Patrick Cox's Tech Digest - View by Tag "drug development"

Early Bottlenecks in Developing Biopharmaceutical Products Delay Commercialization

August 21, 2014

National Institutes of Health (NIH) has published a study in the journal Science Translational Medicine to show how “bottlenecking” pharmaceutical companies early in the drug development slows down the process immensely of getting a drug to market. One of the phenomena observed was that companies often run into so much red tape in the development pathway that they are forced to give up on a product. They normally then sell that technology to another firm to repurpose the technology into another product. This study represents further proof that our current drug development regulations are counterproductive.

“Most Exciting Ever” Novartis Drug Points to Huge Sales

September 2, 2014

A new heart-failure drug from Novartis could have blockbuster potential. The clinical trial results were released at a recent meeting of the European Society of Cardiology and were published in the New England Journal of Medicine. The drug, LCZ696, is used to treat reduced ejection fraction, or the heart muscle failing to properly contract. Given the stellar results recently released, some analysts now suggest the drug could replace a variety of heart failure drugs that have been on the market for over two decades. Novartis next plans to test the drug on preserved ejection fraction patients, or those in whom the ventricles of the heart do not properly relax.

Protein Clouds Gather and Disperse, But Why?

September 3, 2014

Georgetown University and Scripps Research Institute scientists, writing in the Journal of Cell Biology, discuss the possible causes and results of protein “clouds” which gather inside cells, then dissolve as quickly as they appear. In a process known as phase separation, the researchers explain, proteins of various shapes, collectively known as “intrinsically disordered proteins” gather together inside cells for a time and then dissipate. Currently, scientists think this collecting and dissipation of proteins could be related to disease onset, and the next step is to determine if these protein collections can be dissolved with small molecule drugs without harming cells.

Diseased Cells Made to Synthesize Their Own Drug

September 8, 2014

Scientists from The Scripps Research Institute, writing in the German Chemical Society’s journal Angewandte Chemie, report a new disease-fighting process whereby diseased cells could become the manufacture point of the very treatments that repair those cells. Studying their platform on a variation of muscular dystrophy, the researchers were able to turn the same RNA defect that causes the disease into the cellular engine that combats it. If tests progress, this exciting new technology could improve the treatment of not only muscular dystrophy, but also diseases such as ALS and Huntington’s.

Researcher Develops, Proves Effectiveness of New Drug for Spinal Muscular Atrophy

September 16, 2014

University of Missouri-Columbia researchers, writing in the Journal of Human Molecular Genetics, reveal what could be a drug treatment breakthrough for spinal muscular atrophy (SMA), a genetic neurodegenerative disease carried by roughly 1 in 40 people. SMA causes muscles to slowly weaken and degrade over time, usually starting in the legs and working toward the head and neck. Now, however, the new drug candidate from UM-Columbia appears in animal study models to improve severe SMA cases by up to 90%. After patenting the treatment, the next step is to begin experimental trials in humans.

NIH Launches “I-Corps” to Grow Drug, Diagnostic Startups

October 8, 2014

The NIH announced that 21 teams of researchers and entrepreneurs have received funding to help establish the feasibility and commercial prospects of new therapeutic drug, diagnostic, and medical device technologies. The program, called NIH Innovation Corps, aims to assist startup biotech and medical technology companies in establishing a business plan and beginning research. The entrepreneurs are counseled, for example, in areas such as how to define clinical need before spending millions in testing and how to assess and react to intellectual property and regulatory risks prior to pushing ahead with research and development.

Amgen, Sanofi, Ono Link with Academia on Proteins Project

October 28, 2014

Amgen, Sanofi, and Ono Pharmaceutical have teamed up to enter a research collaboration with three academic centers of protein study in an attempt to better understand G-protein coupled receptors (GPCRs). GPCR research is a rapidly growing area of interest, as work in this field received the Nobel Prize for chemistry in 2012. GPCRs control how agents pass through a cell’s membrane; wider understanding of how they work could lead to new drug candidates and ways to treat a variety of diseases.

Fujifilm Says Test Results on Avigan as Ebola Drug by End-2014

November 11, 2014

Reuters reports Japanese drugmaker Toyoma Chemical Co., a division of the Fujifilm conglomerate, has enough doses of its Avigan flu/Ebola drug to treat 20,000 patients and could make up to 300,000 more doses, if necessary. Avigan was approved for use in Japan to treat novel flus, but it has already been used in limited cases in Europe to treat Ebola virus patients. There is also work underway to test the drug in Guinea in 200 mg doses to gauge its effectiveness, and success could lead to wider approval and use of the drug for Ebola treatment yet this year.

Drug Can Repair Spinal Cord Injuries, Study Shows

December 4, 2014

The BBC reports that new research from Case Western Reserve University School of Medicine scientists, appearing in the journal Nature, could lead to compounding breakthroughs in the quest to repair spinal cord injuries and resulting paralysis. Typically, proteins build up in scar tissue at the point of injury in damaged spinal cords and prevent nerve reattachment. In a study on rats, however, using a new drug designed to break the protein buildup, 21 of 26 recovered either some movement or bladder control. With further study, this new treatment could become a powerful tool in repairing spinal cord injury.

