scholarly journals The New Paradigms in Clinical Research: From Early Access Programs to the Novel Therapeutic Approaches for Unmet Medical Needs

2019 ◽  
Vol 10 ◽  
Author(s):  
Cristina Scavone ◽  
Gabriella di Mauro ◽  
Annamaria Mascolo ◽  
Liberato Berrino ◽  
Francesco Rossi ◽  
...  
Keyword(s):  
Clinical Research ◽  
The Novel ◽  
Therapeutic Approaches ◽  
Medical Needs ◽  
Early Access ◽  
Unmet Medical Needs ◽  
Novel Therapeutic ◽  
Access Programs

scholarly journals What do patients with unmet medical needs want? A qualitative study of patients’ views and experiences with expanded access to unapproved, investigational treatments in the Netherlands

2019 ◽  
Vol 20 (1) ◽  
Author(s):  
Eline M. Bunnik ◽  
Nikkie Aarts
Keyword(s):  
Clinical Trials ◽  
Qualitative Study ◽  
Standard Treatment ◽  
Treatment Options ◽  
Safety And Efficacy ◽  
Expanded Access ◽  
Medical Needs ◽  
Investigational Drugs ◽  
Unmet Medical Needs ◽  
Access Programs

Abstract Background Patients with unmet medical needs sometimes resort to non-standard treatment options, including the use of unapproved, investigational drugs in the context of clinical trials, compassionate use or named-patient programs. The views and experiences of patients with unmet medical needs regarding unapproved, investigational drugs have not yet been examined empirically. Methods In this qualitative study, exploratory interviews and focus groups were held with patients with chronic or life-threatening diseases (n = 39), about topics related to non-standard treatment options, such as the search for non-standard treatment options, patients’ views of the moral obligations of doctors, and the conditions under which they would or would not wish to use non-standard treatment options, including expanded access to unapproved, investigational drugs. Results Respondents had very little knowledge about and/or experience with existing opportunities for expanded access to investigational drugs, although some respondents were actively looking for non-standard treatment options. They had high expectations of their treating physicians, assuming them to be aware of non-standard treatment options, including clinical trials elsewhere and expanded access programs, and assuming that they would inform their patients about such options. Respondents carefully weighed the risks and potential benefits of pursuing expanded access, citing concerns related to the scientific evidence of the safety and efficacy of the drug, side effects, drug-drug interactions, and the maintaining of good quality of life. Respondents stressed the importance of education and assertiveness to obtain access to good-quality health care, and were willing to pay out of pocket for investigational drugs. Patients expressed concerns about equal access to new and/or non-standard treatment options. Conclusion When the end of a standard treatment trajectory comes into view, patients may prefer that treating physicians discuss non-standard treatment options with them, including opportunities for expanded access to unapproved, investigational drugs. Although our respondents had varying levels of understanding of expanded access programs, they seemed capable of making well-considered choices with regard to non-standard treatment options and had realistic expectations with regard to the safety and efficacy of such options. Dutch patients might be less likely to fall prey to false hope than often presumed.

scholarly journals Following a potential epileptogenic insult, prolonged high rates of nonlinear dynamical regimes of intermittency type is the hallmark of epileptogenesis

2016 ◽  
Vol 6 (1) ◽  
Author(s):  
Massimo Rizzi ◽  
Itai Weissberg ◽  
Dan Z. Milikovsky ◽  
Alon Friedman
Keyword(s):  
Clinical Research ◽  
Point Of View ◽  
Clinical Perspective ◽  
Therapeutic Approaches ◽  
Nonlinear Dynamical ◽  
Experimental Animals ◽  
Medical Need ◽  
Brain Barrier Permeability ◽  
Dynamical Regimes ◽  
Novel Therapeutic

Abstract The lack of a marker of epileptogenesis is an unmet medical need, not only from the clinical perspective but also from the point of view of the pre-clinical research. Indeed, the lack of this kind of marker affects the investigations on the mechanisms of epileptogenesis as well as the development of novel therapeutic approaches aimed to prevent or to mitigate the severity of the incoming epilepsy in humans. In this work, we provide evidence that in an experimental model of epileptogenesis that mimics the alteration of the blood-brain barrier permeability, a key-mechanism that contributes to the development of epilepsy in humans and in animals, the prolonged occurrence in the electrocorticograms (ECoG) of high rates of a nonlinear dynamical regimes known as intermittency univocally characterizes the population of experimental animals which develop epilepsy, hence it can be considered as the first biophysical marker of epileptogenesis.

scholarly journals Is there a place for mesenchymal stromal cell-based therapies in the therapeutic armamentarium against COVID-19?

