Modern medicine broke another barrier on Monday, Nov. 13, when scientists at the University of California, San Francisco (UCSF) edited genes inside of a man’s body for the first time in human history. The operation, conducted on 44-year-old Brian Madeux at the UCSF Benioff Children’s Hospital in San Francisco, is the beginning of a field of medical practice that could save millions of lives in the near future if it is properly funded and supported. Stagnating statewide budgets for medical research across the country, in addition to the Trump administration’s proposed budget cut of up to $13 billion from medical research institutes, however, threaten to stop the rapid advance of medical technology in its tracks.
Madeux’s treatment was an effort to cure a rare genetic disorder known as Hunter syndrome, by scripting his cells to produce an enzyme called I2S, the lack of which causes a metabolic failure to break down particular sugars in the body. The procedure is the newest form of gene therapy, a process by which a missing gene is inserted into a patient’s DNA so that the affected cells will reproduce with modified DNA until the new gene becomes ubiquitous in the patient’s genetic composition. Traditionally, gene therapy has generally inserted viruses toward targeted locations in the patient’s genome that do not always perfectly meet their mark and have, at times, increased risk of cancer.
The procedure performed on Madeux differed because direct gene editing allows for much greater precision, which is expected to increase effectiveness and reduce the risk of getting cancer. The company that operated on Mr. Madeux, Sangamo Therapeutics, used microscopic gene editing tools known as zinc-finger nucleases to alter the DNA inside the active cells inside patients’ bodies. Sangamo is also hoping to use this technology to treat additional conditions such as hemophilia B, Hurler syndrome, and sickle cell disease.
Traditionally, gene editing has been considered too dangerous, unpredictable or difficult to be done properly; however, new technology advancements such as Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) have shown the promise and safety of this new type of technology. Experts predict that gene editing inside of human patients could be used to effectively treat or even cure more than 10,000 genetic conditions, and even stop hereditary transmission to future children.
CRISPR, like many medical advancements over the past half century, was developed by researchers at the Massachusetts Institute of Technology, with funding and support from the federal government. At institutions like Harvard University, 75 percent of all medical research funding comes from the federal government. Although corporate spending on medical research has more than tripled since 2006, the medical world as we know it is still completely reliant on the federal government’s support to fund research and innovation.
The foundation of American medical research funding as it is known today can be traced back to the founding of the National Institutes of Health (NIH) in 1887 and to former president Harry Truman’s establishment of the National Science Foundation (NSF) in 1950. As federal budgets for medical research have stagnated, pharmaceutical corporations and philanthropists have mostly picked up the tab, bringing their own risks and disadvantages. Pharmaceutical companies mostly engage in what is known as “applied research,” or research aimed toward a “specific commercial goal,” which leads to great innovation, but is too focused on profit to be relied on. And reliance on philanthropists to fund our medical research is not only impractical but also an incredibly unstable revenue stream.
Pharmaceutical research and philanthropist funding, however, are not the biggest threat to medical research today. President Trump’s budget proposal for 2018 proposes to cut funding for the NIH by nearly 20 percent and to “reform” funding to the Centers for Disease Control and Prevention (CDC), possibly to be replaced by block grants to states, presumably to make room for tax cuts for the wealthiest Americans. According to a study done by economist Bruce Weinberg of Ohio State University, the NIH granted more than $3.5 billion for medical research to just nine major universities in the midwest — a critical source of their funding. Any reforms to medical research funding should be aimed at either increasing funding to existing programs or creating additional ones, not siphoning from highly effective agencies that live off of government funding.
CRISPR treatment, and the wide array of new possibilities it has brought, is a product of the past half-century’s prudent medical research policy. We have seen rapid development of medicine in the past 50 years unrivaled by any other period in human history. Gene editing has the potential to save and improve the lives of millions, but it is an incredibly complicated procedure that will require substantial federal funding and support to truly enter the economy as a common treatment. We are in a golden age of medical advancement that we cannot take for granted. Cutting critical medical funding to fill the pockets of wealthy donors will stifle the incredible potential of these technologies and leave millions more waiting, helpless, because no effective treatment for their conditions has been approved yet.
