Medicine › Physiology

Biomedical Ethics and Regulation

Works Count: 74338

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Description

This cluster of papers focuses on the regulation, ethical considerations, and challenges surrounding stem cell therapies and clinics. It covers topics such as patient safety, direct-to-consumer marketing, global governance, translational research, and the ethical implications of unproven therapies. The papers also discuss the need for comprehensive regulatory frameworks to ensure the safe and ethical development of regenerative medicine.

Keywords

Stem Cell Regulation; Clinical Trials; Regulatory Challenges; Patient Safety; Direct-to-Consumer Marketing; Ethical Issues; Global Governance; Translational Research; Unproven Therapies; Regenerative Medicine

Patient Compliance in Medical Practice and Clinical Trials

1991-10-01

Joyce A. Cramer , Bert Spilker

Patient noncompliance with medical regimens is a major factor in therapeutic failure and is particularly detrimental to clinical trials. This text aims to address the entire scope of compliance issues, … Patient noncompliance with medical regimens is a major factor in therapeutic failure and is particularly detrimental to clinical trials. This text aims to address the entire scope of compliance issues, and describes electronic monitoring devices that record actual dose times. Traditional methods of assessing compliance, such as patient history, drug levels, and counting pills or prescription refills, are also discussed. The review of everyday clinical problems facing practitioners everywhere is complemented by discussions on the impact of noncompliance in research and the drug regulatory process. Health care providers in all fields as well as clinical trial researchers should find useful ideas throughout the book.

The Theory and Practice of Industrial Pharmacy

1970-10-01

Sereck H. Fox

Social Organization of Medical Work

2017-07-28

Anselm L. Strauss , Shizuko Fagerhaugh , Barbara Suczek +1 more

Technologies developed to manage long-term, incurable illnesses have radically and irrevocably altered t he organisational structure of health care, presenting us wi th a bewildering array of medical specialities. ' Technologies developed to manage long-term, incurable illnesses have radically and irrevocably altered t he organisational structure of health care, presenting us wi th a bewildering array of medical specialities. '

THE INTERNATIONAL CONFERENCE ON HARMONIZATION GOOD CLINICAL PRACTICE GUIDELINE

1999-02-01

John Dixon

The purposes of the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) Guideline are to protect the rights of human subjects participating in clinical trials and to ensure the … The purposes of the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) Guideline are to protect the rights of human subjects participating in clinical trials and to ensure the scientific validity and credibility of the data collected in human clinical studies. The guiding principle in the guideline is that the rights, safety, and well-being of the trial subject are the most important considerations and should prevail over the interests of science and society . The guideline will have an important and beneficial impact on the clinical trials conducted in the three participating regions (the United States, Europe, and Japan) as well as many other regions throughout the world. In the years to come, it should fulfill its intended purpose of providing for a more economical use of human, animal, and material resources and the elimination of unnecessary delays in the global development and availability of new medicines, and at the same time maintaining safeguards on quality, safety, and efficacy and regulatory obligations to protect public health.

Placebo-Controlled Trials and Active-Control Trials in the Evaluation of New Treatments. Part 1: Ethical and Scientific Issues

2000-09-19

Robert Temple

In recent years, several authors have argued that placebo-controlled trials are invariably unethical when known effective therapy is available for the condition being studied, regardless of the condition or the … In recent years, several authors have argued that placebo-controlled trials are invariably unethical when known effective therapy is available for the condition being studied, regardless of the condition or the consequences of deferring treatment. Some have also disputed the value of placebo-controlled trials in such a setting, asserting that the comparison of new treatment with old treatment is sufficient to establish efficacy and is all that should be of interest. This article considers the ethical concerns about use of placebo controls and describes the limited ability of active-control equivalence (also known as noninferiority) trials to establish efficacy of new therapies in many medical contexts. The authors conclude that placebo-controlled trials are not uniformly unethical when known effective therapies are available; rather, their acceptability is determined by whether the patient will be harmed by deferral of therapy. If patients are not harmed, such trials can ethically be carried out. Furthermore, active-control trials, although valuable, informative, and appropriate in many circumstances, often cannot provide reliable evidence of the effectiveness of a new therapy.

Stem Cells, Tissue Cultures and the Production of Biovalue

2002-07-01

Catherine Waldby

This article examines some of the social and philosophical implications of stem cell technologies. Stem cell technologies promise to transform the way that healthy tissues for transplant are sourced and … This article examines some of the social and philosophical implications of stem cell technologies. Stem cell technologies promise to transform the way that healthy tissues for transplant are sourced and circulated; from a social economy in which citizens donate whole organs to others, to one in which embryos are a major source of therapeutic tissues. This article considers the transformations in concepts of health, bodily relationships and social indebtedness that such a shift might entail. Using the concept of biovalue, this article describes the ways embryos are biologically engineered to act as tissue sources, and considers the relationship between biovalue, health and capital value. It discusses the effects stem cell technologies may have on concepts of the healthy body, particularly on the temporality of ageing, and on understandings of the human more generally.

Late effects toxicity scoring: the SOMA scale

1995-04-01

J.J. Pavy , J. Denekamp , J.G.J. Letschert +7 more

Biopolitics and the molecularization of life

2007-01-01

Bruce Braun

In what ways can it be said of the molecularization of life that it has made our biological existence a political concern in new ways? This essay examines two different … In what ways can it be said of the molecularization of life that it has made our biological existence a political concern in new ways? This essay examines two different answers to this question. The first, exemplified by the work of Nikolas Rose, suggests that the molecularization of life, together with the individualization of risk, has given rise to a new ‘somatic’ self, and a new ‘ethopolitical’ order in which our biological life has becomes our life's work. The second, most evident in growing concern over ‘biosecurity’, posits a vulnerable subject, thrown into an unpredictable molecular world characterized by exchange and circulation and full of ‘emergent’ risks. Whereas the former has arguably led to new forms of governmentality, and new kinds of pastoral power, this paper argues that the latter has been widley taken up as a justification for the global extension of forms of sovereign power whose purpose is to pre-empt certain biological futures in favour of others. An exclusive focus on the former not only risks leaving the latter unexamined, it may leave us unable to consider how the two are related.

PRINCIPLES OF INTERNAL MEDICINE

1955-07-01

Theresa Harrison , Rayhaan Adams , Paul B. Beeson +3 more

Wiskott-Aldrich syndrome

1968-04-01

Max D. Cooper , H. Peter Chase , JamesT. Lowman +2 more

The organization of ground substance and basement membrane and its significance in tissue injury, disease and growth

1949-11-01

Isidore Gersh , H. R. Catchpole

Adv. Drug Delivery Rev.

1997-05-01

Frederick G.P. Welt , Elazer R. Edelman

Endgame: Glybera Finally Recommended for Approval as the First Gene Therapy Drug in the European Union

2012-10-01

Seppo Ylä‐Herttuala

In an Editorial published earlier this year in Molecular Therapy,1 I described the regulatory uncertainty surrounding the evaluation of Glybera (alipogene tiparvovec) by the European Medicines Agency (EMA). Glybera is … In an Editorial published earlier this year in Molecular Therapy,1 I described the regulatory uncertainty surrounding the evaluation of Glybera (alipogene tiparvovec) by the European Medicines Agency (EMA). Glybera is an adeno-associated viral vector engineered to express lipoprotein lipase in the muscle for the treatment of lipoprotein lipase deficiency. As with a typical TV serial, its regulatory saga has now had four acts, leading recently to a final recommendation by the EMA to approve Glybera in the European Union.

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Equipoise and the Ethics of Clinical Research

1987-07-16

Benjamin Freedman

The ethics of clinical research requires equipoise--a state of genuine uncertainty on the part of the clinical investigator regarding the comparative therapeutic merits of each arm in a trial. Should … The ethics of clinical research requires equipoise--a state of genuine uncertainty on the part of the clinical investigator regarding the comparative therapeutic merits of each arm in a trial. Should the investigator discover that one treatment is of superior therapeutic merit, he or she is ethically obliged to offer that treatment. The current understanding of this requirement, which entails that the investigator have no "treatment preference" throughout the course of the trial, presents nearly insuperable obstacles to the ethical commencement or completion of a controlled trial and may also contribute to the termination of trials because of the failure to enroll enough patients. I suggest an alternative concept of equipoise, which would be based on present or imminent controversy in the clinical community over the preferred treatment. According to this concept of "clinical equipoise," the requirement is satisfied if there is genuine uncertainty within the expert medical community--not necessarily on the part of the individual investigator--about the preferred treatment.

The Theory and Practice of Industrial Pharmacy

1970-10-01

Gilbert S. Banker

FDA Regulation of Stem-Cell–Based Therapies

2006-10-18

Dina Gould Halme , David A. Kessler

The authors review existing regulations regarding cell and tissue products and discuss how they expect the Food and Drug Administration to apply these regulations to scientists' efforts to develop and … The authors review existing regulations regarding cell and tissue products and discuss how they expect the Food and Drug Administration to apply these regulations to scientists' efforts to develop and test stem-cell–based therapies.

