Clinical Community Has Crucial Role to Play In the Future of Mitochondrial Replacement Techniques, Say Experts

February 24, 2016
Press Release
For Immediate Release
Contact: Leah Ramsay,, 202.642.9640

Clinical Community Has Crucial Role to Play In the Future of Mitochondrial Replacement Techniques, Say Experts

There is a unique role for the United States medical community to play in determining the future application of, and ethically acceptable approach to, mitochondrial replacement techniques (MRTs), according to a commentary published online by the New England Journal of Medicine.

The United States has a different process for regulating any potential clinical use of MRTs than the United Kingdom, which has also recently addressed policy issues with MRTs, and so the commentary authors emphasize that medical professional organizations, societies, and practitioners will be crucial to determining the ethically acceptable path forward for clinical application of MRTs.

kahn_2014The commentary authors were members of the Institute of Medicine committee which recently released a report commissioned by the Food and Drug Administration (FDA), “Mitochondrial Replacement Techniques: Ethical, Social, and Policy Considerations,” and include committee chair Jeffrey Kahn, PhD, MPH, of the Johns Hopkins Berman Institute of Bioethics.

MRTs, which have not yet been performed in humans, are envisioned as a method to prevent a mother from passing on certain debilitating diseases related to mutated mitochondrial DNA (mtDNA).  The techniques would replace her mtDNA with that of another woman, while still passing on her own nuclear DNA. Mitochondria are inherited solely from the mother.

“If and when initial investigations are undertaken, critical safety and efficacy questions will remain before regulatory approval or clinical use can occur,” the commentary states.

Decisions on how to proceed with initial research uses of MRTs in humans could have a ripple effect for other highly specialized treatments and techniques. “Mitochondrial DNA (mtDNA) disease may the poster child for highly targeted, ‘personalized’ medicine,” the commentary states.

Though MRTs could provide a previously unavailable reproductive option, the authors are careful to note what it cannot achieve.  “MRTs would have no health benefits for people who already have mtDNA diseases, nor would they prevent the occurrence of newly arising mtDNA mutations.”

“The first use of MRTs in humans is uncharted scientific territory, and requires caution and commitment to the safety of any potential offspring born through use of the techniques,” says Kahn, the Levi Professor of Bioethics and Public Policy at the Johns Hopkins Berman Institute of Bioethics. “Any path forward must assess and avoid not only health risks, but take account of psychological effects for the individuals born as a result of these techniques, as well as the social implications of such reproductive technologies.”

The authors highlight two key points made in the IOM report: that safety for the individual conceived by the procedure is the primary ethical concern, and that risk to the human germline through heritable changes should be avoided by limiting initial clinical investigations to transfer of male embryos only. This could change, Kahn notes, as knowledge is gained, and depending on the outcome of ongoing international debate about germline genetic modification.

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NEJM: Mitochondrial Replacement Techniques — Implications for the Clinical Community

IOM Report, “Mitochondrial Replacement Techniques: Ethical, Social, and Policy Considerations

Jeffrey Kahn, PhD, MPH

Media Contact: Leah Ramsay,, 202.642.9640

A Vision of Hope For Young Patients Living With Debilitating Chronic Illness

Media Contact: Leah Ramsay 202.642.9640,

November 10, 2014
For Immediate Release

Johns Hopkins Bioethicists Have Vision of Hope For Young Patients Living With Debilitating Chronic Illness
Scholars from the Johns Hopkins Berman Institute of Bioethics launch a curriculum for training diverse healthcare workers to apply palliative care principles to pediatric patients suffering from sickle cell disease and Duchenne muscular dystrophy.

Palliative care is traditionally focused on care for the terminally ill, but scholars at the Johns Hopkins Berman Institute of Bioethics are aiming to change that by bringing the principles and comfort of palliative care to pediatric patients suffering from chronic illness.  A curriculum launching November 10 will guide healthcare workers in integrating the principles and practices of pediatric palliative care into their long-term care regimen.

Titled “Vision of Hope,” the curriculum uses two specific chronic conditions as case studies: sickle cell disease (SCD) and Duchenne muscular dystrophy (DMD). All content is free and publicly available at

“Young people living with chronic diseases routinely confront challenges that benefit greatly from an expanded repertoire of ethically grounded palliative care practices that are holistic, attending to their emotional wellbeing, spiritual developmental and physical needs,” says Cynda Hylton Rushton, PhD, RN, FAAN, co-principal investigator on the project.

The curriculum provides healthcare institutions with evidence-based curricular materials on pediatric palliative care (PPC) concepts, with flexibility for training interdisciplinary healthcare workers, with all levels of PPC knowledge and experience. The various components include video segments, discussion questions, and interactive activities that can be used together or separately. Three formats are suggested for training sessions: in-service, workshop or a two-and-one half-day retreat.

