20/20 Vision: The Most Critical Discoveries This Century
Oct 22, 2020 Sherry Angel
A devastating disease or injury often challenges doctors—and patients—to overcome seemingly insurmountable odds. But a robust pipeline of research discoveries can bring hope where it is needed most. At the dawn of a new decade, let’s look back with 2020 vision at how some of the biggest medical advances of the past 20 years have emerged from Cedars-Sinai’s labs and transformed patient lives.
A serious health crisis can suddenly change everything—your way of life, your sense of control over your corner of the universe, your outlook for the future. It can throw you into a fight for survival that demands more strength than you ever thought you had.
It can also make you a partner in pioneering, life-changing medicine.
That’s what happens for many people with complex conditions who seek treatment at Cedars-Sinai, where major medical discoveries across many disciplines have created an array of innovative treatment options over the past two decades.
The five patients who share their stories here each played a vital role in their own recovery as they received highly advanced and, in some cases, experimental treatments at Cedars-Sinai.
One patient faced a grim prognosis after being diagnosed with the deadliest form of brain cancer. Another suffered severe pain and other debilitating symptoms from a chronic digestive disorder. Pulmonary arterial hypertension made it difficult for one woman to walk even a short distance. A young man faced two rare, life-threatening blood disorders. And a professional dancer suffered a serious injury that could have ended her career.
It takes trust and courage to be among the first to benefit from a new treatment or approach to medical care. But these patients were all in. Here are their stories.
More 20/20 Breakthroughs
Regenerative Medicine: Trailblazers in Stem Cell Science
They are trailblazers intent on activating the regenerative powers of the human body.
For a decade, researchers at the Cedars-Sinai Board of Governors Regenerative Medicine Institute (RMI) have accelerated progress in an emerging field that focuses on restoring diseased or aged tissues by revitalizing existing cells or transplanting new ones.
Under the leadership of Clive Svendsen, PhD, a renowned stem cell scientist, the institute has made strides toward developing new therapies for Alzheimer’s disease, Huntington’s disease, Parkinson’s disease, amyotrophic lateral sclerosis (ALS), cancer, stroke and other neurological disorders. RMI scientists have also made important discoveries about aging, eye conditions and tissue regeneration for repair of broken bones.
"We are using stem cells to both model and treat human disease," says Svendsen, the Kerry and Simone Vickar Family Foundation Distinguished Chair in Regenerative Medicine. "In our personalized medicine program, we are able to recreate a patient's disease in a petri dish by placing stem cells on tiny chips. We can emulate how cells behave in the human body to learn about what causes diseases and develop novel drugs to fight them."
Cedars-Sinai is one of the world’s largest producers of induced pluripotent stem cells (iPSCs), which are made by reprogramming an adult’s skin or blood cells into an embryonic state. Scientists can then use iPSCs to make any type of cell in the body to create disease models for study or cells for transplanting into patients. This type of innovation will be the focus of the Cedars-Sinai Biomanufacturing Center, scheduled to open this year. Under strict U.S. Food and Drug Administration guidelines, the center will be able to generate cells that can be used in clinical trials to develop new treatments for health issues ranging from cancer to neurological conditions.
RMI researchers are translating basic science into new therapies at a remarkable pace, Svendsen says. Clinical trials now underway include experimental treatments for ALS—a progressive, deadly neurodegenerative disease that causes muscle weakness, atrophy and paralysis—and retinitis pigmentosa, an inherited eye disease that progresses to legal blindness in adulthood and has no known cure.
Sudden Cardiac Arrest: Confronting a Lethal Foe
In 9 out of 10 cases, sudden cardiac arrest (SCA) causes death before help can arrive.
For two decades, Sumeet Chugh, MD, medical director of the Heart Rhythm Center in the Smidt Heart Institute and the Pauline and Harold Price Chair in Cardiac Electrophysiology Research, has been working to overcome these terrible odds.
His mantra: “Find out who is at highest risk so we can fix heart rhythm problems before they trigger cardiac arrest.”
The communities of Portland, Oregon, and Ventura, California, are Chugh’s living laboratories. Through the landmark, long-term Oregon Sudden Unexpected Death Study, and more recently the Ventura Prediction of Sudden Death in Multi-Ethnic Communities study, his group has produced a series of crucial discoveries related to genetics, health issues and lifestyle. The study has linked SCA to obesity, multiple sclerosis and left ventricular hypertrophy (enlargement and thickening in the heart’s left pumping chamber)—and has identified a significantly elevated risk among African Americans.
In 2020, Cedars-Sinai established a dedicated Center for Cardiac Arrest Prevention at the Smidt Heart Institute. Under Chugh’s direction, the center collaborates with researchers within and outside Cedars-Sinai to discover and implement proven, effective prediction tools in the community and the clinic, and to measure the impact of early identification and treatment.
Irritable Bowel Syndrome: No Longer Misunderstood
It’s all in your head.
This was once the prevailing attitude about irritable bowel syndrome (IBS), a chronic condition that causes abdominal pain and bouts of diarrhea and constipation. But Mark Pimentel, MD, executive director of the Cedars-Sinai Medically Associated Science and Technology Program, didn’t believe the debilitating symptoms he saw in the clinic were psychological.
Determined to find a cause and a cure, he followed his instincts down a scientific path that led to a sea change in the attitude toward this disease.
The villain in IBS is not stress—it’s bacteria, Pimentel discovered. His research led to the first IBS treatment to provide long-lasting symptom relief for patients: an antibiotic called rifaximin. He also developed the first blood test for faster, more accurate diagnosis of IBS.
"There is now a test that says this is real—it’s organic, not psychological," he says. “And if you have it, we can treat it. That’s good news for millions of people."
Better Imaging, Better Care
A steady flow of imaging innovations at Cedars-Sinai has given clinicians a clearer view of the inner workings of the human body. Technological advances have led to more accurate diagnosis as well as more effective treatments for a broad spectrum of diseases. These include:
- High-quality 3D MRI images of the heart that produce detailed information on heart function to help cardiologists make the best treatment decisions
- New imaging biomarkers and artificial intelligence that predict pancreatic cancer years before it occurs
- A florescent dye—made from a compound found in scorpion venom—that lights up cancer cells under a high-sensitivity, near-infrared camera developed at Cedars-Sinai, enabling surgeons to see and remove potentially lethal brain tumors that can be hard to distinguish from healthy tissue
- Enhancing the targeting capacity of intensity-modulated radiotherapy to deliver more precise radiation therapy for head and neck cancer, with less toxicity to healthy tissues and structures