Genetic risk and the birth of the somatic individual

2000-01-01

Carlos Novas , Nikolas Rose

This paper considers the implications of the rise of the new molecular genetics for the ways in which we are governed and the ways in which we govern ourselves. Using … This paper considers the implications of the rise of the new molecular genetics for the ways in which we are governed and the ways in which we govern ourselves. Using examples of genetic screening and genetic discrimination in education, employment and insurance, and a case study of debates among those at risk of developing Huntington's Disease and their relatives, we suggest that some of the claims made by critics of these new developments are misplaced. While there are possibilities of genetic discrimination, the key event is the creation of the person 'genetically at risk'. But genetic risk does not imply resignation in the face of an implacable biological destiny: it induces new and active relations to oneself and one's future. In particular, it generates new forms of 'genetic responsibility', locating actually and potentially affected individuals within new communities of obligation and identification. Far from generating fatalism, the rewriting of personhood at a genetic level and its visualization through a 'molecular optic' transforms the relations between patient and expert in unexpected ways, and is linked to the development of novel 'life strategies', involving practices of choice, enterprise, self-actualization and prudence in relation to one's genetic make-up. Most generally, we suggest, the birth of the person 'genetically at risk' is part of a wider reshaping of personhood along somatic lines and a mutation in conceptions of life itself.

World Medical Association Declaration of Helsinki (WMA) - Ethical principles for medical research involving human subjects

2014-01-01

Domján Andrea , Péter Kakuk , Sándor Judit

Medical Nemesis: The Expropriation of Health

1977-10-01

Elliott A. Krause , Iván Illich

An introduction to the study of experimental medicine

1958-05-01

Louis Lasagna

The United States Pharmacopeial Convention

1910-02-24

2002 international ethical guidelines for biomedical research involving human subjects

2003-02-20

Sue Eckstein

Guideline 1: Ethical justification and scientific validity of biomedical research involving human beings Guideline 1: Ethical justification and scientific validity of biomedical research involving human beings

Ethical principles for medical research involving human subjects

2003-02-20

Sue Eckstein

Adopted by the 18th WMA General Assembly Helsinki, Finland, June 1964 and amended by the 29th WMA General Assembly, Tokyo, Japan, October 1975; 35th WMA General Assembly, Venice, Italy, October … Adopted by the 18th WMA General Assembly Helsinki, Finland, June 1964 and amended by the 29th WMA General Assembly, Tokyo, Japan, October 1975; 35th WMA General Assembly, Venice, Italy, October 1983; 41st WMA General Assembly, Hong Kong, September 1989; 48th WMA General Assembly, Somerset West, Republic of South Africa, October 1996 and the 52nd WMA General Assembly, Edinburgh, Scotland, October 2000

The burden of musculoskeletal diseases in the United States

2016-07-27

Edward H. Yelin , Stuart L. Weinstein , Toby King

Pharmaceutics: The science of dosage form design

1989-03-01

Nicholas A. Peppas

FDA approves 100th monoclonal antibody product

2021-05-05

Asher Mullard

Principles for the Buffering of Genetic Variation

2001-02-09

John L. Hartman

An anthropology of biomedicine

2011-04-01

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Biological Relatives

2013-10-16

Sarah Franklin

Thirty-five years after its initial success as a form of technologically assisted human reproduction, and five million miracle babies later, in vitro fertilization (IVF) has become a routine procedure worldwide. … Thirty-five years after its initial success as a form of technologically assisted human reproduction, and five million miracle babies later, in vitro fertilization (IVF) has become a routine procedure worldwide. In Biological Relatives, Sarah Franklin explores how the normalization of IVF has changed how both technology and biology are understood. Drawing on anthropology, feminist theory, and science studies, Franklin charts the evolution of IVF from an experimental research technique into a global technological platform used for a wide variety of applications, including genetic diagnosis, livestock breeding, cloning, and stem cell research. She contends that despite its ubiquity, IVF remains a highly paradoxical technology that confirms the relative and contingent nature of biology while creating new biological relatives. Using IVF as a lens, Franklin presents a bold and lucid thesis linking technologies of gender and sex to reproductive biomedicine, contemporary bioinnovation, and the future of kinship.

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“An International Journal …”

2008-02-01

M. P.R.

Millipore pays US$3.5 M in patent dispute

1998-05-01

Declaration of Helsinki. Ethical Principles for Medical Research Involving Human Subjects

2014-06-01

Solexa Ltd

2004-05-28

Simon T. Bennett

Solexa Ltd is developing an integrated system, based on a breakthrough single molecule sequencing technology, to address a US$2 billion market that is expected to grow exponentially alongside and as … Solexa Ltd is developing an integrated system, based on a breakthrough single molecule sequencing technology, to address a US$2 billion market that is expected to grow exponentially alongside and as a consequence of further technological enhancements. The system, software and consumables will initially be sold to research organizations, pharmaceutical companies and diagnostic companies that will sequence large regions of genomic DNA, including whole genomes, at costs several orders of magnitude below current levels. Solexa expects to launch its first product in 2006, and as it continues to make time and cost efficiencies, additional products will be launched into the expanding markets that will have broad applications in basic research through to healthcare management.

Declaration of Helsinki Ethical Principles for Medical Research Involving Human Subjects

2009-04-01

World Medical Association. World Medical Association Declaration of Helsinki Ethical Principles for Medical Research Involving Human Subjects

2004-01-01

Declaration of Helsinki. Ethical Principles for Medical Research Involving Human Subjects

2009-12-24

World Medical Association

World Medical Association Declaration of Helsinki

2013-10-19

1. The World Medical Association (WMA) has developed the Declaration of Helsinki as a statement of ethical principles for medical research involving human subjects, including research on identifiable human material … 1. The World Medical Association (WMA) has developed the Declaration of Helsinki as a statement of ethical principles for medical research involving human subjects, including research on identifiable human material and data.

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DBG Tissue and DNA Bank

2021-01-01

Rick Levy

The Denver Botanic Gardens' Tissue and DNA Bank is primarily comprised of silica-gel dried plant tissue samples. Plants represented are typically from the Southern Rocky Mountain Region. Tissue samples are … The Denver Botanic Gardens' Tissue and DNA Bank is primarily comprised of silica-gel dried plant tissue samples. Plants represented are typically from the Southern Rocky Mountain Region. Tissue samples are collected as part of floristic survey or population genetics study.

Life as surplus: biotechnology and capitalism in the neoliberal era

2008-07-01

Melinda Cooper

Acknowledgments Introduction 1. Life Beyond the Limits: Inventing the Bioeconomy 2. On Pharmaceutical Empire: AIDS, Security, and Exorcism 3. Preempting Emergence: The Biological Turn in the War on Terror Intermezzo … Acknowledgments Introduction 1. Life Beyond the Limits: Inventing the Bioeconomy 2. On Pharmaceutical Empire: AIDS, Security, and Exorcism 3. Preempting Emergence: The Biological Turn in the War on Terror Intermezzo 4. Contortions: Tissue Engineering and the Topological Body 5. Labors of Regeneration: Stem Cells and the Embryoid Bodies of Capital 6. The Unborn Born Again: Neo-Imperialism, the Evangelical Right, and the Culture of Life Epilogue Notes References Index

19. Department of Internal Medicine

1999-12-31

Haruto Uchino , Noboru Ozono , N. Tanaka

Basic Points to Consider for Cell Storage under the Act on the Safety of Regenerative Medicine in Japan

2025-06-25

Yuki Uno , Kazuaki Nakamura , Morikuni Tobita +4 more | Regenerative Therapy

Spotlight in Plastic Surgery: July 2025

2025-06-25

Brett T. Phillips , Aslan Baradaran , Tara M. Chadab +7 more | Plastic & Reconstructive Surgery

Cardiac function and mortality of stem cell therapy in patients with coronary artery disease who underwent coronary artery bypass graft without heart failure: A meta‑analysis

2025-06-24

Thanut Jansirirat , Sittichai Khamsai , Kittisak Sawanyawisuth | Biomedical Reports

Biomarcadores de última generación y terapias dirigidas en el manejo del cáncer cervical

2025-06-24

Lizette Elena Leiva Suero , Anthony Fernando González Asqui | CienciAmérica

INTRODUCTION. Cervical cancer is the fourth leading cause of cancer-related morbidity and mortality among women worldwide. Human papillomavirus (HPV) infection is recognized as the primary etiological agent, with genotypes 16 … INTRODUCTION. Cervical cancer is the fourth leading cause of cancer-related morbidity and mortality among women worldwide. Human papillomavirus (HPV) infection is recognized as the primary etiological agent, with genotypes 16 and 18 being the most prevalent, accounting for 71% of cervical cancer cases. OBJECTIVE. To evaluate the available scientific evidence regarding advances in biomarkers and targeted therapies for cervical cancer. METHODS. A systematic literature review was conducted using the PRISMA methodology by searching for articles in the WOS, Scopus, PubMed, ProQuest, Embase, Redalyc, Ovid, Medline, DynaMed, and ClinicalKey databases covering the period from 2017 to 2025. RESULTS. The systematic review yielded a total of 31,431 records, of which 95 were deemed eligible; among these, 50 did not provide new data, resulting in the inclusion of 45 scientific articles. DISCUSSION AND CONCLUSIONS. Biomarkers such as miRNAs, lncRNAs, and circRNAs are being used for diagnostic and prognostic purposes in cervical cancer. Likewise, novel immunotherapies and targeted treatments—including monoclonal antibodies, PARP inhibitors, immune checkpoint inhibitors (anti-PD-1/PD-L1, anti-CTLA-4), and vaccines—have shown promising results in patients with advanced-stage cervical cancer.

