Drug Development
Articles
Ex-cell-ent Research Partners
Cells are the basic structural and functional unit of all organisms. Without them, drug development would be stuck in the bad old days before antibiotics. Researchers need squillions of these membrane-bound bundles of molecules to develop new medicines and to make...
Exosomes: Tiny Vesicles With Big Potential
Exosomes: Bio-trash to Bio-treasure We’ve all heard the phrase “One man’s trash is another man’s treasure.” It turns out that what often holds true for the antique business sometimes holds true for biotech as well. This WEEKLY shows how. Term of the Week: Exosome...
Turning On Cellular Garbage Disposals
Proteasomes to the Rescue Many drugs work by stopping overactive proteins that cause disease. The leukemia drug Gleevec, for example, is a small-molecule inhibitor (antagonist) of the protein Bcr-Abl, whose overactivity promotes excessive cell division. Humira...
From Drug Discovery To Approval: Phase IV
Pharma Finish Line: FDA Approval Last week, we focused on the final stage of clinical testing, Phase III trials, where drug developers assess the safety and efficacy of their drug in large patient groups. At the end of Phase III, drug developers face the moment of...
From Drug Discovery To Approval: Phase III
Phase III Is No Guarantee Our last Biotech Primer WEEKLY explored the riskiest part of the human clinical trials pathway: Phase II. About 70% of drugs that enter Phase II never make it out. Most often, it’s because they fail to demonstrate effectiveness. Even making...
From Drug Discovery To Approval: Phase I/II
Phase I and II Clinical Trials Every drug in clinical use today, from the latest CAR-T treatment to older cholesterol-lowering statins, share one thing in common: they have all successfully navigated the rigorous clinical trials process. This is no small feat, as only...
Drug Discovery 301
DRUG DISCOVERY 301 The past two issues, the WEEKLY explored the first two stages of drug discovery. We looked at how pharmaceutical companies identify drug targets, or the molecules (usually proteins) involved in an illness that an ‘as yet undeveloped drug’ will...
Drug Discovery 201
WE WANNA NEW DRUG “One that won’t make me sick/ One that won’t make me crash my car/ and make me feel three feet thick…” Huey Lewis is singing about love, but he voices very human concerns when it comes to the medicines that heal bodies and minds. Last week, the...
Drug Discovery 101
On the Road to New Medicines For most of the 20th century, we discovered new drugs by trial and error. Scientists investigated countless unrelated compounds in animals to see which improved disease symptoms. For instance, in the 1950s and 60s, British scientists at...
The Tight Junction’s Function
A Pathway Lies In The Bridge Between Cells As any high school biology teacher will happily remind us, cells are the basic building blocks of all living things: you, the office ficus tree, the yeast that bubbles your beer. In animals, cells join to form tissues and...
Viruses To The Rescue?
Viruses to the Rescue? Last week, a paper in Nature Medicine described a British teenager whom doctors pulled back from the brink of death. The young woman had developed a deadly, antibiotic-resistant infection following lung surgery. The treatment? Phage therapy, in...
Circadian Rhythm & Disease
AND THE BEAT GOES ON Earlier this week, most areas of North America and Europe observed the annual ritual of “springing forward.” People started setting their clocks ahead one hour in the U.S. in 1918. The federal government aimed to save electricity and increase...
The Gut-Brain Axis
Decoding The Gut-Brain Axis Earlier this week, a newly published study made headlines for showing a direct link between the gut microbiome and depression. For the past several years, scientists have suspected a link between bugs in the gut and neurological health....
MICROBIOME ADVENTURES, PART 2
The most recent Biotech Primer WEEKLY looked at a particularly productive new area of medicine courtesy of our own tiny personal zoo: the microbiome. We considered how resetting a person’s microbiome with a fecal matter transplant (FMT) aka bacteriotherapy can heal such troubling conditions as antibiotic-resistant C. difficile infections and inflammatory bowel disease (IBD). Thankfully for the millions suffering from these health conditions, acceptance of FMTs is growing. Now the research community is turning its attention to the specific components behind the microbiome’s healing potential. Several biotech companies are already developing more precisely-defined therapeutics. This work will foster better quality control and more comfortable formulations. Who wouldn’t prefer swallowing a capsule to undergoing an enema or colonoscopy?