“What makes these palliative care training programs innovative is that they are experiential, interdisciplinary and relational,” explains Gail Geller, ScD, MHS, co-principal investigator on the project. “Relational learning expands the notion of professional competence to encompass the moral realm of human relationships and experience.”

Through the use of video, the Vision of Hope curriculum provides a means to integrate the voices and experiences of patients and families throughout all aspects of the training, Geller says.  Some of the film components of the Vision of Hope project have won honors as stand alone pieces, including awards from the Houston International Film & Video Festival, Cine, and the International Association of Audio Visual Communicators.

“Living with the pain and other serious medical complications of Sickle Cell Disease becomes even more frustrating and emotionally painful when healthcare providers don’t believe the patient’s reports of their pain experience,” says Carlton Haywood Jr., an investigator on the project who both researches and lives with SCD.  “The Vision of Hope curriculum, and the films in particular, shed light on the impact of these added burdens on patients, and we hope to use these patient voices as tools to enact lasting changes in the quality of the care delivered to people suffering from sickle cell disease.”

“We believe the integration of palliative care into lifelong treatment of chronic conditions will give young patients hope for a life filled with what young Mattie Stepanek called ‘heartsongs’,” Rushton says.  During his short life, Mattie J. T Stepanek lived with a rare form of muscular dystrophy and inspired others as a bestselling author, poet, peace advocate, and MDA Goodwill Ambassador. His insights regarding living each day with muscular dystrophy and planning for an uncertain future were foundational to the Vision of Hope project, Rushton explains. Stepanek defined heartsongs as “gifts that reflect each person’s unique reason for being,” distinct from their health and suffering.

The Vision of Hope Project is supported by a grant (1 RC1NR011710-01) from the National Institutes of Health National Institute of Nursing Research and the Stavros Niarchos Foundation. One of the films in the curriculum, On the Edge, received additional support from the Slomo and Cindy Silvian Foundation.

Groundbreaking Professorship in Nursing and Bioethics

Johns Hopkins University will dedicate a new endowed professorship, the Anne and George L. Bunting Professor of Clinical Ethics, believed to be the nation’s first joint professorship in nursing and bioethics.

The ceremony to dedicate the professorship and install the inaugural Bunting Professor, Cynda Hylton Rushton, PhD, RN, FAAN, will be Tuesday March 12, 2013 at 4:00 PM in the Alumni Auditorium at the Johns Hopkins University School of Nursing, 525 N. Wolfe Street, Baltimore, MD 21205.

Johns Hopkins University School of Nursing and the Johns Hopkins Berman Institute of Bioethics have jointly established the Bunting Professorship, and Rushton will work collaboratively with faculty and students of both institutions to identify, analyze and attempt to resolve the ethical dilemmas that arise in caring for patients and their families.

Rushton has been with Johns Hopkins for over 20 years, beginning as a Clinical Nurse Specialist in Ethics in 1991.  She was appointed the first Chair of the Maryland State Council of Quality Care at the End of Life and has been recognized as one of Maryland’s Top 100 Women.  In addition to her faculty appointments in the School of Nursing and the Berman Institute since 1995, Rushton has been program director of the Johns Hopkins Children’s Center Harriet Lane Compassionate Care program since 2000.

“We are at a critical juncture in healthcare; the ethical issues that impact patients, their families and clinicians must become part of the national dialogue in order to illuminate ethically sound solutions and cultivate environments where integrity is preserved,” Rushton says. “I feel incredibly fortunate to have this rare opportunity to engage and explore the ethical foundations of nursing in a new and strategic way.”

George L. Bunting is a long-time member of the Berman Institute’s advisory board and a philanthropic supporter of the university. He served as Chairman of Johns Hopkins Health System and Johns Hopkins Hospital from 1994 – 1998 as well as Johns Hopkins Medicine from 1996 – 1998.   Bunting has been integral to the Berman Institute’s growth into one of the world’s largest and most respected centers of bioethics.

“The Berman Institute is absolutely committed to approaching the ethical challenges of clinical practice from an interdisciplinary perspective, and no health professionals are more experienced than nurses in confronting these challenges,” says Ruth R. Faden, director of the Berman Institute.  “The Bunting Professorship is an important addition to our institute, and we are thrilled to have Dr. Rushton, already a valuable member of our faculty, as the inaugural Bunting Professor

Martha N. Hill, dean of the School of Nursing, notes, “This joint, endowed professorship highlights the increasingly important interprofessional role of nursing and bioethics. Dr. Rushton brings to the professorship the expert nursing perspective that is essential to today’s bioethics scholarship and debate and will be required in shaping tomorrow’s healthcare policies.”

Berman Institute Hosts Working Group On Chemotherapy Shortage

So much effort, angst, and money is expended in the fight against cancer, it is a terrible irony that effective treatments – many of which are critical components in life-saving regimens for pediatric cancer—have become so affordable as to be uneconomical to produce, creating a supply shortage.  Consequently, physicians are faced with grim ethical choices of how to apportion these drugs among equally deserving patients.

A team from the Johns Hopkins Berman Institute of Bioethics, led by Yoram Unguru, MD, MS, MA, also a pediatric hematologist / oncologist at the Herman and Walter Samuelson Children’s Hospital at Sinai, will gather a distinguished group of experts for the working group meeting, to be held January 30, 2013 at the Berman Institute in Baltimore, MD.  The group will work to develop ethical frameworks for decision-making both within and between healthcare institutions related to drug shortages.

“This is a real and significant problem with far-reaching impact on clinical care and research.  Children’s lives are at stake,” says Unguru.  “Physicians are in an untenable position; imagine having to decide which child will receive a necessary treatment and which will not, knowing that this decision will have a negative impact on a child’s life.  Our hope is that this meeting will offer clinicians substantive guidance as they grapple with these and related tragic choices.”

Unguru will be joined by a team from the Berman Institute, including director Ruth R. Faden, as well as an impressive list of experts that includes: 

  • Conrad Fernandez, Professor and head of the division of pediatric hematology/oncology in the Department of Pediatrics, IWK Health Centre, Canada
  • Steven Joffe, Associate Professor of Pediatrics and of Global Health and Social Medicine, Dana-Farber Cancer Institute, Boston Children’s Hospital, and Harvard Medical School 
  • Peter C. Adamson, Chair of the Children’s Oncology Group (COG); Professor of Pediatrics and Pharmacology, University of Pennsylvania School of Medicine; Chief of the Division of Clinical Pharmacology and Therapeutics, Children’s Hospital of Philadelphia
  • Lisa Bomgaars, Director of Clinical Research Operations, Texas Children’s Cancer and Hematology Centers 
  • Eduvigis Cruz-Arrieta, Assistant Clinical Professor of Medical Psychology in Psychiatry and Pediatrics, Columbia University 
  • Eric Kodish, Director of Cleveland Clinic’s Center for Ethics, Humanities, and Spiritual Care 
  • Robert “Skip” Nelson, Deputy Director and Senior Pediatric Ethicist in the Office of Pediatric Therapeutics, Office of the Commissioner, U.S. Food and Drug Administration
  • A. Kim Ritchey, President, American Society of Pediatric Hematology/Oncology; Board of Directors, American Board of Pediatrics;  Professor of Pediatrics, University of Pittsburgh School of Medicine
  • Christine Grady, Chief, NIH Clinical Center Department of Bioethics
  • I. Glenn Cohen, Assistant Professor, Harvard Law School and Co-Director, Petrie-Flom Center for Health Law Policy, Biotechnology and Bioethics
  • Carol Taylor, Senior research scholar and professor of nursing, Kennedy Institute of Ethics at Georgetown University 


Media Contact:
Leah Ramsay

Johns Hopkins Berman Institute of Bioethics

Working group contact:
Yoram Unguru,

Experts Aim to Redefine Healthcare and Research Ethics

January 11, 2013

Johns Hopkins bioethicists lead a call to reform the ethical foundation of the changing American healthcare system

In what they acknowledge as a seismic shift in the ethical foundation of medical research, practice and policy, a prominent group of interdisciplinary healthcare experts has rejected an ethical paradigm that has guided the American system since the 1970s.  A pair of articles in a special report from The Hastings Center Report calls for morally obligatory participation in a “learning healthcare system” more in step with the digital age, along with seven commentaries from other experts responding to their ideas.

In one article, the authors reject the bright-line distinction between medical research and patient care that has been central to the ethical underpinnings of federal human subject research regulations for decades. They argue that it is increasingly difficult to distinguish clinical research from practice and the daily operations of healthcare organizations, and that widely held assumptions about how research differs ethically from practice may be incorrect.  Specifically, the authors challenge the assumption that participation in clinical research by definition offers patients less potential benefits and puts them at greater overall risk than clinical practice, as well as the assumption that research imposes more irrelevant burdens on patients.

In today’s healthcare system, the labels “research” and “practice” are poor proxies for what should be our central moral concerns, the authors argue, and no longer serve as an effective guideline for what requires ethical oversight.  They point out, for example, that over 50 percent of medical treatments are used without sufficient proof of their effectiveness, and approximately 100,000 die annually from healthcare acquired infections.

