A Phase I Study of Palbociclib in Younger Patients with Recurrent or Persistent Brain or Spinal Cord Tumors

Full Title

Phase I Study of CDK 4-6 inhibitor PD-0332991(palbociclib; IBRANCE) in Children with Recurrent, Progressive or Refractory Central Nervous System Tumors


Palbociclib (PD0332991) is an anticancer drug designed to fight cancer by inhibiting certain enzymes called cyclin-dependent kinases (CDKs). These enzymes can fuel cancer growth. Palbociclib is approved for treating advanced breast cancer; its use in this study is considered investigational.

In this Pediatric Brain Tumor Consortium study, researchers are finding the highest dose of palbociclib that can be given safely in children and adolescents with central nervous system (brain and spinal cord) tumors that have come back or continued to grow despite other treatments. Palbociclib is taken orally (by mouth).


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • This study is for patients ages 4 to 21 years.
  • Patients must have a cancer of the brain or spinal cord that has continued to grow or came back despite prior therapy.
  • Patients’ cancers must be positive for the Rb1 protein.
  • Patients who have had more than two prior chemotherapy regimens, radiation therapy to the brain or spine, or intensive chemotherapy with stem cell rescue may not participate.

For more information and to inquire about eligibility for this study, please contact Dr. Stephen W. Gilheeney at 212-639-3973.





Disease Status

Newly Diagnosed & Relapsed/Refractory