The Microbiome Magnified
You'll Never Walk Alone Ready for a fascinating, “earthy” truth about ourselves? Our every surface and crevice teems with microbes—inside and out. In fact, bacterial cells in and on our body outnumber human cells by about ten to one. Most people walk around completely...
Autophagy: The Incredible, Edible Cell?
Autophagy: The Incredible, Edible Cell? True, weird fact: our cells are cannibals. Right now, these itty-bitty sacs of vital fluids inside your body are eating themselves. This phenomenon is called autophagy, or “self-eating.” Autophagy Is Awesome This seemingly odd...
Hacking The Fountain Of Youth
Hacking The Fountain Of Youth Last week, we began to delve into longevity research — the area of biotech which seeks to understand the biomolecular changes that occur with aging and possibly underlie many of the dubious gifts of old age: heart disease, Alzheimer’s and...
Fountains of Healthy Old Age?
Fountains Of Healthy Old Age? Plenty of famous people have uttered (or supposedly uttered) lots of pithy comments about aging. A couple gems: “Age is mind over matter; if you don’t mind, it don’t matter,” said Satchel Paige. “We are always the same age inside,”...
Epigenetics: Writing, Reading, & Erasing
Foundations Of Epigenetics Genetic mutations — changes in the order of the A, C, G, and T nucleotide bases that make up a gene — have been the primary focus of cancer researchers over the last several decades. By sussing out mutations involved in regulating cell...
Swallowing A Biologic Drug?
Swallowing A Biologic Drug? Over the past two decades, biologic drugs — drugs composed of proteins produced by living cells — have become the safest, most effective top sellers within the pharmaceutical industry. Approved to treat a variety of diseases including...
Biotech In Space!
Experimenting In Lower Earth's Orbit Drug discovery in space? If this sounds like a page from science fiction, think again. Leading pharmaceutical companies are now collaborating with NASA to run experiments on the International Space Station (ISS). The focus on bone...
Red Blood Cells: Ready For Double-Duty?
RED BLOOD CELLS: READY FOR DOUBLE-DUTY? Biotech Primer WEEKLY talks a lot about white blood cells, with good reason. These powerful immune cells defend us against pathogens and have recently been adapted to fight cancer as CAR-T cells. What about the body’s...
$8.7 Billion SMA Drug Explained
The Science Behind the Deal Earlier this week, news of the $8.7 billion acquisition of gene therapy company AveXis (Bannockburn, IL) by Novartis (Basel, Switzerland) made big biotech headlines. AveXis’ lead candidate, AVXS-101, is now in Phase III clinical...
From Drug Development to Approval: A Recap
From The Lab To The Patient In this issue of the Biotech Primer WEEKLY we will recap the past seven issues that highlight the journey a molecule takes from the lab to the patient. Beginning in the 1980's, scientists took a new tack in developing drugs. They adopted an...
Market Access
Market Access Primer For the last few weeks we here at Biotech Primer have tracked the progression of a drug candidate from the lab to the marketplace, where only the fittest survive. Winning at clinical trials means earning an official regulatory approval....
Plants That Heal
Nature’s Medicine Cabinet Where does medicine come from? Before it gets to your medicine chest? Before you purchase it from your neighborhood drugstore? Next time you’re hiking through a forest or gazing at your pretty screensaver of the Olympic Peninsula, think of...
Chaperoning The Rare Disease Dance
PROPERLY FOLDING MISFOLDED DISEASE PROTEINS Amicus Therapeutics (Cranbury, NJ) found itself in the news earlier this month when the FDA agreed to review the company’s new drug application for their investigational therapy to treat Fabry’s disease. The drug under...
