Thursday, January 31, 2013

Exjade Approved for Inherited Blood Disorder - Drugs.com MedNews

We know that, Deferasirox (marketed as Exjade) is a rationally-designed oral iron chelator. Its main use is to reduce chronic iron overload in patients who are receiving long-term blood transfusions for conditions such as beta-thalassemia and other chronic anemias. It is the first oral medication approved in the USA for this purpose.
It was approved by the United States Food and Drug Administration (FDA) in November 2005. According to FDA (May 2007), renal failure and cytopenias have been reported in patients receiving deferasirox oral suspension tablets. It is approved in the European Union by the European Medicines Agency (EMA) for children 6 years and older for chronic iron overload from repeated blood transfusions.
Now  Exjade (deferasirox) has been approved by the U.S. Food and Drug Administration to remove excess iron in the blood among people with a genetic blood disorder called non-transfusion-dependent thalassemia (NTDT). Too much iron in the blood can damage vital organs, the agency said Wednesday in a news release. 

Thalassemia typically leads to the production of fewer red blood cells and less hemoglobin, a protein that carries oxygen throughout the body. NTDT is a milder form of thalassemia that unlike other forms, does not require frequent blood transfusions. Thalassemia affects about 1,000 people in the United States, the FDA said............

Wednesday, January 30, 2013

NIH clinical trial begins for treatment of rare, fatal neurological disorder, January 23, 2013 News Release - National Institutes of Health (NIH)

 We know that, Cyclodextrins (sometimes called cycloamyloses) are a family of compounds made up of sugar molecules bound together in a ring (cyclic oligosaccharides). Cyclodextrins are produced from starch by means of enzymatic conversion. They are used in food, pharmaceutical,drug delivery, and chemical industries, as well as agriculture and environmental engineering. Hydroxypropyl Beta Cyclodextrin (HPβCD) is the chief active compound found in Procter and Gamble's deodorizing product "Febreze" under the brand name "Clenzaire".Cyclodextrins are composed of 5 or more α-D-glucopyranoside units linked 1->4, as in amylose (a fragment of starch). The 5-membered macrocycle is not natural. Recently, the largest well-characterized cyclodextrin contains 32 1,4-anhydroglucopyranoside units, while as a poorly characterized mixture, at least 150-membered cyclic oligosaccharides are also known. Typical cyclodextrins contain a number of glucose monomers ranging from six to eight units in a ring, creating a cone shape.



A clinical trial to evaluate a drug candidate called cyclodextrin as a possible treatment for Niemann-Pick disease type C1 (NPC), a rare and fatal genetic disease has already started on 23rd Jan, 2012. Scientists from the NIH’s National Center for Advancing Translational Sciences (NCATS) and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) will conduct the clinical trial at the NIH Clinical Center. Reaching this trial stage required collaboration among government, industry, patient advocacy groups and academic researchers.



Tuesday, January 29, 2013

New drug protects against side effects of chemotherapy

 A drug developed at Linköping University in Sweden protects against the side effects of cancer treatments while strengthening the effects on the tumour. An international drug evaluation is now starting up on a larger group of patients. 

The results of the studies with the compound, known as calmangafodipir  [Ca4Mn(DPDP)5], were published in the latest issue of the cancer journal Translational Oncology with Professor Rolf G. G. Andersson as the main author.


The research was initiated on a substance called mangafodipir MnDPDP (see structure below), which was used as a contrast media in magnetic resonance scans. But pharmacologists at LiU discovered that it also protected healthy cells in connection with cancer treatments.



"We found that the substance could affect the formation of oxygen radicals, which are a cause of side effects in chemotherapy," says Professor Andersson.


For example, the number of white blood cells decreases drastically in almost all the patients, which opens the door to infections that could even be fatal.


The researchers began with cell tests, and then went on to mice infected with cancer cells. The mice were treated with chemotherapy and were administered mangafodipir at the same time. Tumour formation decreased while white blood cells were protected.


One problem was that a large portion of the manganese in the substance was released; as a consequence, the positive effect subsided. The free manganese can also be poisonous and cause brain damage. More at the ffollowing link...

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3542842/

Monday, January 28, 2013

Beta carotene may protect people with common genetic risk factor for type-2 diabetes

Stanford University School of Medicine investigators have found that for people harboring a genetic predisposition that is prevalent among Americans, beta carotene, which the body converts to a close cousin of vitamin A, may lower the risk for the most common form of diabetes, while gamma tocopherol, the major form of vitamin E in the American diet, may increase risk for the disease. 


Sunday, January 27, 2013

Drug combination extends pancreatic cancer patient survival, study suggests

A multi-center Phase III clinical trial demonstrates that Abraxane (below left structure) (nab-paclitaxel) plus gemcitabine is the first combination of cancer drugs to extend survival of late-stage pancreatic cancer patients compared to standard treatment. Their findings show that Abraxane plus gemcitabine (below right structure) was well tolerated and resulted in clinically meaningful outcomes compared to gemcitabine alone, the current standard of care. 


MPACT is the largest phase III clinical trial completed in advanced pancreatic cancer with more than 800 patients. Findings from the study showed a 59 percent increase in one-year median survival rates from less than a quarter of the patients (22 percent) to more than a third (35 percent). The two-year survival rate for this cancer is negligible, less than 4 percent, but that more than doubles (9 percent) with the nab-paclitaxel/gemcitabine combination.

