CAMBRIDGE, Mass.--(BUSINESS WIRE)--Magenta Therapeutics, a biotechnology company developing therapeutics to improve and extend the use of curative bone marrow transplant for more patients, today announced the publication of research led by Jonathan Hoggatt, Ph.D., assistant professor of medicine at Harvard Medical School and the Massachusetts General Hospital (MGH) Cancer Center and Center for Transplantation Sciences, and a principal faculty member at Harvard Stem Cell Institute. Dr. Hoggatt is a scientific co-founder of Magenta.
The research demonstrates that a single administration of a chemokine, GROβ, in combination with plerixafor (also known as AMD3100) resulted in rapid mobilization of robust numbers of hematopoietic stem cells (HSCs) from the bone marrow and into the blood, which were then harvested for transplant in mouse models. Notably, HSCs obtained with GROβ and plerixafor mobilization resulted in faster engraftment and higher donor chimerism, two important factors for the success of a transplant, compared to cells obtained with the current mobilization standard of care, G-CSF. The article “Rapid Mobilization Reveals a Highly Engraftable Hematopoietic Stem Cell” was published in Cell online on December 7, 2017.
Now under exclusive license to Magenta from Harvard University’s Office of Technology Development, the technology described in this paper was co-developed by David Scadden, M.D., the Gerald and Darlene Jordan Professor of Medicine in Harvard’s Department of Stem Cell and Regenerative Biology, co-director of Harvard Stem Cell Institute, and Director of the Center for Regenerative Medicine at MGH. Dr. Scadden is also a scientific co-founder of Magenta.
“Magenta’s mission is to expand the curative power of bone marrow transplant to more patients. Improving the standard of care for mobilization of hematopoietic stem cells has the potential to improve both the process and outcomes of bone marrow transplant, helping extend the procedure to a broader range of diseases that could be cured,” said Michael Cooke, Ph.D., chief scientific officer, Magenta Therapeutics. “At Magenta we are building on this important research in mouse models by investigating the combination of GROβ and plerixafor in non-human primates. We look forward to presenting these primate data this weekend at the annual meeting of the American Society of Hematology (ASH).”
As part of their research, Dr. Hoggatt and colleagues analyzed a single-blind dose-escalating pilot study in healthy human volunteers to assess the tolerability, pharmacokinetics and pharmacodynamics following administration of single agent GROβ. The study showed that GROβ was well tolerated.
“Mobilization of HSCs with G-CSF involves repeated injections and often requires five or more days of treatment and multiple outpatient visits. G-CSF is also associated with bone pain, nausea, headache and fatigue; and is contraindicated in some difficult-to-mobilize patient populations, such as patients with sickle cell disease,” said Dr. Hoggatt. “In this report, we show that a single administration of GROβ with plerixafor resulted in peak mobilization of stem cells within 15 minutes in mouse models. Moreover, mice transplanted with the GROβ-mobilized stem cells experienced robust short- and long-term engraftment, with faster recovery of neutrophils and platelets compared to mice transplanted with stem cells mobilized with G-CSF. These data suggest that the combination of GROβ with plerixafor has the potential to become a more effective and more patient-friendly approach than the current standard of care. We may be able to turn a one-week process into an optimal single day process.”
About Bone Marrow Transplant
Healthy bone marrow stem cells and the blood cells they form are crucial for survival, but certain diseases can affect the bone marrow, interfering with its ability to function properly. A bone marrow transplant is a process to replace unhealthy bone marrow with healthy bone marrow stem cells. Bone marrow transplant can save the lives of patients with blood cancers and genetic diseases and is a potential cure for patients with severe, refractory autoimmune diseases. However, the high risks, toxic side effects and complexity of the procedure currently prevent many patients from being able to benefit.
About Magenta Therapeutics
Magenta Therapeutics is a biotechnology company developing therapeutics to revolutionize bone marrow transplant for patients with autoimmune diseases, blood cancers and genetic diseases. By creating a platform focused on critical areas of transplant medicine, Magenta Therapeutics is pioneering an integrated approach to extend the curative power of bone marrow transplant to more patients. Founded by internationally recognized leaders in bone marrow transplant medicine, Magenta Therapeutics was launched in 2016 by Third Rock Ventures and Atlas Venture and is headquartered in Cambridge, Mass. For more information, please visit www.magentatx.com.