Tthank you very much steps in basic research, biology is becoming increasingly programmable. Two recent scientific breakthroughs show just how powerful the possibilities can be. Genetic modification of plants is allowing the mechanism of photosynthesis to be interfered with, as published in science on August 18 it starts. This could lead to dramatic improvements in plant productivity, and eventually a second green revolution. Meanwhile, changing the genes of people suffering from fatal incurable diseases has also had extraordinary results. A number of gene therapies have arrived or are arriving in clinics to treat blood cancers, spinal muscular atrophy, hemophilia and sickle cell disease. The task now is to spread these benefits far and wide.
The consequences of both developments could be significant. Genetic modification of crops promises cheaper, more nutritious and more climate-resilient food for a hungry planet. Gene therapies offer hope for curing devastating diseases. They also allow for one-time treatments that can be transported to the four corners of the Earth, bringing years, decades or lifetime benefits to the terminally ill. Imagine a cure for aid or sickle cell disease that could be carried to the African continent or the entire Middle East. The associated benefits would be similar to the eradication of smallpox.
This tantalizing promise has been made possible by a tremendous investment in basic research over the years. Basic knowledge of genetics and protein functions and structures has proven to be an engine for discovery in medicine and agriculture. The private sector, of course, plays an essential role in the innovation cycle. But these advances are a reminder that investment by governments and charities is essential in areas that offer little commercial benefit in the short term but that in the long term promise to greatly advance well-being. Much of the success in treating rare diseases is the result of philanthropic efforts, often thanks to fundraising by patients and their families. Research that benefits low-income countries often relies on deep-pocketed philanthropic donors such as the Gates Foundation. Investments made in basic science today reap the benefits of tomorrow’s productivity.
Unfortunately, there is no guarantee that these benefits will be realized. Gene therapies are a remarkable technical achievement. But their huge current cost—often over $1 million to treat a single person—makes them difficult for health care systems to afford, even when they are reserved for rare, fatal genetic diseases. The idea of using them to treat more widespread conditions seems prohibitively expensive.
In the past, new drugs that started out prohibitively expensive have become cheaper. According to the Boston Consulting Group, monoclonal antibodies, useful proteins made in the lab, were expensive when they first arrived, before a decade of advances brought their price down 50-fold. If gene therapy is to live up to its promise, it will have to do even better than this. Even the most efficient photosynthesis will need further investment if it is to be commercialized.
The long-term costs of producing a new green revolution will thankfully be low; plants make more plants in a way that treatments never can. However, gene therapies need innovation to lower the cost of their production, whether in bioreactors or in some completely new way. If they are to become more affordable, new therapies should also experiment with payment mechanisms, such as installment or outcome-based charging. Accumulating international demand for buying therapies could also help lower prices. This is where governments, charities and the private sector can work together.
Finally, regulators also need to realize more quickly and more the societal benefits of helping these technologies reach their potential. Innovations can die out without adequate regulation or time. Regulation of genetically modified crops has been hampered by disinformation campaigns, delaying benefits and increasing costs. Similarly, although experimental drugs undoubtedly need scrutiny, regulators should remember that the alternative in untreatable genetic diseases is often death. Science has made a genetic revolution possible. Now that revolution must flourish. ■