Author Archive for Pharmboy

What is ‘right to try,’ and could it help?

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What is 'right to try,' and could it help?

File 20180207 74506 1dsdxkd.jpg?ixlib=rb 1.1

In this March 18, 2011 photo, Cassidy Hempel waved at hospital staff as she was being treated for a rare disorder. Her mother Chris, left, fought to gain permission for an experimental drug. AP Photo/Marcio Jose Sanchez

Morten Wendelbo, Texas A&M University and Timothy Callaghan, Texas A&M University

After a year in which President Donald Trump devoted much of his health policy attention to the repeal and replace of the Affordable Care Act, Trump used part of his recent State of the Union address to press Congress to focus attention in 2018 on a new health priority – the passage of “right to try” legislation.

Right to try legislation gives terminally ill patients the right to use experimental medications that have not yet been approved by the Food and Drug Administration. In 2017, the U.S. Senate unanimously passed HR 878, legislation that would do just that and the president’s address has pressured the U.S. House to follow suit, where a bill is now being debated in committee.

President Trump’s push for the passage of right to try nationally builds on the efforts of the Libertarian-leaning Goldwater Institute, which has used the broad popularity of the policy to help achieve passage of similar legislation in 38 states, even though it diverges with current federal regulations.

Despite these state gains and the policy’s growing popularity among states, ethical questions remain about the tangible impact of a federal right to try law on Americans with terminal illnesses. Most notably, a growing body of evidence from policy analysts argues that the legislation would unfortunately accomplish very little change for most patients, and it could actually make it harder to get new drugs approved in the future.

How the drug approval process works

Pharmaceuticals in the United States are regulated by the FDA. When a drug company develops a new compound intended for patient use, the medication goes through three phases of clinical trials that often take years to…
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What is CRISPR gene editing, and how does it work?

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What is CRISPR gene editing, and how does it work?

File 20180130 170442 4m9ea.png?ixlib=rb 1.1

Scientists discovered some bacteria can cut the DNA of invading viruses as a defence mechanism. They realised they could use this to cut human DNA.

Courtesy of Merlin Crossley, UNSW

You’ve probably read stories about new research using the gene editing technique CRISPR, also called CRISPR/Cas9. The scientific world is captivated by this revolutionary technology, since it is easier, cheaper and more efficient than previous strategies for modifying DNA.

The term CRISPR/Cas9 stands for Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR associated protein 9. The names reflect important features identified during its discovery, but don’t tell us much about how it works, as they were coined before anyone understood what it was.

What does CRISPR/Cas9 do?

CRISPR/Cas9 is a system found in bacteria and involved in immune defence. Bacteria use CRISPR/Cas9 to cut up the DNA of invading bacterial viruses that might otherwise kill them.

Today we’ve adapted this molecular machinery for an entirely different purpose – to change any chosen letter(s) in an organism’s DNA code.

We might want to correct a disease-causing error that was inherited or crept into our DNA when it replicated. Or, in some cases, we may want to enhance the genetic code of crops, livestock or perhaps even people.

So do we just snip the unwanted gene out and replace it with a good one?

We first have to remember that animals and plants are composed of millions of cells, and each cell contains the same DNA. There is no point editing just one cell: we would have to edit the same gene in every single cell. We’d have to snip out millions of genes and paste in millions of new ones.

And not all cells are easy to get to – how could we reach cells buried in our bones or deep within a brain?

A better approach is to start at the beginning and edit the…
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How robot math and smartphones led researchers to a drug discovery breakthrough

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How robot math and smartphones led researchers to a drug discovery breakthrough

Courtesy of Ian HaydonUniversity of Washington

File 20180112 101514 1gfowf8.jpg?ixlib=rb 1.1

Moving a robot is like manipulating a molecule. Willyam Bradberry/Shutterstock.com

Robotic movement can be awkward.

For us humans, a healthy brain handles all the minute details of bodily motion without demanding conscious attention. Not so for brainless robots – in fact, calculating robotic movement is its own scientific subfield.

My colleagues here at the University of Washington’s Institute for Protein Design have figured out how to apply an algorithm originally designed to help robots move to an entirely different problem: drug discovery. The algorithm has helped unlock a class of molecules known as peptide macrocycles, which have appealing pharmaceutical properties.

