Stem Cell Gene Therapy Safe for Adrenoleukodystrophy

This article originally appeared here.
Share this content:
Stem Cell Gene Therapy Safe for Adrenoleukodystrophy
Stem Cell Gene Therapy Safe for Adrenoleukodystrophy

THURSDAY, Oct. 5, 2017 (HealthDay News) -- Early results suggest that stem cell gene therapy is a safe and effective treatment for boys with early-stage cerebral adrenoleukodystrophy, according to a study published online Oct. 4 in the New England Journal of Medicine to coincide with the 2017 Child Neurology Society Annual Meeting, being held Oct. 4 to 7 in Kansas City.

Florian Eichler, M.D., from Massachusetts General Hospital in Boston, and colleagues enrolled 17 boys with cerebral adrenoleukodystrophy in a single-group, open-label, phase 2-3 safety and efficacy study involving infusion of autologous CD34+ cells transduced with the elivaldogene tavalentivec (Lenti-D) lentiviral vector. Inclusion criteria included having early-stage disease and gadolinium enhancement on magnetic resonance imaging (MRI).

The interim analysis (median follow-up of 29.4 months) showed that all patients had gene-marked cells after engraftment, with no evidence of preferential integration near known oncogenes or clonal outgrowth. All patients also had measurable ALD protein. There were no reports of treatment-related death or graft-versus-host disease. Fifteen of the 17 patients were alive and free of major functional disability, with minimal clinical symptoms. One patient with rapid neurologic deterioration died of disease progression, while another patient with MRI evidence of disease progression withdrew from the study to undergo allogeneic stem cell transplantation but later died of transplantation-related complications.

"Additional follow-up is needed to fully assess the duration of response and long-term safety," conclude the authors.

Several authors disclosed potential conflicts of interest.

Abstract
Full Text (subscription or payment may be required)
Editorial

Share this content:

is free, fast, and customized just for you!

Already a member?

Sign In Now »


Sign up for myCME e-newsletters


Drug Lookup

Browse drugs by: BrandGenericDisease

More in Home

ACG: Diphenhydramine Ups Sedation in Chronic Opioid Users

ACG: Diphenhydramine Ups Sedation in Chronic Opioid Users

Improvement in sedation scores for patients using chronic opioids scheduled to undergo colonoscopy

Online Ratings Not Aligned With Objective Quality Measures

Online Ratings Not Aligned With Objective Quality Measures

In addition, consumer ratings are consistent across platforms for specialist physicians

Most Female Physicians Have Faced Sexist Patient Comments

Most Female Physicians Have Faced Sexist Patient Comments

Sexual harassment by patients can negatively affect physician-patient relationship, quality of care

is free, fast, and customized just for you!

Already a member?

Sign In Now »