scholarly journals Update on the role of copanlisib in hematologic malignancies

2021 ◽  
Vol 12 ◽  
pp. 204062072110060
Author(s):  
Thuy Le ◽  
David Jerel ◽  
Locke J. Bryan
Keyword(s):  
Hematologic Malignancies ◽  
Pi3k Pathway ◽  
Pi3k Inhibitors ◽  
Fda Approval ◽  
Solid Malignancies ◽  
Valuable Treatment ◽  
United States Food ◽  
States Food

Clinical research in hematologic malignancies is continually advancing with emerging concepts in therapy and evolving results from clinical protocols. Targeting of the PI3K pathway remains a valuable treatment across both hematologic and solid malignancies. There are currently four United States Food and Drug Administration (FDA)-approved PI3K inhibitors, with several others in development. Copanlisib is a pan-PI3K inhibitor currently FDA-approved for the treatment of relapsed/refractory follicular lymphoma (FL) following two lines of therapy. Since FDA approval, there have been further investigations into the long-term safety profile of copanlisib, as well as treatment of FL and other lymphoma subtypes, both indolent and aggressive. Here, we review the most recent available data from clinical trials, describe the management of the most common side effects, and explore future concepts. The use of copanlisib as part of a combination therapy for various hematologic malignancies will also be discussed. Copanlisib is a unique drug compared with other PI3K inhibitors, with remarkable potential to improve our armamentarium in cancer treatment.

21st Century Citizen Pharma: The FDA & Patient-Focused Product Development

2018 ◽  
Vol 44 (2-3) ◽  
pp. 309-327 ◽  
Author(s):  
Jordan Paradise
Keyword(s):  
Product Development ◽  
Health And Safety ◽  
The United States ◽  
The Public ◽  
United States Food ◽  
Recent Developments ◽  
States Food ◽  
And Control ◽  
Patient Advocacy Groups

Perpetual debate regarding the delicate balance between access and innovation and the protection of the public health and safety dominate discussions of the United States Food and Drug Administration (“FDA”). Established chiefly as a command and control federal administrative agency, iterative changes in legislation have shaped the FDA's activity in drug, biologic, and medical device regulation over the course of the last one hundred plus years. The most recent fundamental reframing of the agency's authority and directive presented itself in the 21st Century Cures Act, reflecting an important role for patient perspectives in the regulatory process. This Article explores recent developments in patient-focused product development efforts at the FDA and offers modest insights on the increasing role of patients, and patient advocacy groups, in agency decision-making. The Article terms this era “21st century citizen pharma.”

scholarly journals Strategies to prevent anthracycline-induced cardiotoxicity in cancer survivors

2019 ◽  
Vol 5 (1) ◽  
Author(s):  
Neha Bansal ◽  
M. Jacob Adams ◽  
Sarju Ganatra ◽  
Steven D. Colan ◽  
Sanjeev Aggarwal ◽  
...  
Keyword(s):  
The Elderly ◽  
Cancer Diagnostics ◽  
European Medicines Agency ◽  
Cardiovascular Comorbidities ◽  
Thoracic Radiation ◽  
United States Food ◽  
Short And Long Term ◽  
States Food ◽  
Anthracycline Chemotherapy

AbstractCancer diagnostics and therapies have improved steadily over the last few decades, markedly increasing life expectancy for patients at all ages. However, conventional and newer anti-neoplastic therapies can cause short- and long-term cardiotoxicity. The clinical implications of this cardiotoxicity become more important with the increasing use of cardiotoxic drugs. The implications are especially serious among patients predisposed to adverse cardiac effects, such as youth, the elderly, those with cardiovascular comorbidities, and those receiving additional chemotherapies or thoracic radiation. However, the optimal strategy for preventing and managing chemotherapy-induced cardiotoxicity remains unknown. The routine use of neurohormonal antagonists for cardioprotection is not currently justified, given the marginal benefits and associated adverse events, particularly with long-term use. The only United States Food and Drug Administration and European Medicines Agency approved treatment for preventing anthracycline-related cardiomyopathy is dexrazoxane. We advocate administering dexrazoxane during cancer treatment to limit the cardiotoxic effects of anthracycline chemotherapy.

scholarly journals Diagnostic Mycology: Xtreme Challenges

2020 ◽  
Vol 58 (4) ◽  
Author(s):  
Brian L. Wickes ◽  
Anna M. Romanelli
Keyword(s):  
Clinical Laboratory ◽  
Antifungal Susceptibility ◽  
Assay Development ◽  
Invasive Infection ◽  
Fungal Morphology ◽  
Diagnostic Assay ◽  
Diagnostic Assays ◽  
Fda Approval ◽  
United States Food ◽  
States Food

ABSTRACT Developing any diagnostic assay that receives United States Food and Drug Administration (FDA) approval can be a slow and difficult process. FDA-approved assays for fungal diagnosis are generally few in number and are focused mainly on diagnosing candidiasis, which is caused by several species of Candida, in addition to a limited number of systemic mycotic agents. While all microbial diagnostic assays face challenges before they are FDA approved and reach the market, there are a number of challenges to fungal diagnostic assay development that have been difficult hurdles to overcome. These hurdles include template preparation, fungal morphology, how many fungi should be identified in a single assay (scope), taxonomy and nomenclature, discriminating colonizers from invasive infection, combining identification with antifungal susceptibility, and navigating the administrative hurdles required to integrate an assay into a clinical laboratory. Some of these challenges are easier to overcome than others, but all seem to be particularly difficult for fungal diagnostic assays.

The PI3K Pathway at the Crossroads of Cancer and the Immune System: Strategies for Next Generation Immunotherapy Combinations

2018 ◽  
Vol 18 (4) ◽  
pp. 355-364 ◽  
Author(s):  
Dearbhaile C. Collins ◽  
Maxime Chenard-Poirier ◽  
Juanita S. Lopez
Keyword(s):  
Immune System ◽  
Pi3k Pathway ◽  
Growth And Survival ◽  
Cellular Processes ◽  
Combination Strategies ◽  
Integral Role ◽  
Phosphoinositide 3 Kinase ◽  
Growth Factor Pathways

Immunotherapy has led to a paradigm shift in the treatment of some malignancies, providing long-term, durable responses for a subset of patients with advanced cancers. Increasingly, research has identified links between the immune system and critical oncogenic growth factor pathways. The phosphoinositide 3-kinase (PI3K)-AKT-mTOR cascade is frequently hyperactivated in cancer, and plays an integral role in many cellular processes including tumour growth and survival and can underlie resistance to therapies. In this review, we first summarize two key learnings from the initial studies of inhibitors of this pathway, including the profile of immune-related adverse events such as colitis, transaminitis and pneumonitis and the increased incidence of infections with the majority of agents that target the PI3K-AKT-mTOR pathway. We then discuss recent advances in our understanding of the role of this pathway in the tumour micro-environment, and in the regulation of innate and adaptive immune responses, and propose synergistic combination strategies with PI3K-network inhibitors and cancer immunotherapy.

Immunoregulation of Autoimmune Disorders: The Role of Intravenous Immunoglobulins

1993 ◽  
Vol 16 (5_suppl) ◽  
pp. 189-195 ◽  
Author(s):  
G. Luzi ◽  
R. Ferrara
Keyword(s):  
Hematologic Malignancies ◽  
Intravenous Immunoglobulins ◽  
Term Therapy ◽  
Immune Modulators ◽  
Bacterial Diseases ◽  
Immunoglobulin Preparations ◽  
Original Observation ◽  
Long Term Therapy

Modified and intact immunoglobulin preparations are available for therapeutic use. The administration of intravenous immunoglobulins (IVI G) gave positive results in Primary Immunodeficiency Syndromes (PIS) (prophylaxis of viral and bacterial diseases), in treatment of secondary immunodeficiencies (hematologic malignancies, bone marrow transplantation), and in some infections. Adverse reactions have been reported during IVIG infusions, but they are rarely serious and do not represent limiting conditions for a short or long term therapy. After the original observation in thrombocytopenic purpura, IVIG have been used as immune modulators in various autoimmune related disorders. Various mechanisms of action are proposed: blockade and down regulation of phagocytic function via Fc receptor, regulation of idiotype-anti idiotype network, suppression of idiotype synthesis, T-B cell interference towards antigen presentation, increase in suppressor lymphocytes, IVIG-cytokine interaction.

