By Steve Arlington and John Wise, The Pistoia Alliance

As a society, we are benefiting from the remarkable pace of scientific innovation. Starting in 1990, it took scientists thirteen years to sequence the first genome. Now, this process can be completed in one day, testament to the speed of developments. We will see many more innovations like this over the next decade and beyond, some of which we have explored in The Pistoia Alliance’s report, “2030 – Life Sciences And Health Go Digital.” The report brings together insights from over seventy industry experts to predict advances in technology, medical science and healthcare that will significantly change outcomes for a variety of diseases by 2030.

Today, in the midst of the Covid-19 pandemic, it is clear that in the short term, R&D efforts in life sciences and biotech organizations will be occupied by the search for potential new treatments, including efficacious vaccines, and for therapies that can be repurposed. At the same time, in the long term, it is essential that the industry continues its pattern of innovation in many other therapeutic areas, which could benefit millions of patients around the world.

To that end, and looking ahead, we have identified four areas which will meaningfully improve patient care: stem cell therapy, gene therapy, genomics and oncology. In doing so, these patient-centric innovations have the potential to allow society to progress from a “one-size-fits-all” management and palliation of disease, to a personalized, preventive and predictive approach to patient care.

Stem cell therapy

We have already seen a remarkable increase in the development of stem cell therapy and its uses over the past decade. During this time, stem cell therapy has become a useful treatment option; more than 17,000 blood cancer patients have had successful stem cell transplants. Elsewhere, in the realms of research and drug discovery, stem cells have been used successfully to develop organoids, which are cultures that can be crafted to mimic an organ. Over the next ten years, we can expect to see this field of work accelerating rapidly. We anticipate that stem cell therapy will have advanced to the point where organoids that represent over a dozen different organs of the body are being used in a wide variety of research disciplines. Such advances should minimize the need for animal testing as well as some human testing, thereby improving the efficiency, effectiveness and ethics of drug R&D. As organoids can model healthy and diseased organs as well as specific cancers, they may also pave the way for patient-specific drug screening.

Gene therapy

It was only in the mid-2010s that gene therapy emerged as an important therapeutic intervention for rare genetic disorders. Initial research in 2012 generated tremendous interest in the academic community and among venture companies as it showed CRISPR technology could cut DNA in specific locations, which could be used to repair troublesome DNA or disable certain genes. This gave rise to several clinical studies that explored the impact of this technology on other genetic diseases, ranging from sickle-cell disease and beta-thalassemia, to multiple myeloma and inherited forms of blindness.

In 2017, Strimvelis, a gene therapy for treating adenosine deaminase deficiency (ADA-SCID), a rare, inherited pediatric disorder, was approved by the UK’s regulatory healthcare body for clinical use. Going forward we anticipate that the promise of gene therapy will become established in the next decade. Opportunities provided by big data and computational biology, will likely facilitate the evolution of DNA and RNA technologies from primarily research tools to agents in clinical developments. This could lead to advances in several rare disease disorders, such as the GBA-form of Parkinson’s disease and improve outcomes for a variety of liver and retinal diseases.

Genomics 

Breakthroughs in our understanding of genomics coupled with phenomics, the systematic study of phenotypes, is likely to improve the understanding of polygenic diseases, which are diseases that are caused by the combined action of more than one gene. Advancements in this field will enable better ways to diagnose and perhaps cure polygenic diseases such as diabetes or coronary heart disease. By 2030, we also expect that advances in genomics will facilitate the shift towards personalized medicine, so that therapies can be prescribed, indeed manufactured, to meet the needs of the patient and their specific genetic makeup.

Oncology

In the field of oncology research, the work of the ASCO Center for Research & Analytics (CENTRA) has been critical, providing researchers with the infrastructure to analyze real world data (RWD) and deliver deep insights into the cause, diagnosis, treatment and prognosis of cancers. Today’s treatment options of surgery, radiotherapy and chemotherapy to destroy cancers will be reinforced with new therapies such as immunotherapies including oncolytic viruses.

Similarly, advancements in liquid biopsy analysis using next generation sequencing (NGS), will likely equip physicians with a more complete understanding of a tumor’s molecular profile so that cancers are identified and treated earlier, increasing the probability of successful treatment. In 2019, researchers began seriously investigating the potential for proteolysis-targeting chimeric molecules (PROTACs) also known as protein degraders, which destroy rather than inhibit proteins. For example, PROTACs might be deployed to destroy the cancer-fuelling protein MYC. Looking forward to 2030, we may see significant breakthroughs in cancer treatments using such protein slaying drugs.

However, one key factor in ensuring these highly personalized therapeutics become widely available over the next decade, is a simultaneous development in how such treatments are funded. A transition from the traditional one size-fits-all, “pay per pill” model to “payment for outcomes,” will be a much-needed shift. This will help to address affordability issues and drive continued R&D momentum by ensuring the biopharma industry is rewarded for developing such innovative breakthrough therapies and indeed cures.