News Column

Findings from Shinshu University Update Understanding of Clinical Trials and Studies

May 29, 2014

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Current study results on Clinical Research have been published. According to news reporting originating from Matsumoto, Japan, by NewsRx editors, the research stated, "What is known and objective Transthyretin (TTR) is a representative amyloidogenic protein in humans. Rate-limiting tetramer dissociation and rapid monomer misfolding and misassembly of variant TTR result in autosomal dominant familial amyloidosis."

Our news editors obtained a quote from the research from Shinshu University, "Analogous misfolding of wild-type TTR results in senile systemic amyloidosis (SSA) presenting as sporadic amyloid disease in the elderly. The objective of this review is to summarize recent progress in our understanding and treatment of TTR amyloidosis. Literature searches were conducted on the topics of transthyretin, familial amyloid polyneuropathy and clinical trials, using PubMed, the United States clinical trials directory, pharmaceutical company websites and news reports. The information was collected, evaluated for relevance and quality, critically assessed and summarized. The current standard first-line treatment of familial TTR amyloidosis is liver transplantation. However, large numbers of patients are not suitable transplant candidates. Recently, the clinical effects of TTR tetramer stabilizers, tafamidis and diflunisal, were demonstrated in randomized clinical trials, and tafamidis has been approved for the treatment of FAP in European countries and Japan. In addition, gene therapies with antisense oligonucleotides and small interfering RNAs are promising strategies to ameliorate TTR amyloidoses and are currently in clinical trials. What is new and conclusions Liver transplantation to treat the familial TTR amyloidosis will likely be replaced by other less invasive therapies, such as TTR tetramer stabilizers and possibly gene therapy approaches."

According to the news editors, the research concluded: "These newly developed therapies are expected to be effective for not only familial TTR amyloidosis but also SSA, based on their mechanisms of action."

For more information on this research see: Recent progress in the understanding and treatment of transthyretin amyloidosis. Journal of Clinical Pharmacy and Therapeutics, 2014;39(3):225-233. Journal of Clinical Pharmacy and Therapeutics can be contacted at: Wiley-Blackwell, 111 River St, Hoboken 07030-5774, NJ, USA. (Wiley-Blackwell -; Journal of Clinical Pharmacy and Therapeutics -

The news editors report that additional information may be obtained by contacting Y. Sekijima, Shinshu University, Sch Med, Dept. of Med Neurol & Rheumatol, Matsumoto, Nagano 3908621, Japan (see also Clinical Research).

Keywords for this news article include: Asia, Biotechnology, Japan, Matsumoto, Nephrology, Amyloidosis, Gene Therapy, Bioengineering, Clinical Research, Clinical Trials and Studies

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Source: Gene Therapy Weekly

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