Ned Sharpless GBM Awareness Day Speech transcribed
I was really elated to be invited to this. This is really a personal effort to me. I would really love to see some progress in GBM. Probably no cancer pisses me off more than GBM. You know it would be great to relieve it. At the FDA we would love to see those ideas come in so that we could approve them to help people with GBM. So this is really something that I was looking forward to doing. Thank you for having me. I’d like to thank Senator Graham and Senator McSally as well as Senators McConnel, Warren, Sinema, and Markey for their sponsorship of this resolution for this Glioblastoma Awareness Day. It’s a great initiative. And based on my experience, as Comprehensive Cancer Center Director, and cancer researcher, NCI Director, and now Acting FDA Commissioner, huh what a mouthful, I had a chance to get various insights into this disease and see it from many perspectives. I certainly have had the experience of talking to patients and loved ones about the problems with therapy for GBM and the bad outcomes. I know as a doctor how tough this disease is to treat and how limited our options are for our patients. As someone who has directed the resources of the Federal Government to topics related to cancer research related to public health, this cancer, GBM in particular is where I’d like to see us do more to make more progress. Because I think we have it. We used to say at NCI, this is a special time for cancer research. We’ve made a lot of progress in a lot of places, and remarkable pace, but unfortunately GBM is not one of them. So given my recent role at NCI and FDA, I’ve been keenly aware of how hard it is to develop new therapies for this disease. It’s really a place where we really haven’t had the progress we’ve had in other cancers for several reasons. GBM is a cancer where we have this blood brain barrier problem which is an issue, and has limited the efficacy of agents in GBM . . . In my new role at the FDA I can help the development of new therapeutic approaches and diagnostics for GBM based on the science funded by NCI and other sources. Although we are currently in an era of unprecedented drug development for many cancers, progress has lagged in GBM. The therapies we have are too toxic. We recognize for Glioblastoma, it’s not really life threatening, it’s like personally threatening, self threatening. Its a disease that these therapies can rob patients of so much. Patients bear the burden of both a life-threatening illness and a progressive neurologic disease that affects their ability to work and to be with loved ones, and every sense of self. In recognizing the challenges, and the uniqueness of this disease, the FDA has been committing to putting all hands on deck approach to supporting drug and device development aimed at treating patients with GBM. To that end, we’ve worked closely with the National Brain Tumor Society to co-sponsor workshop aimed at how to best design clinical tails and measure outcomes for patients with GBM. We continue to support this partnership in the form of twice yearly round-table discussions designed to bring together the FDA and the academic community to discuss the challenges to produce drug development in GBM. The FDA has participated in the planning the informal discussions with the aim of clarifying regulatory issues and providing our expertise. And the FDA’s Oncology Center of Excellence has established a mechanism to ensure the disease area of expertise is provided when reviewing all products intended to help patients with GBM as well as ensuring consistent advice is provided throughout development on this important topic. The FDA also has several methods to streamline and fast-track review of investigational therapies for a lot of life threatening orphan diseases, and I can think of no better candidate for those kinds of mechanisms than GBM. These include the break-though therapy designation, fast-track review, priority review,and programs designed to move the products through the review process rapidly, as well as accelerated approval where drugs are allowed onto the market based on the activity at certain end points. We’re hopeful that progress will be made in this disease as multiple new technologies are brought to bear on this problem, the new exciting immunotherapies, some new targeted therapies, new types of careful clinical trials with adaptive design and novel to better find agents more quickly and the better use of radiation therapy, surgery, novel devices, imaging, and other therapeutic approaches in this space. It is my belief, you know with all this science and great efforts brought to bear, we will build on previous work as well as the new ongoing science to take the significant but in my opinion too incremental progress we’ve made in this disease thus far and move it to even better therapies that will really make a difference in the lives of patients with this disease. And you know, no one would like to see this progress more than me in that regard. So thank you for the opportunity to speak tonight and let’s get this done.
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