One small step, one giant leap

Roboticists who program movement conceive of it in what they call “degrees of freedom.” Take a metal arm, for instance. The elbow, wrist and knuckles are movable and thus contain degrees of freedom. The forearm, upper arm and individual sections of each finger do not. If you want to program an android to reach out and grasp an object or take a calculated step, you need to know what its degrees of freedom are and how to manipulate them.

The more degrees of freedom a limb has, the more complex its potential motions. The math required to direct even simple robotic limbs is surprisingly abstruse; Ferdinand Freudenstein, a father of the field, once called the calculations underlying the movement of a limb with seven joints “the Mount Everest of kinematics.”

Watch a robotic hand move.

Freudenstein developed his kinematics equations at the dawn of the computer era in the 1950s. Since then, roboticists have increasingly relied on algorithms to solve these complex kinematic puzzles. One algorithm in particular – known as “generalized kinematic closure” – bested the seven joint problem, allowing roboticists to program fine control into…
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How Alzheimer’s disease spreads throughout the brain – new study

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How Alzheimer's disease spreads throughout the brain – new study

Courtesy of Thomas E CopeUniversity of Cambridge

File 20180105 26154 13d7znc.png?ixlib=rb 1.1

Harmful tau protein spreads through networks. Author provided

Alzheimer’s disease is a devastating brain illness that affects an estimated 47m people worldwide. It is the most common cause of dementia in the Western world. Despite this, there are currently no treatments that are effective in curing Alzheimer’s disease or preventing its relentless progression.

Alzheimer’s disease is caused by the build-up of two abnormal proteins, beta-amyloid and tau. Tau is particularly important because it causes neurons and their connections to die, preventing brain regions from communicating with each other normally. In the majority of cases, tau pathology first appears in the memory centres of the brain, known as the entorhinal cortex and hippocampal formation. This has been shown to occur many years before patients have any symptoms of disease.

Over time, tau begins to appear in increasing quantities throughout the brain. This causes the characteristic progression of symptoms in Alzheimer’s diseases, where initial memory loss is followed by more widespread changes in thinking and behaviour that lead to a loss of independence. How this occurs has been controversial.

Transneuronal spread

In our study, published in Brain, we provide the first evidence from humans that tau spreads between connected neurons. This is an important step, because stopping this spread at an early stage might prevent or freeze the symptoms of Alzheimer’s disease.

This idea, called “transneuronal spread”, has been proposed before and is supported by studies in mice. If abnormal tau is injected into a healthy mouse brain, it quickly spreads and causes the mice to manifest dementia symptoms. However, it had not previously been shown that this same process occurs in humans. The evidence from mouse studies was controversial, as the amount of tau injected was relatively high, and disease progression occurred much more rapidly than it does in humans.

Artist’s impression of tau spreading between connected neurons. Author provided


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Designer proteins that package genetic material could help deliver gene therapy

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Designer proteins that package genetic material could help deliver gene therapy

Courtesy of Ian HaydonUniversity of Washington

File 20171205 31063 15cffwi.jpg?ixlib=rb 1.1

Delivering genetic material is a key challenge in gene therapy. Invitation image created by Kstudio, CC BY

If you’ve ever bought a new iPhone, you’ve experienced good packaging.

The way the lid slowly separates from the box. The pull tab that helps you remove the device. Even the texture of the paper inserts matters to Apple. Every aspect of iPhone packaging has been meticulously designed for a pleasing aesthetic experience.

When it comes to genome editing, good packaging is even more crucial.

In a recent article in the journal Nature, a team of bioengineers here at the University of Washington describe a new type of packaging built to protect genetic material, specifically RNA. This designer packaging consists of proteins which self-assemble into soccer ball-like nanostructures known as capsids. These tiny particles encapsulate RNA, allowing it to move around the bodies of mice for hours without being degraded — sidestepping one of the biggest challenges to successful gene editing.

Delivering genetic material

Moving genetic material (DNA or RNA) throughout the body – or targeting it into specific organs and tissues – is a key challenge in human genome editing. In addition to technology like CRISPR, which physically cuts DNA, some potentially lifesaving gene therapies will require the insertion of new genetic elements to serve as templates for repair. But these genetic blueprints face perilous conditions once they enter the body.

