Workshop on Product Development for CNS Metastases

March 22, 2019

IMG_6129-300x225.jpgMetastatic brain cancer can result from several types of primary cancers including but not limited to breast, kidney, lung, and skin cancer (melanoma). Brain and central nervous system (CNS) metastases represent late-stage cancer and are always a serious, if not a life-threatening diagnosis.

More than 80% of cancers have been associated with the ability to metastasize to the brain.
(Journal of the National Cancer Institute – JNCI) These include:

  • Lung – (25-50%)
  • Breast (15-20%)
  • Melanoma (10%)
  • Renal (5-10%)
  • Colorectal (1-5%)

Studies cite that anywhere from 6-28% of cancer patients develop brain metastases. There are too few treatments for cancer patients with brain metastases and also too few clinical trials aimed at developing new treatments. Increased efforts to develop therapeutic products are needed to address this unmet medical need and ultimately help patients. Barriers to product development for CNS metastases include lack of clinical trials specific for this population, exclusion of patients with brain metastases from clinical trials, and lack of standardized response assessments.

In light of all this, the Food and Drug Administration and the National Brain Tumor Society, joined by a strong coalition of patient advocacy and research organizations, collaborated to host a workshop on March 22, 2019 at the FDA campus, with the intent to provide an open forum to discuss key challenges including clinical trial accrual, study design, and endpoint selection for brain metastases research and treatment discovery.

This workshop has generated results, criteria, documentation, and awareness for the challenges of brain tumor mets patients. SEE the SIDEBAR for materials and resources related to brain and CNS metastases. Also, view the pre-workshop video segments featuring key researchers, medical professionals, and clinicians that highlight key sessions of the workshop.

**The coalition includes ABC2, ABTA, Friends of Cancer Research, KCCure, LUNGevity, MBCA, MRA, RANO and SNO

Key Take Aways from the CNS Metastases Workshop:

  • There is enthusiasm for including patients with CNS metastases broadly in clinical trials, and having opportunities for all such patients is a major source of hope for them.
  • There is general consensus that these patients can be more broadly included in trials without compromising safety or trial design, and many past assumptions need to be challenged.
  • There is strong research identifying potential targets for drugs.
  • Additional guidance from FDA would be welcome.
  • There is recognition that more needs to be done to include in trials patients with leptomeningeal disease.
  • There is a need to develop trials and drugs that specifically target brain metastases.
  • Developing new innovative endpoints and selection of endpoints for trials involving brain metastases patients remains a major challenge, and ultimately might depend on the trial, drug, cohort of patients, and other specific facts. Appropriate endpoints may differ between early and late clinical development.
  • Imaging is one area where more work needs to be done to help make radiographic endpoints more feasible in trials with brain metastases patients. This will include refinement and adoption of RANO brain metastases guidelines, as well as the development of standards for acquiring imaging data in trials using new and advanced imaging methods.