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A look at emerging sickle cell disease therapies

Teams at the University of Washington School of Pharmacy and Fred Hutch will model the clinical and economic burden of sickle cell disease and the potential benefits of emerging therapies
microscopic image of red blood cells
A microscopic image of red blood cells from a boy with sickle cell disease. Image courtesy of CDC / Sickle Cell Foundation of Georgia: Jackie George, Beverly Sinclair

The National Institutes of Health’s National Heart, Lung, and Blood Institute funded a unique collaborative — The Sickle Cell Clinical and Economic Impact Consortium — that brings together The Comparative Health Outcomes, Policy, and Economics Institute at the University of Washington School of Pharmacy, Fred Hutchinson Cancer Research Center, the NHLBI, and The Emmes Company. 

The collaborative enables the two teams at The CHOICE Institute and Fred Hutch’s Hutchinson Institute for Cancer Outcomes Research to develop models that will give insight into the clinical and economic benefits of cures for sickle cell disease over the lifetime of the patients. The goal is to clarify the potential long-term benefits of genetic therapies for this genetic disease. Results and public versions of the models will be made publicly available.

About sickle cell disease

About 100,000 people in the U.S. are affected by sickle cell disease (meaning they have two sickle cell genes, or have one and another abnormal gene). A disproportionate percentage of these people are black or Hispanic. About one in 13 African Americans have the sickle cell trait (one sickle cell gene) and are at risk for having a child with the disease. The inherited blood disorder leads to many medical complications and shortens the lifespans of people affected by about 20 to 30 years.

The disease can lead to episodes of excruciating pain, stroke, heart, lung and kidney damage. The trauma of the pain and repeat hospitalizations for young children can cause stigmatization, depression and social isolation. For people living with the disease, early intervention and access to care are vital for their long-term prognosis — physically and psychologically.

“Our findings can inform the development strategies to use gene therapy to treat patients with severe sickle cell disease with the objectives of reducing disease complications, extending survival and improving quality of life. Our team is very excited to apply our expertise in economic modeling to address these important issues.”

— Dr. Joshua Roth, assistant member, Hutchinson Institute for Cancer Outcomes Research, Fred Hutchinson Cancer Research Center

Historically, treatments have decreased the frequency and severity of complications. More recently, some patients have been cured with bone marrow transplantation, but many do not have well-matched marrow donors, and there can be serious complications. There are some promising new curative therapies on the horizon, which make the consortium’s work timely and valuable. 

The Sickle Cell Clinical and Economic Impact Consortium’s work

With detailed feedback from all consortium members and a variety of stakeholder inputs, the teams will develop simulation models for patients from the age of diagnosis over their lifetimes and investigate the state-specific estimates of comorbidities and complications, quality of life, health care utilization and costs. Models will be informed using extensive literature review and datasets from both public and private sources, which will likely make these models the most comprehensive ever to be built around sickle cell disease in the United States. The potential impact of different types of curative therapies will be studied.

portrait of Dr. Joshua Roth
Dr. Joshua Roth is a faculty member in the Hutchinson Institute for Cancer Outcomes Research at Fred Hutch. Photo: Fred Hutch file

The researchers at the CHOICE Institute and Fred Hutch are global leaders in disease modeling, particularly for genetic diseases. The UW School of Pharmacy team is led by Principal Investigator and CHOICE Director Dr. Anirban Basu, with CHOICE Professor Dr. Beth Devine and Seattle Children’s Dr. M.A. Bender serving as co-investigators. Fred Hutch's team is led by Dr. Joshua Roth  along with co-investigator and Hutchinson Institute for Cancer Outcomes Research Director Dr. Scott Ramsey. Both groups have a long history of collaborative work.

“Our findings can inform the development strategies to use gene therapy to treat patients with severe sickle cell disease with the objectives of reducing disease complications, extending survival and improving quality of life,” said Roth. “Our team is very excited to apply our expertise in economic modeling to address these important issues.”

The collaborative agreement (OTA 1OT3HL152448) is funded by the NHLBI for a potential duration of three years of funding totaling $3 million for the UW team.

—Adapted from a UW School of Pharmacy announcement

Read more about Fred Hutch achievements and accolades.

Last Modified, November 08, 2019