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To view a list of all clinical trials for ALD/AMN on the website, click here. To view a list of all clinical trials for ALD/AMN on the EU clinical trials website, click here.  Key word search: adrenoleukodystrophy.

To stay up to date on clinical trial news, join our email list or follow us on Twitter @ALDconnect.

New therapies being investigated for treating ALD/AMN

An ALD Connect workgroup lead by Rachel Salzman, CSO The Stop ALD Foundation, and Kendrick Goss, Scientist II at bluebird bio provided an update at the ALD Connect/ULF meeting in Baltimore July 30 - August 3, 2014. A list of potential treatments were considered and narrowed down to the following:

  • Ampyra, a K+ Channel blocker, is said to improve nerve conduction, however no published data exists for ALD/AMN. It is approved for MS.
  • MD 1003, an anti-oxidant, is energy producing in neurons and repairs demyelination. Data on one AMN patient was reported and a multi-center AMN trial is being launched in Europe (not available in the US). Contact:
  • EPI-743, an anti-oxidant, shown to reduce oxidative stress in vitro. An AMN trial is close to being initiated in Italy. Contact: Keith Van Haren at
  • Natalizumab, an anti-integrin, is known to block lymphocyte infiltration in CNS. In-vitro data has been reported but not published. ALD experts have a strong interest in pursuing. It is approved for MS.
  • AAV, an in vivo gene therapy treatment. In-vitro and in-vivo mouse data has been shown to lower VLCFAs. More work is ongoing to further prepare for a human study (not prior to 2016).
  • Sobiterome, a thyroid agonist that up regulates ABCD2. In-vitro and in-vivo data has been reported but not published. A clinical study is under design.

Team members will continue to consider how to best move forward efforts.

Team members include: 

  • Joshua Bonkowsky, Univ of Utah
  • Nancy Braverman, McGill
  • Marie-Josee Duran, European Leukodystrophy Association
  • Florian Eichler, Massachusetts General Hospital
  • Ali Fatemi, Kennedy Krieger Institute
  • John Fink, Univ of Michigan
  • Kendrick Goss, bluebird bio
  • Stephan Kemp, Univ of Amsterdam
  • Troy Lund, Univ of Minnesota
  • Sanjay Magavi, Vertex
  • Alex McCampbell, Biogen Idec
  • Patricia Musolino, Mass General
  • Asif Paker, bluebird bio
  • Rachel Salzman, The Stop ALD Foundation
  • Keith Van Haren, Stanford University
  • Inna Tzvang, ALD Connect

A provisional assessment of the Neuralgene treatment in Mexico (as of May 1, 2014):

Neuralgene issued a press release stating that they are using their gene therapy platform to treat ALD. They claim to have an Adeno Associated Virus (AAV) targeted at delivering corrected gene copies to neural tissue. They injected their product, PRCN-323, into the spinal fluid of an ALD patient with the intention of "infecting" neural tissue with a correct copy of a gene. They believe that expression of the "corrected" genes might begin in a matter of a few weeks. The press release states that Neuralgene intends to monitor the first patient and treat additional patients in Mexico. The technology pipeline page of their website says that the AAV vector is delivering the ALD gene.
What is known about this treatment?

·         An email sent to the company to ask about the treatment has not yet been answered.
·         Leading ALD physicians were not familiar with the treatment.
·         ALD Connect does not have a relationship with Neuralgene.
·         The trial was not listed in (see explanation below).
·         There is no article published or data generally available to explain what studies have been done in animals to warrant trying it in patients. 
While delivering a corrected version of the ALD gene sounds like a good idea, one must be cautious:

·         Specific details of the procedure need to be evaluated to learn how safety is being ensured.
·         It is not known where the AAV vector was manufactured and whether it was done to a standard that would support safety.
·         It isn't clear what is being measured to demonstrate whether treatment is efficacious.
Should anyone be able to make contact with the company, please help us answer the following questions:

·         Can Neuralgene confirm that ALD gene is being delivered?
·         Can you specify how safety of the procedure is ensured?
·         What kind of patients are you treating? children? adults? with cerebral demyelination? AMN? adrenal insufficiency?
·         What kind of effect are you looking for?
·         What makes Neuralgene think this treatment will work? 
·         Is there pre-clinical data you can share with us? animal-model data?
·         What would it take for this treatment to be available in the US?
·         When will you know if the treatment was helpful to the first patient? how will you share the information?
·         While Neuralgene’s founder is listed as a radiologist, does the company employ a consulting neurologist with expertise in ALD?
·         Is there pre-clinical data in non-human models? is a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world


On February 2, 2015, Viking Therapeutics, Inc., a clinical-stage company based in San Diego, CA, and focused on developing novel, first-in-class or best-in-class therapies for metabolic and endocrine disorders, announced that it has entered into a research collaboration agreement with Stephan Kemp at the Academic Medical Center (AMC) of the University of Amsterdam, Amsterdam, Netherlands.

The collaboration will focus on the development of Viking’s proprietary selective thyroid beta agonists as potential treatments for X-linked adrenoleukodystrophy (X-ALD) and related diseases.
The thyroid beta receptor is known to regulate expression of an alternative VLCFA transporter, known as ABCD2. Various preclinical models have demonstrated that increased expression of ABCD2 can lead to normalization of VLCFA metabolism.

Under terms of the agreement, Viking will provide financial support and access to its proprietary library of thyroid beta agonists for scientists at AMC to evaluate in preclinical models of the disease. Viking will own exclusive rights to all research and associated intellectual property resulting from the collaboration, while AMC will have the ability to publish results, subject to Viking’s consent.

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