BIOPOLITICS IN THE AGE OF GLOBAL PANDEMICS:

2025-06-24

Tony See Sin Heng | Anthem Press eBooks

Geopolitical Data Barriers in Biomedicine: Resolving Sino–U.S. Regulatory Conflicts and Compliance Challenges Under Executive Order 14117

2025-06-23

Tian Mao , Peng Feirong | Biotechnology Law Report

Q&A: Xiaoxing Xi on the wrongful arrest that upended his research and his life

2025-06-20

Toni Feder | Physics Today

Oral habit-breaking product wins award

2025-06-20

| BDJ Team

Public Health Implications of Legal Challenges to the FDA’s Regulation of Laboratory-Developed Tests

2025-06-20

Kushal T. Kadakia , Joseph S. Ross , Reshma Ramachandran | JAMA Health Forum

Importance Laboratory-developed tests (LDTs) refer to in vitro diagnostics developed and used by individual laboratories. LDTs are widely used in modern medicine, with their results informing over 70% of clinical … Importance Laboratory-developed tests (LDTs) refer to in vitro diagnostics developed and used by individual laboratories. LDTs are widely used in modern medicine, with their results informing over 70% of clinical decisions. The US Food and Drug Administration (FDA) has historically claimed regulatory authority over LDTs and, in 2024, finalized new regulations to strengthen oversight of these products. However, the FDA’s rulemaking was vacated in the 2025 court case American Clinical Laboratory Association et al v FDA et al, a decision that has carried substantial implications for public health. Observations The FDA has possessed oversight of in vitro diagnostic tests, including LDTs, since the US Congress passed the Medical Device Amendments Act of 1976. Due to their limited use, the FDA initially exempted LDTs from formal requirements for premarket clinical testing and regulatory review. These regulatory flexibilities enabled substantial innovation in diagnostic technology, enabling the development of LDTs for applications including routine clinical care, complex molecular testing, and rapid response during public health emergencies. However, the exponential growth of LDTs in clinical practice despite little to no clinical evidence of safety and effectiveness has raised public health concerns from the FDA and Congress, with subsequent investigations identifying substantial issues related to test quality and performance. These findings motivated the FDA to issue a new rule expanding its risk-based approach to LDT regulation that was subsequently challenged in the US District Court for the Eastern District of Texas by the American Clinical Laboratory Association and the Association of Molecular Pathology. The court ruled in favor of the plaintiffs and moved to vacate the FDA’s LDT rule, asserting that LDTs constituted services, not products, and were therefore beyond the scope of the FDA’s medical device authorities. Applying the Supreme Court’s recent decision to overturn the Chevron doctrine that deferred to agency interpretation in implementing statute, the court also ruled that the FDA could not regulate LDTs without the express authorization of Congress. Conclusions and Relevance The case of LDTs illustrates the challenges the FDA faces when adapting regulatory frameworks in response to emerging health technologies. The outcomes of recent reforms and litigation carry substantial public health implications for both diagnostic technologies and the FDA’s broader regulatory remit.

Exploring Alternatives to Animal Testing: Scientific, Legal and Ethical Challenges in Developing Novel Alternative Methods with a Focus on Organoids as Potential NAMs

2025-06-20

Inesa Fausch , Daniel Zeyer-Iyengar , Rosa Maria Cajiga Morales +3 more | LEOH - Journal of Animal Law Ethics and One Health

Organoids are multicellular, three-dimensional structures derived from stem cells. They require an extracellular matrix and are capable of recapitulating cell types, organ structure, and organ function. Their characteristics indicate the … Organoids are multicellular, three-dimensional structures derived from stem cells. They require an extracellular matrix and are capable of recapitulating cell types, organ structure, and organ function. Their characteristics indicate the potential for a wide range of applications. Moreover, they have the potential to promote and serve the 3R principle – i.e. the replacement, reduction and refinement of animal testing. To this end, however, organoids have to be accepted as so-called novel alternative methods or new approach methodologies (NAMs). NAM’s main objective is the promotion of the 3R using non-animal research methods. In this paper, we outline and analyze the legal and regulatory framework that governs the 3R-principle, the use of NAMs as well as the use of organoids using the example of Swiss law. In doing so, we identify concrete important limitations on the use of organoids as NAMs: their validation requires substantive financial and personal commitments and even a successful validation does not lead to their widespread adoption. Against this background, the paper makes first proposals to promote the adoption of organoids as NAMs.

Toward equitable regenerative medicine: a health equity research framework for emerging regenerative treatments

2025-06-20

Mohamed Addani , Lindsay R. Beaman , Goldie S. Byrd +2 more | Regenerative Medicine

Regenerative medicine continues to advance rapidly, yet research examining health disparities within this field remains notably limited. The distinctive characteristics of regenerative therapies - complex manufacturing, specialized delivery, and personalized … Regenerative medicine continues to advance rapidly, yet research examining health disparities within this field remains notably limited. The distinctive characteristics of regenerative therapies - complex manufacturing, specialized delivery, and personalized approaches - present unique challenges for equitable health care access beyond traditional disparities. This gap is concerning as many conditions targeted by regenerative treatments disproportionately affect underserved populations. Our analysis of the literature identifies knowledge gaps and demonstrates the critical need for a multi-level approach to health equity research in regenerative medicine. We propose a research framework involving three key steps. First: systematic investigation of field-specific access barriers through established health disparity frameworks, focusing on factors uniquely relevant to regenerative therapies across individual, interpersonal, organizational, community, and societal levels. Second: development and rigorous evaluation of targeted, multi-level interventions addressing these barriers. Third: implementation of evidence-based strategies using equity-focused frameworks to translate findings into practice, ensuring equity considerations are embedded throughout development and delivery processes. By prioritizing health equity research while many regenerative treatments are still developing, the field has an unprecedented opportunity to create inclusive access pathways and ensure its potentially revolutionary therapies benefits all populations.

New AAN Report Reviews the State of Evidence on Gene Therapy Drug for Duchenne Muscular Dystrophy

2025-06-19

Susan Fitzgerald | Neurology Today

Regulatory data protection for repurposed medicinal products

2025-06-19

Żaneta Zemła‐Pacud | Edward Elgar Publishing eBooks

Exclusivity versus confidentiality of regulatory data

2025-06-19

Żaneta Zemła‐Pacud | Edward Elgar Publishing eBooks

Regulatory protection in the pharmaceutical and life science industries: an introduction

2025-06-19

Żaneta Zemła‐Pacud | Edward Elgar Publishing eBooks

The CorNeat Everpatch and the Process of FDA Authorization

2025-06-19

Risa Jampel , Henry Jampel | Ophthalmology

RCR open

2025-06-18

Daniel Bell | Radiopaedia.org

Detailed revisions to the subordinate regulation following the amendment of the Act on the Safety of Regenerative Medicine in Japan

2025-06-18

Satoshi Hosoya , Yuichiro Ukon , Kazuki Morita +3 more | Regenerative Therapy

Dermatologist prescriptions for biologics contribute to thousands of tons of plastic waste

2025-06-17

Micha Nouafo , Gabrielle Rivin , Alan B. Fleischer | Dermatology Online Journal

Zhaomin Xu, MD, MPH

2025-06-17

Scott R. Steele | Clinics in Colon and Rectal Surgery

Overview of Cellular Therapeutics Clinical Trials: Advances, Challenges, and Future Directions

2025-06-16

Minzhe Guo , Bingyi Zheng , Xiaoling Zeng +2 more | International Journal of Molecular Sciences

Cellular therapeutics, encompassing stem cell-based regeneration and engineered immune cell platforms, have demonstrated efficacy in treating degenerative diseases, immune-related diseases, and oncology. However, low engraftment rates and limited long-term efficacy … Cellular therapeutics, encompassing stem cell-based regeneration and engineered immune cell platforms, have demonstrated efficacy in treating degenerative diseases, immune-related diseases, and oncology. However, low engraftment rates and limited long-term efficacy remain critical translational barriers. This review compiled clinical projects on cell therapy in China over the past five years (over 1200 patients across 172 clinical trials) to highlight its rapid development in recent years and illustrate the directions of indications for application. This review also analyzes published clinical achievements all over the world, revealing significant therapeutic improvements in degenerative disorders (40-60% improvement in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) scores and oncology (78% ctDNA clearance, p < 0.001)). We propose integrating traditional Chinese medicine (TCM) bioactive compounds to enhance cell viability via C-X-C motif chemokine receptor (CXCR4) upregulation and mitochondrial biogenesis. Despite mechanistic insights, translational barriers include limited TCM validation (72% lacking single-cell omics) and regulatory misalignment. Future efforts should prioritize randomized trials and standardized TCM-cell therapy protocols to bridge discovery and clinical translation.

