Taking a Closer Look at a First-ever Parkinson’s Stem Cell Study

A friend of mine sent me an article about a self-funded clinical trial using stem cells to treat a patient named George Lopez, who has Parkinson’s. I was intrigued. For many, myself included, the term “stem cells” meant only embryonic stem cells and evoked controversy.

I had numerous questions, but I was hopeful at the same time. This was a study to actually replace the dopamine neurons lost as Parkinson’s progresses — not only to manage symptoms. And the stem cells they used were not embryonic. They were created from Lopez’s body.

A press release read, “Reprogramming a patient’s own skin cells to replace cells in the brain that are progressively lost during Parkinson’s disease has been shown to be technically feasible.”

Many in the research community have found the results of this study were presented in a “clear and unbiased manner,” and it appears to be a “solid scientific study.” However, there are also articles debating various aspects of the study. I began to research this particular study, as well as clinical trials in general and stem cell research.

My editor suggested contacting the organizer of the study. Less than an hour later, Dr. Kwang-Soo Kim agreed to answer some questions that I emailed to him. I had the opportunity to be the voice of others with Parkinson’s who may have the same questions.

So, here is my first experience as a journalist with Parkinson’s.

Stem cells have more variety than I’d thought

Kim referred to this particular study as “very unusual and exceptional” because it was the first trial of its kind. It reported successfully using Lopez’s own skin to create induced pluripotent stem cells, which are cells (usually skin cells) that are reprogrammed to behave like stem cells. Those cells, which are autologous, were then transplanted into the Parkinson’s patient’s brain. This is different from embryonic stem cells (ESC), which is an allogeneic approach.

OK, what do autologous and allogeneic mean? Allogeneic stem cells are not taken from one’s own body, while autologous stem cells are created from one’s own body.

Kim explained: “There are different approaches, such as fetal cells and embryonic stem cells. These approaches could be complementary and not exclusive. For instance, the ESC-based approach may be more economic and fast because the cells can be available for many patients. However, these cells are allogeneic and need immunosuppression. In contrast, autologous cell transplantation does not need immunosuppression … but will take more time and budget. Once FDA approval is obtained, these different approaches can be compared with each other.”

The pay-to-participate trial

Lopez paid $2 million to make the trial happen — he said this was both for his own benefit and to pave the way for others with Parkinson’s. This has proven controversial.

“Medical and scientific groups, such as the International Society for Stem Cell Research, warn against ‘pay to play’ arrangements in which wealthy people buy their way into clinical trials,” STAT’s Sharon Begley noted.

However, Kim said that all pay-to-participate is under strict monitoring and regulation by the U.S. FDA and the Institutional Review Board.

He thinks personal financial status “does not/should not affect future studies and it is not a criteria in our next Phase I/II clinical trial, … which can only handle a small number of patients.”

As for future funding, they are seeking grants and philanthropic donations.

Kim also stated that “the most important issue is whether the patient fits to the criteria of our clinical team.”

As a person who participates in clinical trials, hearing all of that was enough for me. My questions had been answered.

Paving the way to new horizons

Two years after the study began, time seems to be on Kim and Lopez’s side. The cells are still alive, and the study has shown favorable outcomes. However, the one-person model was not a traditional blind study. Lopez knew he was receiving the therapy. Did his mindset create a placebo effect? Why only one patient?

“Since our approach is personalized therapy, even the big scale trial should start from an individual patient,” Kim said. “So, we thought that we better start with a single patient and gain all experiences and information to move on.”

The one-person study introducing personalized therapy seems to have paved the way for additional research. However, Kim noted that a double-blind study is needed to conclude its safety and efficacy.

I am grateful to Lopez who funded the trial and accepted the risks. He and Kim, together with all patients and doctors that participate in Parkinson’s research, have the people with Parkinson’s as their catalyst to keep pressing forward. Sometimes it’s hard to see the forest through the trees, but the goal is the same: a therapy for us all. Ultimately, that is what is important — even if it is one person at a time.

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Note: Parkinson’s News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Parkinson’s News Today or its parent company, BioNews, and are intended to spark discussion about issues pertaining to Parkinson’s disease.