UConn Team Awarded Nearly $3M to Study Bone Loss in Sickle Cell

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by Steve Bryson, PhD |

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To mark World Sickle Cell Day, Marja Hurley, a University of Connecticut (UConn) physician-researcher, has been awarded nearly $3 million in research funding to investigate bone loss in people with sickle cell disease (SCD).

A four-year $2.21 million endowment comes from the National Institutes of Health (NIH) to Hurley as the principal investigator, alongside university co-investigators Liping Xiao, MD, PhD, an assistant professor of medicine and psychiatry, and Siu-Pok Yee, PhD, an associate professor of cell biology.

A second, three-year $535,000 grant was provided by Global Blood Therapeutics (GBT), which developed and markets the approved SCD oral therapy Oxbryta (voxelotor).

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“Our hope is that we can develop a useful therapy to prevent bone loss in sickle cell disease patients in the very near future,” Hurley, MD, a professor of medicine and orthopedic surgery at UConn Health, said in a university press release.

SCD is an inherited disorder characterized by the presence of abnormally shaped, sickled red blood cells, which can block smaller blood vessels and blood flow, triggering painful episodes called vaso-occlusive crises (VOCs). VOCs can lead to infections and anemia, a deficiency in oxygen-carrying red blood cells, among other risks.

Sickled red blood cells can also give rise to problems in the bone marrow, resulting in a damaging lack of blood flow to bone tissue. This can increase the risk of fractures, as well as conditions such as osteoporosis — a bone disease that causes bones to weaken and become more prone to fractures. However, research is lacking into bone loss in SCD.

“Little is known why a high prevalence of sickle cell patients also experience musculoskeletal frailty and brittle and broken bone complications such as osteoporosis,” UConn stated in its release.

With the NIH grant from its National Institute of Diabetes and Digestive and Kidney Diseases division, Hurley’s team will study how SCD leads to a loss of bone mineralization. The researchers will use mouse models to investigate the molecular mechanisms impacting fibroblast growth factor 23 (FGF23), a hormone that regulates phosphate, a key bone component.

The team aims to determine whether FGF23 contributes to SCD-related bone loss and if it can be prevented, supporting the development of new therapies for bone impairment associated with SCD.

GBT’s grant will further boost bone and muscle function research in SCD. Although Oxbryta effectively treats anemia, little is known about its impact on muscle strength and bone mineral density in animals or people. Hurley’s research will explore Oxbryta’s efficacy in treating SCD-related bone loss in mouse models.

“There is much needed intervention to further fuel scientific discoveries to not only curb the pain suffered by sickle cell patients but further enhance their overall musculoskeletal health and well-being,” Hurley said.