Author Correction: Identification of stable housekeeping genes for induced pluripotent stem cells and -derived endothelial cells for drug testing

2025-06-16

Sheena L. M. Ong , Hans J. Baelde , David G.P. van IJzendoorn +2 more | Scientific Reports

CRISPR-based gene and cell therapy designs to improve treatment efficacy and safety for patients with genetic disorders, infectious diseases and cancer

2025-06-16

Dania Aziz | Journal of Rehman Medical Institute

“Clustered Regularly Interspaced Short Palindromic Repeats” (CRISPR) genome editing technology has revolutionized molecular biology and therapeutic development since its adaptation from a bacterial immune defense mechanism. This technological innovation has … “Clustered Regularly Interspaced Short Palindromic Repeats” (CRISPR) genome editing technology has revolutionized molecular biology and therapeutic development since its adaptation from a bacterial immune defense mechanism. This technological innovation has opened new avenues for treating previously intractable diseases, from correcting monogenic disorders like sickle cell disease to engineering immune cells for cancer immunotherapy and targeting persistent viral infections. The objective of this review was to study the role of CRISPR-based gene and cell therapy in the treatment of cancer, genetic and infectious disorders and its implementations to improve the treatment efficacy. A comprehensive search was carried out using data bases such as Cochrane, PubMed and Google Scholar which identified relevant studies published between 2014 and 2024. After screening and applying inclusion-exclusion criteria,18 articles were selected. CRISPR/Cas9, a genome editing tool from bacterial immune systems, shows promise for treating cancer, genetic and infectious diseases. It holds potential for enhancing T-cell therapies and fighting viruses like HPV, hepatitis B and C, and EBV. Challenges include cell toxicity, immune responses, off-target impacts and delivery methods. CRISPR/Cas9 is transforming the way genome is edited and is critical in the progress of cancer research, therapy, and management of infectious diseases. Novel applications include universal CAR T-cells precision gene correction and the elimination of HIV reservoirs. While it holds great promise, many issues still need to be addressed, such as off-target effects and how therapeutic genes are delivered into cells.

Regulating the unseen hand: ai, authorship, and trust in medical science

2025-06-16

Naveen Gautam | Annals of Medicine and Surgery

La medicina regenerativa como alternativa terapéutica para enfermedades crónicas y lesiones graves: Avances, desafíos y aplicaciones clínicas

2025-06-16

Emily Juliet Cueva Criollo | Revista Multidisciplinaria Investigación Contemporánea

In recent years, regenerative medicine has seen remarkable progress and the emergence of new applications, as well as significant challenges. This review article aims to analyze the advances, clinical applications, … In recent years, regenerative medicine has seen remarkable progress and the emergence of new applications, as well as significant challenges. This review article aims to analyze the advances, clinical applications, and challenges in regenerative medicine to drive further development and research in this field. The research adopted a qualitative approach and followed the systematic review protocol based on the PRISMA 2020 guidelines. Key advances include 3D bioprinting and cellular reprogramming, which have enabled the creation of functional tissues and organs in the laboratory, as well as the development of therapies to regenerate musculoskeletal tissues and vital organs from stem cells. Although many experimental studies on cell regeneration face challenges such as immunological compatibility and validation in terms of safety and efficacy, these studies show great potential for more effective and less invasive treatments. According to the Regenerative Medicine 2030 Report, conducted worldwide in 2023, the regenerative medicine therapy market was valued at USD 30.43 billion; and an annual growth of 16.79% is expected from 2024 to 2030. In addition, a deeper understanding of the cellular and molecular mechanisms involved in organ and tissue regeneration has been achieved. The results demonstrate significant advances in regenerative medicine through the use of stem cells, platelet-rich plasma, organoids, and 3D printing, highlighting their application in the regeneration of bone, cartilage, neuronal, skin, and ocular tissue. Ethical and technical regulatory challenges are identified, such as the need for standardization, regulatory approval, and the development of safe and effective protocols for their clinical implementation. While regenerative medicine therapies are emerging as promising therapeutic alternatives for chronic diseases and serious injuries, continued interdisciplinary research is essential to advance their development and consolidate a future in which these conditions can be treated more efficiently and with less invasive methods, thus transforming medical practice

Precision Medicine and the Price of Equity

2025-06-16

Kenna McRae , Josiah Yarbrough | Journal of Science Policy & Governance

Medicine has increasingly moved toward more personalized approaches that utilize individual patient characteristics to tailor therapeutic interventions and predict outcomes. While advancements in precision medicine have garnered significant attention, the … Medicine has increasingly moved toward more personalized approaches that utilize individual patient characteristics to tailor therapeutic interventions and predict outcomes. While advancements in precision medicine have garnered significant attention, the ways that this budding subset of technology intersects with existing health disparities are often overlooked. Precision medicine holds tremendous potential to transform healthcare, but it risks widening existing disparities if not managed inclusively. We highlight three core issues that further marginalize underserved communities: high costs, limited accessibility of precision medicine, and insufficient diversity in research that produces these innovations. We also propose opportunities for state and federal policymakers to mitigate these issues. Without deliberate policy interventions, these innovations may remain accessible only to privileged demographics, excluding underserved communities from life-saving treatments. Key policy recommendations include restructuring reimbursement frameworks, reducing drug prices, fostering diversity in clinical trials, and building strong community partnerships. These measures are essential to transforming precision medicine from a selective advantage to a universally accessible health benefit.

The political economy of compulsory licensing: democracy and regulatory threat in public health

2025-06-15

Sojun Park | Public Choice

Regulatory framework and challenges for live biotherapeutic products in Taiwan

2025-06-13

Shirley Pan , Jia-Chuan Hsu , Kuo-Teng Hung +1 more | Journal of Food and Drug Analysis

493-P: Risk Factors for Failure of Autologous Cell Therapy in Patients with Diabetes and Chronic Limb-Threatening Ischemia

2025-06-13

Dominika Sojáková , Michal Kahle , Jitka Husáková +4 more | Diabetes

Introduction and Objective: Autologous cell therapy (ACT) is a revascularision method for patients with no-option chronic limb-threatening ischemia (CLTI). The aim of our study was to identify patients who benefit … Introduction and Objective: Autologous cell therapy (ACT) is a revascularision method for patients with no-option chronic limb-threatening ischemia (CLTI). The aim of our study was to identify patients who benefit most from ACT and to analyze factors responsible of failure of this therapy. Methods: We performed 159 treatments of 145 limbs in 132 patients with no-option CLTI treated in our center over 15 years. During ACT the patient's bone marrow is harvested to manufacture the final cell product, which is injected into the calf muscles of the ischemic limb. We analyzed all baseline parameters obtained before ACT and compared them with amputation-free survival (AFS). Baseline parameters (BP) were divided into three groups: patent-related BP, ischemia-related BP and ulcer - related BP. Risk factors were analyzed using Kaplan-Meier estimate and Cox proportional-hazard model. Results: Major amputations were necessary to perform in 48 limbs (33.1 %) and 60 (45,5 %) patients died during a 15-year follow-up. The most significant baseline factors for shorter AFS were severe stages of chronic kidney disease (HR 1.23, CI 1.08 - 1.39), severe stages of foot infection in accordance with WIfI classification (HR 1.31, CI 1.05 - 1.64), and the presence of bacteria resistant to oral antibiotics (HR 1.65, CI 1.14 - 2.39). Conclusion: Our study showed that ACT is an effective method for diabetic patients with CLTI. Patients with no kidney damage, without infected diabetic foot, and without resistant bacteria have the greatest chance of success with this treatment. Disclosure D. Sojáková: None. M. Kahle: None. J. Husáková: None. V. Fejfarova: None. R. Jarosikova: None. K. Sutoris: Speaker's Bureau; Bonalive Biomaterials. M. Dubsky: None. Funding Supported by the project CarDia (Programme EXCELES, Project No. LX22NPO5104) - Funded by the European Union - Next Generation EU.

395-P: Dynamically Predicting Renal Failure after Development of Diabetes for Millions across Biobanks

2025-06-13

A. E. Jensen , Sayera Dhaubhadel , Gang Li +4 more | Diabetes

Introduction and Objective: Despite advances in diabetes (DM) care, complications remain high, underscoring the need for better risk prediction. We developed a risk engine in the Veterans Health Administration (VA), … Introduction and Objective: Despite advances in diabetes (DM) care, complications remain high, underscoring the need for better risk prediction. We developed a risk engine in the Veterans Health Administration (VA), validated it in All of Us (AoU), and compared it with Risk Equations for Complications Of Type 2 Diabetes (RECODe). Methods: We analyzed newly diagnosed VA patients using Fine-Gray sub-distribution hazard models for renal failure at 1, 5, and 10-year landmarks (LM), with all-cause mortality as a competing risk. We used a two-step feature selection (logistic regression followed by a minimax concave penalty) to choose variables at LM1, then re-fitted unpenalized models at LM5 and LM10. Medication/comorbidity histories, baseline covariates, and biomarker trends were included. Results: Of the 815,429 newly diagnosed patients at LM1, 3.27% developed renal failure (median age 62; time to renal failure 8.0 years from DM), while 29.2% died without renal failure. At LM1, AUROCs for predicting 1-/5-/10-year renal failure were 0.86, 0.82, and 0.79 in VHA; 0.86, 0.85, and 0.80 in AoU; and 0.87, 0.76, and 0.69 for RECODe in AoU. Other landmarks showed similar performance. Conclusion: Our dynamic risk engine, which continuously updates patient risk at key intervals, significantly improves renal failure prediction among those with diabetes. Its superior discrimination compared to RECODe, with validation in AoU, suggests broad applicability for care optimization. Disclosure A. Jensen: None. S. Dhaubhadel: None. G. Li: None. H. Zhou: Consultant; Merck & Co., Inc. B. McMahon: None. P. Reaven: Research Support; Dexcom, Inc. J. Zhou: None.

556-P: NextGen Fellowship—Strengthening Career Pathways in Diabetes

2025-06-13

P. Bischoff , MARY JANE ROCHE | Diabetes

Introduction and Objective: Current trends in the decreasing number of endocrinologists, the consistency in diabetes funding from the NIH, and the rising prevalence and costs of diabetes elucidate the importance … Introduction and Objective: Current trends in the decreasing number of endocrinologists, the consistency in diabetes funding from the NIH, and the rising prevalence and costs of diabetes elucidate the importance of expanding the pipeline for future leaders in diabetes, encouraging them to consider diabetes care, research, and advocacy as career options. The Diabetes Link, a non-profit committed to supporting young adults with diabetes, offers the NextGen Fellowship (“NextGen”) to provide mentorship, educational sessions, conference attendance, and a network of peers to cultivate the “next-generation” of diabetes leaders. Aim: To assess the effectiveness in educating and proving a clear career pathway of the 2024 NextGen Cohort. Methods: Fellows completed surveys before and after the fellowship which included likert-style questions, ranking activities, and qualitative feedback. These questions polled demographic information; diabetes care, research, and advocacy knowledge; main takeaways of the fellowship; and knowledge of diabetes organizations. T-tests to explore significance before and after the fellowship and among different demographics were used as statistical tests. Results: Fifteen fellows were surveyed across different ages, backgrounds, and career paths, and analyses of their surveys revealed significant increases in diabetes research knowledge (p &lt;.05), advocacy knowledge (p &lt;.001), and familiarity with diabetes organizations (p &lt;.05). Improvements in career confidence, cultural care understanding, and education knowledge were observed, but did not reach statistical significance. Meeting peers along a similar career pathway also positively contributed to fellows’ experience. Conclusion: NextGen provides a strong foundation for young adults interested in pursuing a career in diabetes. This illuminates the importance of providing early career professionals interested in diabetes with experience, equipping them for success in the field and effectively expanding the pipeline of young talent entering the diabetes workforce. Disclosure P. Bischoff: None. M. Roche: None.

1864-LB: Novel Diabetic Kidney Disease Endotypes Derived from Matched Clinical and Multiomics Analysis Reveal Dysregulated Molecular Drivers of Fast Progression

2025-06-13

KARINA GUTHERIDGE , Hannah Alphs Jackson , Andrew Parton +5 more | Diabetes

Introduction and Objective: Diabetic kidney disease (DKD) is a multifactorial, heterogeneous chronic disease. Despite this, current care largely follows a one-size-fits-all approach. We used longitudinal electronic health records and matched … Introduction and Objective: Diabetic kidney disease (DKD) is a multifactorial, heterogeneous chronic disease. Despite this, current care largely follows a one-size-fits-all approach. We used longitudinal electronic health records and matched multi-omics data to identify clinically meaningful DKD endotypes, with distinct molecular mechanisms, to support precision medicine drug discovery. Methods: We analysed data from 450 T2D patients with stage 3/4 CKD from the NHS, UK Salford Kidney Study. Machine learning-enabled time series analysis of longitudinal biochemical data on kidney function and anaemia (median follow-up: 4.5 years) was performed to discover DKD endotypes. Disease progression risk was assessed via Cox proportional hazards models. Metabolomic (Biocrates & Nightingale), proteomic (Olink), and DNA methylation (Infinium MethylationEPIC v1.0) profiles were analysed using linear models, co-expression network analysis, and over-representation analysis for differential enrichment of molecular signals between endotypes. Results: Two distinct DKD endotypes were identified: slow and fast progressors, with significantly different risks for progression to end-stage renal disease or renal replacement therapy (A1vA2: HR=26.9, 95% CI 10.4-69.4, P=9.7×10-12). Fast progressors showed multi-omic enrichment in disease-relevant pathways, including well-evidenced drivers of inflammation and DKD e.g. TNF receptor activity, FDR=3.2×10-4. We also identified novel associations warranting future investigation, including enrichment for dysregulated fatty acid processing (FDR=6.0×10-3) e.g. driven by DBI (FDR=2.1×10-3) and FABP5 (FDR=2.6×10-3) up-regulation. Conclusion: By integrating longitudinal biochemical, clinical, and multi-omics data, we identified novel DKD endotypes with significant prognostic value and distinct molecular signatures. These findings offer insights for precision medicine to improve DKD progression and patient outcomes. Disclosure K. Gutheridge: None. H.R. Jackson: None. A. Parton: None. H. Bull: None. V. Kuzmuk: None. M.L. Sierra: None. P. Kalra: Speaker's Bureau; Pharmacosmos. Advisory Panel; Medice, CSL Vifor. A. Cohain: None.

Abstract P4-04-04: Leveraging Technology to Improve Access to Clinical Trials in Underrepresented Populations within a Safety-Net Institution

2025-06-13

Melissa Howell , Dedra L. Preece , David E. Gerber +2 more | Clinical Cancer Research

Abstract Background: Clinical trials should be accessible to all patients regardless of race, ethnicity and socio-economic status. In the US, Blacks constituted 4-6% and Hispanics 3-6% of participants in cancer … Abstract Background: Clinical trials should be accessible to all patients regardless of race, ethnicity and socio-economic status. In the US, Blacks constituted 4-6% and Hispanics 3-6% of participants in cancer therapeutic trials despite representing 15% and 13% of people with cancer. US Food and Drug Administration draft guidance from April 2022 calls for the improvement of clinical trial enrollment of participants from historically excluded racial and ethnic populations. Safety-net healthcare systems like John Peter Smith Health Network (JPS) in Tarrant County, TX, serve predominantly racial ethnic minorities. At JPS, recruitment into oncology therapeutic clinical trials remains pitifully low. Efficiently identifying patients who meet eligibility criteria in an understaffed, busy clinic in the non-academic setting has been a challenge. Referral responsibility lands on overwhelmed providers or outside companies who lack the ability to effectively screen patients. Therefore, we piloted a program leveraging the expertise of our Information Technology business intelligence (IT) team to support our clinical research team to improve recruitment efforts for an oncology trial. Methods: Collaboration between IT and research teams was established on 5/1/24 with a goal to identify patients for this trial: EMBER-4: A Randomized, Open-Label, Phase 3 Study of Adjuvant Imlunestrant vs Standard Adjuvant Endocrine Therapy in Patients who have Previously Received 2 to 5 years of Adjuvant Endocrine Therapy for ER+, HER2- Early Breast Cancer with an Increased Risk of Recurrence. This study was obtained by JPS via the Cancer Prevention and Research Institute of Texas Clinical Trials Network Award (CPRIT-CTNA) partnership. Using the inclusion and exclusion criteria and applying the Microsoft Structured Query Language (SQL) Service Management Studio software, queries and parameters were developed and run against the Epic Clarity database of patients seen by 15 providers spanning medical oncology, radiation oncology and survivorship clinics, to extract the requested dataset of potentially eligible patients. Results: IT team identified 123 potential eligible patients (pre-screened) for EMBER-4 research study as of 6/5/24. EMBER-4 was open for accrual at JPS on 6/18/24. A roster of pre-screened patients’ next scheduled clinic appointments was also provided by the IT team. Of the 9 pre-screened patients in the first week, 4 qualified for Ember-4, and successfully enrolled two Hispanic patients. 1 of the remining 2 patients is slated to be enrolled into Ember-4 in the third week. Manual screening of all patients in providers’ clinics with a high proportion of breast cancer is performed weekly in the first month to validate IT list. Validation efforts have reflected that the current list from the IT collaboration has indeed included all possible patients for EMBER-4 trial. Pre-screening has been completed on 47 of the 123 potential patients thus far. Our efforts have yielded an additional 4 eligible patients for enrollment in the next 6 weeks for a total of 8 patients meeting all eligibility criteria within the first 9 weeks. Conclusion: These efforts have empowered the clinical research team with statistics regarding diversity among study populations and ability to better track and engage all appropriate patients for the EMBER-4 study. Leveraging the IT team’s expertise to collaborate with clinical research team is a novel method: it has resulted in precision screening capabilities with immediate possibility of increasing enrollment in therapeutic oncology research studies. This method of patient identification can be done in tandem with opening clinical research studies and will be used for future studies at JPS not only in oncology but other specialties as well. We plan to introduce this concept to other CPRIT-CTNA sites. Citation Format: Melissa Howell, Dedra L. Preece, David E. Gerber, Jerry D. Henderson, Kalyani Narra. Leveraging Technology to Improve Access to Clinical Trials in Underrepresented Populations within a Safety-Net Institution [abstract]. In: Proceedings of the San Antonio Breast Cancer Symposium 2024; 2024 Dec 10-13; San Antonio, TX. Philadelphia (PA): AACR; Clin Cancer Res 2025;31(12 Suppl):Abstract nr P4-04-04.

