Janelle's story: Overcoming sickle cell disease with innovative gene therapy

Janelle was 8 years old, sitting in her parents' room, when she first felt it: a dull ache in her lower back that wouldn't let up.

"I thought it would go away, so I didn't really care about it at first," Janelle says. "But then the pain just kept growing."

She curled up on the couch, crying, until her sister found her and called their mom. They headed to the hospital, where Janelle got a clear answer: Her pain was caused by sickle cell disease, a disease she and her family knew too well.

A severe case of a familiar diagnosis

Sickle cell disease causes red blood cells to form in a rigid, crescent shape instead of a flexible, round one – a difference Janelle can explain better than most.

"If you have sickle cell, your red blood cells are like a C-shape instead of like an O-shape," Janelle says. "And when they’re C-shaped, they can't travel through your body normally, so they get stuck and cause pain."

Janelle's older brother was diagnosed at birth with sickle cell disease and was cured with a bone marrow transplant, using donor marrow from their older sister.

But, unfortunately, nobody in Janelle’s family was a donor match.

So in 2022, Janelle joined the donor registry – and waited. As she waited, she was hospitalized 12 times for pain crises and needed four blood transfusions.

"Janelle’s case was so severe that we actually had a specialized pain management team that took care of her," says Samer Zaid Kaylani, M.D., Pediatric Hematologist/Oncologist at Children’s Health℠ and Associate Professor at UT Southwestern.

A new option, without a donor

About a year into her wait for a bone marrow donor, Janelle's care team told her about another path to a cure – one that didn't require a donor match at all.

In December 2024, the FDA approved Lyfgenia, a gene therapy that uses a patient's own genetically modified stem cells, to produce healthy hemoglobin (the protein inside red blood cells).

Because the treatment uses a patient's own cells, there's no need for a donor and no risk of the donor cells reacting to Janelle’s body.

"Lyfgenia introduces a hemoglobin that’s not a sickle cell hemoglobin," Dr. Kaylani says. "So, in a way, you're curing people by producing normal-looking red blood cells."

For Janelle, the news of this option was a relief.

"After over a year of waiting, I wasn’t sure anyone would match me," Janelle says. "So when I found out I could use my own cells, I got excited."

She was also a little nervous.

"I was nervous it might fail. But I decided to go ahead with it – and it worked," Janelle says.

At age 13, Janelle became the first pediatric patient in North Texas to receive Lyfgenia. Before receiving the treatment, her care team did an evaluation to be sure her body was healthy enough to receive gene therapy.

"We went fully by the book and wanted to do everything in the safest way possible," Dr. Kaylani says.

Looking toward the future

Before Janelle's treatment began, Brooke Kemp, RN, Transplant Coordinator at the Pauline Allen Gill Center for Cancer and Blood Disorders at Children’s Health, sat down with Janelle and her family to talk through what the months ahead would look like – and what Janelle hoped her life could look like on the other side.

"We talked for a good hour about her dreams and wishes, and how this therapy could help her achieve those," says Kemp. "I found out she wants to be an actor and was excited to pursue acting – without the fear of pain crises and needing to go to the hospital."

Janelle believes that early conversation, and the ones that followed, helped her feel ready for what was ahead.

"Brooke [Kemp] gave me a lot of information about what to expect with gene therapy and it made me more confident," says Janelle.

Less chemo and less medicine

The process started with collecting Janelle's own stem cells through a procedure called apheresis, where blood is filtered through a machine that separates out the needed stem cells before returning the patient’s blood. Janelle needed three separate sessions for the team to gather enough cells.

After that, Janelle's cells were sent to a manufacturer, where they were genetically modified over about two months.

When they came back, Janelle was admitted for four days of chemotherapy to clear space in her bone marrow. Then, she received her own modified cells in an infusion that took just minutes.

"It was really quick," Janelle says.

Tiffany Simms-Waldrip, M.D., Medical Director of the Cellular and Immunotherapy Program at Children's Health and Clinical Professor at UT Southwestern, says the process continues for weeks after the infusion.

"It takes about three weeks to see the new cells start to grow," Dr. Simms-Waldrip says.

Because gene therapy uses a patient's own cells rather than a donor's, Janelle needed far less chemotherapy than her brother had for his bone marrow transplant – an no need for immunosuppressant medications.

"My brother needed more chemo and a lot more medicines than I did,” Janelle says. “He also had to stay in the hospital longer and had more side effects.”

Home and feeling better fast

Janelle returned home in December 2025, with gifts from her care team in hand.

Her improvement came quickly.

"It didn't take me that long to feel better," Janelle says. "As soon as I made it back home, I started feeling way better."

Because her immune system was still fragile, she had to take extra precautions to reduce her risk of infection for the first few months. That meant changes in her diet as well as limitations on her activities.

But she pushed through, eager for the day she'd be able to go out for pizza, a burger or boba tea.

Since she’s been home, Janelle hasn't had a single hospital visit for a pain crisis.

Her hemoglobin S, the marker doctors use to track how well the sickle-shaped hemoglobin has been replaced, has held steady around 30%, which is well below the 50% mark that usually keeps sickle cell symptoms at bay.

Back on the dance floor

For Janelle, the clearest measure of success has nothing to do with lab values.

Before treatment, she couldn't run more than half a lap without feeling exhausted – and she couldn't participate in sports.

Today, she wrestles alongside a friend and dances with another – activities that once felt entirely out of reach.

Kemp has watched her transformation up close.

"In the beginning, Janelle was quiet. She was skeptical that there was anything out there to help her," Kemp says. "Watching her grow through this journey has been amazing. She’s opened up, and her dreams have widened."

Looking back on everyone who helped her, Janelle doesn't hesitate.

"I’d like to thank my care team more than anything," she says. "They were there for me and took really good care of me while I was in the hospital. They even decorated my room pink, which they knew was my favorite color.”

Explore treatment options for sickle cell disease

For some children with sickle cell disease, treatments such as stem cell transplant and gene therapy may offer a cure. Children’s Health specialists work closely with families to determine which treatment options may be appropriate based on a child’s health, disease severity and other factors. Learn more about our Sickle Cell Disease Program and curative options for the disease.