Thursday, January 3, 2013

FDA Approves Juxtapid - New Orphan Drug for Rare Cholesterol Disorder

In continuation of my update on Juxtapid (lomitapide) 

We know that, Lomitapide (INN) is an investigational drug for the treatment of familial hypercholesterolemia, developed by Aegerion Pharmaceuticals.  It has been tested in several Phase II clinical trials as single treatment and in combinations with atorvastatinezetimibe and fenofibrate. 

The US Food and Drug Administration approved lomitapide on December 21, 2012 as anorphan drug to reduce LDL cholesterol, total cholesterol, apolipoprotein B, and non-high-density lipoprotein (non-HDL) cholesterol in patients with homozygous familial hypercholesterolemia (HoFH).



FDA Approves Juxtapid - New Orphan Drug for Rare Cholesterol Disorder

Wednesday, January 2, 2013

Ability to metabolize tamoxifen affects breast cancer outcomes

Continuous vancomycin optimizes neonate therapy

In continuation of my update on vancomycin...

Tuesday, January 1, 2013

Scientists design small molecules that recognize myotonic dystrophy-associated RNAs

In continuation of my update on RNAs


Killing the message: An approach to direct the cleavage of RNA targets with small molecules in living cells is described (see scheme). A bifunctional small molecule (purple) that recognizes a specific three nucleotide repeat sequence and cleaves that sequence in response to light was shown to be effective at degrading the myotonic dystrophy type 1 (DM1) extended repeat RNAs, affecting biological functions.


Ref : http://onlinelibrary.wiley.com/doi/10.1002/anie.201206888/abstract

Monday, December 31, 2012

Chinese medicine yields secrets: Atomic mechanism of two-headed molecule derived from Chang Shan, a traditional chinese herb

The mysterious inner workings of Chang Shan  a Chinese herbal medicine used for thousands of years to treat fevers associated with malaria  have been uncovered thanks to a high-resolution structure solved at The Scripps Research Institute (TSRI). Described in the journal Nature this week, the structure shows in atomic detail how a two-headed compound derived from the active ingredient in Chang Shan works. Scientists have known that this compound, called halofuginone (a derivative of the febrifugine), can suppress parts of the immune system  but nobody knew exactly how.


The new structure shows that, like a wrench in the works, halofuginone jams the gears of a molecular machine that carries out "aminoacylation," a crucial biological process that allows organisms to synthesize the proteins they need to live. Chang Shan, also known asDichroa febrifuga Lour, probably helps with malarial fevers because traces of a halofuginone-like chemical in the herb interfere with this same process in malaria parasites, killing them in an infected person's bloodstream.


"Our new results solved a mystery that has puzzled people about the mechanism of action of a medicine that has been used to treat fever from a malaria infection going back probably 2,000 years or more," said Paul Schimmel, PhD, the Ernest and Jean Hahn Professor and Chair of Molecular Biology and Chemistry and member of The Skaggs Institute for Chemical Biology at TSRI. Schimmel led the research with TSRI postdoctoral fellow Huihao Zhou, PhD.

Halofuginone (see structure, below) has been in clinical trials for cancer, but the high-resolution picture of the molecule suggests it has a modularity that would make it useful as a template to create new drugs for numerous other diseases.





Gattex Approved for Short Bowel Syndrome - Drugs.com MedNews


We know that, Gattex (teduglutide) is a recombinant analog of human glucagon-like peptide 2 for the treatment of adults with short bowel syndrome....

Gattex Approved for Short Bowel Syndrome - Drugs.com MedNews

Sunday, December 30, 2012

FDA Approves Adasuve (loxapine) Inhalation Powder for the Acute Treatment of Agitation Associated with Schizophrenia or Bipolar I Disorder in Adults



We know that, Loxapine (LoxapacLoxitane) is a typical antipsychotic medication, used primarily in the treatment of schizophrenia. It is a member of the dibenzoxazepine class and structurally related to clozapine (which belongs to the chemically akin class of dibenzodiazepines). Several researchers have argued that Loxapine may behave as an atypical antipsychotic.  Loxapine may be metabolized by N-demethylation to amoxapine, a tetracyclic antidepressant. ........




