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If you are a patient or a doctor interested in finding out about active and upcoming clinical trials on HIBM, please contact us. We will include your details in our database and send you pertinent information.
During 2006, the first clinical therapeutic trial on HIBM was conducted at the National Institutes of Health (NIH). IVIG may have “mild benefits” for some patients, and the decision to obtain IVIG therapy will depend on numerous factors which should be discussed with your doctor. Your doctor may need the published results to help you decide.
You may download the full text of the article at BMC Neurology. The study was made possible by patients referred from HIBM Research Group ( HRG), an ARM funded laboratory.
Research on a mouse model of IBM has offered significant hope for HIBM. See the news release. The result of scientific study regarding N-Acetylmannosamine (ManNAc), a sugar-like molecule, can be downloaded at original scientific article published on June 1, 2007 issue of JCI. The study used a mouse model produced by HIBM Research Group (HRG), an ARM funded laboratory.
ManNAc is not an approved therapy. Upon completion of required safety and toxicology studies according to FDA guidelines, we are hopeful clinical trial will begin in the near future. This project has been selected by NIH TRND program as one of five rare disease projects to pursue. We are hopeful that clinical trial using ManNAc as a promising treatment for HIBM will begin in the next 1-2 years.
Several groups worldwide are working hard to develop gene therapy for HIBM. Initial trials will be conducted on very few patients to evaluate both safety and effectiveness. There is currently a single patient trial ongoing under the direction of Dr. Nemunaitis. The initial results of intramuscular injection has been published. This initial work is very encouraging, and has paved the way for continued development of effective gene therapy for HIBM.
ARM sponsored investigator, Dr. Jacques Tremblay, has been working on developing stem cell based therapy for HIBM for the past several years. Initial trials will be conducted on very few patients. There is currently no projected start date.