Biotech In Space!
Experimenting In Lower Earth's Orbit Drug discovery in space? If this sounds like a page from science fiction, think again. Leading pharmaceutical companies are now collaborating with NASA to run experiments on the International Space Station (ISS). The focus on bone...
100 Drug Development Acronyms
ADME: Absorption, Distribution, Metabolism, Excretion AE: Adverse Event ANDA: Abbreviated New Drug Application API: Active Pharmaceutical Ingredient ATMPs: Advanced Therapy Medicinal Products BLA: Biologic License Application BsUFA: Biosimilar User Fee Act CAT:...
The Medicine Machine
Modern Technology & Mother Nature For thousands of years, nature has been the best medicine cabinet around. Natural products are drugs derived from nature, typically plants or microbes, and have been especially useful in fighting cancer and infectious disease....
Orphan Drugs Lead 2015 Approvals
Treatment Options Broaden Driven by 19 new orphan approvals, a whopping 45 novel drugs were cleared for the marketplace in 2015. Additionally, new approvals opened up treatment options for Americans with diseases ranging from breast and lung cancers to irritable bowel...
Natural Products Drug Discovery: A Nobel-Winning Journey
Drug Discovery Via Natural Products Half of the 2015 Nobel Prize in Medicine went to Satoshi Omura and William C. Campbell for their work leading to the discovery of the drug avermectin, used in the treatment of parasitic diseases. The other half went to Youyou Tu for...
Inching Across The Blood-Brain Barrier
A Constant Hurdle in Drug Delivery When it comes to achieving success in drug development, picking the right drug target and developing an effective inhibitor (or activator) is only half the battle. A drug candidate may appear promising in cell-based testing—and even...
The Science Behind The Blockbuster Drugs Of Tomorrow
Billion Dollar Mechanisms Of Action The Most Promising Drugs of 2015—a Thomson Reuters Cortellis Competitive Intelligence report—includes several new drugs with predicted sales of $1 billion plus by 2019. Those on the list are well on their way to winning or have...
Interchangability Denied To First US Approved Biosimilar
G-CSF: The original Innovator Sandoz's (Princeton, NJ) Zarzio rode the first wave into the U.S. biosimilars market after it received FDA approval last week. Zarzio is similar to Amgen's (Thousand Oaks, CA) Neupogen and both of these medications are recombinant...
Biosimilars: Ready or Not, Here They Come
Last week, the FDA's Oncologic Drugs Advisory Committee unanimously recommended Novartis' biosimilar version of Amgen’s megahit Neupogen (filgrastim), effectively paving the way for the first US biosimilar approval in the near future. Filgrastim is a recombinant...
First-in-Class: 2014 Drug Approvals
The FDA ushered in six new drugs just before the close of 2014, bringing the final tally to 41– the highest rate since 1996, when the agency approved 53 drugs. 2014 drug approvals were notable not only for their quantity, but also their quality. A number of new drugs...
Inhaling Insulin Is A Breath Of Fresh Air
INSULIN INHALED Injections are a part of life for most diabetics. Type 1 patients inject insulin into themselves multiple times per day, and as many as 40% of type 2 diabetics require at least one daily injection. Thanks to the FDA's approval of MannKind Corporation’s...
Mitochondrial Medicine Going Mainstream?
MORE THAN "JUST" A POWERHOUSE When we hear the word mitochondria, most of us remember it described in high school biology class as the “powerhouse” of a cell. It is an apt moniker, and not just because mitochondria are the subcellular compartments that convert glucose...
Alphabet Soup: Drug Development Acronyms
ALPHABET SOUP: NEW DRUG APPROVALS Acronyms abound in the world of new drug approvals. Check out our cheat sheet below for help with some of the most common: BLA: Biologic Licensing Application Planning on developing a biologic drug? Would you like to bring it to...