Before we get to the therapeutic stuff, here is a reminder of
the main problem people with Parkinson’s disease face.

Researchers are reasonably sure that the accumulation of a
protein called alpha-synuclein is responsible for neurons dying
in people with PD. However, there are two competing theories as
to how it builds up and spreads, the threshold theory and the ascending theory (also called the
prion hypothesis). The ascending theory states that
alpha-synuclein spreads from cell to cell, ‘infecting’ cells as
the protein moves up through the brain. The threshold
theory recently put forward by Dr. Ole Isacson and Dr. Simone
Engelender, proposes that alpha-synuclein builds up
independently in each affected cell.Auto Draft

Regardless, an improved understanding of exactly how such
proteins misfold and clump together is at the heart of the
riddle that is Parkinson’s as well as a long list of other diseases. Thankfully a
number of labs around the world have been working on this
sticky problem. Additionally, if anyone wants to help you can
do so very easily from any computer, watch this video to learn
how.

The Age of Personalized Medicine

The ongoing revolution in genetics is playing an
increasingly important role in our understanding of the disease
while also revealing why it varies so much from patient to
patient. There have been dozens of mutations and variants
associated so far with the disease. We are just beginning to
understand the role our genes play in the development of
neurological diseases but an immense amount of progress has
been made in the last 15 years since the human genome was
sequenced. Now that sequencing costs have plummeted to around a
thousand dollars we are on the verge of a new era in medicine
that promises to give patients treatments tailored to their
specific condition.

“Personalized medicine is healthcare based on your unique
genetic and molecular blueprint. Each individual has distinct
genetic makeup, biomolecule
and metabolic profiles, set of gut microbes, and so on.
Similarly, there is no one-size-fit-all in healthcare. How you
stay healthy or how you are treated for disease should be
catered to match your unique profile. Knowledge of your
genomics, proteomics, metabolomics, microbiotics, and other
bioinformatics allow for the
improvement in the quality of life, from disease prevention to
therapy best suited to you.” (from the Personalized Medicine Initiative in British
Columbia.)

A better understanding of genetics will help unlock a cascade of other
problems that surround this disease including mitochondrial dysfunction, lysosomal degradation, neuroinflammationgut bacteria, and epigenetics, among others. And thankfully there is
now a large interconnected global community of researchers
working to solve these problems with more resources and better
tools than in all of human history combined. This growth in a
variety of public and private sector health initiatives across
disciplines has lead a growing number of experts to believe
that we will make more progress in the next decade than we did in
the past century
, which is good reason to be hopeful
considering what medicine was like a hundred years ago.

This medical revolution will be further bolstered by new and
improved imaging techniques. A big part of the problem we
still have with this disease is that we can’t actually see what
is wrong. Every person who has PD has slightly different
symptoms but we don’t really know why primarily because we
can’t accurately see inside patient’s heads. Soon a new line of imaging techniques will be
available that will give surgeons and researchers a much better
understanding of what is going on inside the heads of each
patient.

In addition, there are some immense ongoing collaborations such
as the European human brain
project
 and the U.S. brain
initiative
 that are trying to do for the brain what
the human genome project did for our understanding of the
genome. If successful it will give researchers unprecedented
insight into how our minds are pieced together.

Then there are the new therapies themselves.

Current Treatments That are Getting Better Every
Day

Levadopa – For 50 years now this wonder drug has
brought relief to millions. Of course, problems still persist,
namely in getting it past that stubborn blood brain barrier and
making sure a more steady supply is delivered to reduce on/off
fluctuations. To get around some of those problems we now
have patches, slow release and extended release capsules,
as well as intestinal pumps that deliver a steady flow of the
drug directly into the intestines. Of course this drug is not
an ideal solution as there are nasty side effects that come
from long term use, predominantly dyskenisia which gives people
the motor control of a blob of jelly, but for now, it is still
the best stop-gap solution we have.

