Research Focus

Dr. Brigitte Gomperts is inspired by the unprecedented tools and technologies available through stem cell science and regenerative medicine. “Just a few years ago,” Gomperts says, “our ability to study regenerative medicine and think about regenerative therapies was quite limited. Now with current and developing stem cell technology the possibilities for new treatments for lung diseases seem endless.” Although trained as a pediatric cancer specialist, it was the rapid and ongoing development of stem cell technology that steered Dr. Gomperts down the path of lung disease research.

As member of the UCLA Broad Stem Cell Research Center and an associate professor of medicine, Dr. Gomperts focuses much of her research efforts on the lung stem cells that are responsible for repair and regeneration of lung tissue after injury. She also studies how repair goes awry to cause lung diseases, with the goal of finding new therapies and preventive measures for idiopathic pulmonary fibrosis (IPF) and other related diseases. Dr. Gomperts sees innovation and experimentation in the laboratory as the keys to making significant new breakthroughs for patients.

Creating Disease Models With Stem Cells

In 2011 the Stem Cell Center received a generous gift from David and Diane Steffy to establish the Steffy Family Stem Cell Research Fund for IPF. “My family has been directly impacted by IPF and it is my great hope that patients suffering from the disease may soon have access to new and more effective treatments. So, I chose to personally support Dr. Gomperts and the UCLA Broad Stem Cell Research Center because they are pursuing an exceptionally creative and novel research approach. I am very impressed with Dr. Gomperts’ passion and scientific tenacity and proud that my philanthropy has served as a catalyst for her innovative research.”

IPF is a disease of unknown cause in which thick scar tissue that restricts breathing develops in the lungs and worsens over time. In current mouse models of IPF, the lung scarring eventually goes away, which is inconsistent with the progressive scarring seen in humans with IPF. Thus, without good quality disease models very little progress has been made in treatments for patients.

Dr. Gomperts and her colleagues have worked toward developing a high-quality stem cell based disease-in-a-dish model of IPF that will allow them to study the process of IPF in the laboratory and possibly find the cause of the disease. Stem cells are an important tool because they can become any cell in the body, including lung cells. The knowledge created by Dr. Gomperts’ research could speed the discovery of significant treatments. “I became a doctor to take care of people,” Gomperts says, “then after becoming a doctor I discovered I was not happy accepting the treatments we had, which often were few. When I saw I could also contribute as a scientist by pushing the boundaries of our current medical knowledge and finding new therapies, new diagnostic markers and new ways of moving patient care forward.”

Driven by this desire for innovation, Dr. Gomperts looked at past stem cell research to model diseases of similar complexity as IPF. Using lung and skin samples collected from IPF patients, she and her colleagues derived induced pluripotent stem (iPS) cells and drove them to become lung cells called fibroblasts. This enabled Dr. Gomperts to recapitulate the development of the disease in the laboratory. She and her colleagues now have well-functioning models in a dish, in which the tissue scarring progresses as it does in the human lung, and they can begin to study the causes and possible treatments of IPF.

Lung Regeneration Therapies

For this startlingly creative project, Dr. Gomperts is using iPS cells and working with bioengineers to create a small replacement organ, called a lung organoid, that could be grown in the laboratory, transplanted into a patient, and regenerate healthy tissue that will take over the function of a diseased or damaged lung. The main limitation in current lung transplantation is a patient’s body rejecting the new organ. Thus, a mini-organoid derived from the patient’s own cells would be perfect for transplantation, eliminating the rejection problem and making organ donors unnecessary.

To date, Dr. Gomperts and her colleagues have made iPS cells from fibroblasts taken from patients, which are then differentiated into three different types of lung cells: epithelial cells, which make up the tissue that covers the surface of the lung; fibroblasts, which are the structural support cells; and endothelial cells, which make up the delicate tissue that line the tiny lung blood vessels called capillaries.

“The bioengineers create a template around which we can grow the fibroblasts into alveoli, the tiny breathing sacks of the lung,” Dr. Gomperts explains, “we add the endothelial and epithelial cells to see if they can organize themselves into alveoli that are functional for patient transplantation. The long-term goal is to differentiate all the cells of the lung to grow transplantable lung organoids. Right now we’re at the beginning stages, but with further research that is where we are headed.”

Dr. Gomperts stem cell research has implications for treatments beyond IPF. As a pediatrician, she sees children as possibly the first beneficiaries of this technology because their lungs are smaller and need less surface area. An organoid might easily replace an underdeveloped lung in a premature baby, a child suffering from congenital diaphragmatic hernia (a condition in which the lungs do not develop enough), or a child with bronchopulminary dysplasia (a disease in which the lungs do not have enough alveoli). In this way Dr. Gomperts’ creatively advanced treatment strategy potentially has wide-ranging effectiveness.

A View Into the Future

Although she began her career as a cancer doctor, Dr. Gomperts does not see herself limited only to cancer research. “I think very broadly. I don’t think of myself as boxed in by any subspecialty,” she says, “although much of my current research is not hematology-oncology, I feel unconstrained by any field and try to think about what is helpful to patients overall, from a clinician’s point of view. My lab needs to be helpful for patients, so I think about how what we are doing fits into a clinical scenario. I try to have interesting new ideas and take advantage of the new tools and technologies that are becoming available, to see how they can be used to really push the envelope in new directions to help patients.”

Brigitte Gomperts' research exemplifies the world-class scientific innovation that the Broad Stem Cell Research Center brings to bear on today’s biggest medical problems through the remarkable advancements of stem cell science and regenerative medicine.