This week: Successes for both stem cell and CAR-T therapies, plus funding to evaluate a gene-edited cell therapy candidate.
The news highlights:
First-ever clinical trial investigating stem cell therapy for severe traumatic injury
Single dose of HMGB1 promotes tissue repair through endogenous stem cells
Peripheral arterial disease: stem cell therapy shows long-term success
Sangamo wins grant to evaluate a gene-edited cell therapy candidate for Î²-thalassemia
CAR-T immunotherapy success against colorectal cancer in pre-clinical trials
The University of Texas Health Science Center at Houston (TX, USA) has received funding for a clinical trial to investigate a stem cell therapy for early treatment and prevention of complications post-severe traumatic injury. This will be the first-ever clinical trial of this nature.
The Phase II trial is funded by US$ 2 million provided by the Medical Technology Consortium (SC, USA) and US$ 1.5 million from the Memorial Hermann Foundation (TX, USA). In addition, Athersys, Inc. (OH, USA) is providing the investigational clinical product, MultiStem®, and will provide regulatory and operational support.
Charles S Cox, Jr, principal investigator of the study, commented: “Our prior research demonstrates that administration of MultiStem following acute neurological injury can help improve recovery and reduce the occurrence or severity of certain complications, so we are excited about the clinical potential in this area.”
Researchers from the Kennedy Institute of Rheumatology at the University of Oxford (UK) have shown how administration of HMGB1 protein can promote tissue repair by triggering endogenous stem cells to transition from G0 to GAlert, priming the cells to respond to activating factors released upon injury. It is anticipated that exogenous administration of HMGB1 could benefit patients in clinical scenarios including trauma, chemotherapy and surgery.
Editor’s note: A previous version of this section cited a press release as opposed to the original publication and omitted that there was no control group.
A 5-year study published in STEM CELLS Translational Medicine has highlighted that stem cell therapy to treat angitis-induced critical limb ischemia, a serious form of peripheral arterial diseases, is both safe and effective. Although this study had no control group, it is the first study evaluating the long-term outcomes of treating this disease with purified CD34+ cell transplantation.
“Notably, in 17 patients (65.38%) not only were their limbs saved, but they also fully recovered their labor competence and returned to their original jobs by week 260. [Purified CD34+ cell transplantation] demonstrated long-term efficacy and durability as a treatment of AICLI, not only in achieving limb salvage but also in recovering the labor competence and improving the patient’s quality of life,” remarked Weiguo Fu (Department of Vascular Surgery at Zhongshan Hospital, Shanghai, China), corresponding author of the study.
Further research is required to dismiss the possibly effects of selection bias, investigator bias and clinical differences, and confirm the efficacy of CD34+ cell transplantation
Sangamo Therapeutics (CA, USA) has been awarded US$ 8 million by the California Institute for Regenerative Medicine (CIRM; CA, USA) to carry out evaluations into a gene-edited cell therapy candidate, ST-400, for the treatment of transfusion-dependent Î²-thalassemia. Sagmano expects to begin enrolling patients in the study in the first half of 2018.
“CIRM plays a critical role in funding the rigorous evaluation of new stem cell therapies, and we are very pleased to receive CIRM’s support for the study of ST-400 for the treatment of transfusion-dependent Î²-thalassemia,” commented Edward Conner, Chief Medical Officer at Sangamo. “We believe the precision, efficiency and specificity of zinc finger nuclease gene editing has the potential to differentiate ST-400 from other genomic therapies in development for this disease.”
Researchers at the Sidney Kimmel Cancer Center at Jefferson Health (PA, USA) have demonstrated the success of a CAR-T immunotherapy made specifically to treat GUCY2C-expressing cancers, such as colorectal cancer. In pre-clinical trials in mouse models, the therapy was found to be effective in both killing tumor cells and preventing metastases.
“Colorectal cancer rates are exceptionally high in our region, and advanced-stage disease is difficult to treat. The concept of moving CAR-T cell therapy to colorectal cancer is a major breakthrough, and could address a major unmet clinical need. We are optimistic about the pre-clinical results,” remarked Karen Knudsen, Director of the Sidney Kimmel Cancer Center.
For more weekly cell therapy news, read previous editions of the cell therapy weekly.