Rare Disease Report

Neuroblastoma Treatment Granted Orphan Drug Designation

MARCH 19, 2018
Mathew Shanley
This morning, Cellectar Biosciences announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CLR 131 for the treatment of neuroblastoma.

CLR 131 is the company’s lead Phospholipid Drug Conjugate (PDC) product candidate, and is the first potential therapy that would use a PDC tumor-targeting delivery platform to deliver a cytotoxic radioisotope (iodine-131) directly to the tumor cells. 

In children under 5 years, neuroblastomas can develop anywhere that there is an existing group of nerve cells, or neuroblasts. The condition develops from immature neuroblasts found in a variety of areas throughout the body, and typically progresses in and around the adrenal glands, which rest above the kidneys. According to a study published in the Journal of Nuclear Medicine, CLR 131 had already been tested in models of adult cancers and discovered to target cancer cells with very high specificity.

“Neuroblastoma is the third most common childhood cancer for which there are currently no approved treatments for children with relapsed or refractory disease,” stated John Friend, M.D., chief medical officer of Cellectar in a press release. “The FDA’s granting of orphan drug designation for CLR 131 highlights the significant need for new treatments for children with neuroblastoma, and we believe that the targeted delivery of CLR 131 represents a promising novel approach to its treatment.”

Based upon preclinical and interim Phase 1 study data, treatment with CLR 131 provides an original approach to treating solid and hematological tumors. The drug is being developed so that it can potentially provide patients with therapeutic benefits, including: overall survival (OS), an improvement in progression-free survival (PFS), surrogate efficacy marker response rate, and overall quality of life (QOL).

With the University of Wisconsin Carbone Cancer Center, Friend expects Cellectar to initiate its first Phase 1 clinical trial of CLR 131 at the end of the second quarter of this year. The study will enroll patients with relapsed/refractory pediatric cancers, with two cohorts: one with non-brain-related cancers like neuroblastoma, and a second of patients with malignant brain tumors. All enrolled patients will receive a single 30-minute infusion of CLR 131 and will be followed for up to 85 days with safety and efficacy assessments throughout.

“Finding the right collaborators and academic sites who are engaged in clinical research, but also able to administer CLR 131 as a radiotherapeutic can be a bit of a challenge,” said Dr Friend in an exclusive interview with Rare Disease Report. “Fortunately, in neuroblastoma, one of the second-line therapies that is used off-label is MIBG 131, which is essentially a delivery of a very, very high dose iodine 131 to pediatric patients. A lot of these major academic centers, like the University of Wisconsin, have these MIBG-capabilities.”

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