Is
this the Beginning of gene-editing medicine?
Does the smiling face of Layla Richards
mark a new era in genetic medicine that could change all our lives?
The story of Layla Richards may be just the
beginning of a new chapter in medicine.
Layla Richards developed CLL as an infant
and the day before her first birthday, Layla's parents were told she was going
to die. All treatments for her leukemia had failed
An experimental treatment that had only
been tried on mice was her only hope. Her family, her doctors and a
biotechnology company decided they had to try it.
The biotechnology company genetically
engineered some cells that were designed to kill Layla’s cancer.
Today, because of this experimental
treatment, Layla has no trace of leukemia.
We could be seeing the start of a
revolution in medicine. The same procedure that worked in Layla, could be used
to treat, not only other cancers, but inherited diseases.
Genetic engineering is a whole new field in
which missing DNA be inserted and defective DNA can be corrected. This is what
doctor’s did for Layla.
This revolution may have started around the
turn of the millennium when scientists were saying that gene therapy was going
to transform the world. While it has taken 15 years for gene therapy technology
to develop, the experimental treatment that saved Layla’s life may be just the
first miracle of this new technology.
As with all advances in science, caution is
the word for the day. Gene-based therapy involves changing our DNA. These are
the instructions for building and running every part of our body.
Early attempts to use this technology ultimately
resulted in patients developing leukemia and further attempts were abandoned.
DNA was being inserted, in such a way, that
it disrupted the natural functioning of some cells and they became cancerous.
Since then the process has become much more
precise. The viruses being used can precisely place DNA into safer sites in the genome.
This new precision has been described as the difference between using an ax and
a laser. The viruses used act as a guide that finds its way to specific sites
in our DNA and a pair of molecular scissors that can edit the DNA.
DNA is our blue print of life
Will doctors be able to harness our DNA for new treatments?
A similar technique is already being tested
in HIV-positive patients. The aim is to take the patient's cells out of the
body, give them HIV protection, and then put them back in.
The doctor involved in treating Layla's
leukemia, told the BBC: "The technology is moving very fast, the ability
to target very specific regions of the genome has suddenly become much more
efficient.
"The technology itself has got
enormous potential to correct other conditions where cells are engineered and
given back to patients or to provide new properties to cells that allow them to
be used in a way we can only imagine at the moment."
Re-arming the immune system to target
cancer and a wide range of inherited disorders is in the sights of doctors.
It will be easiest for them to use cells
that can be taken out of the body, modified and reinserted rather than trying
to edit them still in the body.
So diseases of the blood or immune system -
such as MPNs or sickle cell anemia - will be easier targets than kidney or
heart defects.
Doctors are predicting an
"explosion" in the use of such genetic engineering in the next 10
years.
After the overhyped false dawn fifteen
years ago, gene-editing is now, it seems, about to arrive.
