Looking for ways to cut the potential $1 billion annual cost oftreating Gaucher's disease in the United States with GenzymeCorp.'s Ceredase enzyme-replacement therapy, doctors arefinding that lower doses of the drug may be sufficient to treatpatients at far less expense.

The inherited disorder, which affects 20,000 to 40,000 peopleworldwide, is characterized by enlargement of the spleen,erosion of bone, easy bruising, slow blood clotting andsometimes early death. Mental impairment appears soon afteronset.

Writing today in the New England Journal of Medicine, a groupof doctors at the Scripps Clinic in La Jolla, Calif., said the cost oftreating a child for a year would be $191,000 at dosesrecommended by the study group at the National Institutes ofHealth. Adult therapy for the disease would be $382,000.

The La Jolla doctors, together with a colleague practicing in LosAngeles, recommend that patients could receive thrice-weeklyinfusions of a lesser amount of the enzyme instead of therecommended dose every other week. They calculate thesavings at $6,000 a week for an adult treatment.

Physicians at a Jerusalem hospital, also writing in the NEJM,pointed out that the gene for the disease is most frequentamong Ashkenazi Jews. Consequently, many patients reside inIsrael, but "the health care provider organizations have refusedto cover this very costly therapy."

The Israeli doctors reported on two boys they treated for threeand five months, respectively, at a quarter the recommendeddosage to minimize cost. The result in these children, thedoctors wrote, "has been very encouraging," with increasedalertness and mobility, as well as improved blood counts anddecreased organ swelling.

Responding to the letters, the NIH group agreed that lowerdoses may be adequate once an initial course of high-dosetherapy clears the disease products from the body. Yet theycautioned that some of the patients who appear to respond wellto low doses of Ceredase lack spleens.

"Only a formal clinical trial comparing a low dose with a highdose in patients who have undergone splenectomy will provethat the burden and disruption of three treatments per weekare truly worthwhile," the NIH group and colleagues atMassachusetts General Hospital concluded.

"We are committed to lowering the dosage if that is shown tobe effective," said Genzyme spokeswoman Donna LaVoie, notingthat various researchers have published data supporting use ofa lower dose.

LaVoie and the company's product manager for Ceredase,Jennifer Aronoff, said that patients in Israel are being fundedfor the treatment by a combination of government funds andinsurance companies.

Stock of the Cambridge, Mass., company closed at $50, up 25cents, on Wednesday.

-- Roberta Friedman, Ph.D. Special to BioWorld

(c) 1997 American Health Consultants. All rights reserved.