There are roughly 100 autoimmune diseases, plus about 40 more that are immune-based. Total annual direct healthcare costs for these debilitating conditions are nearly double that of cancer treatments, according to the American Autoimmune-Related Diseases Association. For this reason, the stakes in autoimmune disease research are high.
Read on to discover some of the newest innovations affecting common autoimmune diseases.
Much of the focus in lupus research is focused on interferons, which are signaling proteins made and released by cells in response to the presence of pathogens. Interferons have been found to be overproduced in many lupus patients.
"This observation has led to interferon itself being identified as a therapeutic target," says Mary Crow, MD, physician-scientist lupologist, co-chair of the Lupus Research Alliance Scientific Advisory Board, and physician-in-chief of Hospital for Special Surgery. Anifrolumab, in phase 3 clinical trials, is one medication showing promise in this area, she says, noting it's likely that at least one new medication could be approved for use in the next year.
More than 30 clinical trials are ongoing in various phases of lupus drug development, says Rosalind Ramsey-Goldman, MD, DrPH, Solovy Arthritis Research Society Professor of Medicine and medical director of the clinical research unit at Northwestern University in Chicago. In the last six months, several phase 2 trials showed positive results and are likely to move on to the next phase, she adds.
Ustekinumab, which blocks the IL12/23 pathway linked to the pathogenesis of lupus, and previously approved for psoriasis, psoriatic arthritis, and Crohn’s disease, has been evaluated in lupus in a phase 2 trial, Ramsey-Goldman says. One study showed efficacy in the treatment of active lupus and plans are in place for more studies.
Another study is evaluating sirolimus, which is already approved by the FDA for immunosuppression and to prevent rejection after organ transplantation. Ramsey-Goldman says this medication may be useful in lupus to restrict T cell activity.
Two phase 2 clinical trials in 2016 that are now in phase 3 could result in FDA approval for anifrolumab and voclosporin, a calcineurin inhibitor and immunosuppressant that blocks T cells.
Researchers are also working to identify biomarkers that can help clinicians improve diagnosis and better identify those at risk for developing the disease, Goldman says. Biomarkers may also help clinicians predict flares, as well as how patients will respond to different treatments.
The most effective intervention for celiac disease is to follow a strict gluten-free diet, but this can be difficult, says Peter H.R. Greene, MD, director of the Celiac Disease Center at Columbia University in New York, the Phyllis and Ivan Seidenberg Professor of Medicine at Columbia University Medical Center, and attending physician at New York Presbyterian Hospital.
Though a gluten-free diet is not easy, it is highly effective and safe. This means that all potential therapies and treatments have a high standard to meet to be considered appropriate alternatives. Some research is focusing on altering wheat to remove or detoxify gluten, but there is a general dislike for genetically altered foods, Greene says. There is also research looking at the use of anti-glutenase enzymes that could reduce the damage caused by gluten.
Larazotide, which is entering phase 3 trials, is helpful in reducing symptoms of celiac disease. Budesonide and prednisone, along with other steroids, have been used to reduce symptoms in celiac patients. Researchers are also working on a vaccine that involves the injection of peptides that may protect patients from some gluten contamination, Greene notes, adding that study is moving to phase 2 trials.
More than 20 different immunotherapy targets are also being investigated, one in particular for refractory celiac disease—a particularly bad form of the disease that does not respond to a gluten-free diet "There is a lot of interest, and some of the bigger pharma companies are getting involved," Greene says.
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