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By Sandra C. Raymond
Women, men, and children stricken by lupus, the prototypical autoimmune disease, often experience many setbacks as they struggle to overcome the disease. For far too long, those with the disease have been disappointed about how little was being done to find the causes and cure for lupus. However, we believe there are numerous signposts on the road to a cure that signal a new era of discovery and hope.
In this new era, federal funding for lupus research is steadily increasing, and additional biotechnology and pharmaceutical companies are investing money to develop safe, nontoxic, and effective therapies for lupus. And while a cure remains elusive, promising research studies to find a cure have begun.
The federal government is more engaged in lupus than ever before. For example, a new Federal Working Group on Lupus has been established through the National Institute of Arthritis and Musculoskeletal and Skin Diseases to coordinate federal efforts on lupus; the Centers for Disease Control and Prevention is supporting patient registries in Atlanta and in Detroit to ascertain the incidence and prevalence of lupus; the National Institutes of Health has created a program to validate lupus biomarkers; and the FDA has released a much anticipated draft guidance document to provide industry with guidelines on lupus drug development.
The availability of new safe, nontoxic, and effective therapies is a key priority for people with lupus and the health professionals who treat them. Many existing therapies for lupus, although often effective in controlling disease activity, are toxic and cause other potentially serious health effects from their use over time. There has not been a new, FDA-approved therapy for lupus in almost 40 years.
While the number of companies developing new treatments for lupus has increased, there is an urgent need to bring down barriers that in the past have obstructed the progress of clinical trials on lupus. As these scientific barriers are overcome, an increasing number of pharmaceutical and biotechnology companies will make substantive investments to find the causes and cure for lupus.
Industry, however, must be enabled to conduct successful clinical trials of potential new treatments for lupus. Because of the heterogeneity of lupus patients, clinical trials of new lupus drugs require a large number of patients treated over a long period. There is an urgent need to validate biomarkers to efficiently measure the safety and efficacy of new drugs. Validated biomarkers will enable pharmaceutical and biotechnology companies to determine whether their drugs are working within a shorter period of time using a smaller group of patients. There are many candidate markers for lupus, and efforts are under way to validate them for use in clinical trials.
In addition, achieving better outcomes for individuals with lupus by improving early diagnosis of the disease is needed. More than one-half of the people with lupus report they visited three or more doctors over four or more years before being diagnosed. Through increased public awareness of lupus symptoms, better training of health professionals, and improved diagnostic tests, individuals with lupus can receive earlier treatment, impeding progression of the disease and improving their quality of life.
It is also important to have reliable, evidence-based epidemiological information on the incidence and prevalence of lupus among different populations.
Existing epidemiological studies on lupus have not included all the syndromes associated with lupus and all the subsets of the population believed to be disproportionately affected by lupus. The benefits of a national scale study will enable researchers to better study the disease, will encourage industry to identify the market potential of new therapies to treat manifestations of lupus, and will enhance efforts to plan effective educational strategies to reach those individuals at risk for the disease.
This is an exciting time for everyone affected by lupus: patients, family members, physicians, and researchers. The pace of discovery for lupus is escalating, and new scientific pathways have created opportunities to gain insight into the causes of the disease. In this new era of discovery, the Lupus Foundation of America will lead a nationwide crusade to ensure that people with lupus will have hope that this disease will be brought under control and that a cure will be found.
Sandra C. Raymond is president and CEO of the Lupus Foundation of America Inc.