Navigation Links
Gene Therapy Might Treat 'Bubble Boy' Disease

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:
(Date:11/26/2015)... ... November 26, 2015 , ... ... Travel Representative. As a franchise owner, Somu now offers travelers, value and care ... wedding packages, private cruise sales, as well as, cabin upgrades and special amenities ...
(Date:11/26/2015)... ... November 26, 2015 , ... Jobs in hospital medical laboratories ... and offered by healthcare staffing agency Aureus Medical Group . These ... October 2015 among those searching for healthcare jobs through the company’s website, ...
(Date:11/26/2015)... Mass. (PRWEB) , ... November 26, 2015 , ... ... thorough second medical opinion process, participated in the 61st annual Employee Benefits Conference. ... Plans and took place Sunday, November 8th through Wednesday, November 11th, 2015. The ...
(Date:11/26/2015)... ... November 26, 2015 , ... Health-E-minds ... collaborated with Women’s Web – an online resource for Indian women looking ... and emotional well-being relationship, life balance, stress, professional development, and lifestyle. Health-E-minds ...
(Date:11/25/2015)... OAK BROOK, Ill. (PRWEB) , ... November 25, ... ... sometimes larger and potentially more aggressive than those found on mammography, according to ... cases MRI findings of additional cancers not seen on mammography may necessitate a ...
Breaking Medicine News(10 mins):
(Date:11/26/2015)... --> ... att använda SyMRI för att hitta optimal ... med multipel skleros (MS) eller hjärntumörmetastaser och ... för att kunna använda SyMRI Research Edition ... kan man generera flera konstrastbilder från en ...
(Date:11/26/2015)... 26, 2015 ... of the  "2016 Future Horizons and ... Drug Monitoring (TDM) Market: Supplier Shares, ... Opportunities"  report to their offering.  ... the addition of the  "2016 Future ...
(Date:11/26/2015)... , November 26, 2015 ... the "Self Administration of High Viscosity Drugs" ... ) has announced the addition of the ... report to their offering. --> Research ... addition of the "Self Administration of High ...
Breaking Medicine Technology: