Reply To: Rapamycin: Has anyone taken it?

June 11, 2017 at 3:51 pm

There have been over 2000 US Clinical Trials involving Rapamycin/Sirolimus since approx 1997. I just searched the FDA database and none of the studies have used the drug, or in combination with other drugs, for possible treatment of GBS/CIDP or its variants.

The FDA approved the drug in 1999. However, in 2009 the FDA put out a warning on the drug due to a high incidence of severe side affects that included an increased risk of mortality. The drug now carries a warning and many insurance carriers have removed it from coverage under their formulatories.

Two trials studied it to see if it could treat MS: one (conducted at Harvard) was terminated in 2005; the other (conducted by Wyeth) was also ended in 2005 and has no study results posted. I can find no evidence that the drug could be used as a treatment for CIDP or its variants. Although there are international studies that may suggest it could be used for CIDP related conditions (a study in Iran), I personally discount such studies if they are not overseen by a reputable authority.

Rapamycin/Sirolimus is a macrolide compound that is used to coat coronary stents, prevent organ transplant rejection (liver, kidney, etc), and to treat a rare lung disease called lymphangioleiomyomatosis. It was originally developed as an anti-fungal/anti-biotic agent and is still used for such. It inhibits activation of T cells and B cells by reducing the production of interleukin-2 (IL-2), as a result, it has immunosuppressant properties.

There are ongoing clinical trials trying to determine if it can treat various forms of cancer and other conditions. A report of increased longevity in dogs has been suggested in one study. Another study suggests a specialized delivery system for the drug might help treat MS in mice. Whether favorable study results in animals can be reproduced in humans is yet to be known. Nonetheless, the numerous side affects of this drug class may make it too risky for humans in comparison to other break through treatments such as Ocrelizumab.

If there is a viable study on the drug please post a link to it.