Finding a Cure for Leukemia

Arikka Gooch
November 12, 2016

What is leukemia? Leukemia is a cancer of blood cells. It is a malignant

progressive disease in which the bone marrow and other blood-forming
organs produce increased numbers of immature or abnormal leukocytes.
Leukemia can differ between either acute or chronic. Acute leukemia is a
rapid growing cancer that usually progresses more quickly. Chronic leukemia
is a slower growing cancer that worsens over longer periods of time. The
treatment for leukemia is highly dependable on the type of blood cell
affected and if the leukemia is chronic or acute.
Experts say that there are many different known and suspected causes
of leukemia. Some of these include: artificial ionizing radiation, viruses,
benzene, and electromagnetic energy. Leukemia is common in young
children and adults over the age of 50 years old. There are many types of
leukemias that have different effects on each age group.
Acute Lymphoblastic Leukemia (ALL) is the most common type of
leukemia among young children. Survival rates of ALL range from 85%
among children and 50% among adults. Although adults can get ALL, they
are more affected by Chronic Lymphocytic Leukemia (CLL). CLL hardly effects
children and is most common in adults over the age of 55, mainly male.
Experts say that this type of leukemia is incurable, although 75% of treated
CLL patients survive for over five years.
Other common types of leukemia include Acute Myelogenous Leukemia
(AML) and Chronic Myelogenous Leukemia (CML). Acute Myelogenous
Leukemia is a very rare type of cancer. It mostly occurs in males over the
age of 50 years. The main known causes of AML are benzene and high levels
of radiation. It is usually treated with chemotherapy. 40% of treated patients
survive for over 5 years.
Chronic Myelogenous Leukemia occurs mainly in adults. This type of
cancer is treated using gleevec (imatinib). CML is caused by a chromosome
mutation that spontaneously occurs. Experts are unsure what causes the
mutation, but say that 90% of treated patients survive for over 5 years.
There are many symptoms to look for when diagnosing leukemia. One
major sign is poor blood clotting. As immature leukocytes crowd out blood
platelets the patient may bruise or bleed easily and heal slowly. Another sign
is an effected immune system. Leukocytes are crucial for fighting off
infection, but they may be damaged or not working properly. The patient
may experience frequent infections, or their immune system may attack
other properly working body cells.
Anemia is another major symptom of leukemia. Patients may suffer
difficulty breathing or pallor due to the shortage of good red blood cells
growing larger. Patients may also experience nausea, fever, chills, night
sweats, flu-like symptoms, and tiredness. Weight loss is common from an
enlarged liver or spleen causing the patient to eat less. Headache is more

common among patients whose cancerous cells have invaded the central
nervous system.
While some experts are trying to discover the main cause of leukemia,
others are searching for cures. According to a study performed by Robert
Paulson and Sandeep Prabhu, they inject the compound delta-12prostaglandin J3 (J3 for short) into mice that have been infected with
leukemia stem cells, and record the results. We found that by treating the
cells with the J3 compound, we turn on the p53-dependent cell death
pathway, which causes not only the leukemia cells to die, but the leukemia
stem cells to die as well. (Lajeunesse, 2012)
It is essential to kill the leukemia stem cells if you want to cure
leukemia. Chemotherapy kills the bulk cells because theyre dividing much
faster than the stem cells which divide much slower. Killing the stem cells is
important because stem cells can divide and produce more cancer cells and
sometimes more stem cells. The scientists are investigating how stable the
compound is and whether it will break down in the stomach if given orally.
They are curious to see if the compound will work the same way in humans.
Scientists are optimistic that the therapy will translate into humans and
make its way into clinics to help patients live a little bit longer.
In late 2015, one year old Layla was dying from acute lymphoblastic
leukemia after other treatments had failed. Thats when her doctors turned
to an experimental form of gene therapy. They used genetically engineered
immune cells from a donor, which killed off all the cancerous cells in her
bone marrow in just one month. Doctors would remove immune cells from a
Laylas body, and genetically engineer them to attack cancerous cells and
place them back into her body.
ALL cells hold the protein CD19. In these trials, a gene is added for a
receptor called CAR19 which sits on the outside of the T-cells. This action
programs the T-cells to find and kill any cells containing CD19. This is not a
cheap procedure, and according to researcher Waseem Qasim, Layla was
too small and too sick. She did not have enough T-cells left to modify.
However, Qasims team has found a way to modify T-cells from a healthy
donor and properly inject them into patients. They use gene editing to
disable a gene in the donor cells that would recognized all of the recipients
cells as foreign and attack them.
Just after three months, Layla was given a second bone marrow
transplant to restore her immune system. She will continue to have test
regularly until doctors confirm the cancer is gone. Other patients have begun
being treated with these cells. A step forward to finding a cure for leukemia.
A similar study was completed by Michel Sadelain in New York City in
2013. After just 8 days of starting gene therapy, David Apontes, along with 4
other patients, had no signs of their incurable leukemia. The therapy

identifies a molecule unique to the surface of cancer cells, and genetically

engineers a patients immune cells to attack it. Just like in Laylas story,
doctors extracted other immune cells called T-cells from the patients and
treated them with a harmless virus. They were genetically engineered to
attack all cells bearing CD19. When the engineered T-cells were injected
back into the patients, they rapidly killed all B-cells.
Each patient was given additional bone marrow transplants to ensure
they regrow a healthy immune system. Researcher Paul Moss says, Although
its early days for these trials, the approach of modifying a patients T-cells to
attack their cancer is looking increasingly like one that will, in time, have a
place alongside more traditional treatments. Sadelain and his team have
now begun investigating how to attack other cancers. He is looking to target
pairs of molecules that only occur together on cancer cells.
This fatal disease has left families mourning over loved ones. Doctors
have dedicated their time to searching for the perfect antidote. Gene therapy
has become a huge asset in this process, giving over a dozen people longer
time to live. They are continuing to perfect these trials and are hopeful that
one day they will bring this incurable disease to a steady halt.

Resources
Coghlan, Andy. Gene therapy cures leukemia in eight days. New Scientist.
March 26, 2016.
Lajeunesse, Sarah. A Cure for Leukemia? Penn State Ag Science Magazine.
2012.
Le Page, Michael. Gene editing saves girl dying from leukemia in world first.
New Scientist.
November 5, 2015
Nordqvist, Christian. Leukemia: Causes, Symptoms and Treatments. MNT.
January 8, 2016.