Because deadly infections often start when unwanted genetic material from a pathogen makes it into our cells, our bodies have evolved sophisticated ways of quickly detecting and demolishing foreign DNA and RNA molecules. Simply put: Unprotected genetic material doesn’t stick around for very long. In fact, CRISPR itself evolved in bacteria to perform precisely this search-and-destroy function before it was co-opted by scientists as a gene-editing tool.

Biotechnologists have known about this delivery problem for some time. Most researchers have turned to what might sound like…
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DNA has gone digital – what could possibly go wrong?

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DNA has gone digital – what could possibly go wrong?

Courtesy of Jenna E. GallegosColorado State University and Jean PeccoudColorado State University

File 20171207 25358 14upyz5.jpg?ixlib=rb 1.1

Modern advances come with new liabilities. Sergey Nivens/Shutterstock.com

Biology is becoming increasingly digitized. Researchers like us use computers to analyze DNA, operate lab equipment and store genetic information. But new capabilities also mean new risks – and biologists remain largely unaware of the potential vulnerabilities that come with digitizing biotechnology.

The emerging field of cyberbiosecurity explores the whole new category of risks that come with the increased use of computers in the life sciences.

University scientists, industry stakeholders and government agents have begun gathering to discuss these threats. We’ve even hosted FBI agents from the Weapons of Mass Destruction Directorate here at Colorado State University and previously at Virginia Tech for crash courses on synthetic biology and the associated cyberbiosecurity risks. A year ago, we participated in a U.S. Department of Defense-funded project to assess the security of biotechnology infrastructures. The results are classified, but we disclose some of the lessons learned in our new Trends in Biotechnology paper.

Along with co-authors from Virginia Tech and the University of Nebraska-Lincoln, we discuss two major kinds of threats: sabotaging the machines biologists rely on and creating dangerous biological materials.

Computer viruses affecting the physical world

In 2010, a nuclear plant in Iran experienced mysterious equipment failures. Months later, a security firm was called in to troubleshoot an apparently unrelated problem. They found a malicious computer virus. The virus, called Stuxnet, was telling the equipment to vibrate. The malfunction shut down a third of the plant’s equipment, stunting development of the Iranian nuclear program.

Unlike most viruses, Stuxnet didn’t target only computers. It attacked equipment controlled by computers.

The marriage of computer science and biology has opened the door for amazing discoveries. With the help of computers, we’re decoding the human genome, creating organisms with new capabilities, automating drug development and revolutionizing …
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The two obstacles that are holding back Alzheimer’s research

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The two obstacles that are holding back Alzheimer's research

Courtesy of Todd GoldeUniversity of Florida

File 20171115 19772 2rishs.jpg?ixlib=rb 1.1

Family members often become primary caregivers for loved ones with Alzheimer’s disease. tonkid/Shutterstock.com

Thirty years ago, scientists began to unlock the mysteries regarding the cause of Alzheimer’s disease. This knowledge ushered in an era of great enthusiasm that scientists could develop new therapies to either prevent Alzheimer’s or significantly slow the symptoms once present.

Despite continued progress and renewed hope that some therapies now in human trials will modify the course of the disease, the initial optimism of neuroscientists like me has been significantly tempered by reality. Numerous therapies, most with sound scientific basis, have been tested and shown to be ineffective in humans with symptomatic Alzheimer’s disease.

Like the war on cancer, the war on Alzheimer’s disease is not going to be won in a single glorious “battle.” Instead, I believe incremental yet transformative progress will eventually lead to success. Unlike cancer, the scientific community does not yet have any “survivor stories” to buoy our efforts, and it will take a concerted effort by scientists, pharmaceutical companies, government and society to bring about the reality of ending Alzheimer’s disease. Only by recognizing and confronting all of the obstacles impeding development of Alzheimer’s therapies can we be confident that our battle will be successful.

As a physician-scientist and director of the University of Florida’s McKnight Brain Institute who began studying Alzheimer’s disease in medical school in the late 1980s, I appreciate the scope of the scientific advances we have collectively made. I have also come to the sobering realization that translating these advances into real therapies that will make a difference for patients suffering from this devastating disease is an incredibly complex issue which is not all about the science.

