First batches of ULTRA-DD patient-based assay data released - ahead of publication
The ULTRA-DD project has made good on its promise to make its data open source with the publication online of datasets from experiments on autoimmune diseases such as lupus and myositis. Through the experiments, the ULTRA-DD team has identified potential new targets that could inspire the development of new treatments for these diseases. The project hopes that if other researchers probe and use the data, they may uncover further insights that will add to our knowledge of autoimmune diseases and accelerate the development of medicines for these patients, many of whom do not respond well to existing treatments. Currently, additional follow-up studies are ongoing to verify and validate the results.
The data published by ULTRA-DD comes from experiments using technically advanced tests developed by the project partners. During the earlier stages of drug development, scientists commonly rely on tests involving human cancer cell lines cultured in the lab, even for non-cancer diseases, or animal models of the disease under study. However, these tests often fail to deliver results which accurately reflect human biology and disease. ULTRA-DD is working to address the need for better, more reliable tests by developing sophisticated tests that are based on blood and tissue samples obtained directly from patients.
In this case, the ULTRA-DD team studied so-called ‘B cells’ – immune cells which are known to play a central role in lupus and myositis. According to the project’s Scientific Director, Michael Sundström of the Karolinska Institutet, IMI support was critical to this study. ‘There has been a very tight collaboration,’ he said, explaining that the pharmaceutical companies provided guidance on priorities and shared experimental protocols, as well as providing critical research reagents to the academic team. The universities delivered scientific excellence, and the hospitals helped with access to consenting patients and clinical expertise. The project has also forged good direct links with relevant patient organisations.
Meanwhile Professor Sundström adds: ‘These are the first of a series of related datasets to be released, and we believe that our approach of pre-publication access to such data is truly unique. In addition to the data released from Karolinska, additional datasets from our IMI-funded partner laboratory in Oxford, which is working on inflammatory and fibrotic diseases, will also be made public during the next two and a half years under the ULTRA-DD project.’
About lupus and myositis
Systemic lupus erythematosus (SLE) is a systemic inflammatory autoimmune disease affecting many organs of the body, such as the skin, joints and kidneys. The symptoms vary from one patient to another, and can include fatigue and joint and muscle pain. There is no cure for lupus; patients are often treated with corticosteroids and other anti-inflammatory drugs. While these treatments minimise symptoms, they are not effective in all patients.
Idiopathic inflammatory myositis (IIM) is a chronic rheumatic muscle disease that results in skeletal muscle weakness and pain. Corticosteroids represent the main treatment strategy, in combination with antiinflammatory drugs. But while a majority of patients benefit from these treatments, many develop chronic muscle weakness and struggle to carry out every day activities.
Further information and reading:
- ULTRA-DD website: www.ultra-dd.org
- ULTRA-DD factsheet: www.imi.europa.eu/projects-results/project-factsheets/ultra-dd
- Access the online datasets: www.ultra-dd.org/tissue/cellassays
- Preclinical target validation using patient-derived cells. Nat Rev Drug Discov. 2015 Mar;14(3):149-50. doi: 10.1038/nrd4565. https://www.ncbi.nlm.nih.gov/pubmed/25722227