THURSDAY, May 27, 2021 (HealthDay News) – An experimental antibody treatment may help relieve skin symptoms of lupus, an autoimmune disease, a small preliminary trial suggests.
The researchers found that a higher dose version of the drug resulted in “clinically significant” improvement in symptoms for 87% of patients after one month.
But they also pointed out that the results are based on a small “phase 1” trial – a type of study designed primarily to assess the safety of a treatment.
The safety results were “encouraging” and there were “some indications of clinical benefit,” said lead researcher Jodi Karnell, senior research director at Horizon Therapeutics, the company developing the drug.
Now, she says, larger trials are needed to confirm the therapy is working.
The drug, currently known as VIB7734, is a monoclonal antibody – a protein made in the lab that acts as an antibody for the immune system. Such antibodies can be directed against specific substances in the body involved in a pathological process.
Lupus is caused by an autoimmune reaction, where the immune system mistakenly attacks the body’s own tissues.
The most common form is systemic lupus, which can cause inflammation throughout the body, including the skin, joints, kidneys, blood vessels, and the brain.
Another form, called skin lupus, affects only the skin, causing rashes and sores, often on the face and scalp.
There are treatments for these skin symptoms, including anti-inflammatory corticosteroids; antimalarial drugs, which modify the immune response; and immunosuppressive drugs like methotrexate.
But these treatments can have significant side effects, and they don’t always work, Karnell pointed out.
“There is a great unmet need,” she says.
In the United States alone, about 1.5 million people suffer from lupus, according to the Lupus Foundation of America.
A monoclonal antibody is approved for systemic lupus called Benlysta (Belimumab). It blocks a protein in the immune system that is involved in creating autoantibodies (antibodies that attack tissues in the body).
The new monoclonal antibody works in a different way, Karnell explained. It depletes cells of the immune system called plasmacytoid dendritic cells.
These cells normally fight infection by releasing inflammatory chemicals, including type 1 interferons. But uncontrolled activity in cells, pumping out too much interferon, is believed to contribute to autoimmune diseases.
For the Phase 1 trial, Karnell’s team recruited 31 patients with at least one of many autoimmune diseases, including systemic and skin lupus. They were randomly assigned to receive injections of either the monoclonal antibody, at different doses, or a placebo. The injections were given every four weeks, for a total of three.
After one month, the group receiving the highest dose of antibody showed the greatest benefit: seven out of eight (87.5%) had a “clinically significant” reduction in skin symptoms, compared to about 37% of patients receiving a lower dose and 28% of patients on placebo.
The results were published on May 26 in the journal Scientific translational medicine.
Dr Donald Thomas, a rheumatologist who was not involved in the study, warned: Over the years, various therapies for lupus have initially shown promise of only disappointing in late stage trials .
That said, these initial results are encouraging, he noted.
“If they confirmed that in the Phase 2 and Phase 3 trials, it would be a game changer,” said Thomas, of the Uniformed Services University of the Health Sciences, and Arthritis and Pain Associates in PG County, Md.
Skin lupus can affect the quality of life for patients, Thomas said, some with hair loss and scarring from skin damage.
Unlike therapies that target the immune system globally, monoclonal antibodies target specific components of the immune response, Thomas said. This means that they can have fewer side effects and be more effective.
Thomas noted that side effects with Benlysta, the antibody approved for SLE, were “remarkably minimal” overall.
Karnell stressed that the investigational drug worked as expected – depleting dendritic cells and type 1 interferon activity in the blood and skin lesions of patients. The next step is a larger Phase 2 trial, she added.
The researchers also found that patients with high interferon activity early on were those whose symptoms improved with the antibody. So a question for the future, Karnell said, is whether measuring patients’ interferon activity can help identify those who are most likely to benefit from treatment.
If doctors could do this, it would be a breakthrough, according to Thomas.
Today, he says, treatment for lupus often involves trial and error to determine which therapy is working – a fact that is frustrating for patients.
The Lupus Foundation of America has more on treatment options for lupus.
SOURCES: Jodi Karnell, PhD, senior director, research group, Horizon Therapeutics, Dublin, Ireland / Deerfield, Illinois; Donald Thomas Jr., MD, associate professor, medicine, Uniformed Services University of the Health Sciences, Bethesda, Maryland, and rheumatologist, Arthritis and Pain Associates of PG County, Greenbelt, Md .; Scientific translational medicine, May 26, 2021, online
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