Leucine is an amino acid that is available as a nutritional substance. It is sold over-the-counter in many nutritional supplements, in many combinations. For everyone’s knowledge, over-the-counter nutritional supplements are not FDA monitored. The drugs are not purified and are in combination forms. 500mg of leucine in one combination product may really only be 350 mg or 620 mg, for example. There is no FDA oversight in the preparation of these supplements.
The medical community is therefore not recommending starting all DBA patients on these over-the-counter leucine/combo product. However the DBA community is very interested in leucine. Since Dr. Pospisilova presented these data at the DBA International Conference in March the DBAR team led by Dr. Vlachos and the Arturi Foundation have been working on finding out more. The DBAR team is trying to find a company that makes a purified leucine product, get them to be interested in a trial for DBA patients, at a controlled dose with a controlled product.
Everyone is anxious to start leucine, but trying a combination, unpurified product may yield poor results and then everyone will be disappointed, much the less may make others unwilling to try the purified product for fear of failure. Please let’s do this in an orderly way. Waiting is a lot to ask and everyone wants a quick cure but we should try get the best product available for the best results. The Czech Republic patient was a single experience so we have to be careful. The leucine seems to be well tolerated but again it has been only a handful of patients.
I hope this is helpful to all. We will keep you posted.
Adrianna Vlachos, MD
Head, Bone Marrow Failure Program
Associate Head, Stem Cell Transplant Program,
Schneider Children’s Hospital,
New Hyde Park, NY 11040