Simplemind map for sickle cell anemia driver#
Simplemind map for sickle cell anemia trial#

Simply envision a pair of ‘molecular scissors’ that could cut out, insert or change disease-causing problems at the genetic level and offer new hope to patients,” explains Cavey, referring to ongoing research in gene therapy for sickle cell disease with CRISPR. “Because sickle cell disease has a single genetic mutation, it provides unique opportunities in exploring gene editing technology. This includes work with small molecules, as well as gene editing with CRISPR technology licensed from Intellia Therapeutics. Our scientists, for example, are working closely with academic researchers and other organizations to evaluate potential disease-modifying therapies. Novartis is also exploring other ways to target processes involved in sickle cell disease. Recently approved in the US to reduce the frequency of VOCs, or pain crises, in adult and pediatric patients aged 16 years and older with sickle cell disease, Adakveo® (crizanlizumab) is an important part of the Novartis commitment to reimagine medicine for individuals living with the disease. Pushing forward the body of knowledge is important, and I’m hopeful that this work will reduce the frequency of pain crises in many patients.” Relentless pursuit to treat people with sickle cell disease “No one who takes care of patients can fail to see how devastating sickle cell disease can be. “When I heard the results, even just verbally, I thought, ‘This actually could work.’” Sharing this belief, Novartis exercised its right to acquire this biotech company later in 2016 and took on the ongoing development of crizanlizumab – which eventually became part of our larger drug development program in sickle cell disease.

“I was on the phone as we unblinded the Phase II results,” Dr.

Simplemind map for sickle cell anemia trial#

The Phase II trial was then conducted, and the results were available in 2016. The agreement provided Novartis with an exclusive option to acquire the company and its lead asset, crizanlizumab, following the completion of the planned Phase II clinical study. When early clinical data showed promise in 2012, Novartis entered an agreement with the biotech company researching crizanlizumab. This clinical advancement was decades in the making. Rodger McEver, Physician-scientist at the Oklahoma Medical Research Foundation When I heard the results, even just verbally, I thought, ‘This actually could work. I was on the phone as we unblinded the Phase II results. Rodger McEver, a physician-scientist at the Oklahoma Medical Research Foundation in the US, recalls the discovery of P-selectin, which eventually resulted in the development of a monoclonal antibody – called crizanlizumab – that binds to P-selectin on the surface of the activated endothelium and platelets, blocking interactions between endothelial cells, platelets, red blood cells, and leukocytes. P-selectin is one of the cell adhesion proteins found in excess on the surface of activated platelets and endothelial cells in blood vessels due to the inflammatory environment in sickle cell disease – which can lead to vaso-occlusion, potentially causing sickle cell pain crises or VOCs. The discovery of a protein called P-selectin in the 1980s helped advance scientists’ understanding of sickle cell disease beyond the signature sickle-shaped red blood cells. Rodger McEver, a physician-scientist at the Oklahoma Medical Research Foundation

Simplemind map for sickle cell anemia driver#

This, we know now, is a major driver of vaso-occlusion.” “One key process is that of multicellular interactions, where the platelets, lining of blood vessels, and white blood cells all kind of stick to each other. “Our understanding of what drives sickle cell disease has improved considerably over the years,” says Andrew Cavey, Global Program Head of Hematology at Novartis Oncology. The role of the P-selectin protein in sickle cell innovation Discovering a key mechanism behind these clusters was critical to developing a novel treatment approach to this disease. They are triggered, in part, by the formation of these clusters of cells, which can block or reduce the blood flow to organs. Individuals living with sickle cell disease often experience frequent and sudden debilitating pain crises, also known as vaso-occlusive crises (VOCs), which can be associated with life-threatening complications.

Unfortunately, Majara’s experience is not uncommon. Majara is one of many people living with sickle cell disease.