New gene therapy trial for adrenoleukodystrophy

Written on Wednesday 21 August 2013

In 2009 the results of the Gene therapy" class="glossy" title="Procedure involving the insertion of a normal gene into an organism in order to achieve a therapeutic objective. This gene (transgene) can be the normal version of a defective gene causing disease or a gene that produces a protein with any therapeutic action." >gene therapy trial for childhood cerebral adrenoleukodystrophy (ALD) performed in Paris by Pr. Patrick Aubourg and Dr. Nathalie Cartier and largely funded by ELA were published. Now a large follow-up clinical trial using an improved viral vector has been authorized in the US and will be developped by bluebird bio, Inc. If proven efficient, this new trial, should allow getting all the required authorizations to market the drug in the future. You will find below a sumary of the study.




Status: Authorized; patient recruitment active (Boston site)
Drug: Lentiviral vector Lenti-D carrying the normal human ABCD1 gene
Objective: To evaluate the safety and efficacy of the treatment in childhood cerebral ALD

Start date of the study: August 2013
Estimated end of the trial: August 2018

Director of the study: Dr. Asif Paker, bluebird bio, Inc.
Principal investigators

  • Dr. David Williams, Boston Children’s Hospital, Boston, MA, USA
  • Dr. Christine Duncan, Children’s Hospital, Boston, MA, USA
  • Dr. Florian Eichler, Massachusetts General Hospital, Boston, MA, USA

Location: Boston Children Hospital / Massachussetts General Hospital, Boston, MA, USA
Funding: bluebird bio

Reference : ALD-102 study, Starbeam study, NCT01896102

Fifteen children suffering from the cerebral form of ALD at a pre-symptomatic stage

Experimental protocol

  • Collection of bone marrow stem cells from the patient blood and isolation of the CD34+ stem cells
  • Transfer of the gene into the CD34+ stem cells using the viral vector.
  • Myeloablative conditioning of the patient (destruction of the bone marrow).
  • Reinfusion of the corrected cells in the patient (autotransplant).

Inclusion criteria

  • Age: up to 17 years old
  • Gender: Male
  • Active childhood cerebral adrenoleukodystrophy
  • Neurological function score ≤ 1

Exclusion criteria

  • Receipt of an allogeneic transplant or gene therapy.
  • Availability of a willing 10/10 matched sibling donor.
  • Use of statins, Lorenzo’s oil or diets aiming at lowering very long chain fatty acids. Note : subjects must discontinue use of these medications at time of consent.
  • Impossibility to perform MRI studies.
  • Hematological, hepatic, renal, pulmonary or cardiac compromise.
  • Infections and other conditions.

Patient follow-up
Patients will be followed-up several years after their gene therapy treatment.


Additional information

  • Other centers will be opened in the future in the United States and in Europe. When new centers will be authorized, we will update the section «  Clinical trials » of our website.



Tara O’Meara, bluebird bio, Inc. – Telephone : +1 617-797-2555, e-mail :
Colleen Dansereau, Boston Children’s Hospital/Massachusetts General Hospital – Telphone : +1 617-919-7008, e-mail :