Tag: drugs

Bioethics Blogs

Research Ethics Roundup: New England Journal of Medicine Editor Wants Changes in Research Culture, Why Lab Animals Aren’t Simply “Human Stand-Ins,” Proposal for National Research Integrity Board, and FDA Approves Drugs Faster than European Medicines Agency

This week’s Research Ethics Roundup examines the New England Journal of Medicine’s (NEJM) editor-in-chief’s call for a new approach to sharing clinical trial data, how experts are trying to improve drug testing success rates by reviewing pre-clinical research assumptions, a National Academies committee’s call for a nongovernmental body to promote research integrity, and a new study that rebuts critics’ claims that the Food and Drug Administration (FDA) is slow to approve new drugs for the American market.

The post Research Ethics Roundup: New England Journal of Medicine Editor Wants Changes in Research Culture, Why Lab Animals Aren’t Simply “Human Stand-Ins,” Proposal for National Research Integrity Board, and FDA Approves Drugs Faster than European Medicines Agency appeared first on Ampersand.

Source: Ampersand, the blog of PRIM&R.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Research Ethics Roundup: New England Journal of Medicine Editor Wants Changes in Research Culture, Why Lab Animals Aren’t Simply “Human Stand-Ins,” Proposal for National Research Integrity Board, and FDA Approves Drugs Faster than European Medicines Agency

This week’s Research Ethics Roundup examines the New England Journal of Medicine’s (NEJM) editor-in-chief’s call for a new approach to sharing clinical trial data, how experts are trying to improve drug testing success rates by reviewing pre-clinical research assumptions, a National Academies committee’s call for a nongovernmental body to promote research integrity, and a new study that rebuts critics’ claims that the Food and Drug Administration (FDA) is slow to approve new drugs for the American market.

The post Research Ethics Roundup: New England Journal of Medicine Editor Wants Changes in Research Culture, Why Lab Animals Aren’t Simply “Human Stand-Ins,” Proposal for National Research Integrity Board, and FDA Approves Drugs Faster than European Medicines Agency appeared first on Ampersand.

Source: Ampersand, the blog of PRIM&R.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Missing Genes Point to Possible Drug Targets

Every person’s genetic blueprint, or genome, is unique because of variations that occasionally occur in our DNA sequences. Most of those are passed on to us from our parents. But not all variations are inherited—each of us carries 60 to 100 “new mutations” that happened for the first time in us. Some of those variations can knock out the function of a gene in ways that lead to disease or other serious health problems, particularly in people unlucky enough to have two malfunctioning copies of the same gene. Recently, scientists have begun to identify rare individuals who have loss-of-function variations that actually seem to improve their health—extraordinary discoveries that may help us understand how genes work as well as yield promising new drug targets that may benefit everyone.

In a study published in the journal Nature, a team partially funded by NIH sequenced all 18,000 protein-coding genes in more than 10,500 adults living in Pakistan [1]. After finding that more than 17 percent of the participants had at least one gene completely “knocked out,” researchers could set about analyzing what consequences—good, bad, or neutral—those loss-of-function variations had on their health and well-being.

Gene knockouts are expected to occur more frequently in certain countries, such as Pakistan, where people sometimes marry and have children with their first cousins. That makes it much more likely that a person carrying a loss-of-function gene variation will have inherited that same variation from both of their parents.

In the latest study, a team led by Sekar Kathiresan at the Broad Institute of Harvard and MIT, Boston, turned to the Pakistan Rise of Myocardial Infarction Study (PROMIS) in hopes of finding more gene knockouts.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Breakthrough Immunotherapies Seem Like a Dream Come True for Children with Leukemia

Guest Post: Nancy Jecker, Aaron Wightman, Abby Rosenberg, Doug Diekema

Paper: From protection to entitlement: selecting research subjects for early phase clinical trials involving breakthrough therapies

A breakthrough therapy to cure cancer in children suffering from acute lymphoblastic leukemia (ALL) is a dream for many families.  New immunotherapies appear to make this dream a reality. Such therapies use a person’s own immune cells to recognize and combat their disease. In the largest study to date of ALL patients treated with a form of immunotherapy known as Chimeric Antigen Receptor (CAR) T-Cell therapy, a 93% remission rate was reported. Such results are a glimmer of hope for those whose prognoses were previously considered very poor.

