For Harley Kornblum, the ability of a stem cell to renew itself
piqued his interest.
For Jerome Zack, pursuing stem cell research was a natural
transition in his work.
And for Hanna Mikkola, her desire to find a cure for leukemia
led her to UCLA.
These researchers, all from diverse scientific backgrounds, are
three of the many scientists studying stem cells at UCLA’s
Institute for Stem Cell Biology and Medicine.
In collaboration with other institutes and departments at UCLA,
the stem cell institute focuses on three major areas of stem cell
research: neurological disorders, HIV/AIDS and cancer.
The scientists spend their research time examining cells,
peering down microscopes, and looking to cure some of the most
prevalent diseases affecting people today.
Stem cells are unspecialized cells that can differentiate into a
specialized cell. Embryonic stem cells, extracted from the fetus in
the beginning stages after conception, have the potential to
differentiate into any cell or organ, while adult cells, which can
be extracted from any individual, can only develop into the tissue
they came from.
The former is more controversial as they involve the destruction
of the embryo.
As excited as Zack, Kornblum and Mikkola are about the potential
in their field, all three recognize that results are still far in
the future.
But Mikkola said that is no reason to be discouraged.
“It is very real and happening; it is not 50 years
away,” she said. “We can’t tell patients when it
will happen, we can only promise that we do our best and work very
hard.”
Tackling neurological diseases
Kornblum, a pediatric neurologist, studies neurological
disorders such as strokes, spinal chord injuries and brain tumors
in his lab. He said understanding and using stem cells is an
essential part of treating neurological ailments.
Specifically, Kornblum studies brain tumors and the role stem
cells play in causing them.
In the future, scientists could potentially use stem cells in
the lab to create working models of diseases, and by understanding
how a disease develops outside of a human body, scientists can
start to understand what is happening inside the body, he said.
Stem cells can be used to recreate lost cells within the brain
as well, and scientists are looking for a way to control the
cell’s development, which is currently not possible.
Kornblum cites Parkinson’s disease as an example of where
this technology could be used.
“With Parkinson’s, you lose a certain type of cell.
Stem cells can potentially make new copies of that cell and replace
them.”
Kornblum said stem cells can also be used as a type of Trojan
horse molecule, which he and other scientists are working towards
being able to do.
Many neurological disorders occur because the brain lacks a
specific enzyme needed to function properly.
In order to fix the problem, a modified stem cell could
potentially be used to carry the enzyme back into the affected
area.
Using stem cells as a cornerstone for new therapies will be a
step forward from what doctors are able to do now, Kornblum
said.
Many existing therapies work as a temporary solution to a
permanent problem, as they only seek to maintain function or delay
the progression of the disease. But new treatments based on stem
cells may be able to restore neurological function to patients, he
said.
But Kornblum has many hurdles to overcome before such treatments
can be realized because the “brain is one of the most complex
systems we know,” he said.
Fighting AIDS from within the cell
As an HIV/AIDS researcher working at the UCLA stem cell
institute, Zack is no stranger to hurdles.
Zack has been working to see how stem cells can be used to
combat HIV/AIDS, for which there is currently neither a vaccine nor
cure.
One particularly promising method he is researching is the use
of anti-viral genes in stem cells. These genes could potentially
prevent infection by blocking HIV’s entry into the cell or
keeping HIV from expressing itself.
Using an engineered virus, genes could be introduced into the
stem cell where they would be part of the cell’s DNA.
Potentially, the cells could then be reintroduced into the
patient’s body with the hope that as these cells replicate
more and more of the cells in the patient will have the anti-viral
genes, protecting the patient from HIV.
But in the lab experiments Zack has been conducting, not as many
cells are picking up the genes as he would like.
“The chances of the gene getting into the patient and
expressing itself are still fairly low. … (But) by that
technology the gene is at least in some of the cells. Whether the
gene will express itself is still under investigation,” Zack
said.
Zack said he hopes these new therapies will eventually be proven
effective.
“In theory, you would have a life-long set of cells that
are protected from the infection. In the absolute starry-eyed
world, there would be no need for therapeutics,” said
Zack.
However, Zack is quick to acknowledge that the application of
these therapies is still far in the future.
“In the HIV field, you never put a timeline on a new
therapeutic. It’s being tested now but it’ll be years
before it can be used.”
Stopping the spread of cancer
Mikkola studies cancer stem cells, a particular type of stem
cell that causes cancer and can exist anywhere in the body, in the
hope of learning why these stem cells are not doing what they are
supposed to do and finding a way to keep them from misbehaving.
Some researchers believe targeting cancer stem cells is an
important step in curing cancer.
In the case of leukemia, the specific type of cancer Mikkola
studies, cancer stem cells are anomalies of hemetaphoric stem
cells, which eventually differentiate into different kinds of blood
cells.
Mikkola is trying to find out which genes are responsible for
signaling these stem cells to divide and why cancer stem cells do
not seem to respond.
Responding to signals from the body is an important part of the
way cells are regulated. Cells know what to do and what is needed
of them by receiving signals from their environment, but sometimes
the signal will get stuck either in the on or off position. If the
signal is always on or always off, the cell stops listening, and
when that happens, cancer develops.
Mikkola said existing treatments are not necessarily solving the
problem.
Current treatments seek out and target any cells that are
dividing rapidly, which can have damaging affects on the
reproductive and immune systems, as well as causing other side
effects.
But Mikkola said these treatments do not actually target and
eliminate the root cause of cancer, so it will eventually
return.