HSS involving resiniferatoxin receives U.S. patent to alleviate intractable pain

We know that, Resiniferatoxin (RTX) is a naturally occurring, ultrapotent capsaicin analog that activates the vanilloid receptor in a subpopulation of primary afferent sensory neurons involved in nociception (the transmission of physiological pain). RTX causes an ion channel in the plasma membrane of sensory neurons — the transient receptor potential vanilloid 1 — to become permeable to cations, most particularly the calcium cation; this evokes a powerful irritant effect followed by desensitization and analgesia. Research is being conducted at the National Institutes of Health and the University of Pennsylvania to design a novel class of analgesics from the latex of resin spurge (Euphorbia resinifera), a cactus-like plant commonly found in Morocco that contains high concentrations of RTX. Resiniferatoxin has a rating of 16,000,000,000 on the Scoville Scale making it one of the highest rated substances known.

Now U. S. Patent and Trademark Office recently issued a patent to the U. S. Department of Health and Human Services involving resiniferatoxin, or RTX, an experimental compound that represents a potential new class of drugs to alleviate the intractable pain that can occur in people with advanced cancer, severe arthritis, and other extremely chronic conditions.

HSS involving resiniferatoxin receives U.S. patent to alleviate intractable pain

Sanofi Canada announces new option for emergency treatment of anaphylaxis

We know that, Epinephrine (also known as adrenaline/adrenalin) is a hormone and a neurotransmitter. Epinephrine has many functions in the body, regulating heart rate, blood vessel and air passage diameters, and metabolic shifts; epinephrine release is a crucial component of the fight-or-flight response of the sympathetic nervous system. In chemical terms, epinephrine is one of a group of monoamines called the catecholamines. It is produced in some neurons of the central nervous system, and in the chromaffin cells of the adrenal medulla from the amino acids phenylalanine and tyrosine.

Now, Sanofi Canada announces a new option for the emergency treatment of anaphylaxis. Allerject™ is the first and only 'talking' epinephrine auto-injector in Canada. 

 

Sanofi Canada announces new option for emergency treatment of anaphylaxis

Intravenous iron dextran effective for restless leg syndrome

Intravenous low molecular weight iron dextran appears to provide effective long-lasting treatment for some patients with restless leg syndrome (RLS), even for those with normal serum ferritin levels, research indicates.

The results showed that 68% of 25 patients with RLS showed moderate or complete improvement of all RLS symptoms after treatment according to a Korean-translated version of the International RLS Severity (K-IRLS) scale and the PAM-RL device, which records periodic leg movements.

However, as reported in Sleep Medicine, the researchers saw no correlation between increases in cerebrospinal fluid (CSF) ferritin in response to therapy and clinical improvements.

"Although a relatively accessible measure, CSF ferritin is at best a crude measure of CNS [central nervous system] iron status and not necessarily reflective of regionally specific changes in iron status that are assumed to account for the symptom development," say Yong Won Cho, from Keimyung University School of Medicine in Daegu, South Korea, and colleagues.

"In addition, the narrow range of symptom severity in this study may have limited our ability to find any differences between CSF ferritin and subjective measures of the disease."

Intravenous iron dextran effective for restless leg syndrome

Everolimus treatment option for tuberous sclerosis patients

In continuation of my update on Everolimus

Everolimus treatment option for tuberous sclerosis patients

FDA Approves Gleevec for Children with Acute Lymphoblastic Leukemia

In continuation of my update on Gleevec

FDA Approves Gleevec for Children with Acute Lymphoblastic Leukemia

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............

Exjade Approved for Inherited Blood Disorder - Drugs.com MedNews

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.

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

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/

New drug protects against side effects of chemotherapy

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. 

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

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."

Drug combination extends pancreatic cancer patient survival, study suggests