Did you know?
How research is improving the lives of people with chronic disease
It’s never a good time to live with a chronic illness — but it’s getting better. Medical research has transformed chronic disease management for many conditions, reducing suffering and enhancing well-being. And ongoing research promises further advancements. How has treatment for chronic illness changed, and what might the future bring? Stanford Medicine doctors who care for people with lung disease, diabetes and the aftermath of a stroke provide their perspectives.
Chronic obstructive pulmonary disease — COPD
Living with lung disease can make carrying out even simple tasks feel monumental.
The good news is that research has recently led to better treatments for COPD, a group of lung diseases that cause breathing difficulties. The conditions, which include emphysema and chronic bronchitis, are often due to long-term exposure to harmful substances including cigarette smoke.
COPD is a major source of disability and a leading cause of death worldwide. In the U.S., 6.4% of adults have been diagnosed with the disease, according to a 2021 Centers for Disease Control and Prevention survey.
One of the main symptoms of COPD is dyspnea, or shortness of breath, which can have a profoundly negative impact on a person’s life.
Though there’s no cure for COPD, basic research and clinical trials have led to therapies that not only tamp down flare-ups but also, in some cases, eliminate symptoms entirely.
“Dyspnea can cause anxiety, limit daily activities and often leave patients feeling helpless,” said Jennifer Williams, MD, co-director of the Stanford Health Care Chest Clinic and a clinical assistant professor of pulmonary, allergy and critical care medicine. “My interest in pulmonary medicine stems from a deeply personal experience: witnessing a family member struggle with long-term breathing issues. Being by their side through moments of breathlessness left a lasting impression on me.”
For many years, the main medical treatments for COPD have been corticosteroid drugs, which reduce inflammation in the airways, and bronchodilators, which relax the muscles surrounding the airways, opening them up and easing breathing. However, corticosteroids don’t work for everyone and can have side effects that prevent their use.
Though there’s no cure for COPD, basic research and clinical trials have led to therapies that not only tamp down flare-ups but also, in some cases, eliminate symptoms entirely.
“Two things that have happened during my career that have really amazed me are T2 biologics — targeted treatments for people who have both asthma and COPD — and virtual pulmonary rehab,” said Lauren Eggert, MD, director of the airways disease program and a clinical assistant professor of pulmonary, allergy and critical care medicine. “Both of these tools are really transforming patient care and allowing patients to live longer, healthier lives.”
Other advances include enhanced methods to identify specific types of COPD, which helps identify the best treatments; new drugs that are safer than corticosteroids; and new strategies to bypass the diseased parts of the lungs.
“BLVR has the potential to improve shortness of breath and quality of life.”
Harmeet Bedi, clinical associate professor of pulmonary, allergy and critical care medicine and medical director of interventional pulmonology
To help patients with severe emphysema who don’t get relief through other treatments, for example, doctors can turn to procedures like a bronchoscopic lung volume reduction (BLVR) to reroute respiration. The method typically involves inserting one-way valves into the airways to block airflow to diseased parts of the lung.
The minimally invasive procedure addresses the problem of air trapping, which occurs when air gets stuck in the lungs during exhalation. Air trapping leads lungs to expand beyond their normal size, making it harder to breathe.
“BLVR has the potential to improve shortness of breath and quality of life,” said Harmeet Bedi, MD, medical director of interventional pulmonology and a clinical associate professor of pulmonary, allergy and critical care medicine.
However, lung volume reduction isn’t always a good option, especially for people whose lungs exhibit widespread disease or have extensive collateral ventilation — breathing routes that bypass the lung’s usual airways.
“Unfortunately, a large proportion of COPD patients fit into this category,” Bedi said. “R&D for bronchoscopic lung volume reduction is really focusing on therapies that can provide less air trapping regardless of collateral ventilation status. Numerous companies are working on different devices that could be implanted and achieve success in such patients.”
Diabetes
Just a few years ago, GLP-1 receptor agonists burst into the public consciousness as weight-loss wonder drugs.
But the advent was no surprise to diabetes researchers. Seung Kim, MD, PhD, has watched the discoveries underlying the drugs accrue over many years. In 2021, Wegovy became the first GLP-1 drug the Food and Drug Administration approved to treat obesity, but initial applications for GLP-1 drugs were for diabetes, not weight loss. Kim, the director of the Stanford Diabetes Research Center, believes these drugs have the potential to greatly lessen the impact of the condition.
Diabetes affects 1 in 9 people globally, according to the International Diabetes Federation. It leads to a high level of blood sugar, or glucose, which over time can cause damage throughout the body, including to the blood vessels, eyes, heart, kidneys and nervous system.
At the root of the disease is a problem with insulin, a hormone that helps regulate the blood sugar by letting glucose into the body’s cells to be used for energy. If you have diabetes, your body either produces too little or no insulin (Type 1 diabetes) or it fails to make good use of the insulin it does produce (Type 2 and gestational diabetes).
“The finding that drugs targeting the GLP-1 receptor can be used for weight reduction and glucose control has the potential to be transformative for many with Type 2 diabetes … or with conditions like obesity that increase diabetes risk.”
