Ryan Benton has been to the funerals of many friends who lost their lives to Duchenne muscular dystrophy. Most people with the genetic disorder don’t live beyond their 20s.
Benton is 32.
He credits his life to injections of mesenchymal stem cells.
Stem cell therapy is still new and sometimes controversial. At first, Benton received treatments overseas because the U.S. Food and Drug Administration did not allow them here.
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In 2014 he became the first person in the United States to have what the FDA calls a compassionate use exemption to receive stem cell treatments for Duchenne muscular dystrophy. Not long after, a little boy was also approved.
“It was just this overwhelming period to be in where you thought it was kind of the end of the road and suddenly this new path is created and you’re on it and you’re a pioneer for this new treatment that you’d hoped for all your life,” Benton said.
Benton’s family doesn’t want this treatment to just be for him. Rather, they want to help thousands of boys with muscular dystrophy.
They have begun advocating for right-to-try legislation and more access to compassionate care exemptions, closely following changes in legislation and raising money. Right-to-try legislation generally allows patients with terminal or chronic illnesses to have access to investigational treatments that have not made it all the way through the FDA's approval process.
One day they hope to help send people to get stem cell treatment, perhaps even in the United States.
They call their nonprofit, which was started by Benton's siblings, Coming Together for a Cure.
Growing up, Benton heard people say that a cure for his disease would likely be found in his lifetime.
Duchenne muscular dystrophy affects about 1 in every 3,500 males worldwide. Lack of the dystrophin protein causes muscles to deteriorate, including weakened respiratory and heart muscles.
Although his parents had him exercise as much as possible, he began using a wheelchair at age 10.
Benton was 22 when family friend Neil Riordan called.
Riordan's father, Dr. Hugh Riordan, founded the Riordan Clinic, a nutrition-based health facility. Riordan had grown up in Wichita with Benton’s father, George Benton. He now had a doctorate in health sciences and was working in Costa Rica, researching the effect of adult stem cells on a variety of health issues.
He couldn’t make any promises, Riordan told Benton’s parents, but maybe stem cells could hold some hope for their son.
Benton traveled to Costa Rica, where he received his first treatment.
“My hope would be in the future that treating often enough you could keep kids out of wheelchairs, keep kids from having to lose muscle at all,” Riordan said. “Time will tell with the studies.”
In his book “Stem Cell Therapy: A Rising Tide,” Riordan writes that research showed adult stem cells can be injected into a muscle, becoming part of the muscle. While there, they produce dystrophin, the protein Benton’s body lacks.
Mesenchymal stem cells have "regenerative and anti-inflammatory properties," meaning they can aid the regeneration process of muscle cells, Riordan writes.
For Benton, results were almost immediate, his family said.
George Benton said his son often had trouble with his head falling back and not being able to raise it. Just a few days after his first treatment, he could raise his head on his own.
“It’s just hard to believe that over 20 years we were looking all over the world for a cure and it generates from our own neighborhood,” George Benton said.
Benton’s mother and stepfather Sandra and Curt Renard said that after his second treatment he gained weight — something they believed was muscle weight, which should have been impossible in his condition. He became energized and more active.
The results wear off after about four months, so Benton continued traveling to Costa Rica and later Panama for treatments until getting the compassionate care exemption. He now receives treatments at Wichita's Allergy and Asthma Consultants.
About stem cells
Riordan said he thinks things are changing for stem cell therapies after a new FDA commissioner was appointed last year.
Currently, such therapies are treated like new drugs by the FDA, meaning approval takes years and billions of dollars.
Stem cell clinics have popped up across the United States and overseas, marketing unapproved treatments directly to consumers. That hurts the patients by shortcutting the formal review process that ensures a treatment is both safe and effective, said Jack Mosher, scientific affairs senior manager at the International Society for Stem Cell Research.
It’s difficult to know what conclusions can be drawn from a story like Benton’s, Mosher said. Ideally, a trial would have a large number of patients in order to compare the treatment with a control group.
“Without that objective, larger data (set), it’s hard to know if this is really a safe and effective treatment in general,” Mosher said.
People looking into stem cell treatments need to have a “healthy sense of skepticism,” Mosher said, since claims may not be supported by scientific evidence.
Riordan said he speaks out against stem cell clinics that make “spurious” claims. His goal is to seek FDA approval, he said, and his Stem Cell Institute in Panama has developed institutional review board-approved clinical protocols for the conditions treated.
Other controversies arise from misinformation. The Bentons often have to explain the difference between embryonic stem cells (which are derived from a human embryo) and the adult stem cells used in Benton’s treatment. Adult stem cells are derived from adult tissues. Organzations that oppose the use of embryonic stem cells, such as the Catholic Church, generally support the use of adult stem cells.
The cells used in Benton's treatment come from umbilical cords after a healthy, live birth. They are considered adult stem cells since the tissues have differentiated, unlike embryonic stem cells that have not become specific cell types.
The FDA has recently made the compassionate use program easier to participate in, said Eric Anthony, director of policy at the International Society for Stem Cell Research. It approves 99 percent of requests for that program, he said.
At the same time, changes are being sought in state and federal law. Earlier this month, U.S. House lawmakers unveiled a companion bill to a Senate version that would allow terminally ill patients access to treatments that have gone through the first phase of clinical trials. The White House has pushed for right-to-try legislation.
In Texas, lawmakers approved a bill allowing companies and clinics to offer unproven stem cell therapies without the testing and approval required under federal law. Anthony said it will take time before the impact of the law is clear.
At 32, Benton is making music in a studio in his house in west Wichita. He has played in venues around Wichita and says he loves every part of the process of creating new songs.
His exemption from the FDA has expired, so now he's waiting to see what happens next for his own treatment.
Blake Benton, Benton’s brother, said he hopes other states will follow Texas’s example.
It costs a lot of money to travel to Panama for treatments, so perhaps one day people can instead travel to Texas without having to seek an exemption, Blake Benton said.
“Ryan shouldn’t be the only one that is so fortunate,” Blake said. “The promise is finally there that more people can gain access. We are very driven to be as involved and help move the needle as much as we can.”