New therapeutic approach for the treatment of sarcoidosis (Sirolimus)

Medical Xpress / Medical University of Vienna / The Lancet Rheumatology ^ | Jan. 23, 2024 | Anna Redl et al

[ConservativeMind]

A research team has identified a new approach to treating the inflammatory disease sarcoidosis. In a clinical study, the inhibition of a specific signaling pathway showed clear success in the treatment of granulomas in the skin. This opens up new therapeutic paths for sarcoidosis and similar inflammatory diseases.

Sarcoidosis is an inflammatory disease that is difficult to treat and affects around 20 out of every 100,000 people. In this disease, tiny clusters of inflammatory cells called granulomas form in the affected organs. These granulomas can affect normal tissue and lead to inflammation and fibrosis, which ultimately limits the functionality of affected organs.

The researchers focused their attention on possible new therapeutic approaches for sarcoidosis.

They chose the mTOR (mechanistic target of rapamycin) signaling pathway, which is known to regulate metabolism and growth in many cell types, as mTOR activation was observed in granulomas of different tissues from multiple patients. Sixteen patients with granulomas of the skin and other organs were included in a clinical study..

The researchers used the mTOR inhibitor sirolimus, which was first approved in 1999 to prevent organ rejection after kidney transplants. It had already shown promise in preclinical models of sarcoidosis.

Complete remission of symptoms in some cases

Sirolimus was first administered topically (as a cream) and then systemically (as an oral solution) to test its effect on the skin granulomas. Topical treatment proved unsuccessful, perhaps because the granulomas—compact structures in the skin—are difficult to penetrate from the surface.

In contrast, systemic treatment was successful in 7 out of 10 patients, who completed the study, and some even experienced complete regression after four months of treatment and no recurrence up to two years later.

Interestingly, patients who responded to the systemic treatment had higher mTOR expression in their granuloma fibroblasts than those who did not.

[ConservativeMind]

A compete remission was had by some, even two years later. The treatment used 2 mg a day for four months, it appears.

Sirolimus costs under $150 a month.

[ConservativeMind]

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[ETCM]

One of Dr House’s favorite diagnoses, er “differential diagnoses”.

[telescope115]

Sarcoidosis and Ulcerative Colitis cost me my liver. I had a successful transplant 2 1/2 years ago. I still have Sarcoidosis and UC, but will be monitored closely for the rest of my life. My hepatologist cautioned me that since I still have both diseases, my new liver could still be attacked like the first one was.
Plus all the meds I’m taking, hopefully, will keep my liver safe.

I most definitely will keep Sirolimus in mind if I ever have problems again.

Thanks for the info.

[Tacrolimus1mg]

With any luck, your current transplant meds (see my handle) may be in the same family of drugs.

Definitely a discussion to have with your doc.