Dr. Elizabeth O'Day
Public-Private Biobanks Will Unlock the Power of Biospecimens
Patient biospecimens are the fuel that drive medical breakthroughs. By interrogating biospecimens (tissue, blood, urine, cerebral spinal fluid, feces, you name it) from current patients we can uncover new insights and develop innovations to help future patients. This is the bedrock of “precision medicine” and has led to some of our biggest successes. For example, in oncology, the so-called “Philadelphia chromosome” was originally observed in tumor cells from 2 patients with chronic myeloid leukemia (CML), and later was revealed to be present in 95% of all cases. Further research uncovered that the Philadelphia chromosome leads to an unnatural fusion protein termed BCR-ABL that drives CML tumor growth. These insights would later lead to the development and commercialization of the targeted therapy, imatinib, which transformed CML from a disease with a dismal 5-year survival of ~5% to now over 95%. This revolutionary treatment can be traced back to biospecimens from 2 patient donations.
There is a tremendous willingness for patient participation in biomedical research. A study from the UK found that over 87% of patients thought donation was important and 75% wanted to be asked to donate. In fact, when asked 93.8% of leukemia patients, 93.5% of breast cancer patients, and 91% of gastrointestinal cancer patients consented to having their specimens banked for research purposes. Unfortunately, not all segments of the population are participating in medical research, with non-whites being far less likely to participate in medical research. As the future of medicine will require molecular data from the entire population, current lack of donation/participation risks increasing healthy inequity. Efforts across the globe to understand barriers to donation, lack of trust in the process from previously marginalized groups (i.e. “I don’t want to be the next Henrietta Lacks”) and initiatives to safeguard patient privacy are needed to close the gap.
Diagnostic and therapeutic companies (i.e private industry) lead the research, development and commercial efforts to translate the findings from patient biospecimens into groundbreaking products. Yet the overwhelming majority of biobanks which collect, store and maintain patient donations are academic-based- either residing in the lab of individual clinicians or centrally located within hospital/research organizations. The ability to access these patient samples typically requires collaboration with the clinician and negotiation of any future intellectual property (IP). In some cases, industry is barred from accessing specific datasets, limiting industry’s ability to develop products based on real patient data. Patient samples from clinical trials are another valuable source of biospecimens, but are typically owned by biopharma companies, and a combination of consent and IP can prevent further analysis. This is unfortunate as the power of patient samples often comes by aggregating multiple datasets. Commercial biobanks have also emerged where you can directly buy patient biospecimens, but they often lack critical clinical information.
At Olaris, we use banked-patient samples to uncover metabolic “biomarkers of response” (BoRs) that differentiate which patients benefited from a specific therapy compared to those that did not. Our mission is to use BoRs from previous patients to help future patients so we can remove the guesswork in medicine. Our discovery process requires access to quality samples and quality clinical records. We have made use of ALL methods (academic partnerships, commercial resources and non-for-profit marketplaces) to obtain biospecimens. No matter how we source the samples we always ask the same 3 questions 1) are the samples of high quality? 2) is there an audit trail of the sample(s) history? and 3) is there sufficient clinical documentation to support our analysis? In many cases, 1, 2 and/or 3 is lacking. This in turn limits the utility of the sample, and in the extreme, potential medical breakthroughs remain hidden in the biospecimens.
My own experience has made me question if and how we can improve the biobanking process. One solution goes to the very root. At present “private industry” if allowed at all, is viewed as the end-user or customer of a biobank. I believe we should be more proactive in their genesis. Through public-private partnerships we can aggregate resources and expertise so that we can 1) increase quality control and assurance of samples and clinical data, 2) increase transparency to patients of how samples will and are being used through use-cases and audit trails and ultimately 3) increase scientific understanding of disease and accelerate innovation to help future patients.
Public-private biobanking is not a new concept. In the 1970s, after demonstrating that metabolites in urine revealed far ranging health information including sex to onset of disease, Linus Pauling and Art Robinson obtained public and private funding to follow 10,000 patients, collecting patient biospecimens at 6-month intervals over 5-years. Unfortunately, just after approval of the project, the two gentlemen got in a dispute over the role of vitamin C and the biobank was not built.
More recently, the “All of Us” research program embarked on an ambitious campaign to improve the prevention and treatment of disease by following at least 1 million diverse persons over the next decade or more collecting and curating health surveys, electronic health records and biospecimens. Researchers will be approved access to this data. A quick glance at the current approved researchers reveals an impressive list of world-leading academic researchers- notably missing is the same who is who of innovators from industry. That worries me. If we want to be able to translate the scientific findings from this incredible dataset into bona fide products we need industry participation.
Today metastatic breast remains incurable with a 5-year survival rate of only 22%. Imagine if we collected, stored, curated and analyzed a biospecimen from every metastatic breast cancer patient. What if patients, clinicians, academics and industry innovators worked together to ensure rigorous quality and scientific standings were upheld. Think about the discoveries we could make. We would likely uncover novel biomarkers to better diagnosis and monitor the disease, as well as biomarkers to tailor treatments for optimal response. It’s even likely that we would uncover novel therapeutic targets that revolutionize our treatments paradigms just as the discovery of BCR-ABL and imatinib did for CML.
Right now, estimates suggest current biorepositories are underutilized and yet innovators complain about access to adequate to patient samples. Can both be true? Sadly yes. Access and quality assurance to patient samples are impeding innovation. Forming public-private partnerships can change this- we owe it to patients to work together.