There are two significant, nonscientific obstacles – a shortage of funding and patent law – that will require concerted effort by scientists, concerned citizens, society and our lawmakers to overcome.

Funding is improving, but still lagging

Governments of industrialized nations have recognized research funding for…
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Immunotherapy: Training the body to fight cancer

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Immunotherapy: Training the body to fight cancer

Courtesy of Balveen KaurThe Ohio State University and Pravin KaumayaThe Ohio State University

Image 20170202 1665 nzadz6.jpg?ixlib=rb 1.1

An oral squamous cancer cell (white) being attacked by two T cells (red), part of a natural immune response. NIH Image Gallery, CC BY-NC

The human immune system is powerful and complex.

It can identify and destroy invaders of nearly infinite variety, yet spare the more than 30 trillion cells of the healthy body.

Unfortunately, the broken cells of cancer are able to retain, and boost, the “recognize and ignore me” signals of undamaged cells, letting them evade detection by the immune system. As a result, these damaged cells grow unmolested, destroying the normal physiological functioning of tissues and organs.

Armed with new insights into the interactions between cancer and the immune system, research teams are developing novel treatments to harness the full potential of the body’s natural defenses. This is called immunotherapy.

In animal models and clinical trials, breakthrough immunotherapies are emerging, techniques that train the immune system to recognize and attack cancer as the enemy.

One way is through drugs that help the immune system find and destroy cancer cells. Another way is through vaccines that can teach the body to recognize cancer cells.

Recently, studies have paired immunotherapies with modified viruses that attack tumor cells and keep them from returning.

With promising results, such new weapons are providing hope that cancer can ultimately be defeated.

Harnessing the immune system

When foreign cells – like viruses or bacteria – infect the body, the immune system springs into action. It produces antibodies that bind to proteins called antigens on the surface of the foreign cell. Sometimes this is enough to neutralize the foreign cell. In other cases the antibodies bind to the antigen and mark the cell for destruction by T-cells, or both.

Cancer cells also produce antigens. But even though cancer cells are not normal and would otherwise be marked for destruction, antibodies don’t bind to their antigens and the immune system does not destroy…
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Circadian rhythm Nobel: what they discovered and why it matters

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Circadian rhythm Nobel: what they discovered and why it matters

Courtesy of Sally Ferguson, CQUniversity Australia

Today, the “beautiful mechanism” of the body clock, and the group of cells in our brain where it all happens, have shot to prominence. The 2017 Nobel Prize in Physiology or Medicine has been awarded to Jeffrey C. Hall, Michael Rosbash and Michael W. Young for their work on describing the molecular cogs and wheels inside our biological clock.

In the 18th century an astronomer by the name of Jean Jacques d'Ortuous de Marian noted his plants opening and closing their leaves with the cycle of light and dark, with the leaves opening towards the sun. Being an inquisitive chap, he placed the plants in constant darkness and observed that the daily opening and closing of the leaves continued even in the absence of sunlight – indicative of an internal clock.

Subsequent work by others also showed innate daily rhythms in other animals and plants, but the location and inner workings of the biological timing system remained a mystery.

The discovery of a misfiring gene that resulted in disrupted daily rhythms in fruit flies (the unsung heroes of the story) gave the first hint. Over several years, Hall, Rosbash and Young uncovered the machinery of the biological clock.

It’s in your genes.

From the latin circa “about” and diem “a day”, circadian rhythms are internally driven cycles in all living things – including humans – that continue in the absence of external time cues. The sleep/wake cycle is one daily rhythm; core body temperature is another. While we have known since de Marian that physiological systems are controlled internally, the way in which the clock works was a mystery.

The biological clock’s cycle is generated by a feedback loop. Genes are activated which trigger the production of proteins. When protein levels build up to a critical…
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Life frozen in time under an electron microscope gets a Nobel Prize

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Life frozen in time under an electron microscope gets a Nobel Prize

Courtesy of Xavier ConlanDeakin University

File 20171005 21985 15evuaj

The electron microscope’s resolution has radically improved in the last few years, from mostly showing shapeless blobs (left) in 2013 to now being able to visualise proteins at atomic resolution (right) in the present. Martin Ho?gbom/The Royal Swedish Academy of Sciences

The scientists who developed the ability to see some of the building blocks of life under the electron microscope have been awarded the 2017 Nobel Prize in Chemistry.

