As an organization, we have always supported and encouraged the active involvement of patients in their own care. Patient perspectives matter greatly when it comes to decisions being made about health care. Their voices are vital in the clinical research process as new medications are being developed and tested.
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 biologicaldisease-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 JIAcauses 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.
在通过焦点小组的输入设计方案后，一期临床试验方案将提交给FDA批准。该研究将收集干细胞治疗的安全性数据，这可能会导致这种治疗的进一步发展。一旦该方案被FDA和各参与中心的机构审查委员会批准，最后一步——研究登记——就可以开始了。
收集第一阶段研究的安全数据是很重要的。辛格博士解释说:“虽然在中国和荷兰的研究中收集了一些安全数据，但有必要收集美国的安全数据，以便更好地理解干细胞如何工作，以感知损伤和炎症，并调整免疫反应的生物学。这是决定干细胞是否在治疗工具箱中占有一席之地的重要部分。”
Dr. Singer is one of six recipients of the Arthritis Foundation2016 Delivering on Discoveryaward 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.
Related Resources:
- Researchers on the Path to a Cure – Spotlight on Dr. Richard F. Loeser, Jr
- Researchers on the Path to a Cure – Spotlight on Dr. Hongsik Cho
- Researchers on the Path to a Cure – Spotlight on Dr. Christine Beeton
- Researchers on the Path to a Cure – Spotlight on Dr. C. Michael Stein
- Participate in JIA Research