National Advocacy Day 2017
VBCF volunteer advocates joined us for our annual trip to Washington DC on May 23rd to participate in the National Breast Cancer Coalition (NBCC) National Lobby Day. Advocates met with staff from several Virginia legislators including Senators Kaine and Warner; Representatives Beyer (D-8th); Comstock (R-10th); Connolly (D-11th); McEachin (D-4th), Scott (D-3rd); Taylor (R-2nd) and Wittman (R-1st) to discuss the following legislative priorities:
NBCC’s 2017 Legislative Priorities
• $150 million for the Department of Defense Breast Cancer Research Program for FY2018 – As a result of NBCC’s grassroots advocacy, the DOD BCRP was created in 1992 to “eradicate breast cancer by funding innovative, high-impact research through a partnership of scientists and consumers.” The DOD BCRP is widely viewed as an innovative, unique, and efficient medical research model which has proven to be accountable to the public and has produced extraordinary results. NBCC seeks continued funding for this successful program.
• Guaranteed Access to Quality Care for All – Ensuring access to quality, evidence-based health care has been a top priority of NBCC for many years and is an essential component of Breast Cancer Deadline 2020®. Prior to the passage of the Affordable Care Act (ACA), the NBCC grassroots Board of Directors approved a Framework for a Health Care System Guaranteeing Access to Quality Health Care for All which built on NBCC’s Principles for Quality Care. In 2010, NBCC endorsed and advocated for the passage and implementation of ACA which marked important steps forward in access to quality health care for individuals with, and at risk of, breast cancer. The Coalition opposes and will work to defeat any and all efforts to repeal the ACA or replace it with something less expansive. In addition, NBCC remains committed to protecting vital existing programs such as the Medicaid Breast and Cervical Cancer Treatment Program (Medicaid BCCTP) and critically examining any proposed changes to programs such as Medicare in order to determine the impact they would have on this population.
• Ensuring the Participation of Educated Patient Advocates in all Levels of Health Care Decisions – NBCC continues to work to ensure that educated patient advocates who represent a constituency, have a meaningful “seat at the table” in all levels of health care decision making which affects their lives.
Report on the 2017 “Accelerating Anti-Cancer Agent Development & Validation Workshop (AAADV)”
AAADV Workshop: Update on Getting New Drugs to Cancer Patients Faster by Vicky Carr, VBCF Advocacy Committee member
I’m an 8 year survivor of a particularly aggressive breast cancer (Stage 3 triple negative). Working as a breast cancer hotline peer counselor for the past 5 years, I have many friends living with advanced and metastatic cancers. They desperately need hope. I attended “Accelerating Anti-Cancer Agent Development and Validation Workshop” (AAADV) May 3-5, 2017, in Bethesda, MD, to see what I could find out for them….and you.
AAADV is a annual workshop, run by the FDA (Federal Drug Administration) and sponsored by the FDA, ASCO (American Society of Clinical Oncology), AACR (American Association for Cancer Research), Susan G. Komen, and Duke University, bringing together leaders from cancer research centers, drug companies, the FDA, and cancer patient groups to focus on one mission: to speed safe and effective cancer treatments to patients. Thanks to a scholarship from Susan G. Komen, I was able to be a voice for breast cancer patients there, and now bring back what I learned to share with you. Presentations from 7am to 7pm daily, often several simultaneously, means too much to share in one article. Here were some of the highlights:
- learning what the FDA has done in the past few years to shorten drug timelines,
- opening trial eligibility criteria,
- new immunology combos and approaches,
- harnessing Big Data,
- and, targeting Master Regulator proteins.
OPENING TRIAL ELIGIBILITY CRITERIA:
(Modernizing Eligibility Criteria, Nancy Lin, M.D. Dana Farber Cancer Institute, NamAtiqur Rahman, Ph.D., U.S. Food and Drug Administration
The most immediate change is this. One of my good friends didn’t qualify for any clinical trials when her cancer metastasized this year because she had a history of a prior cancer. Another friend has been excluded from trials because of her brain mets. These exclusions, plus others, will soon be a thing of the past, for most cancer trials. As part of former Vice President Joe Biden’s Cancer Moonshot Program recommendations announced this year to accelerate cancer research and make more treatments available to more patients: a new “no patient left behind” philosophy where trial eligibility criteria will be modernized to enroll broader populations, so the results will be more like the wider population to ultimately use the drug. The new rule: “only exclude where safety indicates.” Specifically, patients with brain metastases, comorbidities like liver/kidney/heart dysfunction, prior malignancies, AIDS, or exclusion by age (such as must be at least 18 years old) will no longer be denied access to most cancer clinical trials. Look to see this year not just publication of these recommendations, but implementation of the changes.
NEW IMMUNOLOGY APPROACHES:
(Beyond Checkpoint Inhibitors: New Approaches to Altering the Tumor Environment, Roy Herbst, M.D., Ph.D., Yale University )
Much research going on into how to prolong the responses in today’s immunotherapy-based treatments for cancer and where the field is going forward.
Some of my takeaways:
- Some patients who don’t have high levels of PDL1 expression will still respond well to immunotherapy
- They are seeing patients who progress on an immunotherapy treatment still respond to a different immunotherapy combination approach.
- Brain mets seem to respond to immunotherapy based treatments.