Abstract P4-02-28: Decisional challenges regarding risk-reducing surgery for BRCA1/2 and PALB2 women

2025-06-13

Catarina Relvas , Sofia Fragoso , Madalena Machado +7 more | Clinical Cancer Research

Abstract Women with hereditary breast and ovarian cancer syndrome (HBOC) have an increased risk of breast (BC) and ovarian cancer (OC) (absolute risk &gt; 60% and 39–58% for BRCA1-2 respectively). … Abstract Women with hereditary breast and ovarian cancer syndrome (HBOC) have an increased risk of breast (BC) and ovarian cancer (OC) (absolute risk &gt; 60% and 39–58% for BRCA1-2 respectively). Options for risk management include surveillance and risk reducing surgery (RRS). Previous data reported up to 90-95% BC risk reduction after RR bilateral breast surgery (RRBBS). RR salpingo-oophorectomy (RRSO), besides a risk reduction effect on OC, impacts on overall survival for BRCA1/2 women. At present, most early BC patients with a known BRCA1/2 or PALB2 pathogenic variant discuss breast RRS during their primary surgical plan. In this study we reviewed the Multidisciplinary Group (MD) decisions regarding cancer survivors (excluding BC women during primary treatment) and individuals with no previous cancer diagnosis, highly motivated for RRS. Between August 2020 - December 2023, 216 (215 F; 1 M) pts (median age 49 yrs) were discussed in the MD group. Genetic testing (GT): BRCA1/2 (69%), PALB2 (5%), other genes (19%) and GT not positive (7%). Cases: 114-healthy women, 97-CA survivors (6 with advanced CA) and 5 considered ineligible for RRS (1-NF1; 4-not positive GT). Healthy women: the MD group agreed on RRBBS, RRSO or both in 37, 42 and 35 cases, respectively. After further individual discussion 5 (14%RRBBS), 2 (5%RRSO) and 6 (17% both) women refused RRS. Four RRBBS women (3 BRCA2; 1 PALB2) were diagnosed with unilateral BC in the preoperative MRI, 1 RRSO women had OC diagnosis previous to surgery. Surgical specimens of interest (5): 1- lobular invasive BC (pT1bN0), 2- DCIS, 2- STIC (1 bilateral). Survivors were mostly (84/87%) BC survivors. While considering RRBBS, RRSO or both in 17, 49 and 25 cases, respectively, 2 (12%RRBBS), 2 (4%RRSO) and 3 (10% both) pts refused RRS. Regarding the 6 pts with advanced CA, 4 had sustained CR and RRS was agreed. Four pts (3 early BC) relapsed before RRS. Surgical specimens of interest (7): 1- multifocal BC (pT1cN1mi(sn)); 2- high grade serous fallopian tube CA (1 stage III); 4- STIC. The decision of RRS is a challenge that should measure the competing risks, either regarding age of primary prevention for healthy women, or regarding risk of relapse versus another cancer. This decision should be as tailored as possible to each patient, respecting their individuality and preferences. Citation Format: Catarina Relvas, Sofia Fragoso, Madalena Machado, Margarida Pereira, Bernardo Pereira, Berta Lopez, José Duarte, Sidónia Santos, Hugo Nunes, Fátima Vaz. Decisional challenges regarding risk-reducing surgery for BRCA1/2 and PALB2 women [abstract]. In: Proceedings of the San Antonio Breast Cancer Symposium 2024; 2024 Dec 10-13; San Antonio, TX. Philadelphia (PA): AACR; Clin Cancer Res 2025;31(12 Suppl):Abstract nr P4-02-28.

457-P: Overexpression of SOD2 and P53 Gene Silencing in BM-MSCs–Rescued Hyperglycemic Endothelial Cells

2025-06-13

Seshagiri Rao Nandula , Sabyasachi Sen | Diabetes

Introduction and Objective: Diabetes is associated with endothelial dysfunction. Studies have demonstrated that CD34+ve endothelial progenitor cells (EPCs) and mature endothelial cells such as HUVEC, both are susceptible to apoptosis … Introduction and Objective: Diabetes is associated with endothelial dysfunction. Studies have demonstrated that CD34+ve endothelial progenitor cells (EPCs) and mature endothelial cells such as HUVEC, both are susceptible to apoptosis in hyperglycemic (HG) environment. EPC more than HUVEC. EPCs are critical for revascularization and p53 gene silencing prevents EPC senescence and helps vascular regeneration, as shown by our group. Our goal was to test whether conditioned media (CM) rather than cells itself would help regeneration. SOD2 upregulation also helps EC regeneration in HG by reducing mito-toxic ROS. We hypothesized that, CM obtained from both p53 silenced and SOD2 overexpressed human stem cells such as bone marrow derived mesenchymal stromal cells (BMMSCs) can improve EC regeneration. Methods: Ad-human-P53 (TP53)-shRNA is used to silence P53, Ad-humanSOD2 is used to overexpress SOD2 and Ad-scrambled-null-shRNA was used as control in BM-MSCs. After transduction, cells were grown in media containing 2% exosome free FBS for only 48h and the collected supernatant was concentrated 10-fold to obtain the conditioned media (CM). Endothelial regeneration was tested using endothelial wound healing assay kit ( from Cell Biolabs # CBA-120-T). Results: Both CM and exosomes derived from CM from BMMSCs achieved EC rescue in HG compared to control CM. However, the two experimental conditions did not show significant difference in the number of cells in the scratch area. Interestingly, in both the conditions there was an increase in SOD2 gene expression, on CM or related exosome addition. Conclusion: Both exosomes and unfractionated CM from SOD2 overexpression plus p53 gene silenced BM MSCs rescued the endothelial cells in hyperglycemic conditions., which further emphasizes exosomes from modified stem cells, play a therapeutic role in rescuing endothelial cells from hyperglycemic conditions by increased mitochondrial functional genes such as SOD2. Disclosure S. Nandula: None. S. Sen: None.

Abstract P5-07-08: The ACOSOG Z0011 criteria can be safely applied to Chinese patients: the SAHZU experience

2025-06-13

Xiuzhen Li , Yue Hu | Clinical Cancer Research

Abstract Background: As a world-wide known trial in the sentinel lymph node biopsy (SLNB) field, the American College of Surgeons Oncology Group (ACOSOG) Z0011 trial made a big step to … Abstract Background: As a world-wide known trial in the sentinel lymph node biopsy (SLNB) field, the American College of Surgeons Oncology Group (ACOSOG) Z0011 trial made a big step to de-escalate the axillary surgery. The original intention of this trial was to omit reoperation if the patient had cN0 breast cancer with one or two sentinel-node metastases. A lot of queries were raised to this trial and the controversies about it have never stopped. China has his unique national condition which is different to the western countries. Only methylene blue dye is available in SLNB surgeries in most of the Chinese hospitals. Frozen section is widely used and reoperation is usually unacceptable. The safety of the Z0011 criteria needs to be confirmed when it is applied during surgery and frozen section application. In this retrospective study, we shared our single-center experiences. Patients and Methods: In our hospital (SAHZU), the breast cancer patients with positive lymph nodes involvement in their SLNB surgeries were enrolled in this study since Aug. 2012 to Mar 2022. According to the Z0011 criteria, the patients with T3/T4 tumor, 3 or more positive lymph nodes involvement, mastectomy or neoadjuvant therapy were excluded. A part of the patients accepted preoperative advanced axillary evaluation (SABCS2018, Abstr. P3-03-20) which was composed by ultrasound, CT/MRI scans and fine/core needle biopsy if it was necessary. Results: Totally, 153 eligible patients were enrolled in this study. They were younger, with more ER+PR+ diseases, less vascular invasion and higher tumor grades (P&lt;0.05), compared with the Z0011 patients. More systemic therapy and lymph node radiotherapy were carried out in our cohort than in the Z0011’s. Lymph node metastases were diagnosed by frozen section during their surgery in 88.9% (136/153) of patients and the left were diagnosed by permanent H&E section. Seventy-two patients accepted axillary lymph nodes dissection (ALND) and the other 81 patients accepted SLNB. The clinicopathologic characteristics of the two groups were comparable. Median resected lymph nodes in ALND and SLNB group were 17 and 4, respectively (P&lt;0.001). In the ALND group, 27.8% patients (20/72) were with additional positive nodes and the rate was similar with the Z0011 trial. After median follow-up for 44.9 months, one local-regional recurrence happened in both groups and another two patients died in the SLNB group (P&gt;0.05). In the SLNB group, all the recurrence and death events occurred in the patients without advanced axillary evaluation (3/36 vs. 0/45, P=0.088). Conclusions: The Z0011 criteria can be safely applied during surgery in China in spite of the wide use of frozen section and single tracer of methylene blue dye. Advanced axillary evaluation before surgery may do good to the survival of the patients. Disclosure: Both authors declared no conflicts of interest. Citation Format: Xiuzhen Li, Yue Hu. The ACOSOG Z0011 criteria can be safely applied to Chinese patients: the SAHZU experience [abstract]. In: Proceedings of the San Antonio Breast Cancer Symposium 2024; 2024 Dec 10-13; San Antonio, TX. Philadelphia (PA): AACR; Clin Cancer Res 2025;31(12 Suppl):Abstract nr P5-07-08.