However, the good news is tempered by the fact this potentially lifesaving experimental therapy may not be available to everyone who might benefit. And demand is growing as word spreads. Since CAR T-cell therapy for ALL is available only through clinical trials, do patients have a right to participate? How should we choose among medically suitable candidates?

We have faced these questions before. Most recently, with ZMapp to treat Ebola Virus Disease, azidothymidine (AZT) to treat HIV and AIDS, and Immunitab (Gleevac) to treat Chronic Myleogenous Leukemia. Are patients suffering from devastating, life-threatening diseases entitled to breakthrough therapies?

In a recent paper, we argue that benefit is a continuum, from the complete uncertainty associated with standard research, to an intermediate stage where evidence of benefit mounts and reaches a peak, to a final stage of clearly demonstrated benefit that is sufficient to gain approval for clinical applications.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

A Bioethics View of Executions in Arkansas

by Craig Klugman, Ph.D.

This week the state of Arkansas had planned to execute 8 death-row inmates in 4, back-to-back killings using lethal injection over 10 days. The last execution in Arkansas was 12 years ago, so why the sudden rush? As part of the three-drug cocktail used by this state, their supply of midazolam—an anesthetic—is about to expire. If they do not use the drug by the expiration date, then they can’t use it and the company that makes the drug will not sell it to the state for this purpose.

Arkansas had planned to use a combination of 3 drugs in the execution, midazolam (an anesthetic), vecuronium bromide (a paralytic), potassium chloride (to stop the heart). This cocktail would be used to kill the 8 men.

I say “had planned” because last week, two of the prisoners had judges issue stays on their executions. This move is not unusual as there is often a flurry of court appeals and filings in the time before an execution. What is unusual is that over the weekend, another judge placed a stay on all executions on the request of drug companies and distributors—Pfizer, Fresenius, West-Ward Pharmaceuticals, and McKesson—who do not want their drugs to be used in an execution. McKesson’s concern is that when they learned the reason the state bought the vecuronium bromide, that they refunded the cost and asked for the drug to be returned. Drug manufacturers and distributors have come out against their products being used to kill prisoners. The association is unlikely to be good for sales or brand reputation.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Blinded by the Promise of Stem Cell Treatments

Alan F. Cruess cautions against the use of unproven stem cell ‘treatments.’

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Recently, many of you may have read about three patients who are blind after receiving stem cell ‘treatments.’  The patients were ‘treated’ at a Florida clinic for age-related macular degeneration. This common eye condition is the leading cause of vision loss among people over the age of 50. The clinic harvested stem cells from the patients using liposuction and then injected these stem cells into their eyes. Again, these three patients, are now all blind as a result of this unproven ‘treatment.’

There are two types of age-related macular degeneration: ‘wet’ and ‘dry.’ In recent years, treatment of wet macular degeneration has been transformed by new drugs which can be very effective if they are applied early. Meanwhile, treatment of the more common dry macular degeneration remains elusive. As such, patients with dry macular degeneration may be desperate to prevent and reverse blindness and willing to try emerging regenerative therapies.

Some experimental stem cells treatments to prevent blindness are promising, and they are being studied worldwide in laboratories and highly regulated clinical trial settings. In these settings, the safety and efficacy of experimental treatments can be closely monitored. Yet, the safety and efficacy should be called into question when these so-called ‘treatments’ are marketed outside of the research context. This was the case at the Florida clinic.

Before subjecting oneself or a loved one to any new ‘treatment’ with stem cells patients should be informed about the risks and potential benefits of the proposed treatment.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Individually-randomized controlled trials of vaccines against the next outbreak

Guest Post: Nir Eyal, Marc Lipsitch

Paper: Vaccine testing for emerging infections: the case for individual randomisation 

The humbling experience of international response to Ebola taught the world a thing or two on preparing for Zika and for other emerging infections.

Some of those lessons pertain to vaccine development against emerging infections. One lesson was that vigorous vaccine development should start long in advance of outbreaks. CEPI, the Coalition for Epidemic Preparedness Innovations, was recently launched with an initial investment of half a billion US dollars from the Gates Foundation, Britain’s Wellcome Trust and the governments of Japan, Norway and Germany. There is also growing recognition that best practices on vaccine testing should be developed prior to outbreaks, from a study methodology viewpoint.

By contrast, in Zika, ethical guidelines on response in general and on an aspect of vaccine testing were created only once the pandemic erupted. Shouldn’t ethical disputes, e.g. on trial design for vaccine candidates, be ironed out in advance of emerging infections?

One persistent ethical question in vaccine testing pertains to individually-randomized control in efficacy trials. At the height of the 2014-5 Ebola outbreak, individually-randomized controlled trials were much maligned. Our paper at the Journal of Medical Ethics sets out to defend that approach for vaccine efficacy testing in emerging infections, including highly fatal and untreatable ones in developing countries.

Nearly everyone agrees that scientifically, individually-randomized controlled trials are the gold standard of clinical research. But during the Ebola outbreak, ethicists, leaders, and humanitarian workers opposed them. For testing vaccine against a highly fatal infection without approved drugs or vaccines, they deemed these designs unethical.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics News

How Consent Requirements May Shape Teen Mental Health Research

With comments from our Yoram Unguru. Requiring teens to get permission from their parents to participate in studies about behavioral health may make it harder to understand adolescent psychology – especially when drugs and alcohol are involved – a U.S. study suggests

Source: Bioethics Bulletin by the Berman Institute of Bioethics.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

Penny-Foolish

April 07, 2017

by Sean Philpott-Jones, Chair, Bioethics Program of Clarkson University & Icahn School of Medicine at Mount Sinai

Penny-Foolish

The 2018 federal budget battle has barely begun and already critics – including myself – are questioning the wisdom of Trump’s proposal to drastically cut key agencies like the US Department of State, Health and Human Services, and the Environmental Protection Agency in order to build a wall that no one wants, to buy fighter jets that no one needs, and to give tax breaks that no one earned.

The Trump Administration claims that the President’s proposal will make Americans safer, healthier, and wealthier, but the unprecedented cuts and reallocations in this budget are likely to make us poorer, sicker, and endangered. They are also likely to do irreparable harm to America’s image overseas. Although Ronald Reagan famously described America as “a shining city upon a hill whose beacon light guides freedom-loving people everywhere,” we will soon be seen as the exact opposite as the very programs that save lives, promote equality, combat poverty, and advance human rights – the very values and ideals that Americans cherish – are dismantled.

Consider, for example, Trump’s plan to cut funding from a program that provides life-saving treatment to those living with HIV/AIDS across the globe. Known as the President’s Emergency Plan for AIDS Relief (PEPFAR), that program was created in 2003 by then President George W. Bush. It would not be hyperbole to describe PEPFAR as President Bush’s greatest legacy, redeeming an otherwise disastrous administration best known for embroiling America in an unjust and seemingly unending war in the Middle East and for trigger the worst financial disaster since the Great Depression.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

Bioethics Blogs

The Dangers and Challenges of Weaponizable Neuroscience: A Call for Renewed Engagement

Photo Source: This Image was released by the United States Navy with the ID 021015-N-6996M-109 (http://bit.ly/2oQFUdh)

BY DR. DIANE DIEULIIS and DR. JAMES GIORDANO

The chemical weapon attack in Syria that has killed at least 70 people employed the nerve gas sarin. And, it is believed that it was the nerve agent VX that was used to assassinate Kim Jong-nam in a public airport. These uses of nerve agents violate the international Chemical Weapons Convention (CWC). While the Syrian government signed the CWC in 2013, it was never ratified, and of course, signatory agreement does not guarantee compliance. Nor do such treaties among nation states necessarily provide any security against the development and use of biological and chemical weapons by non-state actors. These events are disturbing and, we believe, portend a larger, and ever growing issue of how such neurological agents could be used, altered and/or developed anew as weapons.

International advances in brain science over the past decade are enabling ever greater capabilities to control neurological processes of thought, emotion and behavior. So, while the CWC and Biological Toxin and Weapons Convention (BTWC) prohibit development of drugs, microbes and toxins that can be made into weapons, these prohibitions are not absolute – many of these substances can be – and are – used in basic neuroscience research, or in research programs that seek to develop defenses against biochemical weapons. What’s more, new tools and methods with which to edit genes, such as CRISPR/Cas9, can make it easier to modify bacteria, viruses or certain toxins to be weaponized.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.