Babies are priceless, but at one week old, baby Lexa Dennis already had an amazing $2 million story to tell. It isn’t about investing or winning the lottery, but about a life-saving drug that she received at Woman’s Hospital in Baton Rouge, La., at just four days old -- the youngest known to receive the Zolgensma cutting-edge gene therapy for babies born with spinal muscular atrophy (SMA).
SMA is a devastating, debilitating, and often fatal disease for babies who are born with it. It is caused by a gene mutation that leads to difficulty performing the basic functions of life, like breathing and swallowing. According to curesma.org, it affects approximately one in 11,000 births in the U.S., and about one in every 50 Americans is a genetic carrier.
“A lot of times it’s considered the ALS of children,” said Lexa’s mother, Andrea James. “That’s thee quickest way to explain it.”
“SMA is a serious illness that causes death or severe muscle weakness if they survive,” said Charlotte Hollman, MD, a pediatric neurologist at Woman’s Hospital. “It’s the most common genetic cause of death in the first year of life.”
Zolgensma is a one-time therapy that works by targeting the genetic root cause of SMA by replacing the function of the missing or nonworking gene. It attaches to the DNA without altering it, helping the motor neuron cells to make necessary proteins. While it can’t reverse symptoms of SMA that have already presented, it can halt new symptoms in their tracks. But, it comes with a price tag. One dose of Zolgensma costs $2.1 million.
Lexa had the benefit of being diagnosed with SMA while still in her mother’s womb, an advantage afforded to her because of her two-year-old brother Axel’s diagnosis with the same genetic condition early in his life. Because doctors already knew Lexa’s parents are both genetic carriers for SMA, she was tested prior to birth.
“Luckily we had physicians that were able to diagnose her prenatally, which was a big step, because most babies are diagnosed at a couple weeks of age or months, and by then the disease has already started to progress,” explained Kimberly Stewart, MD, neonatologist at Woman’s Hospital. “So, the medication may help slow it down or stop it at that point, but the baby has already become symptomatic.”
As soon as prenatal testing confirmed that Lexa was positive for SMA, a coordinated team at Woman’s Hospital -- comprised of physicians, nurses, social workers, pastoral care, and administrators -- began their work developing a plan for the best treatment for Lexa. This was done in collaboration with her mom, physician specialists, lab, pharmacists, and the team that specializes in insurance. The reference lab and the Zolgensma clinical specialists provided the proper education on necessary specimen processing and training on how to administer the drug to our team of doctors and nurses.
“By being born at Woman’s, and having the physicians that took care of mom beforehand, and then the neurologist, neonatologist, social workers, the full team being in the same setting, we were able to work together and get this medicine here before she was a week of age,” said Stewart.
“There are a lot of criteria to get approval from the insurance company,” James explained. “There was a lot of training. It’s a $2.1 million drug, so it’s like carrying gold. Everyone who’s around it, everyone who touches it, has to be trained. It took a lot of patience and a lot of time for people to be prepared.”
On Feb. 5, Lexa received her Zolgensma dose, with the hopes that this will prevent her from ever showing symptoms of SMA.
“This is huge, this is life-changing,” said Hollman. “Woman’s Hospital worked very hard to get this baby the gene therapy very quickly, including getting it approved by Medicaid. We’ve given it locally in Baton Rouge three times, but never to a baby this young. And I believe nationally there hasn’t been a baby this young to get the gene therapy. The earlier, the better, and at four days old we expect a very good outcome.”
“It’s more than I could have asked for, more than I could have hoped for,” James added. “I thank the Woman’s NICU team, maternal-fetal medicine, the neurologist, every piece of the puzzle that worked hard the last few months to get Lexa this treatment. Her outlook is limitless. I think it’s possible that she hits every milestone that any child without a neuromuscular disorder would meet.”