5.4 Hazards and Discomforts 
GTI0101 (04/08/93) 
Page 26 of 35 
This gene transfer protocol would be classified as offering no guarantee of direct 
benefit to patients enrolled on it, but posing little risk. Information obtained from 
this pilot gene transfer study could benefit future patients with Gaucher disease, 
and could possibly allow patients in this study to eventually reduce or discontinue 
Ceridase enzyme replacement therapy. None of the over 50 patients that have 
thus far received retrovirally-transduced bone marrow, peripheral blood or tumor 
infiltrating cells have had any adverse events noted that could be attributed to the 
gene transfer procedure. 
If marrow cells are obtained for transduction, patients will be exposed to the 
minimal risks associated with the procedure. These risks include the risks 
associated with the anesthesia used during the marrow harvest. Other risks 
include infection and bleeding. Pain from the multiple marrow aspirates required 
to harvest the necessary volume of marrow cells desired can be a problem but is 
normally responsive to mild-moderate analgesics. Occasional patients may 
require a blood transfusion after marrow harvest. 
Patients will be exposed to the minimal hazards, including bleeding complications 
and infection, associated with apheresis catheter placement each cycle. They will 
be treated with G-CSF for 7 days every cycle, and may experience bone pain, 
fever, or rash, and less commonly reversible elevation of hepatic enzymes, 
transient depression of platelet numbers, and exacerbation of pre-existing 
inflammatory conditions. During apheresis the patient may experience postural 
hypotension and paresthesias. 
After the end of the treatment portion of the protocol, patients will be requested 
to participate in further follow-up to evaluate the duration and extent of GC 
cDNA transfer and expression. The follow-up will be as an outpatient and will 
require needle sticks for blood samples, liver-spleen sizing, and additional bone 
marrow aspirates. As the patient is involved in a gene therapy trial, a regulatory 
committee has also requested that all patients be followed periodically for the rest 
of their lives for detection of side-effects from the gene transfer; this request 
includes that the patient be aware that an autopsy would be requested when the 
patient dies to measure for possible side-effects including the presence and extent 
of gene transfer. 
Patient Costs : At CHLA, the costs for hospitalization, bone marrow harvest, and 
any laboratory tests which are performed for study evaluation will be borne by 
the Division of Research Immunology/Bone Marrow Transplantation. Neither the 
subject, nor their third party payor will be charged for any of the costs which are 
part of the experimental investigations. The patients or their third party payor will 
continue to be responsible for costs of all routine medical care, including 
Ceredase, if the patient is receiving it. 
Recombinant DNA Research, Volume 17 
[811] 
