{"id":134852,"date":"2025-06-02T13:47:06","date_gmt":"2025-06-02T17:47:06","guid":{"rendered":"https:\/\/www.bumc.bu.edu\/camed\/?post_type=bu-article&p=134852"},"modified":"2026-09-09T12:26:54","modified_gmt":"2026-09-09T16:26:54","slug":"collaboration-between-bu-clinicians-basic-scientists-and-engineers-can-lead-to-new-drugs-to-fight-disease","status":"publish","type":"bu-article","link":"https:\/\/www.bumc.bu.edu\/camed\/news-events\/articles\/2025\/collaboration-between-bu-clinicians-basic-scientists-and-engineers-can-lead-to-new-drugs-to-fight-disease\/","title":{"rendered":"Collaboration Between BU Clinicians, Basic Scientists and Engineers Can Lead to New Drugs to Fight Disease"},"content":{"rendered":"\t
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(L to r) Jean Francis, MD, Vipul Chitalia, MD, PhD, Katya Ravid, DSc.<\/p>\n\t\t\t\n\n\t\t\t\n\t\t\t\t

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\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\tCollaboration Between BU Clinicians, Basic Scientists and Engineers Can Lead to New Drugs to Fight Disease<\/strong>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/h1>\n\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t

Recent studies illustrate how collaborations between basic science and clinical research<\/em> can lead to clinical trials, new treatments<\/em><\/h4>\n\t\t\t\t\t\t\n\t\t\t\t\t<\/div>\n\n\t\t\t\t<\/div>\n\n\t\t\t\n\t\t<\/div>\n\n\t\t\n\t<\/div>\n\n\t\n
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June 2, 2025<\/div>\n\t\t\n\t\t\t\t
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New drugs and therapeutic solutions do not suddenly appear; they are rooted in collaborations between basic and clinical science research. In two examples, BU researchers have seen their basic science research of devastating diseases, for which therapy is currently still limited, advance to the clinical phase.<\/p>\n\n\n\n

\u201cUnderstanding basic mechanisms that lead to diseases can identify targets for the further development of therapeutics and new diagnostic tools,\u201d said Katya Ravid<\/a>, DSc, Barbara E. Corkey Professor of Medicine and founding director of the Evans Center for Interdisciplinary Biomedical Research<\/a>, of which novel Affinity Research Collaboratives (ARCs) are building blocks.<\/p>\n\n\n\n

With central support from the Department of Medicine<\/a>, along with the BU Clinical & Translational Science Institute<\/a>, BU Associate Provost for Research Office<\/a>, BUMC Provost\u2019s Office, and recently Boston Medical Center (BMC), ARCs use interdisciplinary, convergence science approaches, integrating the physical and biological sciences, to address complex medical problems. Ravid said that studies aiming to identify mechanisms behind the pathophysiology of disease are, importantly, funded by the National Institutes of Health.<\/p>\n\n\n\n

There are two main routes for basic science research to make the leap to clinical studies and new drugs and\/or treatments. Biotech companies can learn about advancements through published research and decide to develop them into possible clinical applications. The other pathway involves investigators pursuing their own findings with the support of the school\u2019s office of technology development, and the help of additional funding sources.  <\/p>\n\n\n\n

Ravid\u2019s lab<\/a> studies mutations in blood stem cells, generally associated with aging that cause primary myelofibrosis (PMF). The disease manifests as uncontrolled proliferation of blood cells and the appearance of a very dense, nearly clogged fibrotic bone marrow that hinders the ability to produce a normal balance of different blood cells. Symptoms also include abnormal blood cell counts, an enlarged spleen and damage to veins carrying blood from the spleen to liver. In some cases, PMF can lead to myeloid leukemia.<\/p>\n\n\n\n

\u201cThis is a harsh disease, for which current therapies are still only partially effective\u201d says Ravid. While studying cellular reprograming in the mutated cells, her lab found that lysyl oxidase (LOX) was highly expressed in mutated blood cells of the platelet lineage. Ravid and her lab mentees found that inhibiting this enzyme in experimental models reduced bone marrow clogging and other hallmarks of the disease, as well as identified cellular mechanisms that lead to this favorable outcome.  <\/a><\/p>\n\n\n\n

The published studies from this research attracted the attention of the biotech company Pharmaxis (renamed Syntara in 2023), which provided the Ravid lab with more specific pharmacologic inhibitors of LOX for further testing. Successful results obtained and published by Ravid\u2019s lab prompted a clinical trial  with favorable results in human cohorts, which was published in April in the journal Haematologica.<\/em><\/p>\n\n\n\n

That study concluded that preliminary indications of clinical efficacy, including a reduction in bone marrow clogging collagen, were evident, and that continued investigation was warranted for the LOX inhibitor PXS-5505 as a possible remedy for PMF.<\/p>\n\n\n\n

Another study investigating the role of the protein interleukin-6 (IL-6) in the disease calciphylaxis also resulted in clinical trials. The study was led by Vipul Chitalia<\/a>, MD, PhD, professor of medicine & pathology, and Jean Francis<\/a>, MD, associate professor of medicine and medical director of BMC\u2019s Kidney Transplant Program. They are co-leaders of the Thrombosis and Hemostasis ARC<\/a>.<\/p>\n\n\n\n

Calciphylaxis is a rare disease characterized by dermal microvessel thrombosis, the blockage of small blood vessels in the skin that results in skin tissue death. Calciphylaxis occurs mostly in patients with end-stage kidney disease and those patients suffer high (45-80%) annual mortality with debilitating pain, recurrent hospitalizations, limited mobility, depression and poor quality of life. The molecular events driving calciphylaxis are largely unknown and that has significantly impeded the development of a disease marker and targeted therapies.<\/p>\n\n\n\n

\u201cTo date, there is no approved therapy for calciphylaxis and we only have off-label therapies that are ineffective and not well tolerated,\u201d said Chitalia.<\/p>\n\n\n\n

Along with collaborators, Chitalia and Francis examined skin and blood samples from calciphylaxis patients and investigated a highly potent and prevalent blood clotting pathway that was triggered by IL-6, an inflammatory protein. Researchers found that excess IL-6 initiated thrombosis that caused rapidly expanding skin necrosis.<\/p>\n\n\n\n

The study, which was published in April in the journal Science Translational Medicine<\/a>,<\/em> concluded that results supported exploring IL-6 clot-forming as a marker and possible target for therapeutic medicines, which attracted the interest of pharmaceutical companies that had approved anti-IL-6 antibodies.<\/p>\n\n\n\n

\u201cThis body of work paved the way to treat calciphylaxis with anti-IL-6 therapeutics. We have initiated a pilot study at Boston Medical Center, where our first patient showed encouraging results,\u201d said Francis.<\/p>\n\n\n\n

Ravid said the calciphylaxis research is an example of how ARCs work.<\/p>\n\n\n\n

\u201cThis study is a manifestation of the creativity of our faculty, aided by the strong interdisciplinary research and platforms that we have developed here at the medical school through ARCs that can really bring such research to fruition; first through the basic discovery and publication, and then taking it beyond,\u201d Ravid said. \u201c(With ARCs) we see fruits of collaboration between clinicians, basic scientists and engineers across all BU campuses, in line with our long-standing mission of promoting multidisciplinary, convergence science.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"

Recent studies illustrate how collaborations between basic science and clinical research can lead to clinical trials, new treatments.<\/p>\n","protected":false},"author":903,"featured_media":134860,"comment_status":"closed","ping_status":"closed","template":"","meta":{"bu_prepress_billboard":"","_bu_prepress_primary_term":"","_bu_prepress_primary_term_manual":"Research"},"tags":[465,94],"bu-publication":[367],"medicine-article-category":[],"medicine-topic":[],"news-article-category":[],"news-topic":[],"bu_edition":[390,447,368],"media_type":[],"_links":{"self":[{"href":"https:\/\/www.bumc.bu.edu\/camed\/wp-json\/wp\/v2\/bu-article\/134852"}],"collection":[{"href":"https:\/\/www.bumc.bu.edu\/camed\/wp-json\/wp\/v2\/bu-article"}],"about":[{"href":"https:\/\/www.bumc.bu.edu\/camed\/wp-json\/wp\/v2\/types\/bu-article"}],"author":[{"embeddable":true,"href":"https:\/\/www.bumc.bu.edu\/camed\/wp-json\/wp\/v2\/users\/903"}],"replies":[{"embeddable":true,"href":"https:\/\/www.bumc.bu.edu\/camed\/wp-json\/wp\/v2\/comments?post=134852"}],"version-history":[{"count":6,"href":"https:\/\/www.bumc.bu.edu\/camed\/wp-json\/wp\/v2\/bu-article\/134852\/revisions"}],"predecessor-version":[{"id":146510,"href":"https:\/\/www.bumc.bu.edu\/camed\/wp-json\/wp\/v2\/bu-article\/134852\/revisions\/146510"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.bumc.bu.edu\/camed\/wp-json\/wp\/v2\/media\/134860"}],"wp:attachment":[{"href":"https:\/\/www.bumc.bu.edu\/camed\/wp-json\/wp\/v2\/media?parent=134852"}],"wp:term":[{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.bumc.bu.edu\/camed\/wp-json\/wp\/v2\/tags?post=134852"},{"taxonomy":"bu-publication","embeddable":true,"href":"https:\/\/www.bumc.bu.edu\/camed\/wp-json\/wp\/v2\/bu-publication?post=134852"},{"taxonomy":"medicine-article-category","embeddable":true,"href":"https:\/\/www.bumc.bu.edu\/camed\/wp-json\/wp\/v2\/medicine-article-category?post=134852"},{"taxonomy":"medicine-topic","embeddable":true,"href":"https:\/\/www.bumc.bu.edu\/camed\/wp-json\/wp\/v2\/medicine-topic?post=134852"},{"taxonomy":"news-article-category","embeddable":true,"href":"https:\/\/www.bumc.bu.edu\/camed\/wp-json\/wp\/v2\/news-article-category?post=134852"},{"taxonomy":"news-topic","embeddable":true,"href":"https:\/\/www.bumc.bu.edu\/camed\/wp-json\/wp\/v2\/news-topic?post=134852"},{"taxonomy":"bu_edition","embeddable":true,"href":"https:\/\/www.bumc.bu.edu\/camed\/wp-json\/wp\/v2\/bu_edition?post=134852"},{"taxonomy":"media_type","embeddable":true,"href":"https:\/\/www.bumc.bu.edu\/camed\/wp-json\/wp\/v2\/media_type?post=134852"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}