Parkinson's Patients Preparing For 'Breakthrough' Clinical Trial To Reverse Symptoms
A walk in the park can be exhausting for Chris Whitmer. His once athletic stride has been weakened by Parkinson’s disease.
“Outwardly, you probably notice the tremors the most. You know, the shaking, the stiffness, how somebody walks,” said Whitmer, 58, a married, father of two. “The biggest thing I feel is the fatigue,” he said. “It just makes you extremely tired.”
Over the past dozen years, his symptoms have progressed. The disease is not fatal but it can leave a person completely debilitated.
“I don’t want to think about what I’ll be like 10 years from now, and what it’s going to feel like. I want to be around for my kids,” he said, choking back tears.
Parkinson’s symptoms generally include tremors or shaking, weak or aching muscles, and difficulty walking and speaking, along with depression or apathy. While some symptoms can be eased for a while with medication, there is currently no cure.
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Whitmer’s cells from his skin are his biggest hope. The former tech industry businessman is one of 10 people preparing to undergo a brain cell transplant in San Diego as part of a first-of-its-kind clinical trial.
“I think it’s going to be the first successful treatment of Parkinson’s because everything we’ve done to now with drugs and therapies and deep brain stimulation... just treats the symptoms,” he explained. “This will be the first treatment, I think, that will reverse the symptoms.”
If all goes well, his health will be restored, within months.
Replacing Brain Cells To Reverse Symptoms
Parkinson's kills brain cells that make a substance called dopamine. Dopamine allows nerve cells to communicate with muscles.
“So what we propose to do is replace those neurons in the brain with the people’s own dopamine neurons,” said Jeanne Loring, a stem cell scientist with Scripps Research Institute in La Jolla.
The process starts with taking a small skin biopsy from a patient’s arm. The skin cells are transformed into master cells called “induced pluripotent stem cells,” which can make any cell or tissue in the body, including neurons. The cells can also regenerate themselves.
“The reason pluripotent stem cells are so important is that unlike the stem cells living in your body right now — they are cells that can regenerate certain parts of your body, not very well — these cells can make every cell type in the body including nerve cells,” Loring said.
Loring has mastered the procedure of turning pluripotent stem cells into dopamine neurons. She said other researchers are trying a similar approach, but they are using embryonic stem cells or other cells that are not from the patient.
“The difference between our group, what we’re planning and the others, is that we’re the only ones using a person’s own cells,” Loring emphasized.
“It’s reasonable to hope right now,” she said. "We expect to start seeing some improvements in the patients, maybe 6 months or certainly by a year."
Loring’s experiment, more than 5 years in the making, has been successfully tested in animals. The project is currently under review by the FDA.
If all goes well during the first trial in 2019, she hopes to expand the treatment to a large number of patients soon after.
“Stem cell replacement therapy is a reality that’s going to become a much more, a much larger part of medicine over the next few years,” Loring said.
Building A Foundation
Getting Loring’s research to a potential clinical trial has involved climbing mountains for fundraising, opening a new lab in Torrey Pines to reduce overhead costs and starting a foundation to secure grants and donations.
“It feels like we’re on a freight train now on a downward slope sliding into FDA approval for the clinical trials,” said Jenifer Raub, director of the Summit For Stem Cell Foundation.
The nonprofit was created in 2011 to back the project with a collaboration of patients, researchers, doctors and donors.
Raub, 60, is also a Parkinson’s patient and clinical trial participant. She recalled when her doctor diagnosed her disease 12 years ago.
“I didn’t believe it. I told him he was crazy and I walked out,” she chuckled.
But the disease has taken a toll, she said.
“You find yourself slowing down to the point of not being able to move at all,” Raub said.
Still, she’s filled with hope that she’ll soon be able to enjoy life’s simple pleasures once again.
“Playing with my grandkids and working in the yard,” she said, holding back tears.
It’s that hope that gets her up at 4 a.m. every day working to secure grants, organizing fundraising events and answering emails from hundreds of other Parkinson’s patients wanting to participate in a clinical trial.
Years ago when she applied for the trial, she had some anxiety, she said. But now, there’s no doubt in her mind the neuron replacement therapy will work.
“It’s genetically screened multiple times. It’s it. It’s the answer,” Raub said. “It’s going to be super exciting to have an answer for the outcry of 7 to 10 million people worldwide.”
Moving the Needle of Scientific Discovery
The project has drawn interest from researchers around world, including some top institutes in San Diego County. If successful, induced pluripotent stem cells could also potentially treat multiple sclerosis, ALS, Alzheimer’s and other neurological disorders that currently have no cure.
Stephen Wilson, executive vice president of the La Jolla Institute of Allergy and Immunology, recently reviewed Loring’s research and visited her lab.
“As a scientist who tends to root myself in skepticism and careful observation, I’d be lying if I didn’t say I’m extremely hopeful,” Wilson said. “I really want it to work.”
Wilson’s team, focused on preventing disease through a better understanding of the immune system, is studying the role of autoimmunity in Parkinson’s disease.
“Great science loves great scientist neighbors,” he said. “It’s never the case anymore that a single person in a room all by themselves has this Eureka moment and figures it all out A to Z. There are pieces that your work is going to explain and then gaps that you’re hoping that in parallel someone else can factor.”
Wilson said the project could move the needle of scientific discovery.
“There are some great treatments, there are a lot of trials in all these different areas but this is one of those big ones where it could just suddenly change it for the better,” he said.
“This truly is pioneering work,” Wilson said. “We’re going to find out how well it works, and we’re all going to have to collectively hold our breath a bit because we want it to work.”
No one wants it to work more than Chris Whitmer. He's hoping his walk in life will get easier. Because if it does, others with Parkinson’s could soon follow in his footsteps.