Researchers on the Path to a Cure – Spotlight on Dr. Nora Singer
Patient perspectives play a large role in Dr. Nora Singer’s 3-year Arthritis Foundation-funded project - “Cell-based therapy in systemic onset JIA (sJIA) refractory to conventional “c” and biological “b” DMARDs”. Her project uses stem cells derived from adult cells that are designed to “reset” the immune system. The study will compare the safety and effectiveness of the stem cell treatment to conventional and biological disease-modifying antirheumatic drugs (DMARDs). Currently, stem cell therapy is experimental in the U.S. and regulated by the U.S. Food and Drug Administration (FDA), but is less regulated in other countries.
Systemic JIA causes inflammation in multiple joints, with skin rash, and spiking fevers that last weeks. It affects about 10 percent of children with JIA. Dr. Singer explains why she chose to study sJIA instead of a different form of JIA: “This disease has the highest morbidity and mortality of the different forms of JIA, if you don’t stabilize it early. These are very sick kids. It becomes apparent which kids are responding to traditional treatments and which aren’t. The things we learn from this study may very well apply to other forms of JIA and other autoimmune diseases.”
Since this therapy is not approved in the U.S., some parents are going outside of the country for stem cell therapy for their children. Dr. Singer feels parents and patients could be taking a chance because some of the medical environments in other countries are less sophisticated in their treatment of sick kids and the quality controls on product may vary from center to center.
Dr. Singer’s project has several steps. The first step, which began in January, is listening to focus groups made up of parents and patients to discover what will work best in a protocol to test the therapies. Getting feedback from patients is important in the protocol design process because it will help ensure the concerns of parents and patients are addressed, that the protocol is one that parents might enroll their child in and it engages families to not only take part in the research study, but also in the design of the study.
After the protocol is designed with the focus group input, a Phase I clinical trial protocol is submitted to the FDA for approval. The study will collect safety data of stem cell therapy that may lead to further development of this therapy. Once the protocol is approved by FDA and the respective Institutional Review Boards at the participating centers, the last step - enrollment for the study - can begin.
Collecting safety data with a Phase I study is important. Dr. Singer explained, “While some safety data has been collected in studies in China and more recently in the Netherlands, there is a need to collect U.S. safety data to better understand the biology of how the stem cells work to sense injury and inflammation, and adjust the immune response. This is an important part of determining whether stem cells have a place in the therapeutic toolbox for treatment.”
Dr. Singer is one of six recipients of the Arthritis Foundation 2016 Delivering on Discovery award and a rheumatologist in Cleveland, Ohio who cares for adults and children. She is affiliated with MetroHealth Medical Center and Case Western Reserve University and is a member of the Childhood Arthritis Rheumatology Research Alliance (CARRA), which the Arthritis Foundation also funds.
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