Trial Information

Feasibility Study of Acute Myelogenous Leukemia mRNA Plus Lysate Loaded Dendritic Cell Vaccines

Patient Consent: Arm 1 - Standard-Dose Consolidation

The vaccine will be made from your AML cells which will be killed, frozen, and stored away
when you start the study. When you go into remission, researchers will take your normal
blood cells and culture them in the laboratory until they become "dendritic cells."
Researchers will then thaw your tumor cells and load parts of them into the dendritic cells
and inject the mixture. This type of vaccine will hopefully encourage your immune system to
prevent later relapse of your disease.

Before you can start treatment on this study, you will have what are called "screening
tests." These tests will help the doctor decide if you are eligible to take part in this
study. Blood (about 1 tablespoon) will be drawn to make sure you do not have an infection
with HIV/AIDS. If you do, you will not be eligible for this study.

If you are found to be eligible to take part in this study, AML cells will be collected from
your blood through a vein in your arm. A blood separator device called an apheresis machine
will be used. Each apheresis procedure takes about 3-5 hours. It is similar to donating
platelets to a blood bank. During the procedure the blood with tumor cells will be removed
and then returned through a second line in the other arm. You may have bone marrow
aspirations instead with general anesthesia, usually lasting up to 3 hours. To collect a
bone marrow aspirate, an area of the hip or chest bone is numbed with anesthetic, and a
small amount of bone marrow is withdrawn through a large needle. The AML cells collected by
either the apheresis or bone marrow collection procedure will be used later to make the
vaccine in the M. D. Anderson Cell Therapy Laboratory where they will be frozen and stored
until it is time to make the vaccine.

After the AML cells are collected, you will start receiving your induction and consolidation
chemotherapy treatment. This treatment is standard of care and is not directly related to
your participation in this study. After your first cycle of consolidation treatment, a bone
marrow biopsy will be done to find out if you are in complete remission. To collect a bone
marrow biopsy, an area of the hip or chest bone is numbed with anesthetic, and a small
amount of bone marrow and bone is withdrawn through a large needle. If you are in complete
remission then you will have your normal blood stem cells collected, which will be used to
make the vaccine. All of the normal stem cells will be collected with the same apheresis
machine used to collect tumor cells. Beginning 5 days before the first collection, you will
receive daily injections under the skin of granulocyte colony stimulating factor (G-CSF).
These are given to increase the number of normal stem cells available for collection. On
Days 5 and 6 of G-CSF treatment, your normal stem cells will be collected. They will be
taken to the M. D. Anderson Cell Therapy Laboratory where they will be frozen and stored
until it is time to make the vaccine.

After you complete the apheresis procedure to collect normal cells, you will continue
consolidation therapy (typically 3-5 cycles). The vaccine will be made with your
previously frozen cells. Once thawed, a machine called the CliniMACS will be used to isolate
the cells from your blood that will be grown into the vaccine during an 8-day procedure.

After the last consolidation cycle, you will begin to receive 4 monthly injections of the
vaccine. The first vaccination will be given once your blood counts have recovered from the
final dose of chemotherapy. The remaining 3 vaccinations will be given every 28 days (+/- 7
days). Once it is ready, each vaccine will be injected into a lymph node in your groin. A
special machine called a sonogram (typically used to look at babies in the womb) will be
used to painlessly locate exactly where the injection should go.

About 3-7 days after each vaccine, about 4 tablespoons of your blood will be drawn to
measure the immune reaction to the vaccine. At that time you will also have a skin test
performed to look for the presence or absence of an immune reaction to the vaccine. This
will be done with a small injection under the skin on your forearm. About 48 hours later,
researchers will measure the area around the injection for redness, which would be a sign
that your immune system is reacting to the vaccine.

After you have received all 4 vaccines, you will be seen in the clinic yearly to evaluate
how you tolerated the vaccines.

Routine long-term follow-up exams will be performed by your leukemia doctor as per standard
of care.

This is an investigational study. The vaccines are not commercially available and only
authorized for use in research. A total of 40 patients will take part in this study. All
will be enrolled at M. D. Anderson.

