Umbilical cord-derived stem cells failed to improve lupus patients’ impaired kidney function, according to a small clinical trial in China whose results contrasted with previous studies.
Forty to 77 percent of systemic lupus erythematosus patients have reduced kidney function, according to estimates. The condition, known as lupus nephritis, is linked to increased lupus severity and mortality.
Traditional nephritis treatments include immunosuppressive drugs such as steroids, intravenous cyclophosphamide, and mycophenolate mofetil.
As increasing number of studies are examining stem cells as a therapy, however, with a particular focus on human umbilical cord-derived mesenchymal stem cells, or hUC-MSCs. But few randomized clinical trials have examined hUC-MSC as a nephritis treatment.
Researchers decided to evaluate hUC-MSC ’s effectiveness in a randomized, double-blind trial (NCT01539902) at a medical center in Kunming, China.
The team recruited 18 lupus patients, 16 years or older, with lupus nephritis. Each patient’s lupus fell into the World Health Organization’s Class III or IV disease categories.
Patients were randomly assigned to either hUC-MSC or a placebo. The treatment group was 11 patients and the placebo group six.
All of the patients also received standard immunosuppressive treatment: intravenous methylprednisolone and cyclophosphamide, followed by maintenance with oral prednisolone and mycophenolate mofetil.
Researchers checked the patients’ nephritis every two weeks for the first two months of the 12-month trial, then every month for two to six months, and finally every two months until the end of the study.
The main goals of the trial were to stabilize patients’ nephritis by achieving either full or partial remission of the condition, improving their kidney function, or reducing red blood cell and protein levels in their urine. High levels of those components are markers of kidney impairment.
All but one of the 18 patients completed the trial. The one who didn’t died three months after enrolling.
Patients who received hUC-MSC experienced no more benefits than the placebo group, the researchers reported. Nine of the 12 treated patients, or 75 percent, achieved remission, versus five of the six placebo patients, or 83 percent.
In addition, measures of nephritis improvement were similar in the two groups. These included kidney function, Systemic Lupus Erythematosus Disease Activity Index scores, British Isles Lupus Assessment Group scores, and levels of serum albumin.
Overall, the trial showed that lupus patients treated with hUC-MSC obtained no benefits beyond those that standard immunosuppression delivered. The bottom line was that the therapy failed to replicate benefits of previous hUC-MSC studies of lupus nephritis.
“The trial was abandoned after 18 patients were enrolled when it had become obvious that the trial would be unlikely to demonstrate a positive treatment effect for hUC-MSC,” the researchers said.