Saturday, December 29, 2012

Penn Team Mimicking a Natural Defense Against Malaria to Develop New Treatments

Penn Team Mimicking a Natural Defense Against Malaria to Develop New Treatments

Dantrolene shows promise for treating DMD


 File:Dantrolene Tanaka et al.svg

We know that, Dantrolene sodium is a muscle relaxant that acts by abolishing excitation-contraction coupling in muscle cells, probably by action on the ryanodine receptor. It is the only specific and effective treatment for malignant hyperthermia, a rare, life-threatening disorder triggered by general anesthesia. It is also used in the management of neuroleptic malignant syndrome, muscle spasticity (e.g. after strokes, in paraplegiacerebral palsy, or patients with multiple sclerosis), 3,4-methylenedioxymethamphetamine ("ecstasy") intoxication, serotonin syndrome, and 2,4-dinitrophenol poisoning. It is marketed by JHP Pharmaceuticals LLC as Dantrium (in North America) and Dantrolen (Europe).
  

Friday, December 28, 2012

Dantrolene shows promise for treating DMD



 File:Dantrolene Tanaka et al.svg



We know that, Dantrolene sodium is a muscle relaxant that acts by abolishing excitation-contraction coupling in muscle cells, probably by action on the ryanodine receptor. It is the only specific and effective treatment for malignant hyperthermia, a rare, life-threatening disorder triggered by general anesthesia. It is also used in the management of neuroleptic malignant syndrome, muscle spasticity (e.g. after strokes, in paraplegiacerebral palsy, or patients with multiple sclerosis), 3,4-methylenedioxymethamphetamine   ("ecstasy") intoxication, serotonin syndrome, and 2,4-dinitrophenol poisoning. It is marketed by JHP Pharmaceuticals LLC as Dantrium (in North America) and Dantrolen (Europe).
  

Wednesday, December 26, 2012

FDA Approves Varizig for Reducing Chickenpox Symptoms

FDA Approves Varizig for Reducing Chickenpox Symptoms :

Varizag : http://www.cangene.com/VariZIG

Abiraterone improves outcomes for prostate cancer prior to chemo

Vismodegib team wins Drug Discovery of the Year award

In continuation of my update on Vismodegib

Vismodegib team wins Drug Discovery of the Year award: The British Pharmacological Society proudly announces that its first annual Drug Discovery of the Year award has been won by the discovery team developing vismodegib for the treatment of basal cell carcinoma (a type of skin cancer).

Monday, December 24, 2012

New low-cost combined therapy shows promise against malaria

Molecular parasitologist Stephen Rich at the University of Massachusetts Amherst has led a research team who report a promising new low-cost combined therapy with a much higher chance of outwitting P. falciparum than current modes. He and plant biochemist Pamela Weathers at the Worcester Polytechnic Institute (WPI), with research physician Doug Golenbock at the UMass Medical School, also in Worcester, have designed an approach for treating malaria based on a new use of Artemisia annua, a plant employed for thousands of years in Asia to treat fever.

"The emergence of resistant parasites has repeatedly curtailed the lifespan of each drug that is developed and deployed," says UMass Amherst graduate student and lead author Mostafa Elfawal. Rich, an expert in the malaria parasite and how it evolves, adds, "We no sooner get the upper hand than the parasite mutates to become drug resistant again. This cycle of resistance to anti-malarial drugs is one of the great health problems facing the world today. We're hoping that our approach may provide an inexpensive, locally grown and processed option for fighting malaria in the developing world."
Currently the most effective malaria treatment uses purified extracts from the Artemisia plant as part of an Artemisinin Combined Therapy (ACT) regime with other drugs such as doxycycline and/or chloroquine, a prescription far too costly for wide use in the developing world. Also, because Artemisia yields low levels of pure artemisinin, there is a persistent worldwide shortage, they add.

The teams's thesis, first proposed by Weathers of WPI, is that locally grown and dried leaves of the whole plant, rich in hundreds of phytochemicals not contained in the purified drug, might be effective against disease at the same time limiting post-production steps, perhaps substantially reducing treatment cost. She says, "Whole-plant Artemisia has hundreds of compounds, some of them not even known yet. These may outsmart the parasites by delivering a more complex drug than the purified form."

Rich adds, "The plant may be its own complex combination therapy. Because of the combination of parasite-killing substances normally present in the plant (artemisinin and flavonoids), a synergism among these constituent compounds might render whole plant consumption as a form of artemisinin-based combination therapy, or what we're calling a 'pACT,' for plant Artemisinin Combination Therapy."


Ref : http://www.plosone.org/article/info%3Adoi%2F10.1371%2Fjournal.pone.0052746