611 W. Union Street
Benson, AZ 85602
(520) 586-0800

LaFrontera
member support line
1-520-279-5737
M-F 5pm-8pm
24/7 weekends/holidays

AzCH Nurse Assist Line
1-866-495-6735

NAZCARE Warm Line
1-888-404-5530



SEABHS
611 W. Union Street
Benson, AZ 85602
(520) 586-0800

AzCH Nurse Assist Line
1-866-495-6735

NAZCARE Warm Line
1-888-404-5530


powered by centersite dot net
Medical Disorders
Resources
Basic InformationLookupsLatest News
New Hormonal Pill May Boost Outcomes for Older Breast Cancer PatientsHad COVID? Getting Vaccinated Strengthens Your Antibodies to New VariantsU.S. to Pump $400 Million Into Vaccination Programs for Other CountriesPandemic Sent Americans' Blood Pressure Numbers SkywardWere Cancer Patients Neglected in U.S. COVID Vaccine Rollout?Young People Recover Quickly From Rare Heart Side Effect of COVID VaccineMore Evidence That Pandemic Delayed Cancer DiagnosesHigh Heart Rate Linked to Dementia RiskCOVID Vaccine, Testing Demand Overwhelming PharmaciesOmicron Spreading Through Africa Twice as Fast as Delta DidWith Holidays Ahead, COVID Boosters a Must for People With Weak Immune SystemsKeep Your Holidays Allergy-Free This YearDo Immune-Based Cancer Drugs Work Better in Men?Gene Found in Amish Helps Protect Their HeartsOmicron May Overcome Prior COVID InfectionWindy Days Are Safer Days When It Comes to COVID-19Most Vaccinated Adults Plan to Get Boosters: PollStudy Finds Delta Somewhat Resistant to Vaccines — What About Omicron?Is the Mumps Vaccine Becoming Less Effective?Vaping Can Trigger Gene Changes in Cells: StudyPfizer or Moderna? Head-to-Head Study Shows One Shot Has an EdgeSurvivors of Severe COVID Face Doubled Risk for Death a Year LaterKids With Uncontrolled Asthma at Higher Odds for Severe COVID-19Nearly 7% of U.S. Kids Have Had a Head Injury or ConcussionFirst U.S. Omicron Case Reported in California'Ultra-Processed' Foods Up Odds for a Second Heart Attack or StrokeCDC to Toughen COVID Testing for International TravelersAHA News: Irregular Heartbeat Risk Linked to Frequent Alcohol Use in People Under 40Certain Blood Thinners Can Raise Risk of 'Delayed' Bleeding After Head InjuryFDA Panel Gives Support to Merck's COVID Antiviral PillLong-Haul COVID Can Include Chronic Fatigue: StudyVaccines, Boosters Should Protect Against Severe COVID, Even With Omicron: FauciPfizer to Seek FDA Approval of Boosters for Teens Ages 16-17Regeneron Says Its Antibody Cocktail Likely Weakened by Omicron VariantCOVID May Trigger Heart Condition in Young AthletesMany People With High Blood Pressure May Take a Drug That Worsens It: StudyBiden Pushes Vaccines, Masks as Best Defense Against Omicron VariantHow Easily Can Singing Spread COVID-19?New Insights Into What Might Drive Parkinson's DiseaseHot Days Can Send Even Younger Folks to the ERRed Light in Morning May Protect Fading Eyesight: StudyMerck's COVID Pill Appears Effective, But May Pose Pregnancy Risks: FDAVaccine Makers Already Testing Their Shots Against Omicron VariantWhat Experts Know About the Omicron 'Variant of Concern'Gout Drug Colchicine Won't Help Fight COVID-19What You Need to Know About Stomach CancerFetal Infection With COVID-19 Possible, But UnlikelyCOVID Protection Wanes After 2 Doses of Pfizer Vaccine: StudyRural Hospitals' ERs Just as Effective as Urban Ones: Study1 in 5 Avoided Health Care During Pandemic, Study Finds
Questions and AnswersLinksBook Reviews
Related Topics

Cancer
Men's Health
Women's Health

Ten Years On, Gene Therapy Still Beating Most Cases of 'Bubble Boy' Immune Disease

HealthDay News
by Robert Preidt
Updated: Oct 21st 2021

new article illustration

THURSDAY, Oct. 21, 2021 (HealthDay News) -- Nine of 10 patients with so-called "bubble boy" immune disease who received gene therapy about a decade ago are still disease-free, researchers report.

The gene therapy was developed at the University of California, Los Angeles (UCLA), to treat the rare and deadly immune system disorder formally known as adenosine deaminase–deficient severe combined immunodeficiency (ADA-SCID).

It's caused by mutations in the gene that creates the ADA enzyme, which is crucial for a normally functioning immune system. Exposure to common germs can be fatal for babies with the disease. If untreated, most die within the first two years of life.

With the UCLA gene therapy, blood-forming stem cells are removed from a child's bone marrow and a specially modified virus is used to place healthy copies of the ADA gene into the stem cells' DNA. The cells are then transplanted back into the child's bone marrow.

The therapy is meant to prompt the body to produce a continuous supply of healthy immune cells that can fight infections. The transplanted stem cells are the child's own, so there is no risk of rejection.

Now, in the study published Oct. 14 in the journal Blood, the UCLA researchers reported that nine of the 10 children who received the one-time treatment between 2009 and 2012 as part of a phase 2 clinical trial remain stable.

Most of the children were babies at the time they received the gene therapy. The only one whose immune function wasn't restored by the gene therapy was 15 at the time of treatment.

"What we saw in the first few years was that this therapy worked, and now we're able to say that it not only works, but it works for more than 10 years," senior study author Dr. Donald Kohn, with the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, said in a UCLA news release. "We hope someday we'll be able to say that these results last for 80 years."

Gene therapy for ADA-SCID is not yet approved by the U.S. Food and Drug Administration.

Without it, children with ADA-SCID have to receive twice-weekly expensive and time-consuming injections of the ADA enzyme, or receive a transplant of healthy stem cells from a matched bone marrow donor.

In this study, patients with more copies of the ADA gene in more cells had the best immune function. More research is needed to determine the best way to achieve high levels of the gene in all patients, according to Kohn, a distinguished professor of microbiology, immunology and molecular genetics.

"What these results tell us is that there's a formula for optimal success for ADA-SCID, and it involves correcting more than 5% to 10% of each patient's blood-forming stem cells," he said.

"Knowing that a gene therapy can have this lasting effect in ADA-SCID for more than a decade is important for our path forward as we develop new gene therapies for this and other diseases," Kohn added.

More information

The U.S. National Library of Medicine has more on ADA-SCID.

SOURCE: University of California, Los Angeles, news release, Oct. 15, 2021