BACKGROUND: The prognosis of diffuse intrinsic pontine glioma (DIPG) remains 
poor, with no drug proven to be effective.
METHODS: Patients with clinically and radiologically confirmed, centrally 
reviewed DIPG, who had failed standard first-line therapy were eligible for this 
multicenter phase II trial. The anti-epidermal growth factor receptor (EGFR) 
antibody, nimotuzumab (150 mg/m(2)), was administered intravenously once weekly 
from weeks 1 to 7 and once every 2 weeks from weeks 8 to 18. Response evaluation 
was based on clinical and MRI assessments. Patients with partial response (PR) 
or stable disease (SD) were allowed to continue nimotuzumab.
RESULTS: Forty-four patients received at least one dose of nimotuzumab 
(male/female, 20/24; median age, 6.0 years; range, 3.0-17.0 years). All had 
received prior radiotherapy. Treatment was well tolerated. Eighteen children 
experienced serious adverse events (SAEs). The majority of SAEs were associated 
with disease progression. Nineteen patients completed 8 weeks (W8) of treatment: 
There were 2 PRs, 6 SDs, and 11 progressions. Five patients completed 18 weeks 
(W18) of treatment: 1 of 2 patients with PR at W8 remained in PR at W18, and 3 
of 6 children with SD at W8 maintained SD at W18. Time to progression following 
initiation of nimotuzumab for the 4 patients with SD or better at W18 was 119, 
157, 182 and 335 days, respectively. Median survival time was 3.2 months. Two 
patients lived 663 and 481 days from the start of nimotuzumab.
CONCLUSIONS: Modest activity of nimotuzumab in DIPG, which has been shown 
previously, was confirmed: A small subset of DIPG patients appeared to benefit 
from anti-EGFR antibody treatment.