ALPHA Project: Diversity of Lupus Signs and Symptoms Identified as Major Challenge
Kenneth Getz MBA, Associate Professor, Director of Sponsored Research Programs
Tufts Center for the Study of Drug Development
Tufts University School of Medicine
This blog is one in a four-part that dives into the key takeaways from the Addressing Lupus Pillars for Health Advancement (ALPHA) Project, an initiative with the goal of bringing together international lupus experts to develop and implement strategies to address the critical barriers toward improving outcomes in lupus.
Lupus is an extremely complex disease that is very difficult to diagnose and treat. Despite the fact that approximately five million people worldwide are affected by lupus, the symptoms are non-specific. They may look similar to symptoms associated with other medical conditions such as connective tissue diseases and the symptoms vary a lot from patient to patient. There are even instances where some critically-ill patients show no specific signs of sickness at all. And, the underlying cause of this autoimmune disease remains unknown.
Non-specific symptoms that vary widely between patients is also called ‘heterogeneity.’ And in lupus, heterogeneity has made it very difficult to develop new drugs and treatments. It has been very hard, for example, for clinical research and clinical care professionals to agree on the causes of the disease and how to best treat the symptoms. Since the 1950s, several dozen pharmaceutical companies have been working on the development of new drugs to treat lupus. But in that time only one drug — Benlysta® (belimumab) — has been approved by the U. S. Food and Drug Administration.
The number of people affected by lupus is rising around the world, and the death rate from lupus is consistently three times higher than the general population. The need to unify lupus clinicians and scientists, pharmaceutical companies, and policy makers with the patient community and improve our understanding of the disease to guide development, diagnosis and treatment has never been greater. To address this need, the Lupus Foundation of America, researchers at the Center for the Study of Drug Development at Tufts University School of Medicine (Tufts CSDD) and a global committee of lupus experts collaborated to launch the ALPHA Project in 2018.
The goal of the first phase of the Addressing Lupus Pillars for Health Advancement (ALPHA) Project is to identify the top barriers making progress in the field difficult and to promote global consensus on these challenges. Through interviews and a survey conducted with lupus experts and clinicians around the world, the ALPHA Project confirmed and prioritized top barriers associated with lupus heterogeneity that will lead to the development of a road map for drug development professionals and health care providers – and other stakeholders – to follow in order to better diagnose and care for lupus patients and to develop better new treatments. The top five barriers to improving outcomes in lupus include:
- Lack of diagnostic, predictive and prognostic biomarkers for lupus
- Lack of biomarkers to predict drug response in clinical trials
- Lack of access to clinicians familiar with or aware of lupus
- Barriers to effectively manage lupus due to social factors, especially in lower socioeconomic status areas
- Lack of treatment adherence
Strong agreement around the top barriers represents a major opportunity and call for greater investment and effort among stakeholders to identify and validate biomarkers, which can indicate the presence of diseases such as lupus, and target more personalized approaches to treatment in order to address lupus heterogeneity.
The ALPHA team is now focusing on sharing the key findings from the project with lupus stakeholders and mobilizing patients, advocates, health and research professionals, regulators and policy makers to advance the front line of lupus research and care. For more information, please visit lupus.org/alpha.