The study showed significant improvement among some of the sickest patients including those with increased metastases. Significantly there was no increase in life-threatening toxicity. Other drug combinations that have demonstrated benefit have been limited by increased toxicities.

"This is a major improvement in a cancer with the lowest survival rates among all cancer types," said Dr. Ramesh Ramanathan, medical director of Virginia G. Piper Cancer Center Clinical Trials at Scottsdale Healthcare and principal investigator for the clinical trial in the United States. "Advanced pancreatic cancer is fourth most common cause of cancer death in the United States and throughout the world. It is difficult to diagnose with a majority of the cases diagnosed at a late stage after the disease has already advanced."

Saturday, January 26, 2013

Novel technique reveals dynamics of telomere DNA structure: Chromosome-capping telomeres are a potential target for anti-cancer drugs

In continuation of my update on telomerase
"Most cancer cells use telomerase as one mechanism to maintain uncontrolled growth, so it is an important target for anti-cancer therapeutics," Stone said. "The G-quadruplex structures of telomere DNA inhibit the function of the telomerase enzyme, so we wanted to understand the mechanical stability of this structure."


Researchers used  "magnetic tweezers" system to stretch the DNA molecule, while a fluorescence microscopy technique was used to monitor small-scale structural changes in the DNA. 
"Unlike other DNA structures, the G-quadruplex structure is fairly brittle. It takes very little perturbation to make the whole thing fall apart," Stone said. "We also found that the unfolded state has a highly compacted conformation, which tells us that it still has interactions that favor the folding reaction."
These findings have implications for understanding the molecular mechanisms of telomere-associated proteins and enzymes involved in the unfolding reaction, as well as for rational design of anti-cancer drugs, Stone said. Small molecules that bind to and stabilize telomere DNA G-quadruplexes have shown promise as anti-cancer drugs.

The integration of fluorescence measurements and magnetic tweezers is a powerful method for monitoring DNA structural dynamics, and as biophysical techniques go, it is not hard to implement, Stone said. His lab worked with DNA molecules containing the G-quadruplex sequence from human telomere DNA, attaching one end of the DNA to a glass slide and the other end to a tiny magnetic bead. A magnet held above the sample pulled on the bead, exerting a stretching force on the DNA molecule that varied according to how close the magnet was to the sample.

Ref : http://nar.oxfordjournals.org/content/early/2013/01/08/nar.gks1341


Takeda Receives FDA Approval for Oseni (alogliptin and pioglitazone) for Type 2 Diabetes

In continuation of y update on pioglitazone

Takeda Pharmaceutical Company Limited (Takeda) and its wholly-owned subsidiary, Takeda Pharmaceuticals U.S.A., Inc. today announced that the United States (U.S.) Food and Drug Administration (FDA) has approved Oseni (alogliptin and pioglitazone) for the treatment of type 2 diabetes in adults as adjuncts to diet and exercise.

Thursday, January 24, 2013

NuPathe's Zecuity Approved by the FDA for the Acute Treatment of Migraine

We know that, Sumatriptan (see structure) is a synthetic drug belonging to the triptan class, used for the treatment of migraine headaches. Structurally, it is an analog of the naturally occurring neuro-active alkaloids dimethyltryptamine (DMT), bufotenine, and 5-methoxy-dimethyltryptamine, with an N-methyl sulfonamidomethyl- group at position C-5 on the indole ring. On an 17, 2013, FDA has approved, Zecuity (sumatriptan iontophoretic transdermal system) for the acute treatment of migraine with or without aura in adults. Zecuity is a single-use, battery-powered patch that actively delivers sumatriptan, the most widely prescribed migraine medication, through the skin. Zecuity provides relief of both migraine headache pain and migraine-related nausea (MRN)....



Tuesday, January 22, 2013

Researchers identify potential sources of medicines derived from plants against diabetes

A group of researchers from the university's School of Science, led by Dr Solomon Habtemariam, believe they have identified potential sources of medicines derived from plants which may have fewer adverse side-effects for diabetes sufferers.

The scientists are investigating the properties of two plants found in south-east Asia which they think could have properties that are not only anti-diabetic, but also lipid- or fat-lowering, and so can help tackle obesity. The researchers at Greenwich aim to isolate and identify certain extracts from the plants Cassia auriculata and Cassia alata, which could have 'active ingredients' for treating diabetes. They discovered that one of the compounds isolated from the plant, kaempferol 3-O-rutinoside, (structure below)  has proved to be more than eight times more potent than the standard anti-diabetic drug, acarbose.  



The team also found the plants have anti-oxidant properties, which is beneficial when treating diabetes.


"Our other most interesting finding is that many of the active ingredients from the Cassia auriculata plant work through a process called 'synergism' - in other words, they work together to produce an effect greater than the sum of their individual effects," Dr Habtemariam says. "Overall, this suggests that the crude plant extract has lots of potential to be used clinically for treating diabetes and associated diseases."

The researchers adds that the research  is ongoing and requires further study and validation, in my opinion it is interesting...

Ref : http://www2.gre.ac.uk/about/news/articles/2012/a2410-drugs-for-diabetes-scientists-test-the-power-of-plants