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: jf.dhainaut@ela-fondation.com
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 kpv@stanford.edu
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, ELA
Florian Eichler, Mass General
Ali Fatemi, KKI
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.
Inna Tzvang, ALD Connect