Is There a Treatment?

Currently, there are no effective treatments for HIBM. Researchers at Hadassah, USC, UCLA, UCSD, Johns Hopkins University, Canada, NIH, and Japan are contributing towards finding an effective treatment. You may also wish to look at “Developing Rx” which describes how scientists develop treatment for a disease.

Is There Hope for a Treatment Soon?

Yes. Based on preliminary scientific findings, we believe it is less difficult to develop an intervention for IBM2 than for many common myopathic conditions. This is because HIBM is associated with mutations on an enzyme that is expressed at low levels in skeletal muscle, but many other common muscle wasting conditions are caused by mutations on cellular structural proteins which are expressed at very high levels in muscle.

Current treatment theories revolve around the following basic concepts:

  1. Substrate/Product based therapies. These theories involve administration of small molecules that would increase intramuscular pools of a compound known as Sialic Acid. It may involve administration of Sialic Acid rich compounds (e.g. IVIG - IntraVenous Immune Globulins), Sialic Acid precursors, or other derivatives. Currently, this method is most likely to reach clinical trials in the shortest time.
  2. Gene or gene based therapies. These theories involve administration of normal or hyperactive forms of the GNE/MNK enzyme (the enzyme that is mutated in HIBM).
  3. Cell based therapies. These theories involve use of specialized stem cells capable of regenerating muscle and expressing the normal or hyperactive form of GNE/MNK.

It is not unlikely that treatments based on above concepts, which have failed to prove beneficial for other muscle wasting disorders, may prove beneficial for HIBM.

With additional funding, we can speed up the research significantly. To make a donation for HIBM research, you may send a check payable to “HIBM Research” and contact us, or visit Advancement of Research for Myopathies (ARM). ARM offers scientific grants to research centers who are interested in working towards developing a treatment for HIBM.

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