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Parkinson’s disease patients improve after unprecedented stem cell therapy

 


Wednesday, May 13, 2020 (HealthDay News) -First, scientists treated patients with Parkinson’s disease with their skin cells-diverting them into key brain cells that eradicate the disease.

Two years after receiving treatment, his doctor reported that the patient had no side effects. On the other hand, his condition is stable or slightly better.

“The improvement is modest,” said Kwanskim, a principal investigator who heads the Institute for Molecular Neurobiology at McLean Hospital at Harvard University in Belmont, Massachusetts.

“But he deteriorated rapidly before this treatment and stopped worsening thereafter,” he added.

Kim said his team is planning to study treatments in additional patients.

For now, this patient is one demonstration that this approach is viable. However, James Beck, Chief Science Officer of the Parkinson Foundation, issued some notes.

One of the reasons for this is that, at least because of the technology that exists today, it will be difficult to “scale up” and make widespread use of this therapy.

Bec, who was not involved in the study, goes beyond that and the benefits to the patient are unknown.

The 69-year-old patient in this case reported a rapid improvement in quality of life measurements shortly after the procedure, which may reflect a “placebo effect,” Beck said. . And there is still no evidence that the treatment can slow the progression of Parkinson’s disease, he added.

According to the Parkinson Foundation, Parkinson’s disease affects approximately one million people in the United States.

The cause is unknown, but as the disease progresses, the brain loses dopamine-producing cells. Dopamine is a chemical that helps regulate movement and emotional responses. The most visible symptoms are related to exercise-threat, limb stiffness, and coordination problems, but their effects are widespread and include depression, irritability, memory and thinking problems. Everything gets worse over time.

Drugs and other treatments can reduce their effects, but there is no cure.

The approach used by Kim’s team is explained on May 14th New England Medical Journal.. This is based on the assumption that stem cells can serve as a source of new dopaminergic brain cells. Stem cells are primitive cells that have the ability to mature into different types of somatic cells.

In this case, researchers created so-called induced pluripotent stem cells, or iPSCs. They took cells from the patient’s skin and reprogrammed them into iPSCs that were similar to embryonic stem cells, which are the most capable stem cells to produce body tissue.

These iPSCs were then cultured to inherit the properties of dopaminergic neurons (neurons) that the team surgically transplanted into the patient’s brain.

Since those replacement cells were obtained from the patient’s own body, it was hoped that his immune system would tolerate them-without the need for drugs to suppress the immune response.

And that happened, Kim said. Two years later, imaging tests showed that the transplanted cells were still alive and functional. And when it comes to men’s quality of life, he has benefited. His stride has been improved, incorporating some of the previously abandoned activities such as swimming and cycling.

But Beck pointed out the timing: The patient reported a significant improvement in quality of life shortly after surgery-the time frame was too short for the transplanted cells to be effective.

“That was his perception,” Beck said. “And we know that the symptoms of Parkinson’s disease are really affected by the placebo effect.”

He pointed out another reason that replacing dopamine-producing cells may not be a “magic bullet”: while current drug therapies that act on dopamine help improve exercise problems. , Not useful for a range of symptoms other than exercise.

Kim said a lot of research is still ahead. For example, it is unclear whether the “dose” of dopamine-producing cells might be most effective, or at what point in the disease it might be best to intervene.

Another question is whether the transplanted cells will persist or eventually fail. This is a clear possibility. Whatever process originally caused Parkinson’s disease may still exist.

For now, Beck has emphasized that this procedure is taking place at two “best” medical centers and that treatment is only available outside the research setting. He pointed out that he sells a variety of self-describing “stem cell clinics” therapies throughout the United States that have not been certified or approved by the Food and Drug Administration.

“Some outpatient surgery centers on the road can’t get this,” Beck said.

For more information

Parkinson Foundation Parkinson’s disease management..

Source: Dr. Kwang-Soo Kim, Director, Institute for Molecular Neurobiology, McLean Hospital, Belmont, MA; Dr. James Beck, Parkinson’s Foundation, Miami / New York City Chief Science Officer. May 14, 2020 New England Medical Journal

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