Systemic lupus erythematosus, also known as SLE or lupus, is a chronic inflammatory condition that can affect almost every organ in the body. Commonly, patients with lupus suffer from fatigue, fever, muscle pains, and fluctuations in weight. Perhaps the most noticeable feature of lupus is a butterfly rash that affects the nose and cheeks. In its most severe form, lupus can be life-threatening. As many as half of all patients with systemic lupus erythematosus experience some sort of kidney involvement. One feared manifestation of lupus is an inflammation of the kidney called lupus nephritis. Patients can also experience blood clots in the arteries and veins, and serious inflammation of the gastrointestinal tract, lungs, and/or heart.
There is no cure for lupus. The goals of treatment are to reduce the severity of lupus symptoms, prevent damage to organs, and improve patient quality of life. Many treatments for lupus are merely preventive. For example, patients with lupus know that staying out of the sun or using sunscreen can help prevent skin rashes that often occur during the disease. Unfortunately, the treatments that are commonly used for SLE cause serious side effects. For example, steroids can help control flares, but they are not suitable for long-term use. Immunosuppressants are sometimes helpful, but they can increase a patient’s risk of infection. Newer biologics such as belimumab and rituximab are more selective treatments, but are not helpful for most patients and remain relatively expensive.
Not surprisingly, many patients with systemic lupus erythematosus are unsatisfied with their current treatments. This is frustrating for patients, families, and doctors. Thus, researchers have started testing stem cell therapy to determine if this novel treatment could be effective for treating lupus.
In one such clinical study, scientists collected mesenchymal stem cells from umbilical cord tissue (the substance that usually gets thrown away as medical waste after a baby is born). They then infused the purified stem cells into patients with SLE who had a difficult-to-treat disease (i.e., patients had failed to find relief of SLE symptoms from standard treatments or had life-threatening complications from the condition). The scientists then followed the patients for about eight months on average, though some patients were followed for as many as 28 months after stem cell treatment.
Patients treated with mesenchymal stem cells showed dramatic improvements in a measure of lupus disease activity called the SLEDAI. In fact, patients enjoyed significant improvements in as little as one month after mesenchymal stem cell treatment. This effect lasted for up to two years in some patients. Moreover, patients treated with umbilical cord-derived stem cells had improvements in kidney function and lost less protein in their urine. Impressively, patients treated with stem cells showed improvements in various markers associated with active lupus including serum antinuclear antibody (ANA), anti-double-stranded DNA (anti‐dsDNA) antibody, serum complement C3 and C4, and albumin levels. Patients with lupus know that these blood markers are used to track the severity of the condition.
While one person had severe nausea during stem cell infusion, this passed quickly, and no other treatment-related side effects occurred. In fact, stem cell treatment was well tolerated by all patients in the study. These remarkable results will, of course, need to be repeated and verified in larger clinical trials. Nevertheless, the dramatic improvements seen with umbilical cord mesenchymal stem cells in patients with difficult-to-treat lupus are impressive. While more work needs to be done, patients and doctors are now looking toward stem cell treatments in the hope that this terrible disease can finally be treated effectively.
Reference: Lingyun, S. et al. (2010). Umbilical cord mesenchymal stem cell transplantation in severe and refractory systemic lupus erythematosus. Wiley Online Library. 2010 https://doi.org/10.1002/art.27548.