HYPE?

Media portrayal:

HOPE?

Scientific interpretation:

Reprogrammed stem cell trial has officially begun

Original article: ‘Reprogrammed’ stem cells implanted into patient with Parkinson’s disease: A man in his 50s is the first of seven patients to receive the experimental therapy, David Cyranoski. Nature: November 14, 2018. 

The takeaway

The first clinical trial to use reprogrammed stem cells for Parkinson’s has begun in Japan. The first implantation procedure took place in a patient in October 2018. If no complications arise, another 6 patients will be included in the trial, which should be complete by 2020.

Why is it important?

This is a first of its kind clinical trial using reprogrammed adults cells, and represents a major step toward a sustainable, potentially disease modifying therapy.

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IMPACT

  • Novelty 90% 90%
  • Proximity 80% 80%
  • Deliverability 60% 60%

Impact Opinion

“This is a hugely exciting clinical trial as it involves the first time neurons derived from induced pluripotent stem cells are being injected into the brain. If this approach is successful, it could open the door to a more personalised procedure for cell replacement therapies not only for Parkinson’s, but other neurodegenerative conditions as well. One could invisage a future in which skin cells are collected from an individual, transformed into stem cells and subsequently into neurons, before they are then transplanted into their brain. This approach would hopefully avoid the need for suppressing the immune system (which is currently required in order to stop the immune system from killing the transplanted cells). It is important for the community to understand, however, that this is not a curative treatment. Cell replacement therapies are simply intended to replace lost cells, they will not slow the progression of the condition. But if cell replacement therapy can be used in conjunction with a disease-halting treatment, we will certainly be on our way to stopping Parkinson’s.”

Background

Sourcing stem cells for transplantation has posed major ethical and logistical challenges for years. Researchers in Europe, the US and Japan who were developing protocols for the production of stem cells joined forces in 2014 to form GForce-PD (see our related summary on PM). The aim of this work has been to develop a reliable, safe source of dopaminergic cells for transplantation into the brain of people with Parkinson’s. The source of cells being used in the ongoing Kyoto clinical trial are iPSCs: induced pleuripotent stem cells, derived from reprogramming adult cells (such as skin cells) into a more primitive state from where they have the potential to grow into dopaminergic precursor cells.

The details

Scientists at Kyoto University produced these dopaminergic precursor cells from skin-based iPSCs obtained from an anonymous donor. In October 2018, 2.4 million of these dopamine precursors were surgically implanted into the brain of the first patient. During the 3-hour long procedure, they were placed into 12 different sites in a region of the brain called the putamen. This is where the majority of dopamine is released in the brain to help maintain normal movement. By placing the dopamine precursor cells in the putamen, the researchers are hoping that the cells will provide dopamine where it is most needed.

Next steps

This patient, who is in his 50s, will be observed for six months and, if no complications arise, will receive another 2.4 million dopamine precursors. Eventually, the Japanese team plan to treat another 6 individuals in this manner. The safety and efficacy data from this first of its kind clinical trial should be available by 2020.

Original article: ‘Reprogrammed’ stem cells implanted into patient with Parkinson’s disease: A man in his 50s is the first of seven patients to receive the experimental therapy, David Cyranoski. Nature: November 14, 2018. 

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