As a breast cancer advocate, I am seduced by the lure of translational research and the promises of personalized medicine that will make the one size fits all approach to cancer treatment obsolete and possibly reduce over-treatment, toxicity, and treatment errors.
I will be attending the BIO Technology Transfer Symposium and BIO Investor Forum next week and will look forward to hearing insights from biotech industry leaders. The Tech Transfer Symposium will address opportunities and challenges of biotechnology licensing. Personalized medicine will be the topic of a Therapeutic Workshop on Thursday morning that will focus on the molecular and genetic basis of disease that are driving new product opportunities and categories in patient diagnosis, prognosis, monitoring and treatment.
Genomics opens the window highlighting the heterogeneity of breast cancer subtypes. Molecular spectrum and gene assays offer promising predictive and prognostic profiles, and pharmacokinetic drug targets have shown considerable potential (i.e., Cyp2D6 variants and the choice of optimal adjuvant endocrine therapy).
Many recent advances have been made characterizing molecular mechanisms, determining molecular insights into metastasis, and developing interventions/applications in the clinic.
Although I am thrilled that innovation is headed in this direction, I also realize that translational research scientists and clinical investigators face many struggles:
- Developing tools that will identify women who will most benefit from therapy—For example, taxane modestly improves anthracyline therapy, we are still uncertain which subgroups of patients benefit from taxanes
- Integrating cutting edge technologies as early measures of efficacy
- Identifying and validating biological and genetic markers for tumor response and patient toxicity to better discriminate minimal risk from high risk disease and personalize therapy– Even when a clearly defined population of HER2 positive patients has been identified, we remain uncertain of the optimal duration and schedule of trastuzumab administration
- Interpreting indeterminate and incidental findings, and the metrics used to measure clinical impact
- Managing the unrealistic expectations of desperate patients who are motivated by the hope of therapeutic benefit even when potential benefit seems questionable or unlikely.
- Sharing technologies across disciplines and developing new frontiers for public-private partnerships, data sharing, and biospecimen accrual.
- Bringing a drug to market – Currently, the majority of the 90 Phase I & II breast cancer clinical trials will fail FDA scrutiny. Meeting the responsibilities of drug approval and personalized medicine in a fragmentary and flawed regulatory environment is one of the biggest challenges of experimental therapeutics.
Despite the long and winding road ahead, I have the confidence that serious technological and cost barriers can be surmounted by a strategic focus on collaborative translational research approaches emphasizing high impact biomarker development and qualification as a new means for diagnosing, preventing, targeting, and treating disease.
In this arena of challenge and opportunity, I and groups of advocate stakeholders nationwide are asserting claims as credible participants who in partnership with academic investigators, industry, medical research foundations, and non-profits wish to influence the revamping of research direction and practices, so that progress might be accelerated in the fight against cancer.
We suggest changes in research priorities and culture by:
- Championing adaptive pragmatic trial design and non-profit disease organization research support
- Embracing evidence based practices: distinguishing hope from hype in a regulatory arena
- Promoting patient centered priorities: rethinking communication dissemination and consent practices
- Reinvigorating transparency in recruitment: reframing ethical priorities for urging rapid patient enrollment into clinical trials
- Reassessing Economic Support for Clinical Trials
Advocates have become involved in various types of involvements over the past decades and we can be proud of our contributions. However, much of the progress in the acquisition of cultural competence and representation on national cooperative groups, workgroups, and panels, should not be overstated. Many of the steps taken have been notable–albeit modest baby steps, and there is still much more work to be done.
We all know that advocacy has the highest praise when the goal is to support. Although it is less appealing when the goal is to “act up” and critique, I hope that all biotech investor stakeholders remain open to the nuances of advocacy dialogue, openness, and candor even when inconvenient. Advocates are credible allies who share their passion as trustworthy participants in the translational research process.
Our participation in translational science will enhance the democratization of knowledge, and our interactions and brokerage with scientists and other stakeholders will lead to further needed reforms in research practices, regulatory policies, and interpretative mechanisms for evaluating the metrics of survival outcome and clinical impact.
We face challenging times and a needed paradigm shift in translational design, conduct and support requires all of us to adopt a renewed commitment to the partnership between investigators, industry, patients, advocates and their communities; a partnership based on social responsibility, authentic collaboration, and transparency; a partnership that promotes new products, reduced development costs, and faster product cycles.
Susan Samson, UCSF Breast SPORE Cancer Program Advocate