“Far too often, doctors do their best but simply don’t have the information to tell them which approaches or treatments work best, and patients are suffering for that lack of knowledge,” says Nancy E. Kass, deputy director for public health at the Johns Hopkins Berman Institute of Bioethics, and lead author of the article. “We’re finding that patients are both underprotected from risks in medical treatment and over-protected from low-risk quality-improvement research, bringing progress to a dangerous stalemate that is costing lives,” the authors write.

Instead, the authors say that healthcare should be moving toward a system in which clinical research and clinical practice are integrated, and every clinical encounter is simultaneously an opportunity to provide needed care to patients and also to learn from that to improve the care provided to future patients.

In their second article, the authors put forward a new ethical framework for the integration of research with practice in what the Institute of Medicine calls a learning healthcare system. The framework includes seven obligations, six of which fall on health professionals and institutions, and the 7th on patients:

  1. Respect the rights and dignity of patients
  2. Re­spect the clinical judgment of clinicians
  3. Provide opti­mal care to each patient
  4. Avoid imposing nonclinical risks and burdens on patients
  5. Address health inequalities among populations
  6. Conduct continuous learning activities that improve the quality of clinical care and health care systems
  7. Contribute to the common purpose of improving the quality and value of clinical care and health care systems

The framework includes familiar tenets of both medical and research ethics, but also new obligations that the authors acknowledge “substantially revise traditional conceptions” of the roles played by health systems, providers and patients. “In addition to long-standing ethical obligations to ensure that burdens and benefits of research are fairly distributed and that patients are appropriately respected, our ethical framework directs research towards aggressive efforts to reduce or eliminate unfair inequalities in health outcomes and in the evidence base for clinical decision-making,” notes Ruth R. Faden, director of the Johns Hopkins Berman Institute of Bioethics and lead author of the article outlining the framework.  Among the examples of unfair inequalities the authors say should be addressed by obligation five is the scarcity of evidence for managing chronic illness in pregnant women, as compared to other adults with the same conditions.  Women, and their children, would be well-served by a healthcare system that continually learns from patient care.

“The framework also challenges previous thinking in research and clinical ethics by calling for an ethical obligation on the part of clinicians, administrators, payors and purchasers to conduct research to improve health care quality and value, and on patients to contribute to such research,” says Faden. The authors write, “Just as health professionals and organizations have an obligation to learn, patients have an obligation to contribute to, participate in, and otherwise facilitate learning,” that will improve the quality of the healthcare system.

The authors emphasize, however, that this is not a blanket obligation, regardless of risk.  Some kinds of medical research, such as early testing of drugs still unapproved by the Food and Drug Administration, are not included and should always proceed only with the express, voluntary informed consent of the patient, they say.  The patient obligation is focused on research that poses no additional risk beyond what patients face in clinical care, and would also exclude research that compares different types of treatments, for example, surgery to medical management.  The authors add that the framework also includes obligations to avoid imposing nonclinical risks and burdens on patients (4), and to protect their rights and interests (1).

“There is nearly universal agreement that we need to be able to efficiently learn about the effectiveness and safety of health care as an integral aspect of providing clinical care to patients,” said Sean Tunis, President and CEO of the Center for Medical Technology Policy. “We are hopeful that these papers and commentaries will help to focus and catalyze further debate, leading to changes in policy that accelerate learning while protecting patients.”

Extraordinary opportunities for learning are lost in our current system, the authors say, because physicians and researchers face significant hurdles in capturing the rich information generated from thousands of daily medical encounters with patients due to overly burdensome oversight and consent rules.  The new framework is intended to help reduce these hurdles.

The authors write that they expect their articles will spark debate, and hope they will move the transformation to a learning healthcare system forward, both in its ethical underpinnings and in practice.  They write, “We claim no more than a start on a subject that mer­its extensive investigation, and we welcome suggestions and commentary moving forward…We are in the early days of a progressive realization of a lofty aspirational goal, but given the preventable harm, waste, and uncertainty about clinical effectiveness in health care, efforts to accelerate learn­ing should be given high priority.”

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The authors’ work was supported by grant RC1RR028876 from the National Institutes of Health—National Center for Research Resources.

Media Contact:
Leah Ramsay

Ethical Oversight of Learning Health Care Systems, Hastings Center Report Special Report 43 (s1); January-February 2013.

The Research-Treatment Distinction: A Problematic Approach for Determining Which Activities Should Have Ethical Oversight,Hastings Center Report Special Report 43(s1); s4-s15. January-February 2013

An Ethics Framework for a Learning Health Care System: A Departure from Traditional Research Ethics and Clinical Ethics, Hastings Center Report Special Report 43 (s1); s16-s27. January-February 2013.