Tag: clinical trials

Bioethics News

Paolo Macchiarini, Fraud, and Oversight: A Case of Falsified Stem Cell Research

by Michael S Dauber, GBI Visiting Scholar

According to a recent story by John Rasko and Carl Power in The Guardian, surgeon Paolo Macchiarini’s research in artificial windpipes, previously hailed as pioneering medicine with the promise to save many lives, has been exposed as a fraud. Miacchiarini had previously received public praise for creating artificial windpipes by grafting stem cells onto plastic frames, which allowed him to “grow” new trachea for his patients.

While much of the scientific community was eager to believe Miaccharini had made significant breakthroughs, not everyone was convinced. According to a Swedish TV series called Experimenten, most of Miaccharini’s patients died within a few years of their procedures, and it was unclear that the experimental surgeries actually helped: in fact, they may have made matters much worse. Deeper investigation revealed that Macchiarini had actually falsified much of his data, and that institutional checks that normally prevent fraudulent individuals from being hired had been ignored. For example, according to an “external inquiry,” he was hired by the Karolinska Institute in 2010 despite various fraudulent, concerning, and questionable information on his resume (including a claim from a reference that he had been “blocked from a professorship in Italy”). The report also found that there had been inappropriate contact between Macchiarini and the Karolinska Institute’s Vice-Chancellor during his recruitment.

Even more troubling, the Institute failed to comply with government regulations designed to ensure research and clinical interventions are practiced ethically. According to Rasko and Power, Macchiarini failed to test his artificial airways in animals before implanting them in three human patients, and he did not apply for approval from an institutional review board or other ethics committee, despite the fact that Stockholm’s board was housed at the Institute.

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

Offshore Herpes Vaccine Trial Under Investigation

September 1, 2017

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The government of St. Kitts and Nevis has launched an investigation into the clinical trial for a herpes vaccine by an American company because it said its officials were not notified about the experiments.

The vaccine research has sparked controversy because the lead investigator, a professor with Southern Illinois University, and the U.S. company he co-founded did not rely on traditional U.S. safety oversight while testing the vaccine last year on mostly American participants on the Caribbean island of St. Kitts.

The trial received financial backing from a former Hollywood filmmaker who has asserted the vaccine was highly successful in stopping herpes outbreaks. Since then, a group of investors, including Donald Trump supporter Peter Thiel, have backed the ongoing vaccine research with a $7 million investment that could include additional clinical trials in Mexico and Australia.

Neither the Food and Drug Administration nor a safety panel known as an institutional review board, or an “IRB,” monitored the testing on the 20 human subjects. Now, the government of St. Kitts and Nevis says that the researchers also did not officially seek permission to test the vaccine, which took place from April to August 2016.

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Scientific American

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

Webinar Follow-up: Data and Safety Monitoring: Advanced Issues and Case Studies

In July, PRIM&R collaborated with CITI Program to host the advanced-level webinar Data and Safety Monitoring: Advanced Issues and Case Studies. Expanding on introductory knowledge in the module Data and Safety Monitoring in Human Subjects Research, part of CITI Program’s Biomedical Basic course, this webinar described ways in which the IRB, the data and safety monitoring board (DSMB), the investigator, and the sponsor can work together to ensure scientific integrity and subject safety in clinical trials. Summaries of government and non-government organizations’ guidance as well as interactive case studies offered strategies for handling complex situations that may arise during data and safety monitoring, including when and how to report adverse and unanticipated events, when a DSMB is needed, and what is considered a conflict of interest.

The post Webinar Follow-up: Data and Safety Monitoring: Advanced Issues and Case Studies appeared first on Ampersand.

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

The FDA Approves a Landmark Cancer Drug

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The Food and Drug Administration on Wednesday approved a new therapy to treat leukemia in kids and young adults—a decision whose importance is as much symbolic as it is practical.

Kymriah, from the Swiss pharmaceutical company Novartis, is a cancer therapy that represents several things at once: a game-changing way to treat cancer through genetic engineering, a novel paradigm for the biotech business, and the latest turn in the debate over just how astronomically expensive a life-saving therapy can be.

Kymriah is strikingly effective for young patients with acute lymphoblastic leukemia, or ALL, but it is far more involved than taking a pill or getting an infusion. It requires inserting a human-designed gene into a patient’s own T cells so they recognize and ferociously attack cancer cells. Researchers began modifying T cells for patients in the 1990s—and now the technology called CAR T-cell therapy is finally ready for prime time in treating cancer.

