This article describes three landmark scientific advances that have saved countless lives.

Gene editing – a miracle cure
KJ Muldoon was born in 2024 with a genetic defect that prevents the liver from processing food in his tiny body. Untreated, this rare disease would cause brain damage and eventual death. Only a liver transplant could save KJ. Such a transplant was not possible due to his age and frail condition. His genetic defect needed to be corrected.

Twelve years before KJ’s birth scientists published a new technique, CRISPR-Cas9, that allowed precise correction of genetic defects.
This method could fix the single base pair in KJ’s DNA causing his disease. To treat KJ substantial hurdles were overcome including
1) designing the correct DNA and
2) delivering this new DNA to the liver.
The methods created for delivering the COVID mRNA vaccines provided the delivery system. Scientists raced to develop the protocols and KJ received his first treatment when he was six months old. A year later KJ is thriving.
This miracle cure for a precious baby boy was based on years of research funded by the National Institutes of Health (NIH). This live-saving intervention only helped one individual, but NIH research has helped many others.

Immune checkpoint inhibitors
In 2015 President Jimmy Carter had a skin cancer (melanoma) that metastasized to his brain. Even with aggressive treatment the expected survival was less than one year.
President Carter started a new innovative therapy and died nine years later. He enjoyed an excellent quality of life during this time, continuing his work with Habitat for Humanity and monitoring elections to prevent fraud.
What was the revolutionary treatment?
The new treatment was an inhibitor of immune checkpoints. Immune checkpoints are part of the immune system your body uses to prevent runaway immune reactions that could make you extremely ill after an infectious disease. These checkpoints dampen immune reactions so that many individuals with colds only have a mild fever and cough.

Rampant immune responses may damage organs (sepsis will be discussed in a future column). Immune checkpoint inhibitors remove this “brake” so that immune system attacks and eradicates cancer cells.
There is an old joke: “I’ve cured cancer several times, but only in mice.”
Dr. James Allison, an National Institute of Health-funded scientist, discovered immune checkpoints and blocked these molecules with inhibitors and eradicated cancers in mice. He was able to successfully translate this therapy to humans, and cancer patients now benefit from his research.
Dr. Allison won the Nobel prize in medicine for his pioneering work that has been used to treat hundreds of thousands of patients. But research funded by the NIH has helped even more people with a common disease – diabetes.

Diabetes.
In the United State more than 40 million Americans live with diabetes, a disease due to inappropriate glucose (sugar) control.
As a diabetic, these new therapies have particular relevance in my life. Under normal conditions your body converts the food you consume into digestible elements, including glucose, that move into the blood. Your liver also makes glucose, a process called gluconeogenesis.
To regulate glucose levels, cells in the pancreas secrete insulin, which lowers your blood glucose levels. Most diabetics do not produce sufficient insulin, and prolonged high glucose levels cause organ damage.
While diabetics may be treated by injecting insulin, significant problems occur with this therapy. Insulin must be injected since it is a protein and would be degraded in the gut so that it would no longer be effective. Insulin must be tightly regulated since too much insulin causes blood glucose to plummet. The resulting hypoglycemia may cause seizures and even death.

When I graduated from medical school in 1982 the main diabetes treatment was injecting insulin.
Research funded by the NIH increased our understanding of how the body regulates glucose beyond just secreting insulin. This knowledge allowed the creation of new classes of drugs, drugs that can be taken by mouth. These newer drugs use normal body functions to keep glucose levels within the normal range.

One class, the glucagon-like peptide1 (GLP-1) agonist, e.g. Ozempic, stimulates the pancreas to produce more insulin.
Another class, the sodium glucose cotransporter 2 inhibitors, e.g. Jardiance, causes the kidneys to secrete more glucose into the urine which reduces blood glucose levels.
These new classes of drugs effectively treat diabetes and reduce the number of cardiovascular events such as heart attacks and strokes.
Research funded by the NIH created new classes of drugs that treat a range of diseases, from single genetic defects to cancer to diabetes. One study showed that every one of the 210 drugs recently approved by the FDA were based on research funded by the NIH.
Federally funded research literally saves lives. Your tax dollars have been wisely spent.

Daniel Remick, M.D. is Professor Emeritus of Pathology & Laboratory Medicine at the Boston University Chobanian and Avedisian School of Medicine and former Chair of that Department. He was a Professor of Pathology and Assistant Dean of Admissions at the University of Michigan from 1986 to 2006. His research has been supported by grants from the NIH for more than three decades. Dr. Remick grew up in Duluth and graduated from Central High school in 1971.