They were born without a working germ-fighting system, every infection a threat to their lives. Now eight babies with "bubble boy disease" have had it fixed by a gene therapy made from one of the immune system's worst enemies HIV, the virus that causes AIDS.
Astudyout Wednesday details how scientists turned this enemy virus into a savior, altering it so it couldn't cause disease and then using it to deliver a gene the boys lacked.
"This therapy has cured the patients," although it will take more time to see if it's a permanent fix, said Dr. Ewelina Mamcarz, one of the study leaders at St. Jude Children's Research Hospital in Memphis.
Omarion Jordan, who turns 1 later this month, had the therapy in December to treat severe combined immunodeficiency syndrome, or SCID.
"For a long time we didn't know what was wrong with him. He just kept getting these infections," said his mother, Kristin Simpson. Learning that he had SCID "was just heartbreaking ... I didn't know what was going to happen to him."
Omarion now has a normal immune system. "He's like a normal, healthy baby," Simpson said. "I think it's amazing."
Study results were published by the New England Journal of Medicine. The treatment was pioneered by a St. Jude doctor who recently died, Brian Sorrentino.
SCID is caused by a genetic flaw that keeps the bone marrow from making effective versions of blood cells that comprise the immune system. It affects 1 in 200,000 newborns, almost exclusively males. Without treatment, it often kills in the first year or two of life.
"A simple infection like the common cold could be fatal," Mamcarz said.
The nickname "bubble boy disease" comes from a famous case in the 1970s a Texas boy who lived for 12 years in a protective plastic bubble to isolate him from germs. A bone marrow transplant from a genetically matched sibling can cure SCID, but most people lack a suitable donor. Transplants also are medically risky the Texas boy died after one.
Doctors think gene therapy could be a solution. It involves removing some of a patient's blood cells, using the modified HIV to insert the missing gene, and returning the cells through an IV. Before getting their cells back, patients are given a drug to destroy some of their marrow so the modified cells have more room to grow.
When doctors first tried it 20 years ago, the treatment had unintended effects on other genes, and some patients later developed leukemia. The new therapy has safeguards to lower that risk.
A small study of older children suggested it was safe. The new study tried it in infants, and doctors are reporting on the first eight who were treated at St. Jude and at UCSF Benioff Children's Hospital San Francisco.
Within a few months, normal levels of healthy immune system cells developed in seven boys. The eighth needed a second dose of gene therapy but now is well, too. Six to 24 months after treatment, all eight are making all the cell types needed to fight infections, and some have successfully received vaccines to further boost their immunity to disease.
No serious or lasting side effects occurred.
Omarion is the 10th boy treated in the study, which is ongoing. It's sponsored by the American Lebanese Syrian Associated Charities, the California Institute of Regenerative Medicine, the Assisi Foundation of Memphis and the federal government.
"So far it really looks good," but patients will have to be studied to see if the results last, said Dr. Anthony Fauci, head of the National Institute of Allergy and Infectious Diseases, which helped develop the treatment. "To me, this looks promising."
Rights to it have been licensed by St. Jude to Mustang Bio. Doctors say they have no estimate on what it might cost if it does become an approved treatment.
A similar technique harnessing a modified version of HIV is also being studied as a possible cure for sickle cell anemia, CBS News chief medical correspondent Dr. Jon LaPook reports. In a clinical trial at the National Institutes of Health, nine adults with sickle cell anemia have undergone the gene therapy. So far, all are responding well.
View original post here:
Gene therapy might be a cure for "bubble boy disease ...
