Slovenian researchers achieve world first in autoimmune disease treatment
Researchers at Slovenia's largest hospital have demonstrated that an autoimmune disease can be treated by removing the underlying trigger of the immune response rather than suppressing the immune system, in what they say is a world first.
The clinical team at University Medical Centre Ljubljana's Department of Allergology, Rheumatology and Clinical Immunology successfully treated a teenager with a rare inherited form of systemic lupus erythematosus caused by a deficiency of specific enzyme called DNASE1L3, CE Report quotes The Slovenia Times.
The patient had failed to respond to every available immunosuppressive treatment, including modern biological drugs. Instead of further dampening the immune system, doctors administered an experimental recombinant version of the missing DNASE1L3 enzyme, targeting the root cause of the disease.
Within just six hours, the patient's skin rash disappeared, inflammation of the eyes subsided, arthritis resolved and laboratory markers improved dramatically, according to department head Tadej Avčin, who led the clinical study.
"We are the first in the world to show a different therapeutic approach, where we remove the trigger of the immune response instead of directly suppressing the immune system," Avčin said.
The patient was diagnosed in Ljubljana in 2019 after genetic testing revealed a rare mutation preventing the body from producing DNASE1L3. That discovery allowed the Slovenian team to join an international drug development programme.
Following a review by European and American experts, the patient was selected as the first person worldwide to receive the therapy under a compassionate-use programme because the drug has not yet been approved for general use.
The experimental enzyme was developed by US biotechnology company Neutrolis, which is developing DNASE1L3-based therapies as a non-immunosuppressive treatment for autoimmune diseases including lupus and rheumatoid arthritis.
According to Avčin, this breakthrough opens the door to an entirely new class of therapies for a range of very diverse autoimmune diseases and perhaps even other conditions.
After the successful treatment, clinical studies will continue on a larger group of patients, but they will be conducted at two other hospitals.
The clinical study's results have been published in the New England Journal of Medicine, one of the world's leading scientific journals.
"The concept that removing the trigger can mitigate the clinical symptoms of an autoimmune disease is global breaking news and this is why it has been recognised in a leading journal in clinical medicine," Avčin said.
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