Bloomberg Philanthropies, the Johns Hopkins University School of Medicine, and the New York Stem Cell Foundation (NYSCF) Research Institute have launched an initiative to advance and expand the science of precision medicine, in which diagnostic disease markers are defined with pinpoint accuracy to help researchers understand disease pathways and customize therapeutic approaches. The collaboration will combine the clinical expertise of Johns Hopkins with the stem cell technologies and research capabilities of the NYSCF Research Institute to accelerate pioneering precision medicine initiatives.
“Johns Hopkins is working intensively to realize the great promise of precision medicine for all those in our care, locally and globally,” says Ronald J. Daniels, JD, LLM, president of Johns Hopkins University. “This significant new collaboration with Bloomberg Philanthropies and NYSCF moves us ever closer to that aim as we join together our far-reaching research capacities to advance knowledge and deliver better health outcomes for populations and people around the world.”
The collaboration will also establish an unprecedented cache of human disease models available to researchers worldwide—thus promoting the real-world application of precision medicine and driving a new paradigm for understanding and improving the approach to human disease.
“Bloomberg Philanthropies’ mission is to ensure better, longer lives for the greatest number of people,” says Michael R. Bloomberg, MBA, founder of Bloomberg LP and Bloomberg Philanthropies. “For years, Johns Hopkins University and the New York Stem Cell Foundation have shared that mission—and we’re honored to deepen our partnerships with them as they explore new, innovative ways to save lives through the application of precision medicine.”
Diseases manifest themselves differently in different patients. To understand the basis of such differences and to tailor treatments for specific patients, researchers need more accurate biological tools. Stem cell models provide a ‘biological avatar’ of the patient from which they were created, allowing scientists and clinicians to better understand, define, and account for differences among individual patients and groups of patients.
The new initiative will use induced pluripotent stem cells to study disease characteristics in subgroups of patients, identifying markers that lead to varying disease manifestations. By examining stem cells from seemingly similar patients with different forms of multiple sclerosis, for example, researchers may be better able to understand the full range of the disease’s mechanisms and pathways.
The Johns Hopkins precision medicine initiative currently includes 16 disease-specific centers of excellence; the university is now working to develop 50 more such centers within the next 5 years. Johns Hopkins believes that advances in the study and application of precision medicine have the potential to transform the diagnosis and management of many diseases. A condition that is now categorized as a single disease may actually be multiple diseases that display similar symptoms but require quite different therapies. Using a wide range of data sources, precision medicine seeks to elucidate such differences more fully, so that doctors can treat patients with precisely targeted therapies. At Johns Hopkins, dozens of researchers are bringing this idea to reality across a spectrum of debilitating and life-altering diseases.
For the new collaboration, the process will begin with the full consent of patients in Hopkins centers of excellence who wish to participate. Their biological samples will be collected by the NYSCF Research Institute, where scientists will create stem cell models of disease using the NYSCF global stem cell array, the world’s first end-to-end automated system for generating human stem cells in a parallel, highly controlled process. Integrating robotics and machine learning, NYSCF’s technology reprograms skin or blood cells into stem cells, differentiates them into disease-relevant cell types, and performs genome editing to unravel the genetic basis of disease.
“The NYSCF Research Institute has invented and scaled the most advanced methods of human cell manipulation, which are critical for studying disease at the level of the individual patient,” explains Susan L. Solomon, JD, CEO of NYSCF. “By combining our capabilities with Johns Hopkins’ extensive clinical data and expertise, we will be able to develop effective, personalized therapies for patients suffering from diseases with a high unmet need.”
The stem cells generated by NYSCF will be used to research and drive effective therapeutic and diagnostic development for a wide range of diseases, including Alzheimer’s disease, chronic renal failure, multiple sclerosis, and cancers of the bladder, breast, lung, pancreas, and prostate. The stem cell lines will reside in the NYSCF repository and serve as a resource in perpetuity for the disease research community. The collection will enable scientists to develop unprecedented insights into the biochemical and genetic mechanisms underlying different diseases and their subtypes, thereby illuminating avenues for effective, tailored interventions.
“Stem cell science holds enormous potential for the treatment of a wide range of diseases,” says Paul B. Rothman, MD, dean of the School of Medicine and CEO of Johns Hopkins Medicine. “By combining this approach with Johns Hopkins’ groundbreaking work on precision medicine, we are creating a scientific powerhouse that will help us advance medicine and science at an even faster pace. I am excited to see the discoveries and innovations that will be produced by this collaboration.”
For further information, visit the New York Stem Cell Foundation Research Institute.
Featured image: Stem cells. Three-dimensional illustration © Katerynakon courtesy Dreamstime (ID 123305721).
