When Robin Gordon-Cartier was first diagnosed with multiple sclerosis at age 50, she felt like she was “just waiting to die.”
Her busy life as a harpist, full-time teacher and traveling performer was slowly being eroded by the disease, as she succumbed to daily pain, fatigue and a progressively worsening limp. She stopped performing completely, since she couldn’t take her equipment with her.
Most disturbingly, her hands, which are the key to her intricate instrument, become randomly numb and tingly, as if she has been sitting on them for too long.
“I was going straight downhill and that was it,” said the New Jersey native, now 64. he told DailyMail.com. The first doctor he saw diagnosed him with an advanced form of the disease that is resistant to available treatments and affects approximately 1 million Americans.
But in 2019, a new doctor enrolled her in a medical trial of a promising new treatment that involved using her body’s own cells to fight disease.
Robin Gordon-Cartier was first diagnosed with MS when she was 50 years old. No one in his family has the disease, but he found comfort and community in a co-worker who was also diagnosed with the disease.
Dr. Violaine Harris of the Tisch MS Research Center in New York led the stem cell research. She has been at Tisch since 2004, researching potential therapies for MS.
Today, five years after the injections (six in the course of a year), he has regained enough strength to be able to take his own harp to concerts.
She told DailyMail.com that she is much more stable on her feet, giving her the confidence to travel again, which has allowed her to return to performing at weddings and concerts.
Ms Gordon-Cartier was one of 54 patients who began receiving the new MS therapy in 2019, created by scientists at the Tisch MS Research Center in New York, and which had been in the works since at least 2014.
The treatment, which involves injecting blank slate restorative cells into the spine, “has a huge impact” on the quality of life of MS patients, Dr Violaine Harris, associate director of research at Tisch, who led the study. .
Multiple sclerosis is a condition that occurs when the immune system begins to attack cells in the brain and spinal cord, removing the protective layer that insulates the nerves called myelin.
Over time, this can cause the nerves to break down.
Over time, the brain begins to lose tissue and the nerves find it harder to send signals throughout the body, altering the way different parts of the body communicate.
This can make everything from walking to going to the bathroom more difficult.
The disease can affect many different parts of the nervous system, so symptoms vary widely, but common early signs include weakness and tingling in the extremities, fatigue, mood changes, dizziness, or problems with bladder function.
MS affects approximately 1 million people in the US, according to the National MS Societyand it is four times more common in women than in men.
People rarely die directly from MS, but more commonly they die due to complications caused by the condition, such as severe urinary tract infections (UTIs) or falls.
Experts don’t yet know what causes the neurodegenerative disease, but it’s possible that genetics could play a role, as can a person’s environment.
Dr Harris told DailyMail.com that if doctors knew what caused MS, it would be easier to treat it.
There are currently not enough treatments to alleviate the suffering of people with primary progressive MS, from which approximately 100,000 to 150,000 Americans suffer.
Primary progressive MS is a form of the disease in which people’s disease worsens slowly and steadily, without stopping. In this form of the disease, there is no true relief of symptoms once it is diagnosed.
‘We had no problems enrolling (patients). Any type of treatment is necessary for this type of MS,” said Dr. Harris.
Ms. Gordon-Cartier teaches at a performing arts high school in New Jersey during the week, travels to concerts and weddings on the weekends, and attends conferences.
Dr. Harris with Dr. Saud A. Sadiq, director and chief research scientist at Tisch MSRCNY. Ms Gordon-Cartier said things changed for her in her MS treatment when she met Dr Sadiq, who made her feel there was a way to fight the disease.
The potentially revolutionary treatment works by taking bone marrow, the spongy tissue that produces blood cells in a person’s bones, and applying a complex cocktail of drugs to the tissue to turn it into stem cells.
Stem cells are the blank slate cells that your body can use to make any of the various types of tissues the body needs to function properly, including those in the heart, nerves, kidneys, muscles, and more.
Because stem cells can theoretically become any type of cell in the body, Dr. Harris and her colleagues theorized that they could be used to rejuvenate nerve cells lost to MS.
The first trial, which took place between 2014 and 2016., suggested there was some potential. In it, 20 patients had their bone marrow removed and converted into stem cells, which were then reinjected three times, three months apart between each injection.
In the short term, 75 percent of these patients were able to walk faster, reported less mental confusion, and were able to control their bladder better. However, in the two years of follow-up, these gains stabilized, suggesting that the treatment had no long-term effects.
The phase two trial began in 2018, but was recently published in the journal Stem cell research and therapy. 54 participants had their stem cells injected six times over the course of a year, with two months between each treatment.
In the first year, half of the patients received the treatment and the other half received a placebo.
In the second year, the groups were exchanged.
After receiving treatment, people have better control of their bladder and can walk more quickly and easily, Dr. Harris said.
Watching the videos of people walking side by side showed that “remarkable” progress had been made, Dr Harris said.
They even found that when treated with stem cells, people lost less brain tissue.
But it wasn’t a cure.
It did not decrease people’s overall disability or reverse any of their signs of MS.
Although people walked much better and faster, they still had to use devices to do so.
During the first year of treatment, Mrs. Gordon-Cartier was convinced that she had received the placebo and did not feel any different.
But in the second year, things began to improve. She said she felt energized, more stable and better at moving.
Now, five years after starting treatment, he says the experimental treatment has given him back his strength and life.
However, Dr. Green said the findings did not convince him that this therapy is effective in treating most people with MS.
He said that in the future, if they do the same study again and include other researchers outside the Tisch Center and see similar results, that would be clearer evidence that the treatment is beneficial.
At this time, the study was small, so the differences seen could have been due to chance, Dr. Green added.
“There can always be differences between two groups just by chance,” he explained.
However, he said, it’s possible that patients got benefits from the treatment that science might not detect, like the dog’s brain, which is difficult to measure.
There are also many structural barriers that could make it difficult to use stem cell therapies on a larger scale, Dr. Green said.
If this treatment is proven effective, further testing will still need to be done, meaning it could be years before the therapy is widely available to patients.
Researchers also face the challenge of figuring out how to make personalized stem cells on a large scale, a technology that doesn’t yet exist.
However, despite the challenges, the results of the first two trials have been encouraging enough that the FDA is in the process of approving the treatment for compassionate use, meaning people can apply for its use if they do not They have other options.
Stem cell therapy has also made promising advances in other fields. Recently, researchers in China cured a man of type 2 diabetes by injecting him with his own stem cells that had been engineered to produce insulin.
Before that, small studies have shown that stem cells could help treat erectile dysfunction, lung diseases, and potentially even blindness.
Mrs. Gordon-Cartier knows very well the drawbacks of the treatment. She said the bone marrow harvest was painful and the process to inject the stem cells was uncomfortable.
Even knowing these limitations, he said he would do the treatment again without hesitation: ‘Sign me up.
‘I would do it again, absolutely, because I think it would make me stronger. I just feel stronger. My body feels stronger… I don’t have that scary reality of MS where you feel weak and like you’re going to fall.’