IL-2 Trials

    • IL-2 Trials to improve Immune Function
    • Dr. Soma Jyonouchi from Children's Hospital of Philadelphia and Dr. Jordan Orange from Texas Children’s Hospital recently received a grant from the FDA for continuation of the IL-2 therapy study for WAS and XLT. This is a prospective study treating patients with WAS with IL-2 and using safety as the primary endpoint. Restoration of Natural Killer cell cytotoxicity and effects on cytoskeletal dynamics are secondary endpoints.
    • Below is a note from Dr. Jyonouchi with more details on the study.
    • "There is currently no perfect treatment for individuals with Wiskott-Aldrich SyndromeWAS) or X-Linked Thrombocytopenia (XLT). At the Children’s Hospital of Philadelphia, we are doing a research study, to see if IL-2 ( a type of natural protein produced by the body called a cytokine) can improve the way the immune system works in WAS and XLT. We want to improve the way the immune system works in those affected. We hope that what we learn in this study may help us understand immune cell function in WAS and XLT, and may benefit other patients with this disease in the future.
    • Preliminary data from our study has been promising. Patients thus far have tolerated IL-2 treatments with only mild side effects (no severe adverse events have occurred). Additionally, testing of patient immune function while on this therapy has improved and patients have had clinical improvement as well (fewer infectious complications). Our findings indicate that IL-2 therapy has the potential to become a new treatment option for XLT and WAS patients who do not qualify for stem cell transplantation.
    • We are still actively recruiting patients diagnosed with either XLT or WAS for our study. Since even XLT patients are at increased risk of cancer, autoimmunity and infections, we are especially enthusiastic for recruiting these patients. More details about the study are also on the website:
      • For more information about the study or to refer a patient from your practice, please feel free to contact me at or Brenda Gwafila, RN, at 267-426-9636, or"
    • Here is a link to the research publication demonstrating the enhancement in NK cell function on a patient with WAS . Orange JS et al "IL-2 induces a WAVE-2 dependent pathway for actin reorganization that enables WASp independent human NK cell Function.
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