Today CIRM’s governing Board invested almost $10 million in stem cell research for sickle cell disease and patients with solid cancer tumors.
Clinical trial for sickle cell disease
City of Hope was awarded $5.74 million to launch a Phase 1 clinical trial testing a stem cell-based therapy for adult patients with severe sickle cell disease (SCD). SCD refers to a group of inherited blood disorders that cause red blood cells to take on an abnormal, sickle shape. Sickle cells clog blood vessels and block the normal flow of oxygen-carrying blood to the body’s tissues. Patients with SCD have a reduced life expectancy and experience various complications including anemia, stroke, organ damage, and bouts of excruciating pain.
CIRM’s President and CEO, Maria T. Millan, explained in the Agency’s news release:
“The current standard of treatment for SCD is a bone marrow stem cell transplant from a genetically matched donor, usually a close family member. This treatment is typically reserved for children and requires high doses of toxic chemotherapy drugs to remove the patient’s diseased bone marrow. Unfortunately, most patients do not have a genetically matched donor and are unable to benefit from this treatment. The City of Hope trial aims to address this unmet medical need for adults with severe SCD.”
The proposed treatment involves transplanting blood-forming stem cells from a donor into a patient who has received a milder, less toxic chemotherapy treatment that removes some but not all of the patient’s diseased bone marrow stem cells. The donor stem cells are depleted of immune cells called T cells prior to transplantation. This approach allows the donor stem cells to engraft and create a healthy supply of non-diseased blood cells without causing an immune reaction in the patient.
Joseph Rosenthal, the Director of Pediatric Hematology and Oncology at the City of Hope and lead investigator on the trial, mentioned that CIRM funding made it possible for them to test this potential treatment in a clinical trial.
“The City of Hope transplant program in SCD is one of the largest in the nation. CIRM funding will allow us to conduct a Phase 1 trial in six adult patients with severe SCD. We believe this treatment will improve the quality of life of patients while also reducing the risk of graft-versus-host disease and transplant-related complications. Our hope is that this treatment can be eventually offered to SCD patients as a curative therapy.”
This is the second clinical trial for SCD that CIRM has funded – the first being a Phase 1 trial at UCLA treating SCD patients with their own genetically modified blood stem cells. CIRM is also currently funding research at Children’s Hospital of Oakland Research Institute and Stanford University involving the use of CRISPR gene editing technologies to develop novel stem cell therapies for SCD patients.
Advancing a cancer immunotherapy for solid tumors
The CIRM Board also awarded San Diego-based company Fate Therapeutics $4 million to further develop a stem cell-based therapy for patients with advanced solid tumors.
Fate is developing FT516, a Natural Killer (NK) cell cancer immunotherapy derived from an engineered human induced pluripotent stem cell (iPSC) line. NK cells are part of the immune system’s first-line response to infection and diseases like cancer. Fate is engineering human iPSCs to express a novel form of a protein receptor, called CD16, and is using these cells as a renewable source for generating NK cells. The company will use the engineered NK cells in combination with an anti-breast cancer drug called trastuzumab to augment the drug’s ability to kill breast cancer cells.
“CIRM sees the potential in Fate’s unique approach to developing cancer immunotherapies. Different cancers require different approaches that often involve a combination of treatments. Fate’s NK cell product is distinct from the T cell immunotherapies that CIRM also funds and will allow us to broaden the arsenal of immunotherapies for incurable and devastating cancers,” said Maria Millan.
Fate’s NK cell product will be manufactured in large batches made from a master human iPSC line. This strategy will allow them to treat a large patient population with a well characterized, uniform cell product.
The award Fate received is part of CIRM’s late stage preclinical funding program, which aims to fund the final stages of research required to file an Investigational New Drug (IND) application with the US Food and Drug Administration. If the company is granted an IND, it will be able to launch a clinical trial.
Scott Wolchko, President and CEO of Fate Therapeutics, shared his company’s goals for launching a clinical trial next year with the help of CIRM funding:
“Fate has more than a decade of experience in developing human iPSC-derived cell products. CIRM funding will enable us to complete our IND-enabling studies and the manufacturing of our clinical product. Our goal is to launch a clinical trial in 2019 using the City of Hope CIRM Alpha Stem Cell Clinic.”