THE MLD ERT TRIAL - Keep viewing this page for updates as we receive them. We're all in this together and we'll get there faster, working together.

This year, our families with Metachromatic Leukodystrophy have some hope in the form of a Enzyme Replacement Therapy (ERT) Trial. Zymenex, a company in Denmark has been working diligently at bringing ERT into reality for MLD (and in the near future, Krabbe). You can help make this a reality by donating below.

LATEST NEWS - MAY 2006

First clinical trial with rhASA enzyme replacement therapy for patients with Metachromatic Leukodystrophy

The first clinical trial in patients with Metachromatic Leukodystrophy is expected to be carried out at a specialized phase I clinical trial unit in Copenhagen, Denmark under the supervision of an International Steering Committee and a local team of clinical experts. Currently the International Steering Committee comprises clinical, laboratory and statistical experts from France, UK, Germany, Denmark, and the US.

A study protocol is currently being developed by the Steering Committee for an open label phase I/II safety/tolerability clinical trial and an open label extension trial specifying dosing regimes, endpoints and in- and exclusion criteria.

The patient population will comprise of patients with a confirmed diagnosis of MLD in the late infantile stage of the disease. Severity criteria will include documented impaired nerve conduction velocity at enrolment and exclusion of the most severe stages of the disease.

Apart from safety assessments, the study will include assessments of nerve conduction velocity, nerve biopsy, MRI imaging, biochemical, neurological and motor functional evaluations.

All patients who are initially enrolled for the phase I/II safety/tolerability study will continue to be treated during the extension protocol phase .

The authorities will approve the final protocol during late summer and the trial will be launched in the fall of 2006.

A patient localisation process has been initiated. For more information about participation in the planned clinical trial, families are encouraged to have their referring physicians contact the scientific and medical leadership of the clinical project by email only please:

Dr. Ingeborg Kraegeloh Mann, Tübingen, Germany: (Ingeborg.Kraegeloh-Mann@med.uni-tuebingen.de)

Dr. Patrick Aubourg, Paris, France: (aubourg@paris5.inserm.fr)

Dr. Ed Wraith, Manchester, UK: (ed.wraith@cmmc.nhs.uk)

Any other requests should be directed to Dr. Christian Meyer at Zymenex A/S, Copenhagen, Denmark: (cme@zymenex.com)

The British Trust for The Myelin Project invites all MLD families and organisations to work jointly to bring this trial to our families. We have been trying to contact all groups and families but if we have missed yours out, please contact our office to join us in this fight against time.

Although there will be a limited amount of families to partake in this initial trial, it is our hope that all our MLD families will benefit once the trial phase is over and we can bring this product to market. As yet, no one has been chosen to be on the trial and it will be based on research criteria in each family member and not a first come, first served basis. However, everyone can help expedite the trial by helping us compile family histories for the researchers and also, by helping to raise funds.

If you would like to make a donation, please click on the button below or contact our office.

Please click here to make a donation to the trial: