Making Progress in the Fight Against Lupus
Making Progress in the Fight Against Lupus
08.22.12 | By Matt Bennett
The quest for a cure can be a rigorous process that is laden with a series of setbacks and failures. Fortunately, researchers use what they learn from each of these setbacks to advance the science one step closer to a breakthrough. It is because of the perseverance of these researchers who dedicate years and sometimes decades to the discovery of new treatments, that patients suffering from lupus just received the first FDA approved treatment in 50 years.
As the world's leading private supporter of innovative research on lupus, the Lupus Research Institute (LRI) pioneers discovery and champions scientific creativity to find solutions for this debilitating disease. As part of our "From Hope to Cures " guest blog series, following is a guest blog from LRI Research Director Jennifer Bell, PhD, on the significant research progress made in finding new treatments for lupus and the future directions that give us new reason for hope.
1. In 2011, for the first time in 50 years the FDA approved a new treatment for Lupus. How does the Lupus Research Institute plan to maintain progress toward curing this life-threatening disease?
The Lupus Research Institute funds novel research in lupus - research that provides the critical first steps in the path towards new treatments and a cure. The new ideas we support are too novel, too innovative to win conventional funding and so can be viewed as high risk. Yet we know that 80% of our grants are successful, resulting in pivotal discoveries, substantial follow-on funding from NIH and other organizations and, most importantly, new candidates to diagnose and treat lupus.
Currently there are 30 or so targeted treatments in clinical trials for lupus. These drugs hold promise for safer and more effective treatments to suppress the disease, which patients sorely need. But what is even more critical are interventions that can bring about long-term remission - what can be considered a cure. We cannot work towards curing lupus when we do not know what causes it at the most fundamental level. We are aiming to close this gap with an ambitious new program jump-started this year to specifically support the most innovative projects that can drive towards a cure by uncovering the fundamental causes of lupus.
2. At least seven possible medicines have been turned back in the last several years in the clinical trial stage for Lupus. What kept the Lupus Research Institute progressing toward a better range of treatment options?
Several issues have impeded progress in drug development to treat lupus, primarily the heterogeneity and unpredictability of the disease itself, which makes it difficult to design trials that can deliver on efficacy endpoints. Another issue has been the lack of appropriate biomarkers to monitor disease status and identify the most appropriate patients for the trial. To overcome this obstacle, we have invested substantially in biomarker discovery, leading to nine new exploratory biomarkers of which seven are currently in clinical development.
Difficulty enrolling patients for trials is a major problem, particularly among minorities in a disease that disproportionately affects young women of color. We are working to overcome this obstacle by providing educational materials on trials and helping trial sponsors to access a broad patient population including minorities.
Dialogue among all of the stakeholders in the quest for a cure to lupus is critical to solving these issues and LRI is helping facilitate by bringing together industry, academic researchers, patients and the FDA to discuss the challenges in developing new drugs for lupus.
3. While it took 50 years for the FDA to approve a new medicine for Lupus, how would you characterize more recent progress in the fight against lupus?
Over a decade of intensive work has given the community much reason for hope. When the LRI was formed 12 years ago, there were no clinical trials and little progress in sight. Incredible progress has been made with over 30 companies now involved in developing new treatments for lupus. Because of this acceleration in lupus drug development it is increasingly important for industry and patient organizations to work together to engage a population that has not been extensively exposed to the trial process. As the disease affects predominantly women of color, it necessitates trial enrollment of a historically difficult-to-reach population. Fortunately, the FDA has demonstrated a recognition of the need to test drugs adequately among minorities by requesting that the first new treatment of lupus in 50 years is subject to a Phase IV study of use among African-Americans. The LRI is well positioned to reach a broad patient population, having built a coalition of patient groups throughout the country, but particularly in urban centers where the greatest concentration of lupus patients can be found.
4. What advice do you have for Lupus patients and their families who are seeking more information about the disease and treatment progress?
Whether patients are newly diagnosed or have been living with lupus for years, education on the disease and how it can impact their lives is an important first step in the appropriate management of the disease. Patients and their families can visit the LRI website at LupusResearchInstitute.org to learn about the disease and treatment progress or connect with other patients around the world on our Facebook page. In addition, patients and their families have the potential to make a tremendous difference in their own care and that of future generations by participating in clinical trials. Patients can learn about what trials are and how they work by visiting our website, LupusTrials.org.