Earlier this year I wrote about a new model for healthcare – one of ‘personalized medicine’ – in which all patients receive exactly the right diagnoses and treatments, personalized to them as individuals. So it was particularly interesting to hear, at the recent BioIntegrates conference in London, that the pharma biotech industry, at least, is now unified by the goal of "providing the right therapy to the right patient at the right time".
As a result, we now see biomarker-based approaches being integrated in more than 40% of new prospective drug development programmes. This concept of ‘biomarker-enabled drug discovery’ is helping drug developers to maximize efficiency and minimize risk, as it increases success rates for new drug approvals thanks to higher response rates in specific, selected populations.
Amgen, Gilead and GSK are said to be maximizing on the potential for biomarker-enabled drug discovery, and there is a strong focus on oncology, with Roche, Janssen and Merck & Co all showing strong pipelines of late stage candidates. Due to the natural heterogeneity of different types of cancer, which can be biomarked, the field of personalized oncology has seen the most rapid advancement.
As explained by Maria Antonietta Cerone, Precision Medicine Portfolio Manager at Cancer Research UK, the second-largest funder of cancer research in the world, “Instead of looking at one drug for one type of cancer patient, new molecular profiling technologies allow a granular patient stratification with subgroups of patients within one cancer type potentially benefiting from more targeted drugs.”
Jeff Settleman, PhD, Pfizer’s new Senior Vice President and Group Head of Oncology R&D, said in a recent press release, “There has never been a more exciting time in cancer research,” referring to Pfizer’s “highly promising pipeline that includes a variety of targeted therapies.”
So far so good – clearly, advances in genetics and genomics a playing a significant role in enabling the identification and implementation of biomarkers in medical research. However, how does this R&D translate to market?
In fact, the manufacturing of personalized treatments would be prohibitively expensive due to, for example, the necessarily small batch sizes, were it not for other technological advances that are permitting the growth of this sector in a more affordable manner. Reducing the cost of manufacturing is key for the success of personalized medicine.
According to Thomas Theelen, Business Development Manager at Univercells, commenting specifically for this column, “We have observed that personalized medicine, such as cancer vaccination, is enabled by platform technology developments. Several companies have invented biological platforms allowing rapid integration of cancer neoantigens in viral vectors. Given the relatively small batch sizes and the short timelines to obtain the personalized vaccine, a suitable small-scale equipment platform is needed. Univercells’ manufacturing technology allows [us] to mimic a standard vaccine manufacturing process at bench scale with sufficient yield for one patient. A high volumetric productivity is key since one facility should enable manufacturing personalized vaccines for multiple patients simultaneously. Therefore, the full process fits into a biosafety cabinet, which allows setting up multiple small units in a relatively small area. The downsizing combined with single use equipment also helps to contain costs.”
Another challenge is that the application of biomarker-driven therapies requires the assessment of biomarkers in clinical practice.
“There is a risk of identifying biomarkers that cannot be widely used in clinical practice due to the complex assays required for their identification,” explains Cancer Research UK’s Maria Antonietta Cerone. “Clinical trials allow testing of biomarkers in wider populations but often the assay used in those settings are either too complicated or too expensive to be easily translated into routine care. However, in several cases accompanying in vitro diagnostics test are also developed to facilitate implementation.”
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