A third of all medications stem from this discovery

About 35% of all medications on the market target what’s known as G-Protein Coupled Receptors (GPCRs). This is a family of receptor proteins that sit on the cell surface and allow cells to sense the environment they are in and react to what is going on around them. Most of our biological processes depend on these receptors.

These cell receptors were unknown until they were discovered and characterized by Duke’s Robert Lefkowitz, along with Brian Kobilka of Stanford (who was a postdoc in the Lefkowitz lab) – earning them the 2012 Nobel Prize in Chemistry.

Their work led to the creation of drugs that function by targeting these GPCRs  — including beta blockers, anti-anxiety pills, antihistamines and much more. This research was funded in large part by the National Institutes of Health and the Howard Hughes Medical Institute.

The ‘a-ha’ moment of hearing with cochlear implants

A baby hears a parent for the first time; an adult gets back that sense after experiencing hearing loss – both examples of people whose lives have been changed with cochlear implants.

The cochlear implant is a device that unlocks sounds for those who are deaf or severely hard-of-hearing. More than a million people worldwide have received cochlear implants for one or both ears, and nearly all of them are able to talk on telephones, without needing visual cues such as lipreading. They work by mimicking the ear: picking up sound with a microphone, processing the input from sound waves to electrical signal outputs, and delivering these electrical impulses to the auditory nerve.

Duke’s Blake Wilson has been a pioneer in developing the modern cochlear implant, working on the technology with colleagues at RTI International in Research Triangle Park and co-founding the Cochlear Implant Program at Duke in 1984. He developed methods crucial for the implants to be able to process signals, dramatically improving speech clarity. This research was funded by the National Institutes of Health.

The first treatment for Pompe disease

Pompe disease is a rare genetic disorder that causes muscle weakness. It occurs when the body lacks the protein needed to turn stored sugar (glycogen) into glucose, which our bodies use for energy. Without it, glycogen builds up and damages tissue, especially the heart and skeletal muscle. Some babies are born with symptoms of Pompe; other people develop symptoms later in life.

For years, there was no treatment, and babies born with it would likely not live to their first birthday. But after more than two decades of research, Duke scientists, including Drs. Y.T. Chen and Priya Kishnani, developed the first therapy.  Approved by the FDA in 2006, the drug alglucosidase alfa — sold under the brand name Myozyme — replaces the missing protein, helping the body break down glycogen.

What was once a fatal disease is now treatable and a reason to celebrate.

Each year Kishnani does just that, hosting a reunion at Duke for families whose children are alive thanks to the life-saving treatment for Pompe disease.

The modern ultrasound

Noninvasive methods of seeing inside the body are a crucial advancement in how healthcare professionals monitor and treat patients. Ultrasound is one of those ways: a technology that can take images with sound waves.

At first, two-dimensional images were fuzzy and slow, produced by sweeping a device with a single ultrasound emitter across the body. But in the 1960s, Duke biomedical engineers Frederick Thurstone and Olaf von Ramm created the first devices that used a combination of ultrasound emitters that did not require sweeping. This approach allowed for cleaner and quicker two-dimensional images of organs, such as the heart beating inside the body. Working with cardiologist Joseph Kisslo, they brought the technology into clinical practice first at Duke. In the 1990s, Von Ramm and Duke biomedical engineer Stephen Smith extended the capabilities of these ultrasound systems to create 3D images.

Their techniques have been used millions of times worldwide in multiple medical specialties for a range of applications — from real-time imaging of the heart to monitoring a baby’s development during pregnancy. This research was funded in large part by the federal government, including the United States Public Health Service and the National Science Foundation.

Insight into DNA leads to better cancer treatment

Our DNA is the building block of how our bodies function, and we depend on its accurate daily replication. As in any process, though, there can be errors. A simple defect, if not fixed, can result in genetic diseases or cancer.

Duke’s Paul Modrich and his colleagues deciphered how DNA is fixed when mistakes occur during replication, a feat which earned him the 2015 Nobel Prize in Chemistry. Modrich and others also showed that the slowdown of this process causes the most common form of hereditary colon cancer. With this knowledge, clinicians now identify when specific drugs won’t work for certain cancer patients – which means fewer false starts in therapy. Modrich’s research was funded by the National Institutes of Health and the Howard Hughes Medical Institute.

From airbags to helmets: keeping your head safe

Protective equipment for your noggin relies on understanding how the head and neck react to impacts. Starting in the 1970s, Duke’s James McElhaney, Barry Myers and colleagues have done foundational work in the mechanics of head and neck trauma  — informing the design of football, bicycle and motorcycle helmets; airbags in cars; and even swimming pools. 

The researchers created experimental setups of smacking, crushing and whiplashing models of skulls and neck bones to see how they react to different types of impacts and gain a better understanding of how to protect our heads from injury. 

This research was funded in large part by the National Institutes of Health and the Centers for Disease Control and Prevention.

Help for common type of inflammatory arthritis

Gout may sound like an old-time disease, one that conjures up images of decadent aristocrats of a bygone era. But gout is the most common form of inflammatory arthritis, affecting tens of millions of people globally – even postmenopausal women, as their estrogen declines.

The disease is characterized by painful inflammation of the joints, especially in the feet — often the first symptom of gout is severe pain in the big toe. Some varieties of gout are particularly challenging to treat. 

It was work by Duke Drs. Michael Hershfield, Susan Kelly and colleagues that led to the creation of the drug Krystexxa for this ailment. Used for recurring cases of severe gout, Krystexxa was the first new gout treatment in 40 years when it was approved in 2010. This research was funded by the National Institutes of Health.