Bold Step

First-in-Human Trials for Stem Cell Treatments

Stem cell therapies are a provocative new way of treating incurable diseases. Instead of managing symptoms, cellular medicine has the potential to replace or regenerate damaged tissues and organs.

Left: Implanted immature pancreatic cells derived from human embryonic stem cells could develop into full-fledged insulin producers, offering a new treatment for diabetes. Right: UC San Diego researchers have used neural precursor cells (red and green) from human embryonic stem cells to promote regeneration and improvement of function in rats impaired by an acute spinal cord injury. Martin Marsala/UC San Diego

Clinical trials are mandatory before any new drug or treatment can be approved for patient use. In 2014, three groundbreaking stem cell efforts at UC San Diego made the significant leap from laboratory to first-in-human clinical trials. A twenty-six-year-old woman, paralyzed after a motor vehicle accident, successfully underwent a procedure to test whether injecting neural stem cells at the site of a spinal cord injury is safe and could be an effective treatment. The hope is that the transplanted neural stem cells will develop into new neurons that bridge the gap created by the injury, replace severed or lost nerve connections, and restore at least some motor and sensory function.

Another stem cell effort was the launch of a Phase I trial to assess the safety of a monoclonal antibody treatment that targets cancer stem cells in patients with chronic lymphocytic leukemia, the most common form of blood cancer. The third trial was an unprecedented stem cell-based therapy designed to treat Type 1 diabetes.

Since its creation in 2004, CIRM has approved seventy-four awards to UC San Diego stem cell scientists and programs, totaling almost $156 million.

To speed the development of treatments, the Sanford Stem Cell Clinical Center at UC San Diego Health System was launched in 2013. The center integrates operations at four locations: the UC San Diego Jacobs Medical Center and a nearby proposed clinical space, both scheduled to open in 2016; the UC San Diego Center for Advanced Laboratory Medicine; and the Sanford Consortium for Regenerative Medicine. This “collaboratory” of scientists hail from UC San Diego, the Sanford-Burnham Medical Research Institute, the Salk Institute for Biological Studies, The Scripps Research Institute, the La Jolla Institute for Allergy & Immunology, and other contributing institutions on the Torrey Pines mesa, such as the J. Craig Venter Institute.

In 2014, the Sanford Center was named an “alpha clinic” by the California Institute for Regenerative Medicine (CIRM), the state’s stem cell agency. This designation included an $8 million grant to create a long-term, networked infrastructure for extensive clinical trials of stem cell-based drugs and therapies in humans, including some developed by independent California-based investigators and companies. The state’s three alpha clinics will emphasize public education—in part to combat the marketing of unproven, unregulated, and potentially dangerous therapies. The clinics will also help establish sustainable business models for future approved treatments.