Three Renowned Researchers Share Scientific Successes
Three Renowned Researchers Share Scientific Successes

Three outstanding presentations by researchers at the Philadelphia Lupus Research Alliance Advancing Lupus Care Through Research and Advocacy Conference are summarized here:

Dr. Andrea Knight, Children’s Hospital of Philadelphia
“Lupus in Children: Effects on the Developing Brain”
Dr. Knight focused on the effects of brain inflammation that occurs in at about 25% of kids with lupus and the urgent need for more research in this area.

Key points:

  • Neuropsychiatric problems caused by brain inflammation include depression, anxiety, thinking and memory difficulties, headache, psychosis, seizures and stroke.
  • Using a new MRI technique, another study she led found changes in the developing brains of teens even before they showed any symptoms. The aim of future research would try to stop damage before symptoms appear.
  • Knight is now working on a study funded by the Lupus Research Alliance will use a highly sophisticated MRI to see how changes in psychiatric health among children with lupus are related to physical changes in the brain and thinking ability. She is also exploring if proteins in the blood can detect changes in brain function and how to use this information to diagnose issues sooner and individualize treatment.


Dr. Victoria Werth, University of Pennsylvania School of Medicine
“New Approaches to Understanding and Treating Lupus Erythematosus in the Skin”
Dr. Werth showed the critical need and potential for many new treatments being studied for skin lupus.

Key points:

  • Half of people with skin lupus do not also have systemic lupus but skin lupus is largely under-studied.
  • Studies show that quality of life is greatly affected by skin lupus, particularly in social and emotional health.
  • Werth’s study funded by the Lupus Research Alliance found that when treated with the cancer drug lenalidomide, patients who had not been helped by other drugs saw their symptoms significantly improve in a short time frame. This work has led to further studies finding benefit for people with skin lupus with lenalidomide and a related drug CC-220.
  • There are several completed Phase 2 studies that have shown encouraging results for cutaneous lupus, and ongoing studies for cutaneous and systemic lupus that are looking at skin improvement as an outcome. Clinical trials with newer therapies will likely lead to improved treatment options.
  • Advocacy from the lupus community is needed so the FDA understands that even modest improvement is important for patients with skin lupus when potential new treatments are reviewed for approval.

Dr. Philip Cohen, Temple University
“Treatment of SLE: A Glimpse into the Future”
Dr. Cohen conveyed the tremendous progress being made in the development of new treatments.

Key points:

  • Most current treatments for lupus are non-specific, meaning that they do not target the immune system specifically. Targeted therapies represent the direction of the future.
  • Benlysta is the first monoclonal antibody adapted for lupus and specifically destroys B cells. But all monoclonal antibodies are expensive and cannot be taken by mouth.
  • Based on positive results, Stelara, which is approved for psoriasis, is in late-stage testing for lupus through the Lupus Clinical Investigator Network (LuCIN) formed by the Lupus Research Alliance affiliate Lupus Therapeutics. This work is part of the repurposing program initiated by the Lupus Research Alliance to test drugs as lupus treatments that are approved and proven safe and effective for other conditions.
  • Other therapies in testing include anifrolumab, tofacitinib and bariticitinib which block specific substances released by certain immune system cells and could be taken orally.
  • Researchers are looking at whether we can prevent lupus if we treat before symptoms actually develop.

We hope this report helps convey the hope in the room that was generated by the promising work of these three outstanding researchers!  Watch here for complete program.

 

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