“Rheumatoid arthritis remains an incurable condition with no therapeutics that directly act to restore bone content and reduce cartilage degradation. Safety, often concerning elevated infection as a result of immunosuppression, remains a concern, while many patients respond inadequately or eventually develop resistance to first- and second-line therapies. In addition, biologics require either intravenous or subcutaneous administration, which is often inconvenient and painful for patients.”
“While rheumatologists often focus on treatment targets, for many patients with rheumatoid arthritis (RA), control over pain and fatigue, as well as sustaining physical function and quality of life (QoL), is of primary importance.”
“Despite the wide range of available treatments, RA continues to pose a substantial humanistic and economic burden on patients, and there are still unmet needs across key domains such as pain, physical function, mental function, and fatigue. These findings suggest that there is a need for further treatment advances in RA that address these domains of contemporary unmet need.”
The unmet need in rheumatology: reports from the Targeted Therapies meeting 2016
K.L. Winthrop, V. Strand, D. Van der Heijde, P. Mease, M.K. Crow, M. Weinblatt, J. Bathon, M.H. Buch, G.R. Burmester, M. Dougados, J. Kay, X. Mariette, F.C. Breedveld, J.R. Kalden, J.S. Smolen, D.E. Furst.
“Within RA, the committee was quite enthusiastic about the progress that has been made in the treatment of rheumatoid arthritis. Using a “treat to target” strategy with the rapid dose escalation of methotrexate followed by the addition of either biologic response modifiers or small molecules has changed the course of this disease. The committee recognized that a significant percentage of patients, even with this strategy, still remain with moderate to high disease activity. There is a need for additional therapies which can be added on top of background combination therapy that will enhance the efficacy already noted without increasing the degree of immunosuppression. That said, a major challenge currently is access to care due to high cost of the biologics and new small molecules. This is a universal concern, independent of country of origin. Another unmet need in rheumatoid arthritis is the identification of patients who are in remission who would be candidates for dose reduction. Development of biomarkers or imaging programs that can identify those patients who should be able to reduce their drug dose without a risk of a flare is needed. Additional therapeutics that repair the damage induced in RA, new treatments to increase remission rates, new non-narcotic analgesics and also alternatives to corticosteroids were identified as an unmet need. Lastly, the role of the microbiome in RA, the development of animal models that better reflect human disease and identification of sites remote from the joint that may be driving the articular inflammatory process were also noted as areas of research interest.”
Breakthrough cell treatment offers hope to rheumatoid arthritis sufferers
AUGUST 9, 2016 BY JENNY MARCHAL
“Mesoblast are hoping the success of the stem cell treatment in clinical trials will go on to be developed into a first-line treatment for those suffering from the debilitating disease that can often lead to premature death and an increase risk of heart disease. Dr. Allan Gibofsky, a rheumatologist at the Hospital for Special Surgery in New York said in a statement that the new treatment has promise to fill current unmet needs.”
“The safety and efficacy results of this study are very encouraging and suggest that Mesoblast’s cell therapy has the potential to fill the major unmet medical need [for patients who cannot take biologic treatments.]”
Innovation means understanding and responding to unmet needs
Like many other therapeutic areas, RA treatments are often used in combination. For some patients, the combination of methotrexate and a powerful biologic, such as Remicade (infliximab), will help a patient achieve remission Yet others will either not respond to methotrexate and Remicade, or will have a negative reaction. Understanding how to help nonresponders achieve relief has become a key area of research in RA.
According to Terence Rooney, MD, Medical Director at Lilly Bio-Medicines,
“A substantial proportion of patients treated with methotrexate – commonly used across the disease continuum for 25 years – do not achieve satisfactory disease control, signaling a need for more effective RA treatment options. In addition, studies have shown that some patients who initially respond to biologics lose response over time, and approximately 40 percent of patients with high disease activity never respond adequately to TNF antagonist biologics.”