A company in Luxembourg, Organo-Therapeutics, is directing its focus utilizing human stem cell technologies to create “mini-brains” to develop treatments for Parkinson’s Disease instead of animal experimentation.... CAARE believes human-relevant technologies like Organo-Therapeutics’ mini-brains will serve as a model for other scientists to follow, bringing much-needed treatments to patients, and helping to eliminate the extreme, needless suffering of animals.
Parkinson's Disease is a neurological disorder primarily caused by
degeneration of specific neurons that results in tremors, rigidity and loss
of voluntary movement. Like many other devastating neurological diseases,
treatment is limited and there is no cure. Millions of dollars have been
invested over decades to study animal models with no real success.
Now a company in Luxembourg, Organo-Therapeutics, is directing its focus
on utilizing human stem cell technologies to create “mini-brains” to develop
treatments for Parkinson’s Disease. Founders Javier Jarazo, PhD and Jens
Schwamborn PhD, both professors at the University of Luxembourg, hope to
demonstrate that disease research can be done without the use of animals.
To understand how much more promising this approach is over animal
experiments, it’s necessary to know how animal models are created. Because
scientists do not have a full understanding of what goes wrong in
Parkinson’s Disease, animal research is based on chasing down a number of
theories involving gene mutations, infections, and inflammation. In other
words, they are largely groping in the dark.
Using neurotoxins that cause brain inflammation, or gene insertions that are
suspected to play a role in Parkinson’s, researchers turn animals into
so-called models of Parkinson’s Disease.
By contrast Organo-Therapeutics looks directly at examining the
pathophysiology of human Parkinson’s cells. They create “mini-brains” by
taking donated stem cells from patients suffering from Parkinson’s Disease
and differentiate them into 3D structures resembling the midbrain, the part
of the brain that is primarily affected by Parkinson’s Disease. These
mini-brains will be treated with two drugs believed to prevent the nerve
cell death that is typical in Parkinson’s. Next, the mini-brains will be
analyzed with high-end microscopy and computational methods to confirm the
results.
This precise, clear-cut and human-relevant model is a world apart from
chaotic, ill-defined and brutish animal experiments.
Typically, monkey models are created through the use of neurotoxins, as in
an experiment in which the compound MTPT was injected directly into the
brain to cause a Parkinsonian-like syndrome. Monkeys had electrodes
implanted for additional brain mapping studies, while placed into restraint
chairs during experiments.
These monkeys spend their lives in confinement, afflicted with brain damage,
and tethered by wires protruding from their skulls. There is no way to
measure how much their misery affects the scientific outcomes.
In another disturbing experiment, scientists wanted to see whether a specific gene influenced by infection would show a connection to Parkinson’s Disease. They caused severe sepsis in mice by intravenous infusion of Salmonella, while inducing encephalitis, a painful brain infection, in newborn mice by injecting the virus into their noses. This ruthless and unfocused research was simply to explore a theory, that if correct, would impact only 2% of Parkinson’s patients with that particular gene defect.
Parkinson’s patients are not ill because they received a dose of
neurotoxin. Their disease arises from some intricate and microscopic
dysfunction that occurs within the cell or cell interactions. It’s clear
that a method that can grow and examine cells from Parkinson’s patients is
far superior to damaging animals’ brains to glean information that may have
relevance for Parkinson’s Disease.
In fact, earlier this year, Organo-Therapeutics released a publication
demonstrating for the first time that their mini-brain model exhibits
“disease relevant alterations” characteristic of Parkinson’s Disease.
As Dr. Schwamborn explained in an email to CAARE: “Animal models are not
able to recapitulate complex human diseases like Parkinson’s Disease
adequately enough to be helpful for efficiently identifying new therapeutic
strategies. We are convinced that using human patient specific models, like
our mini-brains will dramatically reduce the failure rate, and therefore
help to bring better medications into clinical testing and eventually to the
patients.”
Organo-Therapeutics is raising funds to begin this exciting work with their
crowdfunding campaign:
Medication development free of animal testing.
CAARE is pleased to have donated to support the campaign and we encourage
others to do so as well. We believe human-relevant technologies like
Organo-Therapeutics’ mini-brains will serve as a model for other scientists
to follow, bringing much-needed treatments to patients, and helping to
eliminate the extreme, needless suffering of animals.
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