2021 ◽  
Vol 12 (1) ◽  
Author(s):  
Kátia Nunes da Silva ◽  
André Luiz Nunes Gobatto ◽  
Zaquer Suzana Munhoz Costa-Ferro ◽  
Bruno Raphael Ribeiro Cavalcante ◽  
Alex Cleber Improta Caria ◽  
...  
Keyword(s):  
Multiple Organ ◽  
The Novel ◽  
Systemic Complications ◽  
Medical Needs ◽  
Potential Efficacy ◽  
Global Spread ◽  
Challenges And Opportunities ◽  
One Year ◽  
Novel Coronavirus ◽  
Unmet Medical Needs

AbstractThe COVID-19 pandemic, caused by the rapid global spread of the novel coronavirus (SARS-CoV-2), has caused healthcare systems to collapse and led to hundreds of thousands of deaths. The clinical spectrum of COVID-19 is not only limited to local pneumonia but also represents multiple organ involvement, with potential for systemic complications. One year after the pandemic, pathophysiological knowledge has evolved, and many therapeutic advances have occurred, but mortality rates are still elevated in severe/critical COVID-19 cases. Mesenchymal stromal cells (MSCs) can exert immunomodulatory, antiviral, and pro-regenerative paracrine/endocrine actions and are therefore promising candidates for MSC-based therapies. In this review, we discuss the rationale for MSC-based therapies based on currently available preclinical and clinical evidence of safety, potential efficacy, and mechanisms of action. Finally, we present a critical analysis of the risks, limitations, challenges, and opportunities that place MSC-based products as a therapeutic strategy that may complement the current arsenal against COVID-19 and reduce the pandemic’s unmet medical needs.

scholarly journals “Scar-cinoma”: viewing the fibrotic lung mesenchymal cell in the context of cancer biology

2016 ◽  
Vol 47 (6) ◽  
pp. 1842-1854 ◽  
Author(s):  
Jeffrey C. Horowitz ◽  
John J. Osterholzer ◽  
Antonia Marazioti ◽  
Georgios T. Stathopoulos
Keyword(s):  
Lung Fibrosis ◽  
Cancer Biology ◽  
Disease Process ◽  
Mesenchymal Cell ◽  
Therapeutic Approaches ◽  
Medical Needs ◽  
Distinct Disease ◽  
Disease Entities ◽  
Fibrotic Diseases ◽  
Unmet Medical Needs

Lung cancer and pulmonary fibrosis are common, yet distinct, pathological processes that represent urgent unmet medical needs. Striking clinical and mechanistic parallels exist between these distinct disease entities. The goal of this article is to examine lung fibrosis from the perspective of cancer-associated phenotypic hallmarks, to discuss areas of mechanistic overlap and distinction, and to highlight profibrotic mechanisms that contribute to carcinogenesis. Ultimately, we speculate that such comparisons might identify opportunities to leverage our current understanding of the pathobiology of each disease process in order to advance novel therapeutic approaches for both. We anticipate that such “outside the box” concepts could be translated to a more precise and individualised approach to fibrotic diseases of the lung.

Adipocytes-released Peptides Involved in the Control of Gastrointestinal Motility

2019 ◽  
Vol 20 (6) ◽  
pp. 614-629 ◽  
Author(s):  
Eglantina Idrizaj ◽  
Rachele Garella ◽  
Roberta Squecco ◽  
Maria Caterina Baccari
Keyword(s):  
Central Nervous System ◽  
Nervous System ◽  
Adipose Tissue ◽  
Gastrointestinal Motility ◽  
Diagnostic Tools ◽  
Therapeutic Approaches ◽  
Motor Responses ◽  
Gastrointestinal Motor ◽  
Treatment Of Obesity ◽  
Novel Therapeutic

The present review focuses on adipocytes-released peptides known to be involved in the control of gastrointestinal motility, acting both centrally and peripherally. Thus, four peptides have been taken into account: leptin, adiponectin, nesfatin-1, and apelin. The discussion of the related physiological or pathophysiological roles, based on the most recent findings, is intended to underlie the close interactions among adipose tissue, central nervous system, and gastrointestinal tract. The better understanding of this complex network, as gastrointestinal motor responses represent peripheral signals involved in the regulation of food intake through the gut-brain axis, may also furnish a cue for the development of either novel therapeutic approaches in the treatment of obesity and eating disorders or potential diagnostic tools.

scholarly journals Methyltransferases in the Pathogenesis of Keratinocyte Cancers

Cancers ◽  
2021 ◽  
Vol 13 (14) ◽  
pp. 3402
Author(s):  
Eun Kyung Ko ◽  
Brian C. Capell
Keyword(s):  
Gene Expression ◽  
Squamous Cell Carcinoma ◽  
Basal Cell Carcinoma ◽  
Cell Carcinoma ◽  
Squamous Cell ◽  
Basal Cell ◽  
Histone Methylation ◽  
Cutaneous Squamous Cell Carcinoma ◽  
Therapeutic Approaches ◽  
Novel Therapeutic

Recent evidence suggests that the disruption of gene expression by alterations in DNA, RNA, and histone methylation may be critical contributors to the pathogenesis of keratinocyte cancers (KCs), made up of basal cell carcinoma (BCC) and cutaneous squamous cell carcinoma (cSCC), which collectively outnumber all other human cancers combined. While it is clear that methylation modifiers are frequently dysregulated in KCs, the underlying molecular and mechanistic changes are only beginning to be understood. Intriguingly, it has recently emerged that there is extensive cross-talk amongst these distinct methylation processes. Here, we summarize and synthesize the latest findings in this space and highlight how these discoveries may uncover novel therapeutic approaches for these ubiquitous cancers.

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