Important new clinical trial for MLD

GlaxoSmithKline (GSK) has just launched a Phase 3 clinical trial to study the efficacy of a cryopreserved formulation of their gene therapy, i.e. freezing the modified cells before transfusion. Recruiting of pre-symptomatic late infantile and early juvenile patients will start immediately.

This study still requires patients to travel to Milano, Italy for their transplant — similar to how previous patients have accessed the San Rafaelle gene therapy.

Laying The Groundwork for sites in other countries

Most notable is that this trial lays the groundwork for patients to eventually access the gene therapy in a local medical center outside of Italy. Their cells will still be genetically modified in Italy — the cryopreservation allows for international transport of the modified cells. In the USA, the next step would be a New Drug Application (NDA) to seek approval for offering the gene therapy in the USA.

Inclusion criterial can be found here. We are happy to discuss this trial and inclusion criteria with any of you tat think you may be eligible. We know the Principal Investigator in Italy well and can help you with the referral process to assess eligibility.

Deeper Dive

For those of you wishing to dig  little deeper, here’s a good article about gene therapy cyropreservation — in general, not necessary reflecting the the GSK/San Rafaelle flow or specific issues.

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About us

MLD Support Association UK was set up to bring hope to families in the fight to eradicate Metachromatic Leukodystrophy (MLD). Our aim is to provide support by way of shared information from people in similar circumstances who have already experienced the effects of the condition and/or any treatments available.

MLD Support Association UK

MLD Support Association UK is a
Registered Charity Number 1150542

Professor Timothy M Cox, MD, FRCP, FMedSci
Suzi Digby (Lady Eatwell) OBE
Iain Stewart MP


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