Navigation Links
Gene Therapy Might Treat 'Bubble Boy' Disease
Date:5/21/2010

Chemo clears way for transplanted cells to help children develop immune systems

FRIDAY, May 21 (HealthDay News) -- Scientists report that they've successfully used gene therapy to treat a small number of patients with the condition known as "bubble boy" disease, which robs children of the ability to fight off germs.

The research is preliminary, and another phase of testing is needed before the therapy can get federal approval. Also, the treatment only appears to work in patients with a particular strain of the disease.

Even so, the findings highlight the fact that the disease is becoming easier to treat and the prognosis is improving for these children, said lead investigator Dr. Donald B. Kohn.

"We're talking about 70 to 90 percent surviving and doing well, and we're hoping to get that even higher as gene therapy gets more effective," said Kohn, a pediatric bone-marrow transplant doctor at the University of California at Los Angeles David Geffen School of Medicine.

When they're born, babies have natural resistance to disease, thanks to immunity that they inherit from their mothers. Over time, if things go well, they develop immune systems of their own. But in some cases the immune systems don't develop properly and the children become especially vulnerable to disease.

The condition is rare, but it became widely known in the 1980s because of the case of the "Bubble Boy," a child named David Vetter who lived in a plastic bubble to avoid being infected by germs. He died at the age of 12, and his treatment remains controversial to this day.

Currently, children with the different types of the immune deficiency condition are treated with stem-cell transplants and gene therapy.

In the new study, researchers treated 10 children who had a form of the condition called adenosine deaminase severe combined immunodeficiency that affects 20 percent of those with bubble boy disease.

Researchers, who began the study in 2001, assigned four patients to receive regular treatment with enzyme replacement therapy. Another six stopped that therapy and received chemotherapy designed to create room in their bone marrow for transplanted cells.

"It would be like if you're in a garden with a high density of plants, and you want to put some new plants in. The best chance for them to grow would be to remove some of the old plants," explained Dr. Mark Kay, head of the gene therapy program at Stanford University School of Medicine.

Researchers hoped the transplanted cells would create a new immune system and, in half of the six patients, they did just that.

The treatment gives the patients a "key missing piece of genetic information" so their bodies can create a normal immune system, Kohn said. And the cells come from the patient's own body so there should be less risk that the body will reject the cells or vice versa, he explained.

The study results came in a phase 1 trial, the first of three study phases that treatments must undergo to get federal approval. The second phase of research has already begun.

The treatment is costly, Kohn noted, requiring months of hospitalization.

Kay said the gene therapy treatment may become the standard way to treat bubble boy disease. It could, he said, allow children to undergo just one treatment -- a "lifelong cure" -- instead of repeatedly taking the enzyme treatment.

The study is scheduled to be released Friday at the annual meeting of the American Society of Gene & Cell Therapy, in Washington D.C.

More information

The Nemours Foundation has more about immune deficiency.



SOURCES: Donald B. Kohn, M.D., professor, department of microbiology, immunology and molecular genetics and department of pediatrics, University of California at Los Angeles David Geffen School of Medicine; Mark Kay, M.D., Ph.D., professor, pediatrics and genetics, Stanford University, Palo Alto, Calif.; May 21, 2010, presentation, American Society of Gene & Cell Therapy annual meeting, Washington D.C.


'/>"/>
Copyright©2010 ScoutNews,LLC.
All rights reserved

Related medicine news :

1. Gene Therapy Shows Promise for Muscular Dystrophy
2. New Laser Technology Combined with Manual Physical Therapy Offer Pain Relief to Chronic Pain Sufferers
3. For Depression, Phone Therapy May Be an Answer
4. Behavior therapy effective in reducing tics in children with Tourette syndrome, study finds
5. What is the role of focal therapy in low-risk prostate cancer?
6. Well-tolerated radiotherapy provides longer life to patients with recurrent brain cancer
7. Combination therapy targets stubborn leukemia stem cells
8. Study demonstrates art therapys effectiveness in pediatric asthma
9. Physical Therapy Online Continuing Education Courses Undergo an Inevitable Evolution
10. Complimentary Injury Screenings Available at All AthletiCo Physical and Occupational Therapy Centers
11. Phase II study of an oral therapy for Gaucher disease yields positive results
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:8/23/2017)... ... August 23, 2017 , ... TSC Advantage has been ... Risk Management Solution Innovations and Security Solution for Government Innovations. Both gold awards ... and its DHS SAFETY Act-designated enterprise security assessment approach. , The ninth annual ...
(Date:8/23/2017)... ... August 23, 2017 , ... The non-profit ... holding an inaugural State of the Science Symposium in partnership with the Global ... 2017. , This symposium provides a forum for global leaders in human nutrition ...
(Date:8/22/2017)... ... August 22, 2017 , ... The Women’s Choice Award, a ... Migraine Relief with the 2017 Women’s Choice Award. The identification by women of an ... out of 4 migraine sufferers are women. In a survey taken by the Women’s ...
(Date:8/22/2017)... ... August 22, 2017 , ... Western ... spoke at a popular international aesthetics conference for medical professionals about the positive ... patients’ health and his growing practice. , Dr. George K. Ibrahim ...
(Date:8/22/2017)... ... August 22, 2017 , ... Each of the past six years, Lightning Labels has sent ... these labels and stickers, demonstrating the variety and creativity of their designs. Submissions this year ... came in. Now, it's time to announce the winners of the sixth annual Photo Contest, ...
Breaking Medicine News(10 mins):
(Date:8/7/2017)... Diplomat Pharmacy, Inc. (NYSE: DPLO ) ... president, effective Aug. 7, 2017. ... other interests and will serve as president emeritus during a ... us in multiple leadership roles since he joined Diplomat with ... has provided decisive, strategic leadership which continues to benefit our ...
(Date:8/7/2017)... , Aug. 7, 2017  Endo International plc (NASDAQ: ... agreements to resolve virtually all known U.S. mesh product ... to resolve the known remaining U.S. claims at reasonable ... beginning in the fourth quarter of 2017 and continuing ... its second quarter 2017 results, the Company intends to ...
(Date:8/4/2017)... 3, 2017  Agragen, LLC, a ... the biopharmaceutical, nutraceutical, and aquacultural feed sectors, announces ... lead drug candidates, AGR131.  This drug is designed ... the blood of patients suffering from inflammatory conditions ... disease. Biological ...
Breaking Medicine Technology: