Drug reduces hospitalizations and cost of treating young children with sickle cell anemia

The study is the largest ever focusing on the economic impact of the drug hydroxyurea (see structure below)

 in children with the inherited blood disorder. The result supports expanded use of the drug to extend the length and quality of life for sickle cell anemia patients of all ages, said Winfred Wang, M.D., a member of the St. Jude Department of Hematology and principal investigator of the multicenter federally funded trial known as BABY HUG.

"We estimate that hydroxyurea cut overall annual medical expenses about $3,000 for each patient by helping patients avoid disease complications that require inpatient hospital care," said Wang, who is first and corresponding author of the Pediatrics study. "We expect those savings will grow along with patients, whose symptoms often increase in severity and frequency as they age."

About 100,000 individuals in the U.S. and millions worldwide have sickle cell disease, which leaves them at risk for premature death and disability. The disease is the most common genetic disorder affecting African-American individuals, but those from other ethnic and racial backgrounds also inherit mutations in the hemoglobin gene. The mutations result in blood cells that are prone to assuming the sickled shape that gives the disease its name and that leave patients at increased risk for episodes of acute pain, stroke, organ damage and other complications.

Drug reduces hospitalizations and cost of treating young children with sickle cell anemia

Scientists fish for new epilepsy model and reel in potential drug

According to new research on epilepsy, zebrafish have certainly earned their stripes. Results of a study in Nature Communications suggest that zebrafish carrying a specific mutation may help researchers discover treatments for Dravet syndrome (DS), a severe form of pediatric epilepsy that results in drug-resistant seizures and developmental delays.

Scientists fish for new epilepsy model and reel in potential drug

Synthetic polymer could stop the spread of HIV

The researchers created the large molecule with several sugar molecules, known as glycopolymers (Glycopolymer is synthetic polymer with pendant carbohydrates). By using different sugars attached to the macromolecule in solution, the scientists were able to investigate which sugar molecules were the most effective in inhibiting the potential binding of the virus.

They then measured how the designed macromolecules compete with the virus to bind to the dendritic cells of the immune system at different concentrations.

"These are preliminary but encouraging results for potentially preventing the spread of the HIV by sexual contact," said Dr Remzi Becer from Queen Mary's School of Engineering and Materials Science.

"We've shown that our synthetic molecule binds to the immune cell, which in turn blocks the virus from attaching and entering. The precisely designed macromolecules could be an ingredient of a condom cream or vaginal gel to act as a physical barrier from allowing the virus into the body."

Dr Becer added: "While this isn't a cure for HIV, it is a novel approach that could dramatically slow down the spread of HIV by sexual contact, and a model that could be replicated to treat other sexually transmitted diseases."

Ref : http://onlinelibrary.wiley.com/doi/10.1002/marc.201300439/abstract

Synthetic polymer could stop the spread of HIV

Alzheimer's 'missing link' found: Promising target for new drugs

Yale School of Medicine researchers have discovered a protein that is the missing link in the complicated chain of events that lead to Alzheimer's disease, they report in the Sept. 4 issue of the journal Neuron. Researchers also found that blocking the protein with an existing drug can restore memory in mice with brain damage that mimics the disease.

Ref : http://dx.doi.org/10.1016/j.neuron.2013.06.036

Alzheimer's 'missing link' found: Promising target for new drugs

AstraZeneca and Bristol-Myers Squibb resubmit Dapagliflozin New Drug Application for the treatment of type 2 diabetes in the U.S

In continuation of my update on Dapagliflozin

AstraZeneca and Bristol-Myers Squibb resubmit Dapagliflozin New Drug Application for the treatment of type 2 diabetes in the U.S

FDA Advisory Committee Unanimously Recommends Approval Of Bayer's Riociguat In Two Pulmonary Hypertension Indications

We know that, Riociguat is (BAY 63-2521) is a novel drug that is in clinical development by Bayer. It is a stimulator ofsoluble guanylate cyclase (sGC). At the moment Phase III clinical trials investigate the use of riociguat as a new approach to treat two forms of pulmonary hypertension (PH): chronic thromboembolic pulmonary hypertension (CTEPH) and pulmonary arterial hypertension (PAH). Riociguat constitutes the first drug of a novel class of sGC stimulators.

FDA Advisory Committee Unanimously Recommends Approval Of Bayer's Riociguat In Two Pulmonary Hypertension Indications

Drug used for blood cancers may stop spread of breast cancer cells

In continuation of my update on Decitabine

A drug used to treat blood cancers may also stop the spread of invasive breast cancer, researchers at Mayo Clinic in Florida have discovered. Their study, published online in Breast Cancer Research, found that in the lab and in animals, the drug decitabine turns on a gene coding for protein kinase D1 (PRKD1) that halts the ability of cancer cells to separate from a tumor and spread to distant organs.

Drug used for blood cancers may stop spread of breast cancer cells

Ref : http://breast-cancer-research.com/content/15/2/R66

Study: Type 2 diabetic patients treated with DPP-4 linagliptin experience reductions in blood glucose levels

Data published in The Lancet showed that elderly people with Type 2 Diabetes (T2D) treated for 24 weeks with the dipeptidyl peptidase-4 (DPP-4) inhibitor linagliptin, marketed by Boehringer Ingelheim and Eli Lilly and Company, experienced significant reductions in blood glucose levels (HbA1c) compared with those receiving placebo. In addition, the overall safety and tolerability profile of linagliptin was similar to placebo, with no significant difference in hypoglycaemia.

Study: Type 2 diabetic patients treated with DPP-4 linagliptin experience reductions in blood glucose levels

Watermelon juice relieves post-exercise muscle soreness

Encarna Aguayo and colleagues cite past research on watermelon juice's antioxidant properties and its potential to increase muscle protein and enhance athletic performance. But scientists had yet to explore the effectiveness of watermelon juice drinks enriched in L-citrulline. Aguayo's team set out to fill that gap in knowledge.

They tested natural watermelon juice, watermelon juice enriched in L-citrulline (ABOVE STRUCTURE) and a control drink containing no L-citrulline on volunteers an hour before exercise. Both the natural juice and the enriched juice relieved muscle soreness in the volunteers. L-citrulline in the natural juice (unpasteurized), however, seemed to be more bioavailable -- in a form the body could better use, the study found.

Watermelon juice relieves post-exercise muscle soreness

Combined therapy could repair and prevent damage in Duchenne muscular dystrophy, study suggests

Results from a clinical trial of eteplirsen, {RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-m5U-   C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G),    5'-[P-[4-[[2-[2-(2-hydroxyethoxy) ethoxy]ethoxy] carbonyl]-1-piperazinyl]-N,N-   dimethylphosphonamidate]}  a drug designed to treat Duchenne muscular dystrophy, suggest that the therapy allows participants to walk farther than people treated with placebo and dramatically increases production of a protein vital to muscle growth and health. The study, led by a team in The Research Institute at Nationwide Children's Hospital, is the first of its kind to show these results from an exon-skipping drug -- a class of therapeutics that allows cells to skip over missing parts of the gene and produce protein naturally....

Combined therapy could repair and prevent damage in Duchenne muscular dystrophy, study suggests

Ref http://onlinelibrary.wiley.com/doi/10.1002/ana.23982/abstract

New approach in the treatment of breast cancer

Scientists at the MedUni Vienna, in collaboration with a working group led by Nancy Hynes at the University of Basel, have discovered a new approach in the treatment of breast cancer: an international team involving the Clinical Institute of Pathology at the MedUni Vienna has been able to demonstrate the activation of a receptor, the Ret protein (Rearranged during transfection), on the surface of breast cancer cells. Increased levels of this protein are associated with a lower likelihood of survival for breast cancer patients.

Ref : http://onlinelibrary.wiley.com/doi/10.1002/emmm.201302625/abstract

Read more about RET Inhibitors at : http://www.cancercommons.org/tag/ret-inhibitors/

New approach in the treatment of breast cancer

GLENMARK-A new way for a new world « New Drug Approvals

Undiminished zest..

“Every year we expect two more molecules to get into clinical trials,” says Saldanha, his zest undiminished by past failures. “In 2008, in a span of one or two quarters, our entire pipeline pretty much got wiped out, but we never lost our commitment and passion.” At that time its most advanced molecule, oglemilast, used for treating patients with chronic obstructive pulmonary disease, had to be abandoned when its Phase IIb trials produced unsatisfactory results. It also had to suspend clinical development of GRC 6211, a compound for treating osteoarthritis pain, because of side effects.

GLENMARK-A new way for a new world « New Drug Approvals

New treatment for brittle bone disease found

We know that, Risedronic acid (see structure) (INN) or risedronate sodium (USAN) is a bisphosphonate used to strengthen bone, treat or prevent osteoporosis, and treat Paget's disease of bone. It is produced and marketed by Warner Chilcott, Sanofi-Aventis, and in Japan by Takeda under the trade names Actonel, Atelvia, and Benet. It is also available in a preparation that includes a calcium carbonate supplement, asActonel with Calcium.

Osteogenesis imperfecta (OI and sometimes known as brittle bone disease, or "Lobstein syndrome") is a congenital bone disorder. People with OI are born with defective connective tissue, or without the ability to make it, usually because of a deficiency ofType-I collagen. This deficiency arises from an amino acid substitution of glycine to bulkier amino acids in the collagen triple helixstructure. The larger amino acid side-chains create steric hindrance that creates a bulge in the collagen complex, which in turn influences both the molecular nanomechanics as well as the interaction between molecules, which are both compromised.^[3] As a result, the body may respond by hydrolyzing the improper collagen structure. If the body does not destroy the improper collagen, the relationship between the collagen fibrils and hydroxyapatite crystals to form bone is altered, causing brittleness.^[4] Another suggested disease mechanism is that the stress state within collagen fibrils is altered at the locations of mutations, where locally larger shear forces lead to rapid failure of fibrils even at moderate loads as the homogeneous stress state found in healthy collagen fibrils is lost.^[3]These recent works suggest that OI must be understood as a multi-scale phenomenon, which involves mechanisms at the genetic, nano-, micro- and macro-level of tissues.

Ref : http://linkinghub.elsevier.com/retrieve/pii/S0140673613610910

New treatment for brittle bone disease found

New role for Tamoxifen in saving high-risk breast cancer patients

In continuation of my update on Tamoxifen...

New role for Tamoxifen in saving high-risk breast cancer patients

Ref : http://jco.ascopubs.org/content/early/2013/08/05/JCO.2012.47.8313

New designer compound JQ1, treats heart failure by targeting cell nucleus

Researchers from Case Western Reserve University School of Medicine and the Dana-Farber Cancer Institute have made a fundamental discovery relevant to the understanding and treatment of heart failure -- a leading cause of death worldwide. The team discovered a new molecular pathway responsible for causing heart failure and showed that a first-in-class prototype drug, JQ1, (see structure)  blocks this pathway to protect the heart from damage.

In contrast to standard therapies for heart failure, JQ1 works directly within the cell's command center, or nucleus, to prevent damaging stress responses. This groundbreaking research lays the foundation for an entirely new way of treating a diseased heart. The study is published in the August 1 issue of Cell.

"As a practicing cardiologist, it is clear that current heart failure drugs fall alarmingly short for countless patients. Our discovery heralds a brand new class of drugs which work within the cell nucleus and offers promise to millions suffering from this common and lethal disease," said Saptarsi Haldar, MD, senior author on the paper, assistant professor of medicine at Case Western Reserve and cardiologist at University Hospitals Case Medical Center.

Heart failure occurs when the organ's pumping capacity cannot meet the body's needs. Existing drugs, most of which block hormones such as adrenaline at the cell's outer surface, have improved patient survival. Unfortunately, several clinical studies have demonstrated that heart failure patients taking these hormone-blocking drugs still succumb to high rates of hospitalization and death. Leveraging a new approach, the research team turned their attention from the cell's periphery to the nucleus -- the very place that unleashes sweeping damage-control responses which, if left unchecked, ultimately destroy the heart.

The team found that a new family of genes, called BET bromodomains, cause heart failure because they drive hyperactive stress responses in the nucleus. Prior research linking BET bromodomains to cancer prompted the laboratory of James Bradner, MD, the paper's senior author and a researcher at the Dana-Farber, to develop a direct-acting BET inhibitor, called JQ1. In models of cancer, JQ1 functions to turn off key cancer-causing genes occasionally prompting cancer cells to "forget" they are cancer. In models of heart failure, JQ1 silences genetic actions causing enlargement of and damage to the heart even in the face of overwhelming stress.

"While it's been known for many years that the nucleus goes awry in heart failure, potential therapeutic targets residing in this part of the cell are often dubbed as 'undruggable' given their lack of pharmacological accessibility," said Jonathan Brown, MD, cardiologist at Brigham and Women's Hospital and co-first author on the paper. "Our work with JQ1 in pre-clinical models shows that this can be achieved successfully and safely."

Ref : http://www.sciencedirect.com/science/article/pii/S0092867413008842

Video : http://www.ted.com/talks/jay_bradner_open_source_cancer_research.html

New designer compound treats heart failure by targeting cell nucleus