Held on the FDA’s White Oak campus, the workshop convened FDA representatives and clinicians to strategize how to advance their updated guideline for including most patients with brain metastases in clinical trial enrollment.
“This group of patients has traditionally been excluded from participating in clinical trials because patients with brain metastases may experience unknown side effects from experimental therapies, which may impact trial results,” said Dr. LeBlang. “We even have focused ultrasound trials that have unfortunately excluded such patients. But now the FDA is not necessarily approving clinical research proposals that exclude patients with CNS metastases, stating that this group of patients should be included in the protocol design and perhaps studied as a separate cohort. This is a true change in the paradigm.”
Another key discussion involved the role of opening the blood-brain barrier to increase the delivery of therapeutics to treat the metastatic brain disease, which occurs most commonly from melanoma, breast, and lung cancers. The use of focused ultrasound was discussed on two expert panels. Researchers have already reported that focused ultrasound can reversibly and safely open the BBB in patients with gliomas, and a new clinical trial for patients with brain metastases is expected to begin this year (see NCT03714243).
Researchers also provided convincing evidence that brain metastases have genetic mutations compared to the primary tumor and the extra cranial deposits. New investigations related to how and when these mutations occur will direct the potential need for different therapies to treat the brain metastases compared to the other systemic metastases.
Key goals of the workshop included the following:
• Better define the problems facing CNS metastases treatment development
• Convene leaders from different cancer fields and different sectors that together are invested in developing and delivering improved therapies to patients
• Raise and amplify patient experience and concerns in clinical research and care delivery
• Elevate the importance of including patients with CNS metastases in clinical trials
• Discover what is needed to help stimulate product development and aid in the evaluation of new therapies
The meeting featured a wide variety of valuable perspectives, including those from several inspiring patients and patient advocacy organizations. Physicians from many specialty areas, representatives from large pharmaceutical companies, and regulatory professionals from various government agencies also contributed. Furthermore, a large number of key professional organizations collaborated to assist in organizing the landmark workshop, including Accelerate Brain Cancer Cure, American Brain Tumor Association, Friends of Cancer Research, Kidney Cancer Research Alliance, LUNGevity Foundation, Melanoma Research Alliance Metastatic Breast Cancer Alliance, Response Assessment in NeuroOncology, and Society for NeuroOncology.
An important next step in this work is the Society for Neuro-Oncology (SNO) Inaugural Conference on Brain Metastases, which is planned for August 2019. Abstracts are now being accepted, and Dr. LeBlang highly encourages the focused ultrasound community to consider submitting their work or collaborating with others in this field.
See Related Story: Society for Neuro-oncology to Hold Inaugural Conference on Brain Metastases >