MEDICAL 
RECORD 
CONTINUATION SHEET for either: 
NIH 25 M-1, Consent to Participate in A Clinical Research Study 
NIH 2514-2, Minor Patient's Assent to Participate in A Clinical 
Research Study 
STUDY NUMBER: 
CONTINUATION: page 2 of_6_page: 
some of your bone marrow cells prior to transplantation. It is important to understand that the 
introduction of this gene will not benefit you in any way. It is possible that this study may lead 
to improved bone marrow transplantation therapies for others in the future. The purpose of the 
study is to assess the safety of this procedure and to determine if “retroviral-mediated gene 
transfer” will enable the successful transfer of a gene into patient’s bone marrow cells. 
EVALUATION AND PROCEDURES 
If you decide to participate in this study, special laboratory techniques will be used to insert 
a gene into a portion of your bone marrow stem cells after they are harvested. These treated 
bone marrow cells are referred to as “marked” bone marrow cells. A gene is part of a 
chromosome (hereditary material) that contains the information a cell needs to make proteins. 
By inserting a gene into your bone marrow cells it will be possible to distinguish the marked 
bone marrow cells from all other cells in your body. 
The following procedure will be used to insert the gene into your bone marrow cells. Your 
bone marrow cells will be obtained by a bone marrow harvest in the operating room using the 
regular procedure for harvest This has been explained to you in a separate consent document for 
autologous transportation. It will not be necessary to obtain any more bone marrow than is 
normally taken during a bone marrow harvest Therefore, there will be no additional needle 
aspirations or additional anesthesia time. The major portion of the marrow harvested will be 
handled in the usual fashion and will not be treated by retroviral-mediated gene transfer. A 
portion of marrow (no more than 30%) will undergo retroviral-mediated gene transfer. In this 
30% portion, the cells to undergo the gene transfer will be separated from the other bone marrow 
and blood cells by attaching a special antibody to them and then physically removing only the 
cells that can pick up the antibody. This procedure is called “immunoadsorption” and it allows 
us to separate the more immature stem cells out of your bone marrow and blood cells. These 
immature cells are the ones used for the gene transfer. The physical separation of the immature 
cells attached to the special antibody is done by a laboratory machine called an 
immunoadsoprtion column. The column to be used in this protocol is experimental and has been 
approved by the FDA for use in transplantation studies such as this. 
Once the immature blood cells are separated and the antibody removed from them, they are 
mixed together in the laboratory with three substances called growth factors which help the 
blood cells divide. Also mixed with the blood cells is the retroviral vector that carries the neo— 
gene into the blood cells. The vector is an organism that carries material from one cell to 
another. Retroviral vectors are prepared from a disabled type of mouse virus that does not have 
the ability to reproduce. The vector enters the blood cells and puts the neo^ gene into the cells 
PATIENT IDENTIFICATION 
CONTINUATION SHEET for either: 
NIH-251 4-1 (10-84) 
NIH-251 4-2 (10-84) P.A.: 09-25-0099 
Recombinant DNA Research, Volume 16 
[135] 
