Leading the way to a cure

Lupus Research Update: 2013 Volume 2

Volume 2, 2013 | In This Issue

Lupus Clinical Trials - People Advancing Science >
Insights into Clinical Trials - One Doctor's Perspective >
Faces of Lupus - Transforming Personal Loss into Hope for Others >
Lupus News Corner >

Insights into Clinical Trials

One Doctor's Perspective

Dr. Andras Perl is in a unique position — he is a leading autoimmune disease expert whose clinical trials benefit from invaluable decades-long experience that bridges both scientific research and patient care.

Direct contact with patients allows Dr. Perl to see the benefits, shortcomings, and side effects of treatments firsthand. One of the things he looks for in patients is the effect a particular intervention has on the signaling pathways that are involved in abnormal immune system function.

"We can see to what extent we can normalize them and how treatment relates to improved disease activity," said Dr. Perl. "I find it very instructive to do clinical work and laboratory investigations — it's really informative. Without clinical trials, everything is completely uncontrolled."

In his more than 30 years of studying various aspects of autoimmune disease, Dr. Perl has been involved in a number of clinical trials organized by pharmaceutical companies. Recently, however, he is the designer and lead investigator of two lupus clinical trials based on the laboratory research that he has been conducting for the past 10 to 15 years.

In his latest clinical trial, Dr. Perl and his team at the Upstate Medical University of the State University of New York are looking at ways to replenish a lost natural antioxidant called glutathione (GSH) in the T cells of patients with lupus.

Recent research suggests that metabolic abnormalities in T-cell differentiation and activation play a key role in autoimmunity and lupus.

Dr. Perl's trial involves treatment with the amino acid N-acetycysteine (NAC). "The reason I chose NAC is that it is one of the amino acid components of GSH — and GSH is the primary antioxidant within all living cells," he explained.

Dr. Perl has done extensive research into new treatments for lupus that avoid the significant side effects of existing therapies. In his current trial, he seeks to prove that NAC improves lupus disease activity by blocking mTOR — a protein that may control signaling pathways in metabolism and the differentiation of lymphocytes within the immune system. mTOR activation may play a central part in abnormal signaling in lupus.

The early findings of Dr. Perl's work — which were published in Arthritis and Rheumatismin September 2012 — hold great promise. Within the context of this clinical trial, Dr. Perl was able to show that NAC gets absorbed into lymphocytes and reverses the depletion of GSH.

The invaluable information gained in understanding lupus would not have been possible without this clinical trial. "You get a straight answer in clinical trials. And working with an agent like NAC is really great because it doesn't have any side effects. I am so encouraged by our initial findings," said Dr. Perl.

Although the ALR does not currently fund clinical trials, the organization has been critical to Dr. Perl's laboratory work by granting him two of our Target Identification in Lupus grants. "The ALR has been instrumental. With its funding, we discovered more about mitrochrondrial dysfunction in lupus. We further characterized it. We discovered mTOR activation in lupus and the vital role that NAC may play," Dr. Perl expressed with gratitude.

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