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UC San Diego Researchers Receive Funding to Pursue Novel Stem Cell-based Treatments

Proposed therapies would address a rare heart condition and a chronic lung disease

The governing board of the California Institute for Regenerative Medicine (CIRM) unanimously approved this week two grants worth more than $2.2 million to University of California San Diego School of Medicine researchers investigating stem cell-based therapies for a rare genetic disorder that affects the heart and a chronic, progressive affliction of the lungs.

The first grant for almost $1.4 million was awarded to Eric Adler, MD, cardiologist, professor of medicine, director of cardiac transplant and mechanical circulatory support at UC San Diego Health and member of the UC San Diego CIRM Alpha Stem Cell Clinic, to pursue the use of genetically modified hematopoietic stem cells to treat Danon disease, a progressive condition characterized by weakening of the heart muscle, weakening of muscles used for movement and cognitive disability.

In many cases, Danon disease is inherited from a parent, and is often misdiagnosed and treated as heart failure. The condition involved dysfunction in lyposomes — organelles found in nearly all animal cells that contain enzymes used to break down many kinds of biomolecules. There is no cure and, in the case of the heart, the condition is fatal unless a heart transplant is performed.

Adler is one of the few researchers in the United States studying the condition. His CIRM grant will support further research into using genetically modified hematopoietic stem cells, which give rise to other blood cells, to treat the condition.

The second CIRM grant for $865,282 (with a recommendation of an additional $527,918 to be awarded at the October board meeting) went to James Hagood, MD, pediatric pulmonologist and chief of pediatric respiratory medicine at Rady Children's Hospital-San Diego and chief of the Division of Respiratory Medicine in the Department of Pediatrics at UC San Diego School of Medicine, to investigate the use of mesenchymal stem cell extracellular vesicles (MSC-EV) as a therapy for pulmonary fibrosis. The latter is a lung disease that occurs when lung tissue becomes damaged and scarred, making it more difficult for the organ to work properly. Current therapies can ease symptoms, such as shortness of breath, dry cough and fatigue, but the damage cannot be repaired.

Mesenchymal stem cells can differentiate into a variety of cell types, from bone and cartilage to muscle and fat. Extracellular vesicles are cell-derived membranous structures that carry information between cells. They play a key role in the development and progression of many pulmonary diseases, including fibrosis. MSC-EV are already entering clinical trials for a number of diseases. In this case, the researchers want to better understand how MSC-EV function and how they can be modified to maximize their anti-fibrotic potential.

CIRM was created in 2004 by California voters with $3 billion in funding support to accelerate stem cell research and treatments. Since 2004, UC San Diego researchers have received at least 99 CIRM awards, totaling more than $215 million. At the center of this effort is the Sanford Stem Cell Clinical Center at UC San Diego Health, host of a “stem cell alpha clinic” in the CIRM-designated Stem Cell Alpha Clinics Network created in California to support stem cell clinical trials. The Sanford Stem Cell Clinical Center was established in 2013 with a $100 million gift from philanthropist T. Denny Sanford.


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