-875 resulted in significant drops in HbA1c compared with placebo, a similar reduction occurred in patients given glimepiride.

At a TAK-875 dose of 25 mg or higher, about twice as many patients (33 to 48 percent) reached the American Diabetics Association target of HbA1c less than 7 percent within 12 weeks, compared with placebo (19 percent) and was similar to glimepiride (40 percent).

Ref : http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(11)61879-5/fulltext

Skin cancer drug, vemurafenib may prolong survival in advanced cases: Study

In continuation of my update on vemurafenib...

According to an international study a new treatment for advanced skin cancer almost doubles survival times. Researchers say 132 patients in the U.S. and Australia who were given the drug vemurafenib gained several extra months of life. The treatment is one of two drugs for late-stage melanoma, approved on fast-track in the US last year, which offer hope for patients with advanced melanoma. Vemurafenib is suitable for about half of patients with advanced melanoma as it targets tumors that express a certain gene mutation. Before that, there had been no new drugs for the cancer for more than a decade...

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FDA panel votes in favor of earlier rejected anti-obesity drug Qnexa

In continuation of my up date on Qnexa

FDA panel votes in favor of earlier rejected anti-obesity drug Qnexa: Qnexa took a step closer to approval on Wednesday, when outside advisers to the U.S. Food and Drug Administration voted 20-to-2 in favor of approval. The panel also recommended the company conduct a study to scrutinize even more closely any risk of heart problems. That study may be required after the drug is approved. Should the FDA ask for further data on heart risks before any approval, it would delay Qnexa's launch further.

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.