Drug Repurposing
Sirolimus was originally approved an immunosuppressive drug used to prevent organ rejection in kidney transplant patients. It works by inhibiting the activation of T cells, which helps to suppress the body’s immune response and reduce the risk of rejection after transplantation. Every Cure co-founder David Fajgenbaum discovered this drug could treat multicentric idiopathic Castleman Disease by experimenting on his own tissue. Since it’s initial discovery it has saved thousands of Castleman patients lives and is currently in clinical trial.
Anifrolumab is a monoclonal antibody medication used in the treatment of systemic lupus erythematosus (SLE), an autoimmune disease that can affect various organs and tissues in the body.Anifrolumab’s inhibition of type I IFN signaling results in a reduction in gene expression is associated with inflammation and tissue damage.
Ruxolitinib is a medication used in the treatment of certain blood disorders, particularly myelofibrosis. Proteomics data concluded that the JAK-STAT pathway seems to be highly active in iMCD patients and inhibiting it may be helpful for stopping the disease. Dr. Fajgenbaum suggested that ruxolinitibcould be considered for two iMCD patients that were out of treatment options. Both patients improved with the initiation of therapy and further research is underway
Eculizumab is a monoclonal antibody medication used in the treatment of certain rare blood disorders, particularly paroxysmal nocturnal hemoglobinuria (PNH). Eculizumab’s ability to inhibit the complement system is a groundbreaking approach in the treatment of hyper-inflammatory diseases and has shown significant clinical benefits for patients with iMCD.
Adalimumab is used in the treatment of various autoimmune diseases and inflammatory conditions such as rheumatoid arthritis. Adalimumab specifically targets and binds to TNF-alpha, a pro-inflammatory cytokine produced by immune cells. In January 2023, the first patient was treated with Adalimumab after preparing for hospice. The medication saved his life. Learn more about this patient’s story here.
Tocilizumab is a medication classified as a humanized monoclonal antibody that targets the interleukin-6 (IL-6) receptor. Tocilizumab is approved for the treatment of various inflammatory conditions such as rheumatoid arthritis. The first patient treated with iMCD with Tocilizumab was in 2010 and continues to be a common treatment for iMCD patients
Pembrolizumab is a humanized monoclonal antibody that targets and blocks the programmed death receptor-1 (PD-1) pathway, a key immune checkpoint, to enhance the body’s natural immune response against cancer cells. In 2016, Fajgenbaum and his team at the University of Pennsylvania discovered this treatment after being hidden in the medical literature for 3 years.
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