Jacques Dubochet, Joachim Frank and Richard Henderson pioneered cryo-electron microscopy, which the Royal Swedish Academy of Sciences said both simplifies and improves the imaging of biological molecules, known as biomolecules.

The 9 million Swedish kronor (A$1.4 million) prize is split equally between Dubochet, at Switzerland’s University of Lausanne, Frank, at New York’s Columbia University, and Henderson, at the MRC Laboratory of Molecular Biology, Cambridge in the UK.

The Academy said the method developed by the three researchers had moved biochemistry into a new era. The technology now allows researchers to generate a high resolution view of biomolecules while they exist in their natural state.

The biological lock and key

The human body is amazingly complex and requires the cooperation of a range of biochemical mechanisms, such as digestion and energy production, in order to function well. These intricate processes involve the use of biomolecules, typically large entities made from amino acids – the building blocks of life.

Importantly, just like the construction of any brick-built house, the configuration or placement of the blocks is critical to how well our construction stands up, or how well our biomolecules function.

Furthermore, biomolecules present their capacity to perform tasks by interacting with other entities, such enzymes, in the body. These are based on a specific configuration, much like how only one key can open a particular lock.

The significant challenge overcome by the award-winning team was to develop the capacity to observe the biomolecules…
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Phil's Favorites

Why Altered Carbon is not about the future - nor is any other science fiction

 

Why Altered Carbon is not about the future – nor is any other science fiction

Netflix

Courtesy of Gavin Miller, University of Glasgow

The hopes and dreams of the technological movement known as “transhumanism” have been brought into the media spotlight thanks to Netflix’s new science fiction series, Altered Carbon (based on Richard Morgan’s 2001 novel).

Transhumanists believe that our species will soon undergo a technological evolution into a new and superior form. While ...



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Zero Hedge

North Korea Bailed At The Last Minute On Secret Meeting With Pence

Courtesy of Zero Hedge

Two weeks ago, followers of geopolitics couldn't help but speculate about the chances of a clandestine meeting between North Korea and the US when the news first broke that North Korean leader Kim Jong Un's younger sister, Kim Jo Yang, would be attending the Winter Games in PyeongChang.

After all, US Vice President Mike Pence was already confirmed to be stopping by South Korea during the beginning of the Games as part of a five-day Asia tour. But the White House was quick to repudiate this chatter, announcing that there were no plans for diplomatic talks, though both US and North Korean rhetoric since then has left the door open for such a meeting.

But as it turns out, just as the White House was denying it, plans for talks were being set in motion...



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Chart School

Sellers Come In But Semiconductors Gain

Courtesy of Declan.

Markets were set up for sellers with most indices experiencing broad selling. However, the one index which looked set up best for shorts - the Semiconductor Index - actually managed to gain.  Anyone taking up Friday's short in the latter Index will have been stopped out but another shorting opportunity may have presented itself. Technicals haven't returned to becoming net bullish but only the ADX remains to shift.


The S&P eased a little lower but didn't return below what was channel support. Te...

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ValueWalk

Bill Nygren's Stock Picks

By VW Staff. Originally published at ValueWalk.

Bill Nygren, Harris Associates U.S. equities CIO and Oakmark Funds portfolio manager, shares his top stock picks and long-term investment strategy.

H/T Dataroma

]]> Get The Full Seth Klarman Series in PDF

Get the entire 10-part series on Seth Klarman in PDF. Save it to your desktop, read it on your tablet, or email to your colleagues.

Bill Nygren's Stock Picks

Pro: Three hot stocks to watch from ...



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Insider Scoop

Stifel Sees Reboot Opportunity For Chipotle, Upgrades From Sell To Hold

Courtesy of Benzinga.

Related CMG Benzinga's Top Upgrades, Downgrades For February 20, 2018 The Market In 5 Minutes: Albertsons-R...

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Digital Currencies

As Bitcoin Nears $11,000, Here's A History Of Its Biggest Ups And Downs

Courtesy of ZeroHedge. View original post here.

The cryptocurrency rebound off Feb 5th's bloodbath lows (below $6,000 for Bitcoin) has been impressive, as a 'mysterious' massive buyer 'bought the dip' and momentum took care of the rest.

With Bitcoin now nearing $11,000 (almost a double off the lows), ...



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Biotech

What is 'right to try,' and could it help?

Reminder: Pharmboy is available to chat with Members, comments are found below each post.

 

What is 'right to try,' and could it help?

In this March 18, 2011 photo, Cassidy Hempel waved at hospital staff as she was being treated for a rare disorder. Her mother Chris, left, fought to gain permission for an experimental drug. AP Photo/Marcio Jose Sanchez

Morten Wendelbo, Texas A&M University and Timothy Callaghan, ...



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Mapping The Market

The tricks propagandists use to beat science

Via Jean-Luc

How propagandist beat science – they did it for the tobacco industry and now it's in favor of the energy companies:

The tricks propagandists use to beat science

The original tobacco strategy involved several lines of attack. One of these was to fund research that supported the industry and then publish only the results that fit the required narrative. “For instance, in 1954 the TIRC distributed a pamphlet entitled ‘A Scientific Perspective on the Cigarette Controversy’ to nearly 200,000 doctors, journalists, and policy-makers, in which they emphasized favorable research and questioned results supporting the contrary view,” say Weatherall and co, who call this approach biased production.

A second approach promoted independent research that happened to support ...



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Members' Corner

An Interview with David Brin

Our guest David Brin is an astrophysicist, technology consultant, and best-selling author who speaks, writes, and advises on a range of topics including national defense, creativity, and space exploration. He is also a well-known and influential futurist (one of four “World's Best Futurists,” according to The Urban Developer), and it is his ideas on the future, specifically the future of civilization, that I hope to learn about here.   

Ilene: David, you base many of your predictions of the future on a theory of historica...



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OpTrader

Swing trading portfolio - week of September 11th, 2017

Reminder: OpTrader is available to chat with Members, comments are found below each post.

 

This post is for all our live virtual trade ideas and daily comments. Please click on "comments" below to follow our live discussion. All of our current  trades are listed in the spreadsheet below, with entry price (1/2 in and All in), and exit prices (1/3 out, 2/3 out, and All out).

We also indicate our stop, which is most of the time the "5 day moving average". All trades, unless indicated, are front-month ATM options. 

Please feel free to participate in the discussion and ask any questions you might have about this virtual portfolio, by clicking on the "comments" link right below.

To learn more about the swing trading virtual portfolio (strategy, performance, FAQ, etc.), please click here ...



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Promotions

NewsWare: Watch Today's Webinar!

 

We have a great guest at today's webinar!

Bill Olsen from NewsWare will be giving us a fun and lively demonstration of the advantages that real-time news provides. NewsWare is a market intelligence tool for news. In today's data driven markets, it is truly beneficial to have a tool that delivers access to the professional sources where you can obtain the facts in real time.

Join our webinar, free, it's open to all. 

Just click here at 1 pm est and join in!

[For more information on NewsWare, click here. For a list of prices: NewsWar...



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Kimble Charting Solutions

Brazil; Waterfall in prices starting? Impact U.S.?

Courtesy of Chris Kimble.

Below looks at the Brazil ETF (EWZ) over the last decade. The rally over the past year has it facing a critical level, from a Power of the Pattern perspective.

CLICK ON CHART TO ENLARGE

EWZ is facing dual resistance at (1), while in a 9-year down trend of lower highs and lower lows. The counter trend rally over the past 17-months has it testing key falling resistance. Did the counter trend reflation rally just end at dual resistance???

If EWZ b...



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All About Trends

Mid-Day Update

Reminder: Harlan is available to chat with Members, comments are found below each post.

Click here for the full report.




To learn more, sign up for David's free newsletter and receive the free report from All About Trends - "How To Outperform 90% Of Wall Street With Just $500 A Week." Tell David PSW sent you. - Ilene...

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About Phil:

Philip R. Davis is a founder Phil's Stock World, a stock and options trading site that teaches the art of options trading to newcomers and devises advanced strategies for expert traders...

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Ilene is editor and affiliate program coordinator for PSW. She manages the site market shadows, archives, more. Contact Ilene to learn about our affiliate and content sharing programs.

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