A double-edged stem cell transplantation therapy is being evaluated at the non-clinical stage as a cure for lupus. Will this make it to the clinic?
A classic presentation of lupus is the butterfly rash of the face. However, this autoimmune disease is more than skin deep. We have discussed how several organs can be affected in a previous blog. In fact, lupus is incurable and available drugs only alleviate symptoms. Furthermore, long-term use of prescribed drugs may cause liver damage and peptic ulcers.
Currently there are 16 clinical trials listed on clinicaltrials.gov designed to evaluate stem cell transplantation as a cure for lupus. Cells under consideration include hematopoietic stem cells from bone marrow and cord blood. The basic premise is that a patient’s defective immune cells that cause lupus can be replaced by grafting a new immune system through stem cell transplantation.
Another approach with stem cell transplantation is to use mesenchymal stem cells. The idea here is that mesenchymal stem cell therapy can be used to immunomodulate or calm the patient’s existing immune system. In this manner, the patient’s T cells and B cells will no longer mount a response against the patient’s own tissue.
Promising therapeutic regimens begin at the bench. Scientists work to show the proposed mechanism in mammalian cell culture. Next, animal models are used to demonstrate the proof of principle. And finally, data is presented to the Food and Drug administration to obtain approval for clinical trials.
A recent study still at the non-clinical animal model stage demonstrated that a therapy combining hematopoietic stem cells and mesenchymal stem cells could be a better alternative to using these cell therapies as single-agents. This double-edged stem cell transplantation approach proved better in a study that evaluated it in a mouse model of lupus.
Whereas allogeneic or mouse stem cells were used in mouse models in this study, several mouse models are also developed to allow for xenogeneic or human stem cell transplantation. Such studies go a step closer to clinical trials in evaluating the therapeutic agents. At HemaCare, we provide human hematopoietic stem cells from bone marrow and cord blood, as well as mesenchymal stem cells. We also provide disease state cells, including those from lupus patients, to enable cutting-edge research that generates promising preclinical data.
1. Liu RH, et al. Supplementing mesenchymal stem cells improves the therapeutic effect of hematopoietic stem cell transplantation in the treatment of murine systemic lupus erythematosus. Transplant Proc. 2014 Jun;46(5):1621-7. PubMed PMID: 24935337.