Seung Kim, professor of developmental biology and of medicine
GLP-1 receptor agonists work by mimicking GLP-1, a hormone produced in the small intestine and likely the pancreas. GLP-1 and its copycat molecules stimulate the release of insulin; suppress a hormone, glucagon, that raises blood sugar; and slow the movement of food through the gastrointestinal tract, thereby reducing hunger. They flip the switch to launch these events by latching onto the GLP-1 receptor, a protein embedded in the surface of cells throughout the body.
“The targeting of satiety is a major step in diabetes care and prevention. The finding that drugs targeting the GLP-1 receptor can be used for weight reduction and glucose control has the potential to be transformative for many with Type 2 diabetes — the most common form of this disease — or with conditions like obesity that increase diabetes risk,” said Kim, the KM Mulberry Professor and a professor of developmental biology and of medicine.
“Only time will tell how durable and useful these agents will be. But a principle for complex diseases like diabetes is that multiple agents working through distinct mechanisms can be very powerful, especially if safely combined.”
Researchers and clinicians are now exploring combining the GLP-1 drugs with other medications for diabetes, such as metformin and SGLT2 inhibitors, as well as exercise and behavior modification.
“The beta cell is exquisitely tuned to controlling a very tight range of glucose and other important metabolites.”
Seung Kim
And some researchers, including Kim, are hunting for ways to cure the disease by providing functional versions of the faulty insulin-producing cells. These cells, known as beta cells, develop in structures within the pancreas known as the islets of Langerhans. Kim’s laboratory is investigating the mechanisms that control islet formation, growth and survival, and asking how this knowledge can be harnessed to generate replacements, including beta cells from human stem cell lines.
People with Type 1 diabetes and some with Type 2 diabetes need insulin from an external source to survive, so they must monitor their blood sugar levels and administer insulin, usually through an injection, often multiple times per day.
Technological solutions such as insulin pumps that automatically monitor glucose and continually supply the insulin make managing insulin easier, but they have drawbacks — among them high cost, potential for malfunction and infection at the infusion site.
“We think the best devices for delivering insulin, ultimately, are the cells that actually know how to do that. The beta cell is exquisitely tuned to controlling a very tight range of glucose and other important metabolites,” Kim said. “Devices can approximate this, but when it comes to controlling glucose, beta cells along with other islet cells are the professionals.”
Stroke recovery
Recovering from a stroke can take weeks, months or even years — with some people experiencing lifelong disabilities.
Challenges survivors face can include paralysis, spastic movements, balance problems, difficulties with speaking and swallowing, memory troubles, and emotional changes including depression. The leaders of the Stanford Stroke Recovery Program are out to eliminate these challenges. Their program runs clinical studies to understand stroke recovery and develop new treatments, and they’re optimistic that new treatments and technologies will transform stroke rehabilitation.
“There are some emerging therapies that I am very excited about but that are still in early phases of development,” said Maarten Lansberg, MD, PhD, co-leader of the stroke recovery program and a professor of neurology and neurological sciences.
Among these is the injection of stem cells into the brain post-stroke, a treatment being explored by Stanford Medicine’s Gary Steinberg, MD, PhD, the Bernard and Ronni Lacroute-William Randolph Hearst Professor in Neurosurgery and Neurosciences. A small, early stage study of 18 patients found that patients experienced improvements in walking, hand and arm use, and coordination. Some patients in wheelchairs even regained the ability to walk. A larger study is underway.
“New technologies such as artificial intelligence and virtual reality will make it possible to deliver personalized intensive rehabilitation therapy to patients in the comfort of their homes.”
Maarten Lansberg, professor of neurology and neurological sciences and co-leader of the stroke recovery program
Another exciting development lies in the integration of technology into rehabilitation. “New technologies such as artificial intelligence and virtual reality will make it possible to deliver personalized intensive rehabilitation therapy to patients in the comfort of their homes,” Lansberg said. This shift could revolutionize how patients engage with their recovery, making therapy more accessible and tailored to individual needs.
“The first thing that comes to people’s minds when thinking about stroke is lack of movement, speech and sensation, and these are indeed large problems that stroke recovery research is addressing,” said Marion Buckwalter, MD, PhD, who co-leads the program with Lansberg. However, less well-researched issues such as memory problems, fatigue and depression affect many stroke survivors.
“New therapies aimed at treating these problems will dramatically increase quality of life for people who’ve had a stroke,” said Buckwalter, a professor of neurology and neurological sciences and of neurosurgery.
Bringing the various advances together can be expected to have an even greater impact, the experts said. “We know that children and young adults recover much better after stroke than older patients,” Lansberg said. “Hopefully, we will be able to create the conditions — by combining new medical therapies with more intensive rehabilitation — in which a patient recovers as well as or better than someone who is 10 years younger.”
“I think in the next five years we will see the first early stage trials for promoting recovery of movement after stroke and the first trials to prevent dementia after stroke,” Buckwalter predicted. “I hope in 10 years we will be testing our first therapies for post-stroke fatigue and will have established treatments to promote rewiring and plasticity for movement and to promote healthy brain blood vessels that sustain normal thinking and memory.”