A 12-year-old is the first patient in the state to receive a new form of gene therapy.
By Maureen Gilmer, Riley Children’s Health senior writer, mgilmer1@iuhealth.org
A 12-year-old Greenwood girl has made history at Riley Hospital for Children.
Elin Lewis has been a patient at Riley for years, undergoing regular blood transfusions for treatment of thalassemia, an inherited genetic condition where the body’s red blood cells don’t contain the necessary proteins to carry oxygen to all parts of the body.
She is now the first patient in Indiana to receive a new form of gene therapy that gives her the chance to live a normal life. A new program at Riley offers the possibility of a cure by collecting the body’s stem cells, re-engineering those cells, then transplanting them back into the body.
The gene therapy program gives hope to families whose children are affected by non-malignant blood diseases such as thalassemia and sickle cell disease that often require regular blood transfusions, as well as other red blood cell disorders like acquired aplastic anemia.
The therapy avoids the risks inherent in traditional stem cell transplant, including the difficulty of finding a good donor match, as well as graft vs. host disease and graft rejection.
Elin’s stem cells were collected back in November at Riley and modified offsite before she returned to Riley in February to prepare for the modified cells to be transplanted back into her body.
“This is such a big step for Riley Hospital to be offering these next-generation therapies,” said Riley stem cell transplant physician Dr. Jonathan Bardahl, who did his fellowship training in stem cell transplant at Children’s Hospital of Philadelphia before arriving at Riley in 2024.
Elin, who was discharged from Riley last week (one month after her infusion), “did an amazing job” and is currently engrafting her gene-modified (GM) stem cells as expected, Dr. Bardahl said this week.
She will follow up in clinic weekly, then transition to monthly appointments for the first year.
“From here, her new GM stem cells will start making more stable hemoglobin, and hopefully she will require no more red blood cell transfusions for the rest of her life,” the physician said.
Dr. Seethal Jacob, who hosts a regular multidisciplinary clinic with Dr. Bardahl and other specialists for patients with thalassemia and sickle cell, is thrilled to see the program get off the ground.
“To see it get through the studies, get FDA approval and now get to the point where we can administer it through our own institution is just wonderful and a testament to all the work our Riley teams and collaborators have done to lift up sickle cell and thalassemia care,” she said in a previous interview.
According to available data, up to 95% of transfusion-dependent thalassemia patients who complete the therapy no longer require transfusions, Dr. Bardahl said.
Elin’s parents, Nathan and Monica Lewis, are grateful to have the revolutionary treatment available so close to home.
“We are very hopeful,” Monica said this week. “It was a really good experience. The nurses were absolutely amazing, they were my heroes, and the whole stem cell team is so wise, so informative.”
The nursing team got Elin through some tough times during her hospitalization, she added, “as they do all the kiddos on that floor.”
The Center Grove middle schooler will finish out the school year remotely, but she is already back on the trampoline practicing her gymnastic stunts, her mom