A medical miracle to treat phenylketonuria (PKU) may be on the horizon. In the U.S. and many other countries, babies are tested to see if they have the rare, inherited disorder that leaves them unable to break down the amino acid phenylalanine.
For those who do have PKU, what follows is a strict diet without meat, cheese, fish, nuts, eggs, beans, or other dietary protein for the rest of their lives. If phenylalanine builds up in the blood major health problems will result: neurological problems, intellectual disabilities, psychiatric disorders, and other serious effects. While there is an Food and Drug Administration (FDA)-approved medication that can help some people with PKU (in conjunction with a strict diet and frequent blood testing), treatment options are incomplete and sparse.
{mosads}A new approach to help PKU patients break down phenylalanine may be possible, however, thanks to synthetic biology, an emerging scientific field that aims to make biology easier to engineer. Synlogic — a biotechnology company — announced positive results in early testing for a treatment which involves drinking genetically reprogrammed bacteria.
Once the modified bacteria find their way into the gut, joining the other helpful bacteria that make up the gut microbiome, they switch on their engineered ability to break down the phenylalanine. So far, the tests demonstrated the safety of the treatment, but there was also good evidence that the bacteria were doing their job to break down phenylalanine. The next step for Synlogic is to enroll patients with PKU in clinical trials.
The promise of synthetic biology extends even beyond medicine and treating diseases, into improving agriculture yields, remediating pollution, and making manufacturing more environmentally friendly.
Synthetic biology already has applications to produce a variety of flavorings, adhesives, detergents, and cosmetics, and other products are on the horizon. There are substantial economic gains at stake, as well: BCC research reported that the global synthetic biology market was $4.4 billion in 2017, and it is anticipated to grow at an annual rate of 26 percent to reach $13.9 billion by 2022.
But where will that economic growth take place, and who will benefit? The U.S. is currently the dominant force in the synthetic biology field, boasting biotech companies like Synlogic as well as major research centers. However, the U.S. can’t afford to be complacent. Other nations have evaluated the potential for synthetic biology to improve their economies, and have invested accordingly, including the UK, France, Japan, Germany, and especially China. China’s science is growing in significance, but the indicators for U.S. science are going the other way.
Chinese scientists now produce more scientific articles than U.S. scientists, China is expected to pass the U.S. in R&D investments by the end of this year, and top-flight scientists from all over the world are getting lured to China with generous funds to set up laboratories. U.S. scientists also have to deal with restrictive visa policies that make international collaborations difficult, and make it hard to retain scientists who have trained in cutting-edge U.S. labs.
The rise of science in China and elsewhere does not have to be a net negative for the U.S., but the U.S. should still seek to turn those indicators around. Advances of science and technology worldwide will bring new possibilities for discoveries and international collaborations for U.S. scientists. Given that biological research is a proven path to learning more about our planet, our bodies, our illnesses, and — it is to be hoped — ways to prevent and cure those illnesses, it is a good thing multiple nations are taking biological research seriously.
There is an additional, subtler reason why the U.S. must maintain its excellence in the biomedical sciences: to set the “rules of the road,” or governance, for biotechnology. Regulations and standards always lag behind technology development, so the rules (standard practices, cultural expectations, publication standards, and safety measures) will necessarily be developed by nations and their experts at the technical leading edge.
For example, when Jennifer Doudna, who is one of the biologists credited with developing the gene editing tool CRISPR, became concerned about unintended uses and effects of the technology, she and others called a meeting to consider the ethical and safety ramifications of human germline editing.
In germline editing, modifications to sperm or egg DNA would not be just applied to one person, but to their progeny. Doudna’s effort led to a U.S. National Academies of Science and Medicine initiative to inform decision making for genome editing, including the clinical, ethical legal, and social implications of their use. A second summit on human genome editing will be held in Hong Kong later this year, co-hosted by the Academy of Sciences of Hong Kong, the Royal Society of London, and the U.S. National Academies. These gatherings are important because they define for the scientific community what the accepted norms are for the scientific community, so that this work can proceed safely and in the public’s interest. In the future, where will those conversations be initiated? Who will decide, and who will prioritize?
Synlogic’s exciting breakthrough has the potential to change lives for the better, and other synthetic biology products could also be on the horizon. As long as synthetic biology research and development flourishes in the U.S., the benefits will also extend to the U.S. economy and security.
If policymakers in the U.S. and U.S. scientific institutions want to see more of these medical miracles, as well as to have a say in the direction of these biotechnologies — about their safety, security, and prioritization — U.S. scientists must be at the technological forefront. To remain there, U.S. scientists need their government’s support.
Gigi Gronvall, PhD, is a senior scholar at the Johns Hopkins Center for Health Security and author of Synthetic Biology: Safety, Security and Promise.