APPENDIX D 
NONTECHNICAL ABSTRACT OF PROTOCOL 
One effective treatment for many types of cancers is autologous bone marrow 
transplantation. In this treatment the patient is treated until the disease can 
no longer be detected. At this point, about 1.5 pints of marrow are removed and 
stored frozen. The patient is then given extremely intensive drug and radiation 
treatment aimed at destroying any cancer cells which may be left. This intensive 
treatment also destroys the patient's remaining bone marrow cells. However, the 
individual is "rescued" by injecting back the marrow that has been stored for 
them. 
Although this treatment has proven to be encouraging, there are two 
problems. The first is that the stored marrow, although taken when the patient 
has no visible cancer, may still contain some cancer cells. These unseen cancer 
cells could then regrow and cause the disease to return. In addition, the 
stored marrow is sometimes slow to grow. This means the patient lacks vital bone 
marrow cells for a longer period of time. Without these marrow cells the patient 
cannot adequately fight infection and may suffer bleeding. These problems , are 
often life threatening. 
The aim of the present protocol is to get information that will allow 
clinicians to devise ways to overcome or lessen these problems. Genetic markers 
will be used to trace the development and behavior of cells within the marrow. 
As the marrow cells harvested from the patient look the same as the cells 
remaining within the body, a way is needed to mark these cells. Gene marking is 
the only way to distinguish the returned cells from cells remaining in the 
patient's body. About 30% of the patient's marrow that is removed will be marked 
with a marker gene. These marked cells will then be stored with the untouched 
marrow and both will be given back to the patient after their intensive drug 
treatment . 
If the patient does suffer a return of their cancer, we will look at their 
cancer cells for the marker gene. If no marker gene can be found, then the 
cancer probably regrew from cells left in the patient's body. This is possible 
even though no visible cancer cells may have been seen in the patient. The 
doctors would then know that they needed to work on better methods to get rid of 
the cancer cells left in the body. If the gene is found in the cancer cells, 
then wq know cancer cells were hiding with the returned marrow cells. If we know 
cancer cells are hiding in the returned marrow, the doctors can work harder to 
kill the cancer cells before returning the marrow to the patient. No matter 
which answers are obtained, the doctors will then be able to develop better 
treatments for similar cancer patients. 
After marrow transplantation with marked marrow we will also look for the 
appearance of the marker gene in the patient's blood and marrow. By studying the 
normal cells which contain the marker gene, the doctors will learn more about the 
transplanted marrow cells. We will learn better ways to treat the patient before 
removing the marrow. We will also learn how to best treat the patient after the 
transplant so that the returned marrow grows as quickly as possible. The more 
that is learned about the marrow cells, the better the doctors will be able to 
use the marrow cells to help the patient. 
The doctors hope that by using a gene to mark the marrow removed from a 
patient that they will learn how to better treat the patients. This knowledge 
will not only help patients with cancers similar to the ones studied, but also 
many other patients as well. 
Recombinant DNA Research, Volume 14 
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