The ABCs of the FDA: A Primer on the Role of the United States Food and Drug Administration in Medical Device Approvals and IR Research

2015 ◽  
Vol 26 (9) ◽  
pp. 1324-1330 ◽  
Author(s):  
Ashley Adamovich ◽  
Susie Park ◽  
Gary P. Siskin ◽  
Meridith J. Englander ◽  
Kenneth D. Mandato ◽  
...  
Keyword(s):  
United States ◽  
Medical Device ◽  
Drug Administration ◽  
Food And Drug Administration ◽  
The United States ◽  
United States Food ◽  
States Food

scholarly journals A comparison of mutagenic potential of Aji-no-Moto with a traditional chemical mutagen on microsporogenesis in barley (Hordeum vulagre L.)

2021 ◽  
Vol 13 (4) ◽  
pp. 1378-1382
Author(s):  
Vivek Singh
Keyword(s):  
The United States ◽  
Chemical Mutagen ◽  
Hordeum Vulgare L ◽  
Mutagenic Potential ◽  
Long Term Effect ◽  
Methane Sulphonate ◽  
United States Food ◽  
States Food ◽  
Higher Doses

Aji-no-Moto or Mono Sodium Glutamate (MSG) is a flavour enhancer being used extensively in South East Asian cuisine. The Federation of American Societies for Experimental Biology for the United States Food and Drug Administration (FDA) has concluded that MSG is safe when "eaten at customary levels" but there is still great confusion regarding its toxicity at higher concentrations. Therefore, it was decided to assess the mutagenic efficacy of MSG on a plant system and present the findings as a model for probably similar effects in the animal model. For this, a traditionally popular genus for genetic studies, Hordeum vulgare L. or winter barley, was used as the model system. The studies of microsporogenesis were done in order to see the long term effect. The sets were compared with experimental sets of plants grown from seeds treated with a traditional chemical mutagen Ethyl Methane Sulphonate (EMS). The study revealed that MSG does not induce much genotoxic effects at lower doses and the chromosomal damages induced were very few. However, at higher doses, it almost equals the effects of EMS in terms of heritable genetic damage. The work is significant as MSG continues to be one of the most popular flavouring agents and does not face any challenge to its biosafe status. However, the clastogenic and chromotoxic effects of higher doses of MSG as observed in the study are in total contradiction to the popular belief.

scholarly journals Oxymetazoline, Mupirocin, Clotrimazole—Safe, Effective, Off-Label Agents for Tympanostomy Tube Care

2020 ◽  
Vol 99 (1_suppl) ◽  
pp. 30S-34S
Author(s):  
Glenn Isaacson
Keyword(s):  
Nasal Spray ◽  
Tympanostomy Tube ◽  
Off Label ◽  
Highly Active ◽  
Tympanostomy Tubes ◽  
United States Food ◽  
States Food ◽  
The Common ◽  
Mupirocin Ointment

Objectives: Only a few medications have a United States Food and Drug Administration indications for prevention and/or treatment of infections in patients with tympanic perforations or tympanostomy tubes. We examined 3 off-label agents that have become important in tympanostomy tube care hoping to demonstrate the effectiveness and safety of each in experimental assays and human application. Methods: Computerized literature review. Results: (1) Oxymetazoline nasal spray applied at the time of surgery is equivalent to fluoroquinolone ear drops in the prevention of early postsurgical otorrhea and tympanostomy tube occlusion at the first postoperative visit. (2) Topical mupirocin 2% ointment is effective alone or in combination with culture-directed systemic therapy for the treatment of tympanostomy tube otorrhea caused by community-acquired, methicillin-resistant Staphylococcus aureus. (3) Topical clotrimazole 1% cream is highly active against the common yeast and fungi that cause otomycosis. A single application after microscopic debridement will cure fungal tympanostomy tube otorrhea in most cases. None of these 3 agents is ototoxic in animal histological or physiological studies, and each has proved safe in long-term clinical use. Conclusions: Oxymetazoline nasal spray, mupirocin ointment, and clotrimazole cream are safe and effective as off-label medications for tympanostomy tube care in children.

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