Patient Consent: Arm 2 - Autologous Peripheral Blood Progenitor Cell Consolidation

The vaccine will be made from your AML cells which will be killed, frozen, and stored away
when you start the study. When you go into remission, researchers will take your normal
blood cells and culture them in the laboratory until they become "dendritic cells."
Researchers will then thaw your tumor cells and load parts of them into the dendritic cells
and inject the mixture. This type of vaccine will hopefully encourage your immune system to
prevent later relapse of your disease.

Before you can start treatment on this study, you will have what are called "screening
tests." These tests will help the doctor decide if you are eligible to take part in this
study. Blood (about 1 tablespoon) will be drawn to make sure you do not have an infection
with HIV/AIDS. If you do, you will not be eligible for this study.

If you are found to be eligible to take part in this study, AML cells will be collected from
your blood through a vein in your arm. A blood separator device called an apheresis machine
will be used. Each apheresis procedure takes about 3-5 hours. It is similar to donating
platelets to a blood bank. You may have bone marrow aspirations instead with general
anesthesia, usually lasting up to 3 hours. To collect a bone marrow aspirate, an area of
the hip or chest bone is numbed with anesthetic, and a small amount of bone marrow is
withdrawn through a large needle. The AML cells collected by either the apheresis or bone
marrow collection procedure will be used later to make the vaccine in the M. D. Anderson
Cell Therapy Laboratory where they will be frozen and stored until it is time to make the
vaccine.

After the AML cells are collected, you will start receiving your induction and consolidation
chemotherapy treatment. This treatment is standard of care and is not directly related to
your participation in this study. After your first cycle of consolidation treatment, a bone
marrow biopsy will be done to find out if you are in complete remission. To collect a bone
marrow biopsy, an area of the hip or chest bone is numbed with anesthetic, and a small
amount of bone marrow and bone is withdrawn through a large needle. If you are in complete
remission then you will have your normal blood stem cells collected, which will be used to
make the vaccine. All of the normal stem cells will be collected with the same apheresis
machine used to collect tumor cells. Beginning 5 days before the first collection, you will
receive daily injections under the skin of granulocyte colony stimulating factor (G-CSF).
These are given to increase the number of normal stem cells available for collection. On
Days 5 and 6 of G-CSF treatment, your normal stem cells will be collected. They will be
taken to the M. D. Anderson Cell Therapy Laboratory where they will be frozen and stored
until it is time to make the vaccine.

After you complete the apheresis procedure to collect normal cells, you will continue
consolidation therapy with high-dose standard busulfan and cyclophosphamide chemotherapy.
You will also have an autologous peripheral blood progenitor cell transplant. The
chemotherapy and transplant are standard of care.

After the chemotherapy and transplant when your blood counts have recovered, you will begin
to receive 4 monthly injections of the vaccine. The vaccine will be made with your
previously frozen cells. Once thawed, a machine called the CliniMACS will be used to isolate
the cells from your blood that will be grown into the vaccine during an eight day procedure.

The first vaccination will be given once your blood counts have recovered from the final
dose of chemotherapy. The remaining 3 vaccinations will be given every 28 days (+/- 7
days). Once it is ready, each vaccine will be injected into a lymph node in your groin. A
special machine called a sonogram (typically used to look at babies in the womb) will be
used to painlessly locate exactly where the injection should go.

About 3-7 days after each vaccine, about 4 tablespoons of your blood will be drawn to
measure the immune reaction to the vaccine. At that time you will also have a skin test
performed to look for the presence or absence of an immune reaction to the vaccine. This
will be done with a small injection under the skin on your forearm. About 48 hours later,
researchers will measure the area around the injection for redness, which would be a sign
that your immune system is reacting to the vaccine.

After you have received all 4 vaccines, you will be seen in the clinic to evaluate how you
tolerated the vaccines.

Routine long-term follow-up exams will be performed by your leukemia doctor yearly as per
standard of care.

This is an investigational study. The vaccines are not commercially available and only
authorized for use in research. A total of 20 patients take part in this study. All will be
enrolled at M. D. Anderson.