Abstract P1-01-12: Impact of Preoperative BRCA Testing and National Health Insurance Coverage on Surgical and Risk-reducing Decision-making in Japanese BRCA-mutated Breast Cancer Patients

2025-06-13

Kumiko Kida , Junko Takei , Misato Suzuki +7 more | Clinical Cancer Research

Abstract Introduction: BRCA1 or 2 (BRCA) germline mutations cause increased risks of breast and ovarian cancers. Therefore, preoperative BRCA testing could influence surgical strategies and preventive interventions for breast cancer … Abstract Introduction: BRCA1 or 2 (BRCA) germline mutations cause increased risks of breast and ovarian cancers. Therefore, preoperative BRCA testing could influence surgical strategies and preventive interventions for breast cancer patients with BRCA mutations. In Japan, BRCA genetic testing and risk-reducing surgeries became eligible for national health insurance coverage in 2020, previously conducted as costly private healthcare. When Japanese national insurance coverage is approved, patients' out-of-pocket expenses are reduced to 30% or less. This study aimed to investigate the impact of preoperative BRCA genetic testing and national health insurance coverage on surgical decision-making and risk-reducing surgeries among Japanese populations. Methods:Japanese women with BRCA germline pathogenic mutations and early breast cancer who underwent definitive surgery from 2004 to 2024 were retrospectively identified from institutional databases at St. Luke’s International Hospital in Tokyo, Japan. Factors including clinicopathologic information and surgical decisions were analyzed. We assessed whether undergoing BRCA testing before or after breast cancer surgery influenced the choice of surgical procedures and risk-reducing surgeries. Additionally, we examined whether approval for national health insurance coverage affected these decisions. The chi-square test was used for statistical comparisons. Results: A total of 273 breast cancer patients with germline BRCA mutations were identified (BRCA1: 120, BRCA2: 153). Among them, 167 patients (61.2%) underwent BRCA testing prior to breast cancer surgery. Surgical treatments included breast-conserving surgery (BCS) for 78 patients (28.6%) and mastectomy for 195 patients (71.4%). Among the 167 patients who learned of their BRCA mutation before breast cancer surgery, while BCS was feasible for 113 patients based on preoperative imaging, 24 patients (21.2%) chose BCS, and 89 patients (78.8%) opted for mastectomy due to their mutation results. The breast-conserving rate was 14.4% for patients with preoperative BRCA testing compared to 50.9% for those tested after surgery (p&lt;0.0001). Risk-reducing mastectomy (RRM) was performed in 115 patients (52.0%, excluding cases with bilateral breast cancers). Among patients with preoperative testing, 94 (63.1%) underwent RRM, including 83 (55.7%) who had RRM simultaneously with breast cancer surgery. Risk-reducing salpingo-oophorectomy (RRSO) was performed in 98 patients (61.3%, excluding cases with previous non-preventive oophorectomy), including 67 (41.9%) who had RRSO simultaneously with breast cancer surgery. Patients who learned of their BRCA mutation after breast cancer surgery were subsequently less likely to undergo risk-reducing surgeries compared to those with preoperative testing: 29.2% underwent RRM and 50.5% underwent RRSO (p=0.0001 and 0.097, respectively). Before national health insurance approval, 46.6% underwent preoperative BRCA testing, 28.0% underwent RRM, and 17.5% underwent RRSO simultaneously with breast cancer surgery. After approval, these rates increased to 86.9% for preoperative BRCA testing, 51.7% for RRM, and 43.0% for RRSO (p&lt;0.0001, 0.0004, and &lt;0.0001, respectively, compared between before and after approval). Conclusions: Most Japanese patients who learned of their BRCA mutation before their breast cancer surgery opted for mastectomy instead of BCS due to their mutation status. Preoperative BRCA testing and national health insurance coverage significantly increased the likelihood of undergoing risk-reducing surgeries. These findings emphasize the key role of preoperative BRCA testing and national health insurance coverage in informing surgical decisions and enhancing preventive care. Citation Format: Kumiko Kida, Junko Takei, Misato Suzuki, Megumi Okawa, Risa Kasahara, Asaka Wada, Yuka Kajiura, Fumi Akitani, Kyoko Shiota, Michiko Yamanaka, Atsushi Yoshida. Impact of Preoperative BRCA Testing and National Health Insurance Coverage on Surgical and Risk-reducing Decision-making in Japanese BRCA-mutated Breast Cancer Patients [abstract]. In: Proceedings of the San Antonio Breast Cancer Symposium 2024; 2024 Dec 10-13; San Antonio, TX. Philadelphia (PA): AACR; Clin Cancer Res 2025;31(12 Suppl):Abstract nr P1-01-12.

1058-P: The Need for Clear Ownership Laws for Medical Data Generated by Wearable Diabetes Devices

2025-06-13

David C. Klonoff | Diabetes

Introduction and Objective: No specific legislation in the United States and various EU Member States explicitly recognizes ownership rights of medical data generated from wearable diabetes devices. Clear medical data … Introduction and Objective: No specific legislation in the United States and various EU Member States explicitly recognizes ownership rights of medical data generated from wearable diabetes devices. Clear medical data ownership laws are needed to define the rights of patients as well as those who wish to use patient-generated data. Methods: We performed a PubMed literature to identify stakeholders with a claim to ownership of data generated by wearable diabetes devices, such as continuous glucose monitors, insulin pumps, and exercise trackers, as well as legal bodies in the United States and Europe with jurisdiction over ownership of medical data. Results: We identified three legal bodies with jurisdiction over ownership of patient generated diabetes data: 1) the United States Health Insurance Portability and Accountability Act 2) The United States Federal Trade Commission, and the European General Data Protection Regulation. We identified five stakeholders (patients, clinicians, researchers, public health professionals, and companies), each with unique claims and reasons (See Table). Conclusion: For solutions, increased authority by patients have to block data sharing, could make their data less available for will be for research and product development and vice versa. Clear ownership laws are needed for patient generated diabetes data so that this rapidly accumulating data can be ethically shared to maximally benefit all stakeholders in medical data ownership. Disclosure D.C. Klonoff: Consultant; Synchneuro, Thirdwayv, Tingo, Afon, embecta, Glucotrack, Lifecare, Novo Nordisk, Samsung.

Abstract P1-01-11: Use of a Digital Navigator to Deliver Patient-Centered Genetic Services in Mammography

2025-06-13

Tara Schmidlen , Emilie Simmons , Vivian Pan +7 more | Clinical Cancer Research

Abstract The National Accreditation Program for Breast Centers (NAPBC) 2024 Standards require implementation of a protocol to identify and manage patients at increased risk for breast cancer due to personal … Abstract The National Accreditation Program for Breast Centers (NAPBC) 2024 Standards require implementation of a protocol to identify and manage patients at increased risk for breast cancer due to personal risk factors or family history. In response, the University of Illinois Health partnered with Nest Genomics to implement a comprehensive digital platform to streamline and scale genetic services. The Nest Digital Patient Navigator for Hereditary Cancer Risk Assessment (HCRA) was introduced into a routine mammography setting in November 2023. To date, 4153 patients underwent HCRA with 17% meeting genetic testing (GT) criteria. Successful patient identification led to a new challenge: bottlenecks in access to genetic counseling (GC). To streamline care and empower patients to select their preferred follow-up approach, post-assessment digital navigation was implemented in January 2024 and offered to GT eligible patients. Patients receiving screening mammograms completed HCRA in the waiting room prior to imaging. Patients who met GT criteria were asked via digital navigator to choose their preferred follow-up mechanism: digital pre-test education, virtual group GC, one-on-one GC, or not interested. The digital pre-test education module was developed by genetic counselors at Nest and UIC and utilized a teach-back approach to ensure understanding of the benefits and limitations of GT, possible GT results, how results impact care, and GT logistics. A GC reviewed completed HCRAs and follow-up preferences. Patients consenting to GT after digital pretest education received a phone call and MyChart message to confirm consent and inform them the test was ordered and a saliva kit shipped. For patients requesting GC services, appointments will be scheduled. GT eligible patients not engaging with the digital navigator received standard referral and GC scheduling. In a pilot phase from January to early May 2024, 96 patients eligible for GT were sent a link to the digital navigator via text or email to capture GT follow-up preference. Only one third of those invited (n=31, 32%) engaged and only 38 (14%) selected a follow-up preference. To increase engagement, beginning mid-May, the workflow was updated such that patients meeting GT criteria select a follow-up preference immediately after completing the HCRA and only those selecting digital pretest education are sent a link via text or email. Additionally, a Spanish version of the HCRA was added at this time. These changes increased the percent of patients who selected a follow-up preference from 14% to 74%. In total, from January through June 2024, 156 patients selected a follow-up preference via digital navigator, with most (n=98, 63%) opting for digital pre-test education, 4 selecting group GC, and 54 selecting one-on-one GC. Of the 73 completing digital pre-test education, 31 consented to GT, 7 were not interested in GT, and 19 were unsure about GT. Of the 31 who consented, 26 had a test order placed, 2 had prior GT, 2 had GT in progress, and 1 declined GT after GC outreach. Of the 4 patients requesting group GC, 1 completed and had GT ordered, 1 missed and has not rescheduled, and 2 are scheduled. Of the 54 patients requesting one-on-one GC, none have been scheduled yet due to an administrative backlog. Pairing post-assessment digital navigation with HCRA allows patients to access education and GT based on individual preferences. Most patients completing digital HCRA and meeting GT criteria opted for digital pre-test education over one-on-one or group GC, allowing them to have a test ordered sooner. This digital solution has the potential to reduce bottlenecks and prioritizes limited GC availability for those who prefer one-on-one or group counseling. Further work to optimize reminders to boost engagement with the digital navigator and to improve return of sample kits to the laboratory is underway. Citation Format: Tara Schmidlen, Emilie Simmons, Vivian Pan, Neha Awati, Lara Balay, Iris Fietko, Angelina Izguera, Genesis Rios, Moran Snir, Pamela Ganschow. Use of a Digital Navigator to Deliver Patient-Centered Genetic Services in Mammography [abstract]. In: Proceedings of the San Antonio Breast Cancer Symposium 2024; 2024 Dec 10-13; San Antonio, TX. Philadelphia (PA): AACR; Clin Cancer Res 2025;31(12 Suppl):Abstract nr P1-01-11.