- Impressive results from the KEYNOTE trials for non-small-cell lung cancer: 45% response rate to immunotherapy vs. 28% response rate to chemotherapy; and a clear survival rate over chemotherapy with estimated rate of overall survival at 1 year 70%. (Note: The multiple KEYNOTE trials are still on-going in breast cancer; early results are promising)
- Determining the best combination immunotherapies: Today there are 1,067 open cancer immunotherapy trials with 7 PD1/PDL1 agents and various combinations. What’s the best way quickly prove which combinations are most effective? There is a need to consider using novel endpoints, such as achieving complete response for more than 6 months
- Research looking into predictors of response to immunotherapy: Amount of PD-L1 receptors a tumor expresses should probably only be one of the biomarkers looked at. (In lung cancer) only about 25% of those high in PDL1 respond well…AND there are many others who respond even though they don’t have high PDL1 levels. Another biomarker predictive of who will respond to immunotherapy is measurement of TILS (tumor-infiltrating lymphocytes)
- Research Going Forward:
- Biomarker Analysis: Defining the profile of non-responders – why were they resistant to immunotherapy?
- People who respond to immunotherapy…then become resistant to it – what causes resistance to immunotherapy? Then develop strategies for this.
- How best to use PD-1 inhibitors in first-line cancer therapy? (which do we do first, immunotherapy? Chemotherapy? A combination?)
- What combination immunotherapy approaches are most effective?
- Targeting the immunosuppressive microenvironment to make immunotherapy more effective
ACCELERATED APPROVAL: Pre-conference required reading, from Friends of Cancer Research online course “FDA 101”.
Since 1992, the FDA has put in place 4 programs to speed availability of drugs to treat serious illnesses (like cancer): Priority Review, Accelerated Approval, Fast Track Status, and, most recent (2012), Breakthrough Therapy Designation. My takeaway is that the new cancer drugs I pay most attention to are those that get Breakthrough Therapy Designation, as that is only given to drugs with preliminary data showing substantial improvement over existing drugs.
From 2012 to 2017, the FDA has given 55 drugs Breakthrough Therapy Designation. For breast cancer, this includes Palbociclib (Ibrance) [2013, for ER+,HER2- breast cancer], (2016, for HR+, HER2- advanced breast cancer), Keytruda (Pembrolizumab) [2016 for cancers that express the PD-L1 receptor], and Immu-132 [2016, for triple negative breast cancer].
Summary of the other programs:
Priority Review speeds up review from 10 months to 6 months.
Fast Track Status gives researchers more frequent communication with the FDA and rolling review of the drug application (where the lengthy drug application can be submitted in parts).
Accelerated Approval allows the FDA to base initial drug approval on its effect on an earlier intermediate or surrogate endpoint, such as tumor shrinkage or pathologically complete response after chemotherapy, rather than waiting many more years until all trial patients have lived their lifespan to determine years added to lifespan.
Link to learn more: https://www.fda.gov/forpatients/approvals/fast/ucm20041766.htm
FROM BIG DATA TO SMART DATA:
(Expanding the Role of Data Science in Drug Discovery and Development, Sean Khozin, M.D, U.S. Food and Drug Administration, Howard Fingert, M.D., Takeda Oncology, Martin Murphy, D.Med Sc, Ph.D., Project DataSphere
Updates on efforts to build a “Healthcare Cloud” and Extensive Data Repository for Cancer against which data analysis can be used as a tool to determine better/best cancer drugs for patient subpopulations.
I was most encouraged to hear about Project DataSphere, whose mission since 2014 has been to broadly share cancer clinical trial datasets with all researchers. Launched with just a few Phase 3 datasets, it now has over 83 clinical trial datasets, representing more than 57,000 patients’ worth. The goal is to contain more than 6,000 datasets representing 100,000 patient lives of data.
Link to read more: https://www.projectdatasphere.org/projectdatasphere/html/home
I’m very encouraged to see researchers beginning to come on-board with sharing their data. In many ways, progress-to-date is groundbreaking and monumental, as historically researchers’ data has been guarded closely as “theirs”. As patient advocates, we can continue to insist that it is OUR data, not the researchers’, and most of us want it as broadly available as possible (while of course protecting privacy), if it can be used to potentially help future patients. My concern with the big data push is there also needs to be a focus on collecting the RIGHT data – big data for cancer can only pan out if the stuff that matters is captured, and if it’s captured in a standardized manner, otherwise predictions will not be accurate, useful, or broadly applicable, and will end up as an expensive, but not profitable, investment.
TARGETING MASTER REGULATOR PROTEINS:
(A Systems Biology Approach to Cancer Drug Discovery and Development: Master Regulators as Drug Targets , Andrea Califano, PhD., Columbia University, Kevin Kalinsky, M.D., Columbia University.)
Prioritizing drugs to be used by actionable proteins. New cancer drug development may need a new approach beyond finding a mutation in a person’s cancer and targeting that mutation with a drug. Researchers are running out of new high frequency driving mutations to target, and response to a drug is frequently followed by relapse of the cancer when the tumor becomes drug-resistant. The presentors’ research indicates presence of a mutation is not as predictive of a response to a drug as measuring the proteins being expressed (gene expression signature). My key takeaway was doctors may in the future be able to prioritize the best cancer drugs at this moment in time for any particular patient based on determining the key proteins driving their cancer at the moment and targeting those proteins. Then as the tumor changes and different proteins are expressed, drugs can be changed to match. This research is still early, with researchers just starting to learn, for example, the master regulator proteins of different kinds of breast cancer (like the top 10 master regulatory proteins for basal breast cancer vs. luminal breast cancer vs. TNBC).
I’ve heard many of these women say their goal is to just live long enough until the next new drug becomes available, and if you can just live until next week who knows what will be available, as there are so many new drugs in the pipeline. Based on what I learned at AAADV, new treatment options for the breast cancer patient living with metastatic cancer, particularly new immunology combinations and approaches and expanded trial eligibility for existing drugs, are becoming available in 2017, with more to follow. And there many professionals collaborating across fields to speed new drugs to cancer patients.