Expert opinion on biological therapy
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Gout is a painful inflammatory arthritis with a prevalence of approximately 4% in the United States, affecting an estimated 8.3 million adults. The past 20 years have shown significant increases in the number of patients with gout and its incidence may still be increasing. Current treatment options to control the pain and inflammation of acute gout include nonsteroidal anti-inflammatory drugs, colchicine and corticosteroids, although patients are often unresponsive to, intolerant of, or have contraindications for, these therapies. Additional treatment options are therefore needed for this population with difficult-to-treat gout. ⋯ Canakinumab has been found to be superior to triamcinolone acetonide in acute gout and to colchicine in gout attack prophylaxis in reducing pain and risk of new gout attacks. Canakinumab's long half-life contributes to its prolonged anti-inflammatory effects.
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Ebolaviruses cause severe viral hemorrhagic fever in humans and non-human primates (NHPs), with case fatality rates of up to 90%. Currently, neither a specific treatment nor a vaccine licensed for use in humans is available. However, a number of vaccine candidates have been developed in the last decade that are highly protective in NHPs, the gold standard animal model for ebola hemorrhagic fever. ⋯ The most pressing remaining challenge is now to move these vaccine candidates forward into human trials and toward licensure. In order to achieve this, it will be necessary to establish the mechanisms and correlates of protection for these vaccines, and to continue to demonstrate their safety, particularly in potentially immunocompromised populations. However, already now there is sufficient evidence that, from a scientific perspective, a vaccine protective against ebolaviruses is possible.
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Expert Opin Biol Ther · Jun 2012
Exosomal RNA as biomarkers and the therapeutic potential of exosome vectors.
Exosomes are nano-sized (40 - 100 nm), extracellular vesicles, of endosomal origin. They are released by cells and found in many body fluids, including plasma. Exosomes contain proteins, microRNAs (miRNAs), and messenger RNAs (mRNAs) that can be transferred between cells. The discovery that exosomes contain RNA, and that this encapsulated RNA could potentially be transferred over distances in vivo, reinforced the importance of exosomes in cell-to-cell communication. ⋯ The data reviewed here suggest that exosomal RNA has the potential to play an important role in the diagnosis, prognosis, and treatment of diseases in the future.
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von Willebrand factor (VWF)-related disorders are generally distinguished as either acquired (von Willebrand Syndrome; VWS) or congenital (von Willebrand Disease; VWD). VWD is the most common inherited bleeding disorder and is due to deficiencies and/or defects in VWF. VWS arises from a large variety of causes. ⋯ All these aspects reflect important but sometimes overlooked issues when using replacement therapy, or attempting to follow expert guidelines. Recombinant VWF has been developed and is undergoing clinical trials, and this promising therapy may change the VWD management landscape in the near future.
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Brentuximab vedotin , a novel anti-CD30 antibody-drug conjugate, delivers a cytotoxic agent into CD30(+) cells. CD30 expression is characteristic of anaplastic large cell lymphoma (ALCL) and Hodgkin lymphoma (HL). ⋯ Brentuximab vedotin provides targeted therapy to CD30(+) lymphomas, including ALCL and HL, with high response rates and manageable toxicity, predominantly myelosuppression and peripheral neuropathy.