Transfusion medicine

After 10 years in remission, Derek Ruff’s cancer returned; this time as stage IV colon cancer. Despite aggressive rounds of chemotherapy, palliative radiotherapy and immunotherapy; his disease progressed. In February 2019; as part of a phase I clinical trial at Moores Cancer Center at UC San Diego Health; Ruff became the first patient in the world to be treat for cancer with a human-induce pluripotent stem cell (iPSC)-derive cell therapy call FT500.

Cancer immunotherapy

“This is a landmark accomplishment for the field of stem cell-based medicine and cancer immunotherapy. This clinical trial represents the first use of cells produced from human induced pluripotent stem cells to better treat and fight cancer;” said Dan Kaufman, MD, PhD; professor of medicine in the Division of Regenerative Medicine and director of cell therapy at University of California San Diego School of Medicine.

“This is a culmination of 15 years of work. My lab was the first to produce natural killer cells from human pluripotent stem cells. Together with Fate Therapeutics; we’ve been able to show in preclinical research that this new strategy to produce pluripotent stem cell-derived natural killer cells can effectively kill cancer cells in cell culture and in mouse models.”

Fate Therapeutics

The off-the-shelf, iPSC-derived natural killer (NK) cell cancer immunotherapy received U.S. Food and Drug Administration approval to move into clinical trials in November 2018. Kaufman has been collaborating with Fate Therapeutics over the past four years to bring iPSC-derived NK cells to patients with cancer.

NK cells are specialize immune cells that are very potent at killing cancer cells. While NK cells can be isolate from the blood of donors or patients; therefore this trial uses NK cells entirely produced in the lab from human iPSCs. Since human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body; therefore these cells can be use as starting material to mass produce NK cells ; at significant scale in a cost-effective manner.

Inhibitor based immunotherapies

Work in Kaufman’s lab, in collaboration with Fate and investigators at the Masonic Cancer Center; University of Minnesota; defined conditions to efficiently produce NK cells at a scale suitable for clinical use. Since FT500 does not need to be matched to a patient like other T-cell therapies; Because researchers say FT500 can be administered in the outpatient setting as an “off-the-shelf” cell product; reducing the time and resources needed to treat patients.
The FT500 clinical trial is a two-arm study with up to 64 patients for the treatment of advanced solid tumors. Because It is design to assess the safety and effectiveness of multiple doses of FT500 administer over multiple cycles as a mono therapy and as a combination treatment with one of three checkpoint inhibitor-based immunotherapies nivolumab; therefore pembrolizumab or atezolizumab in patients where previous treatments have not work or who have confirm disease progression on checkpoint inhibitor therapy.