Featured Research

from universities, journals, and other organizations

Potential treatment for sickle cell disease

Date:
November 10, 2011
Source:
University of Michigan Health System
Summary:
Few options are available to prevent the painful episodes and organ damage that are common with sickle cell disease. But a new lab study has revealed a key trigger for producing healthy red blood cells that could lead to new treatments for sickle cell patients.

A University of Michigan Health System laboratory study reveals a key trigger for producing normal red blood cells that could lead to a new treatment for sickle cell disease.

The study, conducted in mice, appears in this week's early edition of the Proceedings of the National Academy of Sciences, and holds promise for preventing the painful episodes and organ damage that are common complications of sickle cell disease.

According to the U-M study, increasing the expression of the proteins, TR2 and TR4, more than doubled the level of fetal hemoglobin produced in sickle cell mice and reduced organ damage.

"The vast majority of sickle cell disease patients are diagnosed early in childhood when adult hemoglobin normally replaces fetal hemoglobin, but the severity of the disease can differ markedly, correlating most strongly with the level of fetal hemoglobin present in red cells," says pediatrician and lead study author Andrew D. Campbell, M.D., director of the Pediatric Comprehensive Sickle Cell Program at the U-M Comprehensive Cancer Center.

Sickle cell is an inherited blood disorder impacting millions of patients worldwide that causes normal red blood cells to change shape to a crescent moon.

The result is debilitating pain episodes, chronic organ damage and a significantly shortened life span.

Sickle cell disease occurs in 1 out of every 500 African American births and 1 out of every 36,000 Hispanic American births, according to the Sickle Cell Disease Association of America, Inc.

It's most common among those with an ancestry to Africa, South and Central America, the Caribbean islands, India, Saudi Arabia and Mediterranean countries such as Turkey, Greece and Italy.

But a small number of sickle cell patients are born with a high enough fetal hemoglobin level to moderate complications.

The study team, that included experts in hematology oncology, cell and developmental biology and pathology from the U-M and the University of Tsukuba, Japan, demonstrated a potential method for boosting the fetal hemoglobin levels by modulating TR2/TR4 expression.

"While the average fetal hemoglobin was 7.6 percent in the sickle cell mice, the TR2/TR4 treated sickle cell mice had an average fetal hemoglobin of 18.6 percent," says senior study author James Douglas Engel, Ph.D. , professor and chair of the U-M's Cell and Development Biology Department.

It's the first time specific proteins have been targeted to prevent a disease, he says.

Anemia and red blood cell turnover all improved within the TR2/TR4 treated mice. Additional studies, including clinical trials, would be required to determine if the method could help humans.

"Currently hydroxyurea is the only FDA approved drug known to increase the levels of fetal hemoglobin within sickle cell disease patients and a substantial number of patients do respond to it," says Campbell, the pediatric hematology oncology specialist. "But the long term consequences for hydroxyurea are unknown, especially in children."


Story Source:

The above story is based on materials provided by University of Michigan Health System. Note: Materials may be edited for content and length.


Journal Reference:

  1. A. D. Campbell, S. Cui, L. Shi, R. Urbonya, A. Mathias, K. Bradley, K. O. Bonsu, R. R. Douglas, B. Halford, L. Schmidt, D. Harro, D. Giacherio, K. Tanimoto, O. Tanabe, J. D. Engel. Forced TR2/TR4 expression in sickle cell disease mice confers enhanced fetal hemoglobin synthesis and alleviated disease phenotypes. Proceedings of the National Academy of Sciences, 2011; DOI: 10.1073/pnas.1104964108

Cite This Page:

University of Michigan Health System. "Potential treatment for sickle cell disease." ScienceDaily. ScienceDaily, 10 November 2011. <www.sciencedaily.com/releases/2011/11/111102082806.htm>.
University of Michigan Health System. (2011, November 10). Potential treatment for sickle cell disease. ScienceDaily. Retrieved July 28, 2014 from www.sciencedaily.com/releases/2011/11/111102082806.htm
University of Michigan Health System. "Potential treatment for sickle cell disease." ScienceDaily. www.sciencedaily.com/releases/2011/11/111102082806.htm (accessed July 28, 2014).

Share This




More Health & Medicine News

Monday, July 28, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Trees Could Save More Than 850 Lives Each Year

Trees Could Save More Than 850 Lives Each Year

Newsy (July 27, 2014) A national study conducted by the USDA Forest Service found that trees collectively save more than 850 lives on an annual basis. Video provided by Newsy
Powered by NewsLook.com
Google's Next Frontier: The Human Body

Google's Next Frontier: The Human Body

Newsy (July 27, 2014) Google is collecting genetic and molecular information to paint a picture of the perfectly healthy human. Video provided by Newsy
Powered by NewsLook.com
What's To Blame For Worst Ebola Outbreak In History?

What's To Blame For Worst Ebola Outbreak In History?

Newsy (July 27, 2014) A U.S. doctor has tested positive for the deadly Ebola virus, as the worst-ever outbreak continues to grow. Video provided by Newsy
Powered by NewsLook.com
Losing Sleep Leaves You Vulnerable To 'False Memories'

Losing Sleep Leaves You Vulnerable To 'False Memories'

Newsy (July 27, 2014) A new study shows sleep deprivation can make it harder for people to remember specific details of an event. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:
from the past week

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins