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The value-for-money assessment and funding arrangements for high-priced drugs in an era of uncertainty: a comparative analysis of national health technology assessment agencies in South Korea, England, Australia, and Canada.

BackgroundInnovative health technologies have increasingly emerged as a promising solution for patients with untreatable or challenging conditions. However, these technologies often come with expensive costs and limited evidence at the time of launch. This study assessed how these high-priced drugs with limited evidence were appraised and introduced in South Korea, England, Australia, and Canada, where cost-effectiveness analysis (CEA) generally plays a central role in pricing and reimbursement decisions.MethodsThe study analysed 22 high-priced drugs (24 indications) introduced in South Korea,

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… Treatment Access, Health Economics, and the Wave of a Magic Wand. Current oncology (Toronto, Ont). 2022;29:1176–89. 26. Sehdev S, Chambers A. Is It Time to Commit to a Process to Re-Evaluate Oncology Drugs? …

Exploring novel therapeutic strategies: Could psychedelic perspectives offer promising solutions for Alzheimer's disease comorbidities?

The increasing prevalence of dementia within an ageing global population, combined with prolonged life expectancy, accentuates Alzheimer's disease (AD) as a multifaceted healthcare challenge. This challenge is further compounded by the limited therapeutic options currently available. Addressing the intricacies of AD management, the mitigation of comorbidities has emerged as a pivotal facet of treatment. Comorbid conditions, such as neurobehavioral symptoms, play a role in shaping the clinical course, management, and outcomes of this pathology; highlighting the importance of comprehensive care

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… Effects and safety of Psilocybe cubensis and Panaeolus cyanescens magic mushroom extracts on endothelin-1-induced hypertrophy and cell injury in cardiomyocytes . Sci Rep . 10 ( 1 ): 22314 . doi: 10.1038/s41598-020-79328-5 . 33339902 PMC7749179 O’Regan MC , Clow A. 2004 . …

Перспективы Талкетамаба и его полезности в лечении множественной миеломы: безопасность, эффективность и место в терапии.

Несмотря на недавние достижения, большинство пациентов с множественной миэломой в конечном итоге развивают сопротивление имеющимся методам лечения, подчеркивая необходимость новых терапевтических стратегий. G Класс рецептора с протеиновыми соединениями C 5 Член D (GPRC5D) стал жизнеспособной новой терапевтической целью для клеток миэломы, которая привела к клиническому развитию талькетамаба, первого GPRC5D-directed bi специфичный Т-активист (TCE). В августе Талкетамаб получил ускоренное утверждение 2023 Управление по контролю за продуктами питания и лекарствами. Помимо ожидаемой краткосрочной

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… Corticosteroids were used for management of oral inflammation, as well as magic mouthwash (compounded product that contains at least 3 of an antihistamine, anesthetic, antacid, antifungal, corticosteroid, and/or antibiotic). …

The Impact of NLRP3 Activation on Hematopoietic Stem Cell Transplantation.

NLR family pyrin domain-containing 3 (NLRP3) is an intracellular protein that after recognizing a broad spectrum of stressors, such as microbial motifs and endogenous danger signals, promotes the activation and release of the pro-inflammatory cytokines IL-1β and IL-18, thus playing an essential role in the innate immune response. Several blood cell types, including macrophages, dendritic cells, and hematopoietic stem and progenitor cells (HSPCs), express NLRP3, where it has been implicated in various physiological and pathological processes. For example, NLRP3 participates in the development a

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… Currently, the most accurate guidelines for the assessment and staging of acute GvHD are the Mount Sinai Acute GvHD International Consortium (MAGIC) criteria and the NIH 2014 criteria for chronic GvHD [ 49 ], the description of which is beyond the scope of this article. …

Epigenetic Modifiers as Novel Therapeutic Targets and a Systematic Review of Clinical Studies Investigating Epigenetic Inhibitors in Head and Neck Cancer.

The survival rate of head and neck squamous cell carcinoma patients with the current standard of care therapy is suboptimal and is associated with long-term side effects. Novel therapeutics that will improve survival rates while minimizing treatment-related side effects are the focus of active investigation. Epigenetic modifications have been recognized as potential therapeutic targets in various cancer types, including head and neck cancer. This review summarizes the current knowledge on the function of important epigenetic modifiers in head and neck cancer, their clinical implications and di

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… Supic G., Kozomara R., Zeljic K., Jovic N., Magic Z. Prognostic value of the DNMTs mRNA expression and genetic polymorphisms on the clinical outcome in oral cancer patients. Clin. Oral Investig. 2017;21:173–182. doi: 10.1007/s00784-016-1772-9. 12. …

Antibody structure and engineering considerations for the design and function of Antibody Drug Conjugates (ADCs).

