We often describe the U.S. healthcare system as one of the best systems in the world. In many ways, this is true. But there are plenty of areas where the U.S. system is woefully behind. One of those areas is regenerative medicine. There are very good things happening in other parts of the world, things that are forcing patients to travel overseas for treatments they so desperately need.
A good case in point is a six-year-old girl from Hartsville, Tennessee. She was born with spina bifida, a rare and often debilitating condition in which the spinal cord doesn’t develop properly while a child is in the womb. Stem cell treatments for spina bifida are not yet approved in the U.S., leaving the family of the little girl from Tennessee to search elsewhere. Their search eventually led them to Thailand and a clinic offering a stem cell treatment provided by a Chinese company.
Our Regulatory System is Broken
Before we get to the results of the little girl’s treatment, let’s talk about the U.S. regulatory system. Our current system requires an investment of millions of dollars to get a new drug, therapy, or medical device to market. The system looks at both safety and efficacy.
So why has the treatment being used in Thailand not been approved by U.S. regulators? Because it hasn’t proved efficacious. In other words, regulators aren’t sure it works. Note that there are no alarming side effects that have regulators excessively worried about patient safety. The problem is one of efficacy. Some patients involved in U.S. studies have experienced positive results while others have not.
This makes no sense. When you’re talking about something as serious as spina bifida, patients should have the right to try alternative treatments. Obviously, no one wants the FDA to approve an unsafe treatment. But if safety isn’t the issue with a particular treatment, patients should have the option of trying it.
The Tennessee Case
Six-year-old Ana Murray was born paralyzed from the waist down as a result of spina bifida. She’s also had problems with her bladder and kidneys. Ana even has a shunt in her brain to prevent fluid buildup.
Ana’s mother, unsatisfied with the treatments her daughter was receiving here in the States, began researching other options. She learned of a stem cell treatment facilitated by China’s largest biotechnology company, a treatment that produced multiple success stories.
Mom Christine Murray then began personally contacting some of the families who had already undergone the treatment. She spoke with one family whose child went from being fed via a feeding tube to eating normally. Another family spoke of a child finally being able to walk after treatment.
The Murray’s had their daughter tested here in the U.S. to see if she would be a good candidate for the treatment. She was. So they set about raising the $40,000 necessary to travel to Thailand. In the months following her treatment, Ana has started moving her left leg and flexing her left foot. She can now stand with assistance.
She’s no longer having kidney issues. Her muscle function has improved in both legs to the point that she can now transfer out of her wheelchair at times. She’s not walking, but already her life has been improved by the treatment.
Ana Murray had to go all the way to Thailand to receive a stem cell treatment unavailable here in the U.S. Her parents are now hoping to raise an additional $40,000 for the second treatment. That’s good. And yet their case is a clear example of how broken our regulatory system is.