Incurable diseases are nothing if not a drag. They’re not managable diseases like diabetes and heart disease: They often show up due to genetics or environmental exposures. And they cost the U.S. health system billions of dollars a year, plus the untold human expense of medication, management, and misery.
This is a big reason why the California Institute for Regenerative Medicine (CIRM) has incurable disease in its crosshairs. Just this week, they announced a five-year, $8 million gift to City of Hope, establishing a clinic that will work specifically to find cures for those diseases that are currently incurable—among them AIDS, lymphoma, and other cancers—using advanced stem cell therapies.
The CIRM has actually had stem cell-based approaches in its wheelhouse for quite sometime. Just last year, it approved a $70 million plan to develop the Alpha Stem Cell Clinics Program, of which these clinics will be a part. The idea is to create one-stop centers dealing in clinical trials for stem cell-based therapies for disease.
Two trials are already up and running at the site, both using stem cells to target AIDS. One combines stem cells and gene therapy using small RNA molecules that block the genes HIV needs to infect immune cells. The goal is to get the immune system to produce its own HIV-resistant T cells. By exposing the patient’s system to altered stem cells, it’s hoped the body will pick up the material it needs to manufacture its own defenses. The other approach uses an enzyme called a zinc-finger nuclease, or ZFN, to alter the patient’s stem cell genes so they no longer produce a key protein the virus requires to infect cells.
"We are committed to finding cures and treatments to diseases that are, for now, incurable," said John Zaia, the Aaron D. and Edith Miller Chair in Gene Therapy, chair of the Department of Virology, and principal investigator for the stem cell clinic. "This grant recognizes City of Hope's commitment to and leadership in this endeavor, [and] enables us to pursue the crucially important work of bringing the promising potential of stem cell treatments to fruition."