Iopofosine I 131 may improve survival in pediatric brain cancer
Early data: Experimental therapy shows promise for hard-to-treat glioma

The experimental therapy iopofosine I 131 may improve survival outcomes in children and young adults with hard-to-treat glioma, according to new clinical trial data announced by the therapy’s developer, Cellectar Biosciences.
“The data from our CLOVER-2 study demonstrate encouraging signs of tumor volume reduction, disease control, and extended survival in children and young adults with relapsed or refractory high-grade gliomas — an area of devastating unmet need,” James Caruso, president and CEO of Cellectar, said in a company press release. “We remain committed to advancing iopofosine I 131 as a targeted radiotherapeutic option for patients with few alternatives.”
Iopofosine I 131 developed as potential treatment for several cancer types
Iopofosine is a small molecule designed to deliver a radioactive compound to cancer cells, thereby killing the cells. Cellectar is developing the therapy as a potential treatment for several types of cancers, including gliomas, which are cancers that form in the brain and spinal cord due to the uncontrolled growth of cells called glia that support nerve activity.
The new data come from a Phase 1b clinical trial called CLOVER-2 (NCT05610891), which is testing iopofosine in 14 children and young adults with high-grade glioma that is relapsed or refractory, meaning it has come back after prior lines of treatment or has failed to respond to treatment.
The new data cover six evaluable patients. Results showed the average progression-free survival time (PFS) — that is, the length of time patients were alive without their cancer growing — was 5.4 months. The average overall survival time was 8.6 months, though since some patients are still alive, this number may change as time goes on.
Among the three patients who received additional treatment cycles (at least four infusions of iopofosine), average PFS and overall survival times were 8.1 months and 11.5 months, respectively.
Presenting these promising interim results at AACR’s Special Conference on Pediatric Cancer before an audience of pediatric oncologists and other researchers in the field was a meaningful milestone for Cellectar.
To put these numbers in perspective, the average expected PFS for pediatric patients with relapsed/refractory high-grade glioma is less than three months, and the expected overall survival time is less than six months, according to Cellectar.
These data were shared at the American Association for Cancer Research (AACR) Special Conference on Pediatric Cancer, held recently in Boston.
At the presentation, the researchers specifically highlighted the cases of two patients — a 25-year-old man and a 15-year-old girl —who remain alive more than a year after starting iopofosine treatment, with their tumors shrinking by more than 50% and about 44%, respectively.
The researchers also shared safety data highlighting a manageable profile for iopofosine. The most commonly reported side effects have been low blood cell counts, and no fatal reactions to the drug have been reported.
“Presenting these promising interim results at AACR’s Special Conference on Pediatric Cancer before an audience of pediatric oncologists and other researchers in the field was a meaningful milestone for Cellectar,” Caruso said.