Antibody-drug conjugates (ADCs) are emerging as effective tools in cancer therapy, combining the antibody's exquisite specificity for the target antigen-expressing cancer cell together with the cytotoxic potency of the payload. Much success stems from the rational design of "toxic warheads", chemically linked to antibodies, and from fine-tuning the intricate properties of chemical linkers. Here, we focus on the antibody moiety of ADCs, dissecting the impact of Fab, linkers, isotype and Fc structure on the anti-tumoral and immune-activating functions of ADCs. Novel design approaches informed by

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… and high toxicity, often resulting in adverse effects, poor quality of life, early discontinuation and reduced clinical efficacy. 1 Targeted treatments in the form of tumor antigen-specific and checkpoint inhibitor antibodies, envisioned for over 100 years since Ehrlich proposed the concept of “the magic …

Design and implementation of mHealth applications for older people with cancer: A scoping review.

ObjectiveThis scoping review aimed to systematically synthesize the evidence on the functions, design, implementation strategies, and evaluation approaches of mobile health (mHealth) applications (apps) developed for older adults with cancer, to inform future research and practice.MethodsA scoping review was conducted using Arksey and O'Malley's methodological framework, with an updated systematic search of five databases (Medline, Web of Science, CINAHL, Embase, and the Cochrane Library) from January 1, 2008, to February 28, 2025. The review adhered to the PRISMA-ScR guidelines for scoping re

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… .) | (1), (4) | (2) c, (3) c, (4) b | (1) a, (3) b | (1) ab, (2) b MAGIC®, Older patients with ovarian cancer. | Vogel RI et al., 47 2019, United States | An interventional study ( n ​= ​104) | Information support (The app was designed to provide genetic counseling information to patients.) | (2), (3 …

Phase I study of IMGN901, a CD56-targeting antibody-drug conjugate, in patients with CD56-positive solid tumors.

Background IMGN901 is a CD56-targeting antibody-drug conjugate designed for tumor-selective delivery of the cytotoxic maytansinoid DM1. This phase 1 study investigated the safety, tolerability, pharmacokinetics, and preliminary activity of IMGN901 in patients with CD56-expressing solid tumors. Methods Patients were enrolled in cohorts of escalating IMGN901 doses, administered intravenously, on 3 consecutive days every 21 days. A dose-expansion phase accrued patients with small cell lung cancer (SCLC), Merkel cell carcinoma (MCC), or ovarian cancer. Results Fifty-two patients were treated at do

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… McNiff JM Cowper SE Lazova R Subtil A Glusac EJ CD56 staining in Merkel cell carcinoma and natural killer-cell lymphoma: magic bullet, diagnostic pitfall, or both? J Cutan Pathol 2005 32 541 545 10.1111/j.0303-6987.2005.00378.x 16115052 10. …

Immunological Targets in Generalized Myasthenia Gravis Treatment: Where Are We Going Now?

BackgroundGeneralized myasthenia gravis (gMG) is a heterogeneous autoimmune disorder marked by antibody-mediated disruption of neuromuscular transmission. Despite advancements in immunosuppressive therapies and biologics, a subset of patients remains refractory, necessitating more targeted and personalized treatment strategies.ObjectiveThis review aims to synthesize current knowledge of the immunopathological mechanisms across gMG subtypes and to explore emerging therapeutic targets tailored to these diverse disease phenotypes.MethodsA narrative review was conducted, integrating recent finding

An overview of multiple myeloma: A monoclonal plasma cell malignancy's diagnosis, management, and treatment modalities.

Multiple Myeloma (MM) is a plasma cell cancer with high mortality and morbidity rates. Its incidence rate has increased by 143% since 1975. Adipokines, cytokines, chemokines, and genetic variations influence the development and progression of MM. Chromosomal translocations cause mutations associated with MM. The pathogenesis of MM is complicated by novel issues like miRNAs, RANKL, Wnt/DKK1, Wnt, and OPG. Conventional diagnosis methods include bone marrow biopsy, sPEP or uPEP, sIFE and uIFE, and sFLC assay, along with advanced techniques such as FISH, SNPA, and gene expression technologies. A n

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… To change growth rates or trigger death, monoclonal antibodies, a new family of protein treatments first nicknamed “magic bullets,” impede ligand binding and signalling. …

A comprehensive overview on antibody-drug conjugates: from the conceptualization to cancer therapy.

Antibody-Drug Conjugates (ADCs) represent an innovative class of potent anti-cancer compounds that are widely used in the treatment of hematologic malignancies and solid tumors. Unlike conventional chemotherapeutic drug-based therapies, that are mainly associated with modest specificity and therapeutic benefit, the three key components that form an ADC (a monoclonal antibody bound to a cytotoxic drug via a chemical linker moiety) achieve remarkable improvement in terms of targeted killing of cancer cells and, while sparing healthy tissues, a reduction in systemic side effects caused by off-tum

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… In 1907, German nobel laureate Paul Erlich postulated that there could exist compounds that would selectively target pathogenic microbes, such as bacteria, while sparing normal cells, a concept gone down in history as “zauberkugel” (magic bullet) ( Schwartz, 2004 ). …

Radiation-Induced Oral Mucositis.

Radiation-induced oral mucositis (RIOM) is a major dose-limiting toxicity in head and neck cancer patients. It is a normal tissue injury caused by radiation/radiotherapy (RT), which has marked adverse effects on patient quality of life and cancer therapy continuity. It is a challenge for radiation oncologists since it leads to cancer therapy interruption, poor local tumor control, and changes in dose fractionation. RIOM occurs in 100% of altered fractionation radiotherapy head and neck cancer patients. In the United Sates, its economic cost was estimated to reach 17,000.00 USD per patient with

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… The “magic mouthwash” (lidocaine, diphenhydramine, magnesium aluminum hydroxide) and morphine mouth washes are preferable and have been reported by patients to be effective in alleviating pain in RIOM ( 106 – 108 ). The application of corticosteroids mouthwashes has shown promising results. … Last updated NCT02508389 | A Study of GC4419 Protection against Radiation Induced Oral Mucositis in Patients with Head & Neck Cancer | Radiation-Induced Oral Mucositis | 23 November 2015 NCT00698204 | Cox-2 Inhibition in Radiation-Induced Oral Mucositis | Oral Mucositis | 7 May 2014 NCT00814359 | Magic …

Targeting CD38 Across Autoimmune and Plasma Cell-Driven Immune-Mediated Diseases: Rationale, Clinical Evidence, and Emerging Therapeutic Landscape.

Autoimmune diseases remain a major cause of chronic morbidity despite substantial advances in targeted immunomodulatory therapies. In many autoantibody-mediated conditions, disease refractoriness and relapse are driven by long-lived plasma cells, which are largely resistant to conventional immunosuppression and upstream B cell-directed strategies. CD38, a surface molecule highly expressed on plasmablasts and plasma cells and functionally involved in immunometabolic regulation, has emerged as a promising therapeutic target to overcome this limitation. Clinical interest in anti-CD38 therapy has

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… Felzartamab: a magic bullet to treat microvascular rejection? Kidney Int. 2025;108(5):730–4. 64. Domínguez-Pantoja M, López-Herrera G, Romero-Ramírez H, Santos-Argumedo L, Chávez-Rueda AK, Hernández-Cueto A, et al. …

Замена иммуноглобулина: Понятия управления несколькими миеломами.

Immunoglobulin (Ig) - заместительная терапия (IGRT) - состоит из применения низкодозированных поликлонных иг человека для лечения первичной и вторичной гипогаммаглобулинемии, которая связана с периодическими инфекциями и иммунной дисфункцией. ИГРТ восстанавливает физиологические уровни антител и вызывает иммуномумудуляторный эффект путем укрепления клеток иммунного эффекта, тем самым уменьшая инфекцию. Здесь мы описываем фармакологию различных составов иг с уделением особого внимания их механизму действий как низкодозированный ИГРТ, включая прямой антимикробный эффект и иммунодуляторную функци

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… Passive Immunization: Toward Magic Bullets; pp. 1403–1434. 44. Chiu M.L., Goulet D.R., Teplyakov A., Gilliland G.L. Antibody Structure and Function: The Basis for Engineering Therapeutics. Antibodies. 2019;8:55. doi: 10.3390/antib8040055. 45. Suzuki M., Kato C., Kato A. …

Precision Reimbursement for Precision Medicine: Using Real-World Evidence to Evolve From Trial-and-Project to Track-and-Pay to Learn-and-Predict.

Basic scientists and drug developers are accelerating innovations toward the goal of precision medicine. Regulators create pathways for timely patient access to precision medicines, including individualized therapies. Healthcare payors acknowledge the need for change but downstream innovation for coverage and reimbursement is only haltingly occurring. Performance uncertainty, high price-tags, payment timing, and actuarial risk issues associated with precision medicines present novel financial challenges for payors. With traditional drug reimbursement frameworks, payment is based on an assumed

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… Randomized controlled trials versus real world evidence: neither magic nor myth . Clin. Pharmacol. Ther. 109 , 1212 – 1218 ( 2021 ). 33063841 10.1002/cpt.2083 PMC8246742 5 Lautscham , G. & Mullner , S. Beyond oncology – precision medicine for autoimmune disease. …

Anakoinosis: Communicative Reprogramming of Tumor Systems - for Rescuing from Chemorefractory Neoplasia.

