Thursday May 30, 2013 during the annual biotech convention ‘Knowledge for Growth’ professor Jo Vandesompele and dr. Els Vanheusden shared their insights and experience on how to go beyond innovative scientific research and how to work towards a clinical, medical solution.
In their talk "Cancer biomarkers: surviving the journey from bench to bedside" both speakers presented the model efficiently used by the Fournier-Majoie Foundation to optimize the transition from biomarker discovery to biomarker clinical development.
Jo Vandesompele is Professor in Functional Cancer Genomics and Applied Bioinformatics at Ghent University, Belgium since 2007 where he supervises a team of 15 researchers. He is author of more than 140 scientific articles in international journals, including pioneering publications in the domain of quantitative PCR. For his research work on neuroblastoma, a childhood cancer originating from nervous system cells, he received a first grant (340 000 €) from the Fournier-Majoie Foundation in 2009 and – together with Frank Speleman - a follow-on valorization grant (€417.000) in 2013.
Els Vanheusden, MD, COO is managing operations at the Fournier-Majoie Foundation, a Venture Philantrophy Organisation. The mission of the Foundation is to recognize and support entrepreneurs who are willing to develop solutions to significantly benefit cancer patients. Since 2007, Fournier Majoie Foundation has built a portfolio of 9 cancer biomarkers, at different stages of development.
You have missed the talk? You can read below what both speakers shared.
Professor Jo Vandesompele begins his presentation with an appalling fact: “One in four children diagnosed with neuroblastoma dies. Besides, today’s standard for assessing the survival probability of children suffering from neuroblastoma is insufficient.”
Aware of this medical need, prof. Vandesompele and his team decided to take action. The team is determined to identify those high-risk patients that will benefit from the existing therapy from those that won’t, via a molecular signature. Their ultimate goal is to separate the ultra high-risk patients who do not benefit from existing therapy and make them eligible for new molecular targeted drugs. Since 2009, their research supported by the Foundation has identified two tissue based gene-expression signatures: one based on messenger RNA and the other based on microRNA (1, 2). For both signatures, patent applications have been filed.
While the performance of the tissue signatures is outstanding, a serum based signature would better meet the medical need.
In addition, prof. Frank Speleman, co-lead of the follow-on valorization project rewarded in 2013, is actually completing the discovery of a DNA methylation signature and both research teams hope to combine DNA methylation, miRNA and mRNA expression signatures into a single integrated classifier.
The current tests based on biopsies are invasive. “It is especially difficult to apply these tests on patients with overt metastases for which we don’t do biopsy because it is just useless additional stress for the child” said Jo Vandesompele. Furthermore those tests require good quality tissue samples that aren’t always easy to collect and store. A serum-based test would be both non-invasive and easier.
The project’s evolution is illustrative for the support of the Fournier-Majoie Foundation: it goes beyond the regular financial support and adds a large array of expertises to enable and accelerate the early project development phase. “The foundation adds a lot professionalism and realism in the daily project management” prof. Vandesompele adds.
Finally, professor Vandesompele shared some important lessons learned along the journey with Fournier-Majoie Foundation. To be able to clinically validate a test, it is very important that researchers secure access to high quality samples as well as foresee complex storage needs at the very beginning of the project, even when not dealing with rare diseases like neuroblastoma. Another important parameter is to add medical feedback and insights from key opinion leaders from the early stages on. For instance what the clinicians are expecting in terms of sensitivity and accuracy? It is very important to align medical expectations with the researcher’s expectations. That is one of the reasons why Jo Vandesompele’s team is for instance transitioning from an invasive tissue based test to a non-invasive serum based test. Finally the professor emphasized that it is also important to look beyond your current research; are applications beyond the pure diagnostic/prognostic utility possible? The Foundation stimulates and challenges with regard to those aspects.
According to dr. Els Vanheusden it is important that researchers work with the clinical endpoint in mind. She refers to the GOBIOM database in which approx. 20.000 existing biomarkers are listed: to date only about 100 biomarkers have been validated for use in the clinic (of which 7 are multiplex In Vitro Diagnostic tests). Researchers are often unaware of the barriers associated with biomarker development. Just to name a few: lack of clinical insight; deficient study/sample populations; inappropriate statistical methods; lack of assay robustness; stringent regulatory requirements, including the set-up of quality assurance and quality control systems; limited understanding of the market /competition and insufficient freedom to operate…This is where the Foundation can add value to research projects.
Fournier-Majoie Foundation enters projects at the stage of early development only. Research teams must have completed the discovery phase and patents have been filed. We then accompany the early development (qualification, verification and validation studies) up to the moment industry partners become interested. .The foundation has a clear focus on cancer only, and more particularly on diagnostics (biomarkers/imaging markers/technology platforms) and health-IT solutions to significantly benefit cancer patients.
“We use a venture philanthropic model” dr. Vanheusden adds, “over the past years, the foundation has supported the development of several biomarker-projects with both financial support and knowhow provided by experts in the market. We define the VP model also by the fact that - if a successful project will result in financial returns, these returns will be for 100 % reinvested on new development projects”.
This approach is unique on the continent and we believe it will help cancer biomarkers surviving the journey from bench to bedside.