WP1066 for Ependymoma Earns Rare Pediatric Disease Designation


Earlier this year, WP1066, a p-STAT3 inhibitor, received three separate Rare Pediatric Disease designations for the treatment of medulloblastoma, atypical teratoid rhabdoid tumor, and diffuse intrinsic pontine glioma. Now, according to OncLive, WP1066 has once again received this designation from the FDA. This time, the designation centered around the treatment of patients with ependymoma, a rare tumor which often occurs in the brain or spinal cord.


According to Moleculin Biotech:

WP1066 is our flagship Immune/Transcription Modulator. It is the first drug we know of that, in laboratory models, appears to both directly induce tumor cell death and stimulate the immune system to kill tumors.

Further, the company explains:

Pre-clinical testing of WP1066 demonstrated high levels of antitumor activity in a wide range of tumors, including pancreatic cancer, glioblastoma, and melanoma, as well as stomach, renal, ovarian and head & neck cancer.

As a result, WP1066 also earned Orphan Drug designation for the treatment of patients with a variety of brain tumors. In the case of ependymoma, researchers believe that the therapy is able to improve the efficacy of immunotherapy when used in conjunction.


Ependymoma is a rare primary tumor which forms in the spinal cord or brain. As explained by St. Jude’s Children’ Research  Hospital, ependymoma tumors:

are thought to develop from certain cells that normally mature into ependymal cells. Ependymal cells line the ventricles (fluid-filled spaces in the brain) and the central canal of the spinal cord.

While ependymoma can affect those of all ages, these tumors are more commonly found in children. Brain ependymomas are typically diagnosed in children, while spinal tumors are found in adults. Ependymomas rarely form or spread outside of the central nervous system (CNS). Currently, treatment options are somewhat limited and consist of surgery, chemotherapy, or radiation. Symptoms include:

  • Nausea and vomiting
  • Dizziness
  • Headaches
  • Back or neck pain
  • Unsteady gait
  • Muscle weakness, especially in the arms, legs, or trunk
  • Confusion and irritability
  • Difficulty with bowel or bladder control
  • Blurred vision
  • Seizures

Learn more about ependymoma here.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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