Promising Compound Rapidly Eliminates Malaria Parasite

December 8, 2014

St. Jude Children’s Research Hospital scientists, releasing data in the Proceedings of the National Academy of Sciences, report that a new drug compound currently in testing can essentially eliminate malaria in 48 hours. In a mouse study, a single dose of the compound was found to destroy 80% of malaria parasites in 24 hours. At 48 hours, traces of malaria were undetectable. This breakthrough new treatment works by “tricking” the immune system to quickly destroy red blood cells associated with malaria infection while leaving healthy cells unharmed.

Pfizer Enhances Focus on Rare Bleeding Disorder, Cancer Immunotherapeutics

December 9, 2014

Pfizer announced it has entered a collaboration agreement with Spark Therapeutics for gene therapy and hemophilia B research. Spark will focus on clinical development of drug candidates and Pfizer will manage regulatory procedures and assist with commercialization should a drug candidate prove successful. Pfizer also has begun a cancer immunosuppression research program with iTeos Therapeutics and has licensed compounds for research, which could be used to help develop drugs that direct the immune system to shut down cancers.

First Step Toward Pill for Obesity Taken, Researchers Report

December 9, 2014

Scientists at Harvard Stem Cell Institute, writing in the journal Nature Cell Biology, report the discovery of two compounds that could help the body turn white, adipose-fat tissue into brown, adipose-fat tissue. These compounds, the researchers reveal, could prove useful in fighting obesity because more brown, fat-burning tissue would help eliminate what would otherwise become white, fat-storing tissue. With further testing and development, this work could become a powerful new tool in the fight against obesity.

Small Molecule Successfully Targets Telomerase to Destroy Cancer Cells

January 5, 2015

Scientists at UT Southwestern Medical Center, writing in the journal Cancer Discovery, report new advances in targeting telomerase with small molecule drugs to destroy cancer cells and slow down tumor growth. In a mouse study, the researchers found that the 6-thiodG molecule slowed the growth of cancer tumors by signaling to the cancer cell via the telomere that damage had taken place to the cell itself, causing it to stop dividing and eventually die. With further research, this new method of telomerase targeting could prove useful in new cancer treatments.

Predicting Superbugs’ Countermoves to New Drugs

January 5, 2015

Duke University researchers, writing in PNAS, discuss the creation of software that can predict changes to a bacterium prior to it being treated with existing or new drugs. This new technology could allow drug development, which stays one step ahead of infectious bacteria’s ability to mutate itself out of being treatable with available drugs. The researchers also suggest it could become possible to coax a mutation away from one drug and right into another, which could prove useful in defeating the growing problem of antibiotic resistance.

“Flying Carpet” Technique Uses Graphene to Deliver One-Two Punch of Anticancer Drugs

January 7, 2015

New work appearing in the journal Advanced Materials discusses the creation of a graphene film containing two anticancer drugs, which proved successful in delivering two types of drugs to tumors in a mouse model. One drug, for example, works best when attached to the outer membrane of cancer cells and was applied to the surface of the graphene film. The other, used to target the nucleus, was bound to the graphene itself. This new technique for delivering combination-therapy cancer treatment could lead to compounding breakthroughs in anticancer research.

Ebola: New Vaccine Trial Begins

January 7, 2015

The BBC reports that trials of a new Ebola virus vaccine have begun under the direction of Oxford University scientists. 72 trial subjects received the first dose of the vaccine, with a second dose scheduled for one to two months in the future. Prior tests on monkeys found the vaccine provided complete protection against one form of the Ebola virus, and the primary and follow-up dosing is designed to first prime, then reestablish immune system response in humans.

Antibiotics: US Discovery Labelled “Game Changer” for Medicine

January 8, 2015

According to new work from Northeastern University scientists appearing in the journal Nature, a new method for uncovering antibiotic candidates has produced positive results in early testing, including one new antibiotic which could be the biggest breakthrough in the industry in decades. By isolating and then growing bacteria in soil, the researchers identified 25 new antibiotics which, if successful in human testing, could help defeat the growing problem of antibiotic resistance. The last new antibiotic came into wide use in 1987, highlighting the need for new antibiotic treatments.

Roche Seals Licensing Deal for Antibiotic-Boosting Drug to Battle Superbugs

January 14, 2015

Reuters reports Roche has entered a licensing deal with Japanese and Canadian drugmakers related to the development of OP0595, a new antibiotic candidate that could make up to 65% of existing antibiotics stronger and more useful in the fight against drug-resistant “superbugs.” Reuters also notes that as many major pharma companies have departed the antibiotic space in recent years due to the lack of significant advances, Roche has remained committed via development and licensing deals like the one announced yesterday. According to the CDC, “superbug” infections strike over 2 million people in the US annually, resulting in approximately 23,000 deaths.

Asthma Drug Shows Promise in Treatment of Obesity and Diabetes

January 14, 2015

University of Michigan scientists, releasing data in Nature Communications, discuss how an asthma drug that is currently in clinical trials came to be regarded as a useful tool in the fight against metabolic disorders like obesity. In studies on mice, the researchers determined that the asthma drug increased the rate at which the mice metabolized fat while also causing fat cells to release interleukin-6. Interleukin-6 was then shown to travel to the liver where it reduced the production of glucose. If results translate to humans in the studies underway, this drug could assist in the treatment of both obesity and diabetes.

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