696-P: Teaming Up for T1D through Telehealth (Triple T)—Initial Findings from an Intervention to Support Families following T1D Diagnosis

2025-06-13

Kelsey R. Howard , Kimberly P. Garza , Nicholas David W. Smith +4 more | Diabetes

Introduction and Objective: Few psychosocial interventions exist for families in the first year after a T1D diagnosis. Initial findings from the Triple T pilot, a telehealth intervention designed to support … Introduction and Objective: Few psychosocial interventions exist for families in the first year after a T1D diagnosis. Initial findings from the Triple T pilot, a telehealth intervention designed to support family coping during this first year are reported. Methods: Inclusion criteria: child with a new diagnosis of T1D and fluency in English. Families participated in 6 telehealth visits (1, 2, 3-, 6-, 9-, and 12 months post-diagnosis) focused on supporting healthy coping and had access to a video library addressing common concerns (e.g., diagnosis disclosure, managing activities, school). Descriptive analyses of psychosocial outcomes and qualitative analyses of exit interviews are reported. Results: Thirty-one youth aged 2-17 (M=9.03; SD=4.6) and their caregivers participated. Youth were 54.8% female and identified as White (71%), Hispanic or Latinx (12.9%), Black (9.7%), or multi-racial (6.5%). The majority (77.4%) of caregivers were mothers. One-third (35.5%) of families reported an annual household income of &lt; $100,000. At baseline, many parents reported elevated levels of depression (38.7%) and anxiety (36.7%); however, fewer reported elevated symptoms at 12 months (24%). At 12 months, some youth (21.4%) and parents (13%) reported elevations in diabetes distress and no youth, but some parents (20%), reported elevated family conflict. Participants reported Triple T was helpful, providing emotional support and validation as they adjusted to life with T1D and provided resources to answer difficult questions, helped them not feel alone, built skills to help their children when they were struggling, and reduced distress. Parents and teens reported improved communication and increased understanding of each other’s experiences. Conclusion: Findings from this pilot suggest that families perceived multiple benefits from participating in Triple T. Compared to previous studies, rates of elevation in psychosocial challenges were lower. Disclosure K.R. Howard: Stock/Shareholder; Abbott, AbbVie Inc. Other Relationship; Association of Diabetes Care & Education Specialists. K.P. Garza: None. N.W. Smith: None. M.C. Suhs: None. J.S. Merrick: None. J. Weissberg-Benchell: None. M. Feldman: None. Funding Breakthrough T1D (2-SRA-2020-985-S-B)

Regulatory Frameworks in Science, Technology, and Medical Innovation

2025-06-13

Hewa Majeed Zangana , Marwan Omar , Jamal N. Al-Karaki | IGI Global eBooks

The convergence of science, technology, and medical (STM) innovation has transformed contemporary society, offering unprecedented benefits while introducing complex legal, ethical, and governance challenges. Regulatory frameworks serve as critical mechanisms … The convergence of science, technology, and medical (STM) innovation has transformed contemporary society, offering unprecedented benefits while introducing complex legal, ethical, and governance challenges. Regulatory frameworks serve as critical mechanisms for balancing innovation with public interest, ensuring safety, efficacy, and equity in rapidly evolving domains. This chapter explores the multifaceted regulatory landscapes that govern STM enterprises, highlighting international standards, national legislation, and sector-specific protocols. It also addresses the tension between fostering innovation and enforcing compliance, especially in areas such as biomedical research, emerging technologies, and data-driven health systems. Through comparative insights and case-driven analysis, this chapter aims to equip stakeholders with a nuanced understanding of how regulatory systems can both constrain and catalyze responsible innovation in STM fields.

New Genetic Tests, New Research Findings: Do Patients and Participants Have a Right to Know–and Do They Have a Right Not to Know?

2025-06-13

Roger Brownsword | Routledge eBooks

Abstract P4-02-03: Transparency for patient assistance and compassionate use programs for high-cost intravenous and oral therapies in breast cancer patients at an NCI-designated cancer center

2025-06-13

Jina Yun , Lynn Symonds , Hannah Linden | Clinical Cancer Research

Abstract Background: Financial toxicity (FT) is a growing concern for cancer patients and affects overall survival, treatment adherence and quality of life. 1 Approaches to address FT include incorporating cost … Abstract Background: Financial toxicity (FT) is a growing concern for cancer patients and affects overall survival, treatment adherence and quality of life. 1 Approaches to address FT include incorporating cost information into treatment decision making, which relies on accurate cost information as well as transparency into the process and timeline for treatment initiation. 1 Objectives: the primary objective is to identify the rates of patient assistance program (PAP) approval for oral anticancer medications (OAMs) and approvals for compassionate use high-cost intravenous (IV) therapies in breast cancer patients. The OAMs and high-cost IV therapies of interest include palbociclib, ribociclib, abemaciclib, fam-trastuzumab deruxtecan, trastuzumab, pertuzumab, pembrolizumab, goserelin, leuprolide, liposomal doxorubicin, and sacituzumab govitecan. The secondary objectives include characterization of insurance approval, copay information, time to manufacturer PAP or EAP approval, clinical decision making (switching or discontinuing therapeutic agents) due to lack of PAP or EAP approval for OAMs and high-cost IV therapies in breast cancer patients. Methods: A retrospective chart review was conducted at Fred Hutchinson Cancer Center (FHCC) for breast cancer patients referred to the Patient Assistance (PA) team for OAMs and high-cost IV therapies of interest within the study period of 1/1/2023 to 6/31/2024. Data was collected for therapy of interest, insurance type, insurance approval, copay information (if available), date of PAP initiation and determination, other patient assistance, off-label indication, and clinical decisions made based on PAP denial. Results: For OAMs, 79 referrals to the PA team for 74 distinct patients were evaluated. For high-cost IV therapies, 17 referrals to the PA team for 15 distinct patients were evaluated. For OAMs, 48 were approved by insurance and 8 were not. Copay information was available for 49 patients and the median copay was $2520 (range $0 to $15,680). The median time from PA initiation to notification of PA determination was 19 days (range 0 to 200 days). Patients referred to the PA teams were insured by a Medicare Part D plan (53.2%), Medicaid (2.5%), private insurance (21.5%), or had no insurance (8.9%). Most patients identified as white (83.5%) with English as their primary language (88.6%). Of the 8 patients denied PA, one patient declined therapy, one patient switched to another agent in that class. For high-cost IV therapies, 1 out of 15 patients pursued PA due to insurance denial while 8 out of 15 patients pursued PA as they had no insurance. The median time from PA initiation to notification of PA determination was 17 days (range 4 to 221 days). Conclusion: Mitigation of financial toxicity should include transparency and metrics for time to drug approval for patients. Our review showed that the median time to patient assistance approval for OAMs and high-cost IV therapies was 19 and 17 days, respectively, and this can be in addition to the time to initial insurance denial or determination. This review also identifies patients with Medicare as a population that may benefit from upfront financial navigation to expedite paperwork around the drug approval and acquisition process, as their median copay was $2520. The scope of this review should be broadened across all OAMs and high-cost IV therapies to provide patients and clinicians clear expectations and opportunities for patients and clinicians to identify and participate in quality improvement efforts and patient advocacy with manufacturer programs. References: Khan HM, Ramsey S, Shankaran V. Financial toxicity in cancer care: implications for clinical care and potential practice solutions. J Clin Oncol 2023; 41: 3051-3058. Citation Format: Jina Yun, Lynn Symonds, Hannah Linden. Transparency for patient assistance and compassionate use programs for high-cost intravenous and oral therapies in breast cancer patients at an NCI-designated cancer center [abstract]. In: Proceedings of the San Antonio Breast Cancer Symposium 2024; 2024 Dec 10-13; San Antonio, TX. Philadelphia (PA): AACR; Clin Cancer Res 2025;31(12 Suppl):Abstract nr P4-02-03.

Defending academic freedom and showing solidarity with US researchers

2025-06-12

Anita Björklund Carlstedt , Tina Helle , Greta Häggblom‐Kronlöf +7 more | Scandinavian Journal of Occupational Therapy