Mid-Size Biotech Rare Disease Case Study: Phase II Pediatric Study With Selected Metabolic Disorders Aims To Increase Survival Rate

Cmed is currently working on a Phase II trial with a US Pharmaceutical company involving pediatric patients with selected inherited metabolic disorders undergoing stem cell transplantation.

Inherited metabolic diseases (IMD), including Hurler syndrome, cerebral adrenoleukodystrophy (cALD), globoid cell leukodystrophy (GLD or Krabbe) and metachromatic leukodystrophy (MLD), are a group of rare, inherited disorders that result in a progressive loss of neuromotor and cognitive abilities. Untreated, these disorders are lethal.

The only accepted therapy for Hurler syndrome, MLD, GLD, and cALD is allogeneic hematopoietic stem cell transplantation (HSCT), after which immature cells of hematopoietic origin migrate to tissues, including the central nervous system. HSCT has a high risk of complications and death. Deaths from transplant-related complications occur in 20% to 30% of patients.

Unrelated Umbilical Cord Blood (UCB) donor programs are most often utilized as treatment. The amount of necessary cells (stem and progenitor) within a UCB unit is infinitely small and therefore takes a very long time to be effective for the patient, a primary factor of the deaths observed.

The study aims to magnify the necessary cell population within a UCB to quicken the time to reach transplant effectiveness and ultimately aims to positively impact the transplant success and survival rate.