- Faulty Circuits (preview) [Last Updated On: April 7th, 2010] [Originally Added On: April 7th, 2010]
- Faulty Circuits (preview) [Last Updated On: April 7th, 2010] [Originally Added On: April 7th, 2010]
- Rare flowers and common herbal supplements get unmasked with plant DNA barcoding [Last Updated On: April 20th, 2010] [Originally Added On: April 20th, 2010]
- Rare flowers and common herbal supplements get unmasked with plant DNA barcoding [Last Updated On: April 20th, 2010] [Originally Added On: April 20th, 2010]
- Biomarker Studies Could Realize Goal of More Effective and Personalized Cancer Medicine [Last Updated On: April 27th, 2010] [Originally Added On: April 27th, 2010]
- Biomarker Studies Could Realize Goal of More Effective and Personalized Cancer Medicine [Last Updated On: April 27th, 2010] [Originally Added On: April 27th, 2010]
- Schizophrenia shares genetic links with autism, genome study shows [Last Updated On: May 12th, 2010] [Originally Added On: May 12th, 2010]
- Schizophrenia shares genetic links with autism, genome study shows [Last Updated On: May 12th, 2010] [Originally Added On: May 12th, 2010]
- Alzheimer's: Forestalling the Darkness with New Approaches (preview) [Last Updated On: May 28th, 2010] [Originally Added On: May 28th, 2010]
- Alzheimer's: Forestalling the Darkness with New Approaches (preview) [Last Updated On: May 28th, 2010] [Originally Added On: May 28th, 2010]
- Large-Scale Autism Study Reveals Disorder's Genetic Complexity [Last Updated On: June 12th, 2010] [Originally Added On: June 12th, 2010]
- Large-Scale Autism Study Reveals Disorder's Genetic Complexity [Last Updated On: June 12th, 2010] [Originally Added On: June 12th, 2010]
- Cancer Therapy Goes Viral: Progress Is Made Tackling Tumors with Viruses [Last Updated On: June 24th, 2010] [Originally Added On: June 24th, 2010]
- Cancer Therapy Goes Viral: Progress Is Made Tackling Tumors with Viruses [Last Updated On: June 24th, 2010] [Originally Added On: June 24th, 2010]
- Vaccines Derived from Patients' Tumor Cells Are Individualizing Cancer Treatment [Last Updated On: June 26th, 2010] [Originally Added On: June 26th, 2010]
- Vaccines Derived from Patients' Tumor Cells Are Individualizing Cancer Treatment [Last Updated On: June 26th, 2010] [Originally Added On: June 26th, 2010]
- A genome story: 10th anniversary commentary by Francis Collins [Last Updated On: June 29th, 2010] [Originally Added On: June 29th, 2010]
- A genome story: 10th anniversary commentary by Francis Collins [Last Updated On: June 29th, 2010] [Originally Added On: June 29th, 2010]
- Hair Trigger: How a Cell's Primary Cilium Functions as a Molecular Antenna [Last Updated On: June 30th, 2010] [Originally Added On: June 30th, 2010]
- Hair Trigger: How a Cell's Primary Cilium Functions as a Molecular Antenna [Last Updated On: June 30th, 2010] [Originally Added On: June 30th, 2010]
- DNA Drugs Come of Age (preview) [Last Updated On: July 16th, 2010] [Originally Added On: July 16th, 2010]
- DNA Drugs Come of Age (preview) [Last Updated On: July 16th, 2010] [Originally Added On: July 16th, 2010]
- 2 Genes Linked to Embryonic Brain Impairment in Down's Syndrome [Last Updated On: July 22nd, 2010] [Originally Added On: July 22nd, 2010]
- 2 Genes Linked to Embryonic Brain Impairment in Down's Syndrome [Last Updated On: July 22nd, 2010] [Originally Added On: July 22nd, 2010]
- Stem Cells from Reprogrammed Adult Cells Found to Bring Along Genetic Defects of Their Donors [Last Updated On: October 11th, 2010] [Originally Added On: October 11th, 2010]
- Was Darwin a Punk? A Q&A with Punker-Paleontologist Greg Graffin [Last Updated On: October 11th, 2010] [Originally Added On: October 11th, 2010]
