[CIS PIDD] [cis-pidd] IL-2 therapy trial for WAS/XLT

[Jyonouchi, Soma C]

Dear Colleagues,

Here at the Children's Hospital of Philadelphia, we are currently recruiting U.S. patients for an FDA RO1 funded Phase I clinical trial to treat patients with Wiskott-Aldrich syndrome (WAS) or X-linked thrombocytopenia (XLT) with low dose recombinant IL-2 therapy.  Patients with grade 1-4 WAS are eligible for this study (patients cannot have malignancy, autoimmunity or be scheduled for HSCT).  Safety is the primary endpoint for the trial but we are also studying improvement in immune function as a secondary endpoint.   IL-2 production in a WAS patient has previously been shown to be reduced (administration of IL-2 in this patient resulted in improved immune function).  We are quite pleased that for the 4 patients who have been treated to date in our study, no serious adverse events have been observed following administration of IL-2.

Jordan Orange and I are the two principal investigators for this study.  Additional information for this trial is available at www.clinicaltrials.gov: http://clinicaltrials.gov/show/NCT00774358

If you have any WAS/XLT patients who would be interested in learning more about the study, please feel free to contact me (jyonouchi at email.chop.edu) or our study coordinator Brenda Gwafila (gwafilab at email.chop.edu).

Sincerely,

Soma Jyonouchi, MD
Children's Hospital of Philadelphia



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