MPS I – Hurler post HSCT Enzyme Replacement Therapy Clinical Trial – Due to Report June 2016
This is an Enzyme Replacement Therapy clinical trial for children with Hurler disease who have had a bone marrow or cord blood transplant and have progressive musculo-skeletal disease.
The three UK children on this trial have had their baseline tests at the University of Minnesota in the USA and two of the children received their first weekly infusion of Aldurazyme on 7 August 2013.
Hurler disease or MPS IH is a Mucopolysaccharide disease fIrst described in 1919 and causing devastating progressive neurological and physical disability and death in early childhood if not treated with a bone marrow or cord blood transplant in the first 18 months of life.
Bone Marrow Transplant (BMT) or cord blood transplant (CBT) is not cure. These treatments are proven to preserve the brain in many of the young children with Hurler disease but not to be effective in preserving the musculo-skeletal disease. Therefore there is an urgent unmet clinical need.
Hurler disease causes rapid neurological degeneration and Enzyme replacement therapy (ERT), Aldurazyme, does not cross the blood brain barrier.
Aldurazyme is already a licenced treatment for the attenuated MPS I diseases, Hurler Scheie and Scheie disease. With over a decade of experience treating children and adults with these diseases there is evidence of Aldurazyme halting or improving musculo-degeneration. This is one of the hypotheses of this Phase I/II clinical trial.
The children received their weekly Aldurazyme at Great Ormond Street Hospital and Manchester Children’s Hospital. After 6-8 weeks the children then received their enzyme replacement therapy at home with the support of the home care nurses. The three UK children travelled five times to the United States during the two year trial for assessments and are now continuing to receive Aldurazyme whilst they wait for the clinical trial to report.
The clinical trial results are now being analysed and the results should be published in 2016. If the results demonstrate safety and efficacy a multinational Phase III/IV clinical trial is likely to take place.