Specific therapy for the nonneuronopathic manifestations of Gaucher disease has been available since 1991 firstly in the form of the macrophage targeted placenta-derived glucocerebrosidase (alglucerase, Ceredase
[R], Genzyme Corporation, MA) (7), and subsequently (1994 in USA and 1997 in Europe) by recombinant human enzyme, imiglucerase (Cerezyme[R], Genzyme Corporation, MA) (8).
Brady's first discovery led directly to Ceredase
, a drug for treating the devastating symptoms of Gaucher's Disease, which afflicts fewer than 10,000 people worldwide, generally killing them before they reach adulthood.
For Brian, one of only about 5,000 Gaucher disease patients in the world, the introduction of Ceredase was truly a miracle.
Before Ceredase and its successor drug Cerezyme, average life expectancy for people born with Gaucher disease was 41 years.
And he's read up on how Ceredase and Cerezyme came to be.
As is often the case in medicine, the science behind Ceredase and Cerezyme can be traced to the pioneering work of one person, Dr.
A woman suffering from Gaucher's disease, a rare life-threatening illness that weakens bones and swells organs, told the press that it costs her $350,000 a year to buy Ceredase
, the drug that keeps her alive.
, a drug for treating a rare enzyme deficiency called Gaucher's disease, now cost an average of $140,000 a year.
The nonrecombinant products ceredase
and adenosine deaminase (PEG-ADA) illustrate the potential impact of chronic lifetime administration of drugs.
The recent arrival on the market of a modified glucocerebrosidase (Ceredase
) for Gaucher's Disease and last year's approval of PEG-adenosine deaminase for severe combined immunodeficiency disease illustrate the growing capabilities of the pharmaceutical industry to provide endogenous compounds that patients are lacking.