Expert opinion on biological therapy
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Expert Opin Biol Ther · Nov 2012
ReviewNabiximols in the treatment of spasticity, pain and urinary symptoms due to multiple sclerosis.
Over the last two decades, experimental and clinical data suggest a therapeutic benefit of cannabis-based medicines for a variety of multiple sclerosis (MS) symptoms. Clinical trials, both with synthetic or plant-derived cannabinoids, have demonstrated clinical efficacy of cannabinoids for the treatment of spasticity, neuropathic pain and bladder dysfunction. Nabiximols, a 1:1 mix of delta-9-tetrahydrocanabinol and cannabidiol extract from cloned chemovars, was licensed in the UK in 2010 and has also been approved in other European countries and Canada. The European Federation of Neurological Societies recommends that cannabis should be used only as a second or third line treatment in central neuropathic pain. ⋯ Despite a number of trials with very promising results, there are still concerns related to relative paucity of data on long-term safety. Also, the long-term efficacy information in terms of the control of symptoms of a disease in which the natural history is progression is sparse. Therefore, further studies are required to improve the current knowledge of nabiximols.
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Expert Opin Biol Ther · Sep 2012
ReviewDecoy activity through microRNAs: the therapeutic implications.
microRNAs (miRNAs), small noncoding RNAs, are deregulated in several diseases including cancer. miRNAs regulate gene expression at a posttranscriptional level by binding to 5'UTR, coding regions or 3'UTR of messenger RNAs (mRNA), inhibiting mRNA translation or causing mRNA degradation. The same miRNA can have multiple mRNA targets, and the same mRNA can be regulated by various miRNAs. ⋯ The miRNA decoy functions have implications for the design of therapeutic approaches in human diseases, including specific ways to overcome resistance to drug therapy and future miRNA-based clinical trials design.
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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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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.