Research Updates

MicroscopeResearch News
ELA is pleased to fund a new clinical trial for male patients with Adrenomyeloneuropathy (AMN). The clinical trial will look at demonstrating clinical efficacy of MD1003 drug treatment and to evaluate the safety of the treatment for male AMN patients.

MD1003 will be given to 60 patients with AMN with no cerebral involvement across France, Spain and Germany. The trial will last two years with patients split into two groups. First group taking a placebo and second treated with MD1003 drug. In the second year, all male patients will receive MD1003 drug.

You can read more about MD1003 clinical trial on ELA website:

Clinical Trials
List of Clinical Trials in the USA
List of Clinical Trials funded by ELA

Interesting clinical trial: A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Childhood Cerebral Adrenoleukodystrophy (CCALD). Sponsor: Bluebird Bio. For more information, please visit their Clinical Trials Page.

UPDATE: First boy undergone treatment for Bluebird Bio’s study in the US. You can find their press release here.

Interesting clinical trial: MD1003 trial in adrenomyeloneuropathy (AMN). Sponsor: European Leukodystrophy Association. Developed by MedDay Pharmaceuticals, a new drug called MD1003 or biotin will be trialled in France, Spain and Germany in male patients with adrenomyeloneuropathy (AMN). You can read more about the trial on European Leukodystrophy Association’s website.

Interesting clinical trial: MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD). Sponsor:
Masonic Cancer Center, University of Minnesota. Dr Paul Orchard is developing a clinical trial looking at transplantation for active, advanced cerebral adrenoleukodystrophy (cALD):

Gavin Metcalf – Lead Database Researcher and Science Advisor
Gavin Metcalf, a Doctoral Student at Imperial College London, is currently involved in active research into Cancer Biomarker development via the use of fluorescent biosensors. He is also an active Researcher and Science Advisor for ALD Life. You can read his full biography here.

He will be voluntarily keeping us updated with appropriate research he has been compiling on Adrenoleukodystrophy (ALD). Please feel free to keep updated with his latest research work below. If you have any scientific questions about ALD, he’s volunteered to answer your questions. Please send an email to

List of appropriate research of ALD Clinical TrialsReferences
Current ALD Therapeutic Options
Potential ALD Therapeutic Options Part 1 and Potential ALD Therapeutic Options Part 2
List of ALD-AMN specialist researchers
Mutations in ABCD1, X-ALD website


Unable to display Facebook posts.
Show error

Error: (#4) Application request limit reached
Type: OAuthException
Code: 4
Please refer to our Error Message Reference.