Clinical trials for a new Danish stem cell treatment could mark the end of Parkinson’s disease

Last year, 49-year-old Jette Oppelstrup invested in a bathroom on the ground floor of her townhouse in Herlev, a Copenhagen suburb. In the lower left corner of the mirror above his sink in the new bathroom are two stickers that read: “FUCK PARKINSON, NEVER GIVE UP”.

Oppelstrup was diagnosed with Parkinson’s disease in 2017. The disease rarely strikes people under the age of 60 and is best known for afflicting people with noticeable tremors on their hands and slowed movement. In the future, she may not be able to go up to her old bathroom as often as nature calls her.

But for people with Parkinson’s today, unlike just a decade ago, the phrase “never give up” is no longer just a motivational saying. A new treatment for Parkinson’s disease that uses stem cells to artificially create new dopamine-producing nerve cells is expected to enter clinical trials in human patients later this year. Due to the long course of Parkinson’s disease, many patients who are diagnosed today may live long enough to be eligible for this type of treatment and have their lives spared the difficult later stages of the disease – and may -to have their motor function restored.

That is, of course, if the treatment is successful.

Jette Oppelstrup’s bathroom mirror that reads “FUCK PARKINSON NEVER GIVE UP”.

Sebastien Skov Andersen

A decade in the making

Nearly one million people are living with Parkinson’s disease in the United States alone, according to the Parkinson Foundation. About 60,000 people are diagnosed with Parkinson’s disease each year, and that number is expected to reach 1.2 million by the end of this decade. It is the fastest growing neurological disorder in the world, with the global number of people diagnosed doubling from 3 million to 6 million between 1990 and 2015. If this trend continues, the number of cases will doubled again by 2040.

“If the rate at which the growth of Parkinson’s disease continues, we will exceed the capacity to manage all the consequences of leaving a chronic neurodegenerative disease unchecked,” said Michael S. Okun, a neurologist at the University of Florida. and one of the world’s leading Parkinson’s scientists, told The Daily Beast.

After decades of research, what we know so far is that Parkinson’s disease is caused when dopamine-producing nerve cells in the brain die too quickly. Often referred to as the “happiness hormone,” dopamine is essential for relaying signals from the brain that instruct different parts of the body to move. A shortage of dopamine will cause tremors and slowed movement. These symptoms only get worse over time and make it extremely difficult to perform even simple activities in the later stages of the disease.

And Parkinson’s disease can also lead to cognitive effects, such as short-term memory loss, difficulty staying focused, and problems with impulse control.

For all these reasons, “it’s hard to really trust that a treatment will be developed in time,” Oppelstrup told The Daily Beast. “But I will be the first in line for practice. I just have to find where to register.

Jette Oppelstrup was diagnosed with Parkinson’s disease in 2017. She is skeptical that treatment will be available in time in her lifetime, but says she will be first in line for treatment.

Sebastien Skov Andersen

The new trials are the culmination of more than 10 years of work by scientists from Lund University in Sweden and the University of Copenhagen in Denmark, with support from Danish pharmaceutical giant Novo Nordisk. This raises high hopes that we may finally have something to transform the lives of millions of people with Parkinson’s disease.

The treatment itself works by harvesting stem cells from excess fertilized eggs from in vitro fertilization (IVF) clinics. These cells are derived from the early stages of fetal development, which means they can be cultured into any other type of cell found in the body. This includes dopamine-producing nerve cells that are lost in Parkinson’s disease.

“What we then do is add a series of very specific proteins, called growth factors, so that the cells turn into the necessary dopamine-producing nerve cells in the Petri dish,” said Agnete Kirkeby, a neuroscientist. from the University of Copenhagen who helped pioneer the new treatment, told The Daily Beast. “Then the cells are surgically inserted into the brain using a special type of needle and will hopefully produce dopamine in place of the patient’s dead nerve cells.”

According to Kirkeby, the team has been testing this therapy in animal models since 2012. Implanted cells have been shown to survive transplantation in rats and communicate with other cells in the body to restore dopamine production and, by extension, motor function.

The process of developing such a treatment is long because the amount and sequence of growth factors used must be incredibly precise in order to develop stem cells into nerve cells. Finding the right combination of growth factors comes down to educated trial and error editing.

Another treatment known as deep brain stimulation, where implants are used to deliver direct electrical stimulation to certain parts of the brain, has already been successful in delay or lessen physical symptoms of Parkinson’s up to several years. Since neurosurgeons have already established a safe surgical method to implant these electrodes, they can use a similar method to safely cut into the brain and deliver the stem cells, Kirkeby explained.

“We hope to start trials this year, and if they go well, the stem cell treatment can hopefully hit the shelves and be used to treat patients with Parkinson’s disease in eight to ten years. “, she said. If the treatment is approved after initial trials, Novo Nordisk is expected to move the treatment to larger scale trials.

It’s not just that the treatment could bring relief to millions of people around the world. Since Parkinson’s disease has such a long natural history and patients can be severely debilitated for years and years, being a caregiver is a full-time job. Many may need time off work to care for loved ones, and the physical toll this care takes on their bodies can lead to their own future health issues.

“We are facing a problem which, if left unaddressed, will have catastrophic consequences. Not only because of patient suffering, but also the economic consequences and the ability of our healthcare systems to defend themselves,” Okun said.

High hopes, realistic expectations

Yet, while there’s a fair amount of hope for what new stem cell treatment might do for patients and their families, there’s a history of new therapies dying out under rigorous testing.

“Very rarely do we actually cure a disease,” Ray Dorsey, a neurologist at the University of Rochester Medical Center, told The Daily Beast. “Much more often we find a way to prevent them. Think about what it takes to cure someone with Parkinson’s disease after half of their nerve cells die. I just think we need to take a cool, rational look at this and say it’s really hard to do.

And while we have a clear picture of the dopamine connection, we have very little understanding of exactly why nerve cells will start producing less dopamine in some people. Age is obviously a big correlate, and some research has found associations with the use of certain pesticides and metals due to the disproportionately high number of rural dwellers with Parkinson’s disease. Some scientists believe that if we lived long enough, all humans would eventually develop Parkinson’s disease as cells begin to die due to old age.

Karin Christiansen, 57, and her husband Keld Hansen, 65, live in Odense, Denmark’s third largest city. In 2018, Karin was diagnosed with Parkinson’s disease after several years of living with symptoms including severe pain in her right leg and difficulty keeping her balance.

Karin Christiansen and her husband Keld Hansen.

Sebastien Skov Andersen

“The worst thing is the fear of disappearing in my mind,” Christiansen told The Daily Beast, referring to the risk of developing dementia as a long-term symptom of Parkinson’s disease. Even though she is participating in closed trials for another new experimental treatment that has significantly helped relieve her pain and regain control of her bodily functions, she finds it hard to believe that any treatment could “save” her at all. time, not even eradicate cells.

“I’m afraid that once you’ve been sick it’s hard to get back to normal,” Hansen added. “It’s his brain that’s being destroyed, and I’m not sure it’s possible to restore it.”

So while rats may soon be swapped with humans and stem cell treatment may be imminent, Karin tries not to pay too much attention to it. In all likelihood, giving her hope would only end in disappointment, she said.

Instead, she isn’t so much looking forward to what treatments like this might do for her, but rather what they might do for future generations.

“It’s probably mostly people who will be diagnosed in the future who will be lucky enough to benefit,” Christiansen said.

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