Riding a New Wave of Translation

Clinicians, patients and the FDA are pressing biotech and pharma scientists to deliver more thoughtful and comprehensive translational strategies that can help to answer multidimensional clinical questions. This process is constantly hungry for innovation.

Next generation sequencing (NGS) has taken academic centers, biotech hubs, clinics, and investors by storm. Faster, better, more efficient sequencing opened promising translational avenues for complex diseases, genetic disorders and the microbiome. The speed and thoroughness with which NGS penetrated biotech, academia and pharma was amazing to witness. With massive promotion and impressive investments, NGS has become the defining trend of translational science – along with its promise to characterize human diseases and transfer the power of genomic research into the clinic.

The illusion that we see the landscape objectively is a powerful one, so it can be hard to accept that, while genomics tech is greatly contributing to the field, it has a lot of limitations. Moreover, genomics has yet to deliver on all its promises. Ultimately, genomics only gives us a partial solution to more fundamental questions about highly heterogeneous and fast evolving cancers, infections, autoimmune and viral diseases, and strategies to treat them. For example, neither genomics nor transcriptomics can deliver a complete and comprehensive answer to how a tumor will differentiate, proliferate, metastasize and adapt to therapy. Supported by generous funding, genomics has created millions of “me too” methods and companies, which cannot truly be called “innovation”. In my view, it’s time to think outside of the genomics box. If we really want to see a new wave of innovation, intelligent thinking should not be fragmented or driven by trends in technology.

What is the next step for translational science? The human proteome is far larger and more comprehensive than the genome. Moreover, the proteome is ultra-sensitive to intra- and extra-cellular stimuli and environmental factors. This gives us a significantly wider window of opportunity than genomics when it comes to designing translational strategies to resolve critical questions about patient stratification, complex diseases and response to therapeutic agents. Multiplexed and multidisciplinary technologies for robust exploration of the proteome in blood and other liquid biopsies are becoming powerful tools for translational science, molecular diagnostics and clinical trials.

It is more challenging to work with the proteome than with the genome or transcriptome. The proteome requires more sophisticated strategies, complex techniques and highly skilled scientists. That is where modern biotech, engineering and information technologies come in. The comprehensive integration of molecular biology with nanotechnology and powerful algorithms, search engines, and big data management systems can lead to outstanding opportunities that I believe will address translational science questions and personalize medicine needs in a more efficient and thoughtful manner than all previous efforts taken together.

Who will accelerate this next phase of translational science? I think it unlikely that old-fashioned, conservative institutions like the NIH will contribute a great deal to this process – conceptually novel, high-risk projects are not members of the “NIH club”. Instead, I foresee a key role for biotech hubs like Silicon Valley, which constantly integrate new angles of science with information technologies and high-tech inventions. In Silicon Valley, we aren’t afraid to take a risk, and to mix and match different approaches to find better solutions for high unmet medical needs. Innovation is our religion here, and I believe cutting-edge high-tech hubs and startup ecosystems will be the forerunners in the next wave of transformation in translational science.