Featured Research

from universities, journals, and other organizations

Gene therapy plus chemotherapy ups odds for success in treating 'bubble boy' disease

Date:
September 14, 2010
Source:
American Society of Gene & Cell Therapy
Summary:
Gene therapy combined with chemotherapy may lead to immune system improvement in patients with 'bubble boy' disease, according to a new study.

Gene therapy combined with chemotherapy may lead to immune system improvement in patients with bubble boy disease, according to a study to be presented at the American Society of Gene & Cell Therapy 13th Annual Meeting.

Related Articles


Beginning in 2001, researchers from the University of California Los Angeles and the National Human Genome Research Institute, a division of the National Institutes of Health, treated 10 patients with adenosine deaminase severe combined immunodeficiency (ADA-SCID), commonly referred to as bubble boy disease.

Four patients received ongoing enzyme replacement while six patients had their enzyme replacement therapy stopped and received low doses of chemotherapy, which reduces bone marrow density to create room for the transplanted cells to multiply. Fifty percent of the latter group demonstrated a restored immune response that parallels normal levels. The remaining patients show some independent production of the adenosine deaminase enzyme, but not enough to elicit independent immune responses.

"This trial extends prior findings and shows that ADA gene transfer can lead to restoration of a functionally protective immune system," said Donald Kohn, MD, the clinical trial sponsor and principal investigator at UCLA. "Because our trial was done in two phases, we can directly compare the effects of these approaches and show that withholding the enzyme and giving pre-transplant chemotherapy is markedly more effective."

Patients with ADA-SCID lack the enzyme adenosine deaminase and, as a result, have little or no immune system, leaving them vulnerable to infection and illness. ADA-SCID is one of the most promising conditions for treatment with gene therapy and has been the source of early successes in the field. Earlier trials in London and Milan have also showed restored immunity in patients treated with the gene therapy protocol.

A Phase II trial on the treatment is underway; three patients have been treated thus far. Research also continues into more efficient vectors for gene transfer, which scientists hope will result in a quicker, more robust immune response.


Story Source:

The above story is based on materials provided by American Society of Gene & Cell Therapy. Note: Materials may be edited for content and length.


Cite This Page:

American Society of Gene & Cell Therapy. "Gene therapy plus chemotherapy ups odds for success in treating 'bubble boy' disease." ScienceDaily. ScienceDaily, 14 September 2010. <www.sciencedaily.com/releases/2010/05/100524105403.htm>.
American Society of Gene & Cell Therapy. (2010, September 14). Gene therapy plus chemotherapy ups odds for success in treating 'bubble boy' disease. ScienceDaily. Retrieved October 24, 2014 from www.sciencedaily.com/releases/2010/05/100524105403.htm
American Society of Gene & Cell Therapy. "Gene therapy plus chemotherapy ups odds for success in treating 'bubble boy' disease." ScienceDaily. www.sciencedaily.com/releases/2010/05/100524105403.htm (accessed October 24, 2014).

Share This



More Health & Medicine News

Friday, October 24, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

IKEA Desk Converts From Standing to Sitting With One Button

IKEA Desk Converts From Standing to Sitting With One Button

Buzz60 (Oct. 24, 2014) IKEA is out with a new convertible desk that can convert from a sitting desk to a standing one with just the push of a button. Jen Markham explains. Video provided by Buzz60
Powered by NewsLook.com
Ebola Protective Suits Being Made in China

Ebola Protective Suits Being Made in China

AFP (Oct. 24, 2014) A factory in China is busy making Ebola protective suits for healthcare workers and others fighting the spread of the virus. Duration: 00:38 Video provided by AFP
Powered by NewsLook.com
WHO: Millions of Ebola Vaccine Doses by 2015

WHO: Millions of Ebola Vaccine Doses by 2015

AP (Oct. 24, 2014) The World Health Organization said on Friday that millions of doses of two experimental Ebola vaccines could be ready for use in 2015 and five more experimental vaccines would start being tested in March. (Oct. 24) Video provided by AP
Powered by NewsLook.com
Doctor in NYC Quarantined With Ebola

Doctor in NYC Quarantined With Ebola

AP (Oct. 24, 2014) An emergency room doctor who recently returned to the city after treating Ebola patients in West Africa has tested positive for the virus. He's quarantined in a hospital. (Oct. 24) Video provided by AP
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:

Strange & Offbeat Stories


Health & Medicine

Mind & Brain

Living & Well

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