Aldagen is conducting Phase III clinical trials at Duke University on its supplemental stem cell known as ALD-101.  The study will determine if this supplemental stem cell accelerates cell engraftment in children who have received umbilical cord blood transplants for inherited metabolic diseases, such as MLD.  Rapid engraftment would shorten the period of time a patient would be at risk for infection and bleeding after a transplant, so it would therefore increase transplant survival rates.  For more information on this clinical trial, click here. 

The Evanosky Foundation is continuing to sponsor pre-clinical work at Duke University which is targeted at developing cellular therapies for treatment of children with leukodystrophies.  In the coming year we expect to see initiation of a Phase I clinical trial that will administer cells intrathecally (in the spinal fluid) for children with advanced leukodystrophies.  Individuals or organizations who are interested in funding this or other research should contact Bob Evanosky at the Evanosky Foundation (evanoskyfoundation@evanoskyfoundation.org). 

Many clinical trials are being conducted that, while not directly related to MLD, may be applicable to the treatment of patients with this or other leukodystrophies.

• Duke University is conducting a Phase I clinical trial to test feasibility of collection, preparation and infusion of a baby's own umbilical cord blood cells if the baby is born with signs of brain injury.  Babies will be followed for neurodevelopmental outcome.  For more information about the neonatal hypoxic-ischemic encephalopathy study, click here.
• Aldagen is conducting Phase I and II clinical trials of ALD-301 (specially processed stem and progenitor cells) to determine if injection of the cells are effective in generating new small blood vessels to improve circulation in patients with atherosclerotic vascular disease in the heart and ulceration or tissue necrosis.  To learn more about the ALD-301 trial, click here.
• Recent studies suggest that it may be possible to grow new blood vessels in the heart with the use of a particular type of stem cell, aldehyde dehydrogenase bright stem cells.  For more information on the Phase I clinical trial being conducted by the Texas Heart Institute and Aldagen, click here.
• To read an article by Duke University's Vinod K. Prasad and Joanne Kurtzberg about trends in transplantation of inherited metabolic diseases that appeared in the medical journal Bone Marrow Transplantation, click here.
• For information on the FDA's Investigational New Drug (IND) Application Process, which all new drugs and treatments must go through prior to use in humans, click here.  The description is lengthy, but it will provide insight on the development and procedures involved in bringing new therapies to fruition.
• To read a paper discussing the study of transplanted ALDH cells engrafting in multiple tissues and promoting cellular repair in damaged tissues, click here.
• To read a case study that appeared in the online journal Haematologica which suggests the possibility of improved transplant engraftment with the use of mesenchymal stromal cells (MSCs), click here.