- Parkinsonian Power Failure: Neuron Degeneration May Be Caused by a Cellular Energy System Breakdown [Last Updated On: October 11th, 2010] [Originally Added On: October 11th, 2010]
- Was Darwin a Punk? A Q&A with Punker-Paleontologist Greg Graffin [Last Updated On: October 11th, 2010] [Originally Added On: October 11th, 2010]
- Desperation Drives Parents to Dubious Autism Treatments (preview) [Last Updated On: October 13th, 2010] [Originally Added On: October 13th, 2010]
- Revolution Postponed: Why the Human Genome Project Has Been Disappointing (preview) [Last Updated On: October 26th, 2010] [Originally Added On: October 26th, 2010]
- Controlling the Brain with Light (preview) [Last Updated On: October 26th, 2010] [Originally Added On: October 26th, 2010]
- Optogenetics: Controlling the Brain with Light [Extended Version] [Last Updated On: October 26th, 2010] [Originally Added On: October 26th, 2010]
- Clear New Insights into the Genetics of Depression [Last Updated On: November 7th, 2010] [Originally Added On: November 7th, 2010]
- TEDMED 2010: Technology and the people [Last Updated On: November 7th, 2010] [Originally Added On: November 7th, 2010]
- Bacteria, the anti-cancer soldier [Last Updated On: November 7th, 2010] [Originally Added On: November 7th, 2010]
- Clear New Insights into the Genetics of Depression [Last Updated On: November 7th, 2010] [Originally Added On: November 7th, 2010]
- TEDMED 2010: Technology and the people [Last Updated On: November 7th, 2010] [Originally Added On: November 7th, 2010]
- Bacteria, the anti-cancer soldier [Last Updated On: November 7th, 2010] [Originally Added On: November 7th, 2010]
- Scientific regress: When science goes backward [Last Updated On: November 29th, 2010] [Originally Added On: November 29th, 2010]
- Can You Live Forever? Maybe Not--But You Can Have Fun Trying [Last Updated On: December 26th, 2010] [Originally Added On: December 26th, 2010]
- How to Fix the Obesity Crisis (preview) [Last Updated On: February 14th, 2011] [Originally Added On: February 14th, 2011]
- Personalizing cancer medicine [Last Updated On: February 14th, 2011] [Originally Added On: February 14th, 2011]
- New Salmonella strain delivers gene-based therapy to fight virus in mice [Last Updated On: February 14th, 2011] [Originally Added On: February 14th, 2011]
- How to Fix the Obesity Crisis (preview) [Last Updated On: February 14th, 2011] [Originally Added On: February 14th, 2011]
- Personalizing cancer medicine [Last Updated On: February 14th, 2011] [Originally Added On: February 14th, 2011]
- New Salmonella strain delivers gene-based therapy to fight virus in mice [Last Updated On: February 14th, 2011] [Originally Added On: February 14th, 2011]
- Steps toward a Bionic Eye [Last Updated On: February 20th, 2011] [Originally Added On: February 20th, 2011]
- Steps toward a Bionic Eye [Last Updated On: February 20th, 2011] [Originally Added On: February 20th, 2011]
- Giving HIV a Poor Reception: New AIDS Treatment Tinkers with Immune Cell Genes [Last Updated On: March 6th, 2011] [Originally Added On: March 6th, 2011]
- Giving HIV a Poor Reception: New AIDS Treatment Tinkers with Immune Cell Genes [Last Updated On: March 6th, 2011] [Originally Added On: March 6th, 2011]
- Smaller, cheaper, faster: Does Moore's law apply to solar cells? [Last Updated On: March 27th, 2011] [Originally Added On: March 27th, 2011]
- Smaller, cheaper, faster: Does Moore's law apply to solar cells? [Last Updated On: March 27th, 2011] [Originally Added On: March 27th, 2011]
- New Drugs for Hepatitis C on the Horizon [Last Updated On: April 10th, 2011] [Originally Added On: April 10th, 2011]
- Can we capture all of the world's carbon emissions? [Last Updated On: April 10th, 2011] [Originally Added On: April 10th, 2011]
- New Drugs for Hepatitis C on the Horizon [Last Updated On: April 10th, 2011] [Originally Added On: April 10th, 2011]
- Can we capture all of the world's carbon emissions? [Last Updated On: April 10th, 2011] [Originally Added On: April 10th, 2011]
- Drug-resistant genes found in cholera and dysentery strains in New Delhi water supply [Last Updated On: May 1st, 2011] [Originally Added On: May 1st, 2011]
- Fast Track to Vaccines: How Systems Biology Speeds Drug Development (preview) [Last Updated On: May 1st, 2011] [Originally Added On: May 1st, 2011]
- Drug-resistant genes found in cholera and dysentery strains in New Delhi water supply [Last Updated On: May 1st, 2011] [Originally Added On: May 1st, 2011]
- Fast Track to Vaccines: How Systems Biology Speeds Drug Development (preview) [Last Updated On: May 1st, 2011] [Originally Added On: May 1st, 2011]
- Autism's Tangled Genetics Full of Rare and Varied Mutations [Last Updated On: June 19th, 2011] [Originally Added On: June 19th, 2011]
- A New Look at Obsessive-Compulsive Disorder (preview) [Last Updated On: June 19th, 2011] [Originally Added On: June 19th, 2011]
- Autism's Tangled Genetics Full of Rare and Varied Mutations [Last Updated On: June 19th, 2011] [Originally Added On: June 19th, 2011]
- A New Look at Obsessive-Compulsive Disorder (preview) [Last Updated On: June 19th, 2011] [Originally Added On: June 19th, 2011]
- Close Encounters of Science and Medicine [Last Updated On: July 3rd, 2011] [Originally Added On: July 3rd, 2011]
- Close Encounters of Science and Medicine [Last Updated On: July 3rd, 2011] [Originally Added On: July 3rd, 2011]
- New Report Details Uphill Battle to Solve the U.S.'s Pain Problem [Last Updated On: July 24th, 2011] [Originally Added On: July 24th, 2011]
- New Report Details Uphill Battle to Solve the U.S.'s Pain Problem [Last Updated On: July 24th, 2011] [Originally Added On: July 24th, 2011]
- A Breath of Fresh Air: New Hope for Cystic Fibrosis Treatment (preview) [Last Updated On: August 7th, 2011] [Originally Added On: August 7th, 2011]
- A Breath of Fresh Air: New Hope for Cystic Fibrosis Treatment (preview) [Last Updated On: August 7th, 2011] [Originally Added On: August 7th, 2011]
- Sickle Cell Anemia: Stem Cell Gene Therapy - Donald Kohn [Last Updated On: August 18th, 2011] [Originally Added On: August 18th, 2011]
- Sickle Cell Anemia: Stem Cell Gene Therapy - A Patient's Perspective [Last Updated On: October 8th, 2011] [Originally Added On: October 8th, 2011]
- Gene therapy improves stem cell transplantation - Video [Last Updated On: October 14th, 2011] [Originally Added On: October 14th, 2011]
- THE NEW MORGELLONS HAIR - Video [Last Updated On: October 14th, 2011] [Originally Added On: October 14th, 2011]
- Studying Mental Illness in a Dish [Last Updated On: November 13th, 2011] [Originally Added On: November 13th, 2011]
- The Puzzle of Pancreatic Cancer: How Steve Jobs Did Not Beat the Oddsbut Nobel Winner Ralph Steinman Did [Last Updated On: November 13th, 2011] [Originally Added On: November 13th, 2011]
- Did Alternative Medicine Extend or Abbreviate Steve Jobs's Life? [Last Updated On: November 13th, 2011] [Originally Added On: November 13th, 2011]
- Calendar: MIND Events in November and December [Last Updated On: November 13th, 2011] [Originally Added On: November 13th, 2011]
- Studying Mental Illness in a Dish [Last Updated On: November 13th, 2011] [Originally Added On: November 13th, 2011]
- The Puzzle of Pancreatic Cancer: How Steve Jobs Did Not Beat the Odds?but Nobel Winner Ralph Steinman Did [Last Updated On: November 13th, 2011] [Originally Added On: November 13th, 2011]