Sirolimus used to cure physician's Castleman Disease

#1

“Fajgenbaum was having his last rites read to him, and his family braced for his death from Castleman disease, a rare inflammatory illness that impacts the lymph nodes and can severely damage other organs. But, in a rare stroke of skill and luck, Fajgenbaum was able to repurpose a generic drug, sirolimus, and go into remission.”

Article is interesting in the paths he used to find a treatment using AI.

Clinical trial Sirolimus New Sirolimus Clinical Trial - CDCN by the same patient - Dr David Fajgenbaum

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#2

It would be interesting to find out the dosage schedule he is using to control his disease and any potential side effects he has experienced.

#3

There is a follow up study where 3 patients with Castleman’s disease were kept at the following blood level:
'Sirolimus trough was maintained at 5–10 ng/mL." https://cdcn.org/wp-content/uploads/2019/11/JCI-Identifying-and-targeting-pathogenic-PI3KAKT-mTOR-signaling-in-IL-6-blockade–refractory-idiopathic-multicentric-Castleman-disease.pdf I believe this means a pretty high daily dose.

For the clinical trial, Sirolimus in Previously Treated Idiopathic Multicentric Castleman Disease - Full Text View - ClinicalTrials.gov There appears a daily dose of either 5mg or 2.5mg dose daily.

“Oral sirolimus: For adults, loading dose of 5 mg/m^2, rounded to the nearest mg, on day 1. For adults, starting on day 2, oral sirolimus daily at 2.5 mg/m^2/day (rounded to the nearest mg), target trough level 10-15 ng/mL by HPLC, for 12 months. For children, 2 mg/m^2/day, target trough level 5-15 ng/mL by HPLC. This study is a Phase II open label study of daily administration of sirolimus in up to 24 evaluable male or female adults. Participants with iMCD who have failed previous therapy will take daily oral sirolimus for 12 months”

Very high doses.

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