Disruptive technologies, such as communicative reprogramming (anakoinosis) with cellular therapies in situ for treating refractory metastatic cancer allow patient care to accelerate along a totally new trajectory and highlight what may well become the next sea change in the care of patients with many types of advanced neoplasia. Cellular therapy in situ consisted of repurposed drugs, pioglitazone plus all-trans retinoic acid or dexamethasone or interferon-alpha (dual transcriptional modulation) combined with metronomic low-dose chemotherapy or low-dose 5-azacytidine, plus/minus classic targete

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… The intention is not to look for new anti-proliferative cytotoxic agents or classic targeted therapies that can be used at maximum tolerated doses, the ‘magic bullets’, but to look for drugs from the available clinical tool that can be used to support the redirection of tumor-specific communication tools …

Emerging disease-modifying therapies for sickle cell disease.

Sickle cell disease afflicts millions of people worldwide and approximately 100,000 Americans. Complications are myriad and arise as a result of complex pathological pathways 'downstream' to a point mutation in DNA, and include red blood cell membrane damage, inflammation, chronic hemolytic anemia with episodic vaso-occlusion, ischemia and pain, and ultimately risk of cumulative organ damage with reduced lifespan of affected individuals. The National Heart, Lung, and Blood Institute's 2014 evidence-based guideline for sickle cell disease management states that additional research is needed bef

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… Early observations also found magnesium supplementation improved sickle RBC hydration in patients with SCD through inhibition of K-Cl co-transport and reduction of dense sickle RBC. 46 However, in the Magnesium for Children in Crisis (MAGiC) study ( NCT01197417 ), which included young adults up to 21 …

Immunotherapy in hematologic malignancies: past, present, and future.

The field of immunotherapy in cancer treatments has been accelerating over recent years and has entered the forefront as a leading area of ongoing research and promising therapies that have changed the treatment landscape for a variety of solid malignancies. Prior to its designation as the Science Breakthrough of the Year in 2013, cancer immunotherapy was active in the treatment of hematologic malignancies. This review provides a broad overview of the past, present, and potential future of immunotherapy in hematologic malignancies.

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… Donnall Thomas and Paul Ehrlich can synergize, where what has been described as the “chemotherapist’s bluntest weapon” can be combined with novel immunotherapies to achieve what is truly the “magic bullet” for patients with hematologic malignancies. Acknowledgements Not applicable. …

Breaking into HIV-1's Epigenetic Vault: Cure Strategies to Eliminate the Viral Reservoir.

Human immunodeficiency virus type 1 (HIV-1) is a retrovirus that integrates into the host cell's DNA as a provirus. Transcription from the provirus is regulated in large part by cellular proteins and epigenetic factors. These may be repressive or permissive to productive infection. The host factors that regulate this balance are therefore attractive targets for HIV-1 therapeutics. Indeed, proviral chromatin is the focus of two of the current HIV-1 cure strategies. "Shock and Kill" uses latency reversal agents to open the provirus's chromatin, promoting high levels of gene expression that induc

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… Virion production in HIV-1-infected MAGIC-5 cells, a HeLa-derived line expressing CCR5/CXCR4 and CD4 [ 358 ], was nearly abrogated, as was proviral transcription, in cells treated with siPromA [ 359 ]. …

Antibody-drug conjugates in cancer therapy: mechanisms and clinical studies.

Antibody-drug conjugates (ADCs) consist of monoclonal antibodies that target tumor cells and cytotoxic drugs linked through linkers. By leveraging antibodies' targeting properties, ADCs deliver cytotoxic drugs into tumor cells via endocytosis after identifying the tumor antigen. This precise method aims to kill tumor cells selectively while minimizing harm to normal cells, offering safe and effective therapeutic benefits. Recent years have seen significant progress in antitumor treatment with ADC development, providing patients with new and potent treatment options. With over 300 ADCs explored

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… INTRODUCTION Since the proposal of the “magic bullet” concept by German scientist Paul Ehrlich in 1913, 1 the development of antibody‐conjugated drugs (ADCs) has experienced both successes and setbacks. … FIGURE 1 Timeline of landmark event in antibody–drug conjugates development over the past 100 years since the “magic bullet” proposed in 1910. …