Of several dozen ALL patients in a clinical trial for Kymriah, 83 percent were cancer-free after three months. It is a lifeline for patients in which traditional treatments like chemotherapy and bone-marrow transplants had failed. When the FDA’s advisory committee initially voted in favor of approving Kymriah, one member called it “the most exciting thing I’ve seen in my lifetime” for childhood leukemia. Novartis is hardly the only company interested in CAR T. Kymriah is the first approved therapy, but several clinical trials—mostly notably Kite Pharma’s for lymphoma—are right behind it.

(To clear up any possible confusion about terminology: The FDA and others have chosen to call CAR T-cell therapy a form of gene therapy—and thus deemed it the first gene therapy to be approved in the United States.

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

CAR-T cells: A drive to the future of cancer treatment

Conrad Fernandez describes the ethical challenges related to the use of CAR T-cell therapy for cancer patients.

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I am a pediatric oncologist and over the years have looked after hundreds of children with cancer – ranging in age from newborns into their early 20s. About a third of these children have suffered from leukemia. During my career of more than 25 years, I have seen my share of sadness and joy. Roughly one in five of these children have died – most often because of resistance intrinsic to their cancer but sometimes as a consequence of the toxicity of cancer therapy. These toxicities may occur acutely during the treatment (such as severe infections) or more insidiously appear years or decades later. A novel treatment approach that would overcome this resistance while avoiding chemotherapy toxicity would be most welcome.

A few years ago, I sat in a plenary session of the American Society of Hematology annual meeting (the preeminent hematology meeting in the world) where early phase CAR T-cell therapy was discussed. CAR (chimeric antigen receptor) T-cells are genetically reprogrammed immune cells that normally have the job of fighting infection or other foreign intruders into our bodies. CAR T-cells are manufactured to target a subtype of leukemia that is called B-cell leukemia – a type especially common in childhood. I thought to myself to take special note of what I was hearing, as this marked the potential for a paradigm shift in how we approached treatment of leukemia and perhaps other cancers. It is for these relapsed and refractory B-cell leukemia patients that the FDA’s Oncologic Drugs Advisory Committee (ODAC) has just recommended approval of CAR T-cell therapy – the first recommendation for approval of its kind.

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

CAR-T cells: A drive to the future of cancer treatment

Conrad Fernandez describes the ethical challenges related to the use of CAR T-cell therapy for cancer patients.

__________________________________________

I am a pediatric oncologist and over the years have looked after hundreds of children with cancer – ranging in age from newborns into their early 20s. About a third of these children have suffered from leukemia. During my career of more than 25 years, I have seen my share of sadness and joy. Roughly one in five of these children have died – most often because of resistance intrinsic to their cancer but sometimes as a consequence of the toxicity of cancer therapy. These toxicities may occur acutely during the treatment (such as severe infections) or more insidiously appear years or decades later. A novel treatment approach that would overcome this resistance while avoiding chemotherapy toxicity would be most welcome.

A few years ago, I sat in a plenary session of the American Society of Hematology annual meeting (the preeminent hematology meeting in the world) where early phase CAR T-cell therapy was discussed. CAR (chimeric antigen receptor) T-cells are genetically reprogrammed immune cells that normally have the job of fighting infection or other foreign intruders into our bodies. CAR T-cells are manufactured to target a subtype of leukemia that is called B-cell leukemia – a type especially common in childhood. I thought to myself to take special note of what I was hearing, as this marked the potential for a paradigm shift in how we approached treatment of leukemia and perhaps other cancers. It is for these relapsed and refractory B-cell leukemia patients that the FDA’s Oncologic Drugs Advisory Committee (ODAC) has just recommended approval of CAR T-cell therapy – the first recommendation for approval of its kind.

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

Book Review: Cells Are The New Cure (BenBella Books, Inc., 2017). ISBN 9781944648800.

$26.95. Reviewed by Michael S. Dauber, MA

 

Cells Are The New Cure, written by Robin Smith, MD, and Max Gomez, PhD, is a book about the history of medical research on cells, both human and non-human, and recent developments in these techniques that have made cellular medicine one of the most promising fields for therapeutic exploration. While the book’s title suggests an exclusive focus on the healing aspects of genetic modification and human stem cell therapy, the text is much more than that: it is a roadmap for understanding the origins of such techniques, the current state of affairs in cellular and genetic therapies, the administrative landscape investigators must traverse in conducting research, and the areas in which we still need to make progress.

