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While Michael Fox, left, may be among the more famous Parkinson's disease patients,
it's ailment impacts 7 million to 10 million worldwide. There is no cure.
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The emails pour into Elena Batrakova’s computer daily.
Batrakova, a researcher at UNC-Chapel Hill’s Eshelman School of
Pharmacy's Center for Nanotechnology in Drug Development, didn’t have a
personal stake in Parkinson’s disease, a degenerative neurological disorder.
There’s no father, brother, child suffering from the ailment, which can impact
everything from motor skills to mental clarity. There's no childhood
friend-turned-patient to inspire the mission.
But the emails, hopeful messages from patients and their
relatives, give her that personal connection.
“You feel that you’re
doing something that is very important,” she says, pausing. “I just think that
it’s a dream of maybe every investigator to see at least one person whom you
can help by your work. So I’m just really eager and hopeful that one day I
could look in the eyes of this person and he’ll tell me he is thankful because
it’s working.”
Batrakova is hopeful about a technique that could achieve the
very thing those Parkinson’s patients are desperate for: A treatment.
“We are not inventing new drugs,” she explains. “We are taking
old, good drugs, which cannot be delivered because of the blood brain barrier …
and we formulate them into drug delivery systems.”
The blood brain barrier is exactly what it sounds like – a
barrier that protects your brain and, in doing so, acts as a wall. But there’s
one particle that can get through – macrophages, white blood cells. When
inflammation occurs, they’re signaled and respond.
“So I realized, why don’t
we use macrophages as a drug delivery system?” she says. “It’s a very potent,
powerful, nature-prepared mechanism.”
And, when genetically modified to produce anti-inflammation
proteins and neuron growth factors, they could – or so the theory goes – repair
nerve cells. As cell death causes Parkinson's symptoms, the applications for
diseases sufferers could be huge.
“Basically, you have a Trojan horse that goes across the blood
brain barrier to the inflammation site, and it’s bringing something very
powerful,” she says.
The macrophages can then be injected intravenously into the
patient, where they will be able to teach neurons to make the protein for themselves by delivering their own version of an instruction manual: DNA.
It’s complicated – and not just the science. A long regulatory
process stands between the science and its use in actual patients. Currently,
the team, armed with a $50,000 grant from the N.C. Biotechnology Center, is
about to go into preclinical testing, researching how to use the intravenous
method in genetically engineered mice.
Even if everything goes perfectly, it will be years before she
can respond to those emails with real trial data.
http://www.bizjournals.com/triangle/blog/techflash/2015/09/unc-researchers-mission-to-treat-parkinsons.html
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