Most of the life-saving drugs we use today are non-living entities. We modulate our bodily functions by hijacking the chemistry underlying our biology. This is because we have historically lacked the understanding and ability to control the fundamental unit of life – the cell. But, after years of scientific and medical advances, we are now ready to unleash the power of using cells as therapeutic “drugs”.
In recent years, cell therapies have emerged as efficacious treatments for end-stage blood cancers, saving thousands of lives and providing hope to patients. But these results only mark the beginning of what will be an extraordinary revolution in medicine, as we look at potential cures for solid-tumour cancers such as ovarian cancer, genetic diseases such as sickle-cell anaemia and autoimmune diseases such as Crohn’s disease. In light of these discoveries, 2019 will mark the year that we come to see the cell as a central part of the standard pharmacological repertoire.
Two great waves defined the success of the pharmaceutical industry in the 20th century: the development of small-molecule drugs, such as Lipitor, which is used to lower cholesterol; and that of biologics, such as the anti-inflammatory drug Humira.
The first wave used chemistry to understand how small-molecule drugs affect our bodies. Early pharmaceutical firms were arguably more skilled at complex chemistry than biology, and to this day, pharma companies still employ some of the top chemists in the world. The second wave was the discovery and use of biologics – peptides, proteins and antibodies that are manufactured by living organisms and engineered using insights from genomics and proteomics.
In 2019 we will see a third wave come into its own – cell therapies. We can now manipulate large numbers of cells with advanced microfluidics, read genomic information via sequencing, introduce new genes with viral vectors, edit the genome with CRISPR and make sense of all this information with computational biology and bioinformatics. With these breakthroughs we are entering a time where we will conceivably be able to program cells to do complex tasks, such as repairing wounds, distinguishing cancerous from benign tissue, and delivering drugs to sites where they detect disease. And we have already begun to build the manufacturing and logistical infrastructure to deliver these therapeutic cells to patients all over the world.
Such developments have not gone unnoticed by industry and government. First-generation cell therapies have led to multibillion-dollar company exits and sparked a burst of research and investment in novel treatments in many areas. And the US’s Food and Drug Administration, via its Center for Biologics Evaluation and Research, is smoothing the way for the rapidly evolving field of cell and gene therapies. It is often easier to approve new therapies in this space than in the well-trodden path of small molecules.
In 2019, we will finally move out of the installation phase of this technology cycle and into the deployment phase. No longer will we be asking if we can provide these treatments, but instead we will be asking how we can provide them – for all patients who are eligible. There will be big debates about costs, as cell-based therapies are still expensive and so are inaccessible to many. But there will also be huge demand. These therapies have the potential to save thousands of lives and many families and communities will be pushing hard to access them.
Francisco Gimenez is a principal and Joe Lonsdale a general partner at 8VC, a San Francisco-based biotech VC fund
This article was originally published by WIRED UK
