After a months-long fight with Texas Medicaid over coverage of a gene therapy, Afghan refugees now have a chance to save their infant son. After initially balking, Texas officials have agreed to pay for the costly treatment, the boy’s family and his doctor told STAT.
At the end of last week, N. Pashai’s cell phone rang. It was from a Minnesota area code. He answered, and through an interpreter, he heard the words he and his family have spent the last few months praying for. Doctors at M Health Fairview, a research hospital in Minneapolis associated with the University of Minnesota, were going to attempt to cure his 4-and-a-half month-old son Sufyan with an experimental gene therapy.
“Even as she said it, I still felt doubt,” Pashai told STAT through an interpreter on a video call. “I kept asking her again and again, are you sure it’s the full approval, the full approval?”
During the United States’ long war against the Taliban, he worked security for an elite unit of American-trained Afghan special forces, and due to the sensitive nature of this work asked to be identified only by his first initial. In 2021, he immigrated to Texas with his wife and oldest son, Mohammad, after having been evacuated from Kabul when the city fell. For the past few months, they have been in a battle of a different kind with their new home state’s public insurance provider.
In October, he and his wife learned that Mohammad, now 3 years old, has metachromatic leukodystrophy — an ultra-rare genetic disorder that attacks the central nervous system. By the time MLD symptoms appear, like they had in Mohammad, they are irreversible and almost certainly fatal. The following month, a DNA test revealed that his youngest brother, Sufyan, also had the same disease-causing mutation. But he didn’t have to share Mohammad’s death sentence.
Orchard Therapeutics has offered its multimillion-dollar MLD treatment, Libmeldy, to the Pashais free of charge. And specialists in Minnesota who have successfully treated a handful of patients with the therapy through the Food and Drug Administration’s compassionate use program, believe Sufyan is a good potential candidate for the gene therapy because he is not yet showing symptoms. But to secure a slot in the company’s cell engineering queue, the team at M Health Fairview needed assurances that the costs of the associated procedures — including bloodwork, cell harvesting, chemotherapy, and hospital stays while Sufyan recovers, which could add up to around $300,000 — would be covered.
Doctors at M Health Fairview had been going back and forth with representatives from Texas Medicaid and the managed care organization that administers the state health care program for more than two months. A request to pay for an initial consultation with the Minnesota team was denied in mid-December. The decision was overturned after STAT published a story drawing attention to the family’s appeal. But Texas Medicaid was slow to agree that gene therapy and all the procedures required to receive it were a medical necessity in Sufyan’s case.
At one point, it seemed as though every provider in Minnesota that might care for the child — and ultimately bill for the care provided — would have to be credentialed as a Texas Medicaid provider, including surgeons, brain specialists, ICU staff, and possibly dozens of other doctors and nurses. That process generally takes months, months that Sufyan may not have.
The issue was finally resolved last week, according to Paul Orchard, a pediatric hematologist at M Health Fairview. On January 30, his team reached an agreement with Texas Medicaid to provide funding for the entirety of the gene therapy procedure for Sufyan.
“A date for the cell manufacturing has been secured, and based on this, within the next two weeks the family will arrive in Minneapolis to begin the procedure,” Orchard said.
A spokesperson from Texas Health and Human Services was not immediately available to comment.
The Pashai family is now making preparations to travel to Minneapolis for Sufyan to be assessed in person and for a multi-day procedure to collect his blood stem cells. These will be sent to Orchard Therapeutics’ manufacturing facilities in Italy, where they will be engineered to express a functional copy of the gene that makes an essential enzyme for clearing toxins out of the central nervous system.
If all goes well, the Pashais will return to Minnesota for Sufyan to receive an infusion of those cells, after receiving chemotherapy to prepare his bone marrow to accept them. That will be followed by a two to three month hospital stay while he recovers and his blood and immune systems rebuild themselves. Friends of the family have set up a GoFundMe to help with travel costs.
By that time, Libmeldy may already be an approved medicine in the U.S. The FDA is expected to make a decision by March 18. The gene therapy, which costs between $2.6 and $3.5 million in Europe, where it has been on the market since 2020, is likely to strain a market structure that was not built to price curative, one-time treatments. Although it may save money in the long run, compared to the health care costs associated with caring for kids with MLD as they progressively become more debilitated, the current model — by placing the responsibility for purchasing treatment on a patient’s health insurer at the time of treatment — often disincentivizes insurers from covering the therapy; patients often change health insurers, so the initial provider may not see the benefits of the reduced future costs.
Public insurers, like Medicaid, are more likely to be strained by the sky-high price tags. Doctors like Orchard, who specialize in delivering gene therapy, are concerned that this could lead to kids with private insurance getting access to Libmeldy, while those on Medicaid get left behind.
“It’s going to be an equity issue,” Orchard said. “It’s exciting to have new things to offer that might be more efficacious and safer, but we have to figure out a way to pay for them.”
For the Pashais though, those worries suddenly feel far away. “We are so thankful for this approval, it’s just a huge relief,” N. said, allowing a wide smile to break out above his neatly trimmed beard as he looked down at Sufyan, cradled gently in his lap. “Today we are happy.”