The HeritX Global Initiative to Prevent Inherited BRCA Cancer launched with a historic conference at Cold Spring Harbor Laboratory’s Banbury Center
The setting for HeritX’s inaugural conference in November 2015 was both idyllic and symbolic. Cold Spring Harbor Laboratory’s Banbury Center is renowned for launching historic initiatives including the Human Genome Project. The HeritX meeting at Banbury convened many of the world’s top scientists for the first meeting worldwide ever devoted to the prevention of BRCA cancers. (Click here for the full conference agenda and attendee list)
BRCA genes are the most prominent of the inherited cancer genes that expose carriers of a BRCA mutation to a greater than 90 percent risk of not only one but several types of cancer, including breast, ovarian, pancreatic, prostate and skin cancer (BOPPS). Public awareness of BRCA and exploding interest in genetic testing mean a ever-increasing number of people are discovering their risk. People who face an overwhelming risk of multiple cancers do not want to wait for cancer to strike, they want to control their destinies through prevention. Today, cancer prevention is limited to surgical removal of body parts that are at the highest cancer risk. These options are traumatic and only feasible for some cancers. Better options are urgently needed.
One of the most powerful stories shared at the conference was that of Matt, who is 45, handsome and athletic, having been a former pro athlete. Matt carries a BRCA2 mutation and was recently diagnosed with aggressive early onset prostate cancer, which is common in men with BRCA2 mutations. Matt spoke candidly about his treatment for his cancer and about the fact that men like him have no preventive options whatsoever for preventing the prostate cancer, pancreatic cancer, male breast cancer or melanoma that are caused by their BRCA mutations.
HeritX is spurring the international research community to launch a quest to prevent all cancer types caused by BRCA mutations and in a way that keeps people healthy and whole. Consensus from this Banbury Conference is that this is a challenging but achievable goal made possible at this point in time because (1) unlike with sporadic cancers, we understand the first causal step of developing inherited BRCA cancers, the inherited gene defect, which allows us to focus our research to intervene; (2) after 20 years of research, scientific understanding of the BRCA genes and their function is ripe to move to the next level; and (3) dramatic advances in biomedicine—gene editing, immunotherapy, engineering, technology and big data analysis, to name a few—have created a new and continually evolving armament of tools that can be deployed in the search for true prevention.
“This is a particularly important point in time to hold this meeting. For 20 years, we’ve known about the increased risk of developing various cancers in people who carry the BRCA1 and 2 genes. The problem is, the biomedical research community hasn’t yet translated this into effective prevention; but things are changing. We are now equipped with new genomics tools and other biological tools to begin this work.”
~ Dr. David Parkinson, President & CEO, Essa Pharmaceuticals, Inc.
The conference, entitled “Preventing Inherited BRCA Cancer: A Think Tank for Innovative Strategies, Milestone Objectives and Research Priorities” was co-chaired by:
- Dr. Alan Ashworth, a member of the team that discovered BRCA2, President of the UCSF Helen Diller Comprehensive Cancer Center, and co-director of the UCSF Center for BRCA Research,
- Dr. Lawrence Brody, Chief Scientific Officer for Inherited Diseases Research at the National Institutes of Health, whose laboratory first reported on the high frequence of BRCA mutations in the Ashkenazi Jewish community, and
- Dr. Thomas Bock, Chief Executive Officer of HeritX.
The Conference opened with a panel of BRCA patients discussing the impact of BRCA on themselves and their families. The panel included not only Matt, the 45-year-old BRCA2 positive man with aggressive BRCA2 prostate cancer; but also Dr. Irina Bock, a gynecologist and BRCA1 breast cancer and melanoma survivor; Dr. Pamela Munster, a UCSF oncologist, BRCA2 carrier and breast cancer survivor; and Joi Morris, author, research advocate, and BRCA2 carrier. The families represented by these patients also included members with pancreatic cancer. The patients discussed the significant life-altering consequences of cancer treatment and surgical prevention as well as their fears for their children and their fears regarding the cancers for which there is no prevention, including prostate cancer, pancreatic cancer and melanoma.
This patient session oriented the scientific discussion to meeting the needs of patients: specifically, the need to prevent all types of BRCA cancers in a way that keeps people healthy and whole. Feedback from the research participants was that this session was an “ah ha” moment that focused their attention on the need for comprehensive non-surgical prevention for these patients. One participant said this was “the most eye-opening session” he had ever attended in the BRCA field.
“The time is right to think differently about this problem and HeritX has the right approach to have a particular type of community – in which the fundamentals are put into place to try to solve a problem and overcome difficulties that are seemingly unsurmountable.”
~ Dr. Maynard Olson, Professor Emeritus of Medicine and Genome Sciences, University of Washington, co-originator of the Human Genome Project
HeritX is a very different kind of nonprofit that not only brings the voice of the BRCA community to this project but also brings the true bench to bedside view of the integration of disciplines needed to create transformative change.
HeritX is a different kind of nonprofit that not only brings the voice of the BRCA community to prevention research but also has an expert bench to bedside perspective across the multiple disciplines needed to create transformative progress for patients. The HeritX team has led more than 100 therapy development programs in cancer and orphan diseases, across entire R&D continuum from basic research to FDA approval, and has established relationships across academia, the NIH, the biopharmaceutical industry and patient organizations. HeritX’s integrative approach was reflected in the diversity of the Banbury participants, who included not only leading researchers inside and outside the BRCA field in academia but also experts from the private sector whose expertise included drug development, regulatory affairs and FDA approval.
”HeritX has a unique approach to tackling this problem, focusing on questions that research needs to answer, in order to bring the power of emerging cancer therapeutics and emerging diagnostic tools such as genomics to bear.”
~ Dr. David Parkinson
All conference discussions focused on the single unified goal of scientifically mapping out a research and development path to an FDA-approved medical prevention for all types of BRCA cancers. The specific outcomes of this conference included:
- Alignment on the targeted profile of the therapy for preventing inherited BRCA cancer.
- Creation of research strategies to develop such a preventive therapy. Eight research strategies were identified and defined including: how to potentially reconstitute the gene function; how to provide the gene product; how to protect a BRCA carrier from the earliest steps of cancer development before cancer starts such as through vaccination.
- Definition of the first specific research questions and milestones goal for the identified prevention strategies; agreement on research priorities.
- Identification of acceleration opportunities and potential hurdles to address early in order to prevent future delays.
- Specific commitments to move forward with research on these identified knowledge gaps were made by a number of participants including those working in the BRCA field as well as research leaders in other fields.
“The key insight from this conference was the development of a list of 3 or 4 questions we didn’t know about, that are actually solvable. We were able to sit there as a group and collaborate to roughly design experiments that answer the questions, allowing us to move on to the next step.”
~ Dr. Lawrence Brody, Chief Scientific Officer for Inherited Diseases Research, National Institutes of Health
HeritX’s founders feel a fundamental responsibility to prevent inherited cancer because it affects their own families together with millions of families worldwide, sweeping across genders and generations. With the support of the BRCA community and public, the HeritX team will spark, support and sustain groundbreaking research and deploy current and emerging technologies across biomedicine and engineering to accelerate progress.