Treatments for LAL Deficiency

No treatments for LAL Deficiency have been studied in clinical trials, which means that there are no licensed medicines approved by the Regulatory authorities like the FDA.  As is the case for other diseases where there are no approved treatments, other treatments are used in an attempt to control or help  some of the signs and symptoms of the disease. 

In patients with early onset LAL Deficiency, Wolman Disease:
• Antibiotics may be prescribed to address pyrexia (a fever) or documented infections
• Changes in diet including stopping of either breast milk or normal formula that contain high amounts of triglycerides and cholesterol esters may be tried.  Special formulas which are fatty ester-free have been recommended to manage the abnormal fat accumulation in the gut and other organs. (Wolman, 1995) 
• In the absence of alternatives, bone marrow/hematopoietic cell transplantation has been performed in babies with early onset LAL Deficiency. While a very small number of babies have survived following this, the procedure still has a very high mortality (greater than 80%) due to the severity of the disease and the serious acute side effects of some of the medicines used.  Additionally, the procedure carries the inherent risks associated with graft-versus-host disease (GVHD).

In patients with late onset LAL Deficiency, CESD:
• High fat levels (LDL “Bad cholesterol “ and Triglycerides ) are treated using:
      • A strict low fat diet that excludes foods rich in cholesterol and triglycerides
      • Lipid lowering drugs such as Statins, cholestyramine and ezetimide
• Liver transplantation