New partnership to advance cell replacement therapy for Parkinson’s
Smartcella, Karolinska Institute team up to further develop novel treatment
Swedish-based Smartcella has entered an agreement with a research team from the Karolinska Institute for the exclusive rights to advance a cell replacement therapy for Parkinson’s disease into clinical development and commercialization.
The Karolinska Institute team in Sweden, led by Johan Ericson, PhD, a professor of developmental biology, has developed a new protocol that uses pluripotent stem cells to produce neuron progenitor cells. These nerve-cell progenitors then mature into dopamine-producing neurons — nerve cells that are gradually lost in Parkinson’s.
In preclinical studies, where the cells were implanted into the brain of a Parkinson’s rat model, progenitor cells were able to generate dopaminergic neurons and relieve motor symptoms associated with the disease.
“Johan’s research around dopamine-producing neurons shows a clear improvement compared to existing methods,” Niklas Prager, Smartcella’s CEO, said in a company press release. “By combining Johan’s protocol, which has better results and efficacy, with our [good manufacturing practice] … and process development capabilities we will have an advantage in bridging the gap between labs and clinics.”
Partnership aims to advance therapy toward clinical testing in patients
Ericson, who said “the preclinical results we have so far are truly encouraging,” noted that the team is looking forward to advancing the therapy.
“We are confident that our translational approach holds great promise for the establishment of a competitive and therapeutically [highly] effective cell product to treat Parkinson’s disease in the future,” said Ericson, who also noted that the research team has “adapted the cell manufacturing process into a scalable format” that will aid in its development.
“With the process development, scale-up capabilities and knowledge of Smartcella, we will be able to accelerate the path [toward] clinical studies in humans,” Ericson said.
Parkinson’s disease is caused by the progressive dysfunction and death of dopaminergic neurons, the nerve cells that produce dopamine, in a region of the midbrain called the substantia nigra. Dopamine is a neurotransmitter, or signaling molecule, involved in motor control.
As Parkinson’s disease progresses, patients experience a gradual deterioration of their motor functions. Over time, the effectiveness of available treatments, which primarily aim to alleviate these symptoms, tends to diminish.
Cell-based therapies have emerged as a potential treatment option for Parkinson’s by aiming to replace the lost dopaminergic neurons. These therapies use pluripotent stem cells, which possess the unique ability to differentiate into various cell types, including dopaminergic neurons.
We are confident that our translational approach holds great promise for the establishment of a competitive and therapeutically [highly] effective cell product to treat Parkinson’s disease in the future. … With the process development, scale-up capabilities and knowledge of Smartcella, we will be able to accelerate the path [toward] clinical studies in humans.
Ericson’s team has developed a pluripotent stem cell-based technology that differs from other protocols used to obtain dopaminergic neurons from stem cells. Their approach uses retinoic acid, a molecule involved in neural development.
According to the researchers, their protocol increases the yield of dopaminergic neurons after transplant, resulting in smaller transplants with hallmark characteristics of existent dopaminergic neurons in the brain.
Prager called the new agreement with the research team “an important addition to our project pipeline.”
“The prospect of delivering allogeneic [donor derived] innovative and safe therapies to Parkinson’s disease patients, with the potential to transform their lives, is both groundbreaking and increasingly within reach,” Prager said.
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