Deep Brain Stimulation
This science-fiction wonder
has become the undisputed Queen
of modern treatments. It has already proven itself to be a
miracle worker, re-animating hundreds of thousands with its
electric wizardry. It too is steadily improving, from John
Palferman’s book, Brain Storms, “Instead of
implanting devices that simply deliver a continuous electrical
stimulation, they are developing technologies that deliver
stimulating jolts only when required. ..The idea is to design
DBS so that the system can monitor the electrical activity in
the basal ganglia, and when it detects an abnormal signal, it
can respond automatically with an appropriate stimulation. A
smart device…”

Therapies Soon to be Available (2 to 5 Years)

New Drugs – There is a long list of promising drugs that are already
in clinical trial
. Some of these drugs have the
potential to not only offer symptomatic relief but hit the holy
grail that is actual disease modifying therapies.

Neuromodulation techniques – A number of
novel neuromodulation techniques are being tested for
clinical use. The most prevalent is called
transcranial magnetic stimulation in which magnets are
attached to the outside of patient’s heads that send a
focused electric current deep into the target areas of the
brain. Already an approved therapy for depression, TMS is now being tried in PD.

Therapies on the Horizon (5 to 10 years)

Immunotherapies – The relatively recent identification
of alpha-synuclein as playing a key role in disease formation
has lead researchers to believe that we may be able to harness the bodies immune system to stop
the protein from clumping while also mitigating the bodies
natural inflammatory responses that damages neurons.

Pharmacogenetics – The genetic revolution has
spurred the development of a relatively new field of
pharmacology called pharmacogenetics. Eventually, instead of making
one drug for everybody, we will be able to tailor drugs to
better fit each person’s unique condition.

Stem Cell Therapies – Though there were a series
of trials in the 90’s that had mixed results,
recently a number of labs around the world have begun
reexamining the therapeutic potential of stem cells. This is
thanks in part to the 2007 discovery of a new type of stem
cell called IPS cells which allow researchers to grow
fully functioning stem cells from patient’s own skin cells.
This has opened the door to a new set of therapies while also giving us
better disease models. Since those first trials we
have also made a series of other advances in our understanding
of how to use stem cells which has lead to some stunning results in trials on other
apes
. Some labs are hoping to push forward
with human trials starting at the end of this year.

Gene Modification Therapies – As discussed earlier,
the field of genetics is blowing up and one of the biggest
benefits to society that will come from it is a new set of
therapies called gene modification therapies. The most
popular one today is called CRISPR, a technique that already
allows researchers to cut and paste genetic code, changing the
genome of living organisms. A
number of articles
have come out touting these kind of
gene-editing techniques as the future of
medicine
. This first use of CRISPR was in a
lung cancer patient in China last
fall, but it is also being used to help us understand neurodegenerative
disorders
 including Parkinson’s disease.

Future Treatments (10+  years)

Direct Programming – In conjunction with gene therapy,
direct programming is believed to be the final solution to
the problem of neurodegeneration. It is a subset of the new
field of synthetic biology that will
eventually allow us to change cell types in living organisms.
For example, in people with Parkinson’s disease we will be
able to reprogram other healthy cells in the affected
area, such as glial cells or astrocytes, and directly turn them
into dopamine-producing cells.

And If All Else Fails, Perhaps Robots Will Figure It Out

When it comes right down to it, the reason why we have not been
able to cure a lot of the diseases that are still with us
today, such as neurodegeneration or cancer, is that there are
an incredible number of factors to consider when trying to
treat them, possibly too many for any human, or even any group
of humans, to make sense of. But there might be a solution to
this problem as we are now figuring out ways to export more and
more of our intellectual abilities into computers. Already
computers have become as good as humans at diagnosing certain
conditions
, and a staggering number of healthcare
companies
 have now invested heavily in
applying artificial intelligence to the medical
industry. This, along with further advances in nanotechnologyhas a lot of potential in helping us
understand diseases such as Parkinson’s and may reveal novel
insights into how to treat them.

As you can see, there is plenty in the pipeline. While there
may not be any magic bullet, there is no doubt that we will
continue to see improvements in the treatment of Parkinson’s
disease that will benefit millions. While it is important to
remain skeptical of all the promises being made, there is very
good reason to believe that afflictions such as Parkinson’s
disease may one day be a thing of the past.


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