Smith and Gomez make an argument that is structurally simple yet gripping: they suggest that targeted therapies involving stem cells and genetic modifications are the future of medicine by pointing to the immense amount of studies in those fields that have yielded beneficial results. While many readers might acknowledge this fact even before reading the book, many may not be aware of the full extent of the knowledge we have gained from research on cells and genetics, or the myriad ways this knowledge has been applied. Of course, Smith and Gomez cover the big diseases that most people think of when imagining medical research: cancer, heart disease, neurodegenerative conditions, etc. However, the book also contains detailed information about how we age, what may cause certain allergies, how the body repairs itself, and the ways stem cell therapies, genetic editing techniques, and other complex medicines that build on these methods can be used to treat these conditions.

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

From the Director: Highlights of PRIM&R’s Ethics of Data Access, Use, and Sharing for Human Subjects Research Workshop

On March 1, 2017, PRIM&R held a day-long Ethics of Data Access, Use, and Sharing for Human Subjects Research Workshop in partnership with the Multi-Regional Clinical Trials Center of Brigham and Women’s Hospital and Harvard University (MRCT Center). I am pleased to announce that the proceedings and other information from the day are now available on PRIM&R’s website.

The post From the Director: Highlights of PRIM&R’s Ethics of Data Access, Use, and Sharing for Human Subjects Research Workshop appeared first on Ampersand.

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

Cyber Security & Implanted Bio-Monitoring Devices

Dylan Roskams Edris describes risks associated with implantable remote bio-monitoring devices.

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Over the past few years, there has been a rapid growth in the development of implantable remote bio-monitoring systems for medical purposes. For example, implantable monitors that allow for constant monitoring of blood glucose and heart status are being tested in clinical trials. A defining feature of implantable monitors for medical care is the ability to transmit the data they collect wirelessly without patient intervention. Suzanne E. Spaulding, former Under Secretary for the U.S. Department of Homeland Security, said that: “It has been predicted that by 2020 the internet will expand to include 50 billion connected devices.”  We can expect that this will include the expansion of bio-monitoring systems.

The appeal of such technology is clear. For chronic conditions like diabetes, the early detection of hypoglycemia can let the patient or healthcare professional take emergency action or notify paramedics before the patient has a potentially fatal seizure. In addition, if a physician or sufficiently sophisticated medical system can access data related to a chronic condition on a remote basis preventive measures can be taken to stop dangerous acute symptoms from occurring in the first place.

However, the uptake of implantable remote bio-monitoring also poses several risks. First, there is a risk that bio-monitoring devices could be illegally hacked or intercepted. If the operation of a device is dependent on the transfer of information between the device and some centralized healthcare system then interference with information going in either direction could affect the device’s function.

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

Pregnant Women Absent from Zika Vaccine Trials

August 15, 2017

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This uncertainty is a major reason behind researchers’ hesitancy to expose pregnant women to newer vaccines. Women do indeed get vaccinated while pregnant—against the flu or tetanus, diphtheria, and pertussis (Tdap), for example. But “the overall safety for flu and Tdap vaccines was established in very large populations prior to being administered to pregnant women,” says August.

Some vaccines—such as the flu vaccine—included pregnant women in clinical trials. And, notably, pregnant women were included in Phase 3 trials for the new respiratory syncytial virus (RSV) vaccine that prevents a devastating, potentially fatal infection that targets infants.

But recommendations for vaccination during pregnancy are not always backed by clinical trials. “Historically, many of the recommendations have relied on observational data,” writes Johns Hopkins bioethicist Carleigh Krubiner in an email to The Scientist. She, along with August, is part of the working group who wrote the Wellcome Trust-funded guidelines for Zika vaccine administration in expectant moms. In these cases, pregnant women were either intentionally or unintentionally given vaccines, then mother and child were observed.

… Read More

See also: PREGNANT WOMEN & THE ZIKA VIRUS VACCINE RESEARCH AGENDA: ETHICS GUIDANCE ON PRIORITIES, INCLUSION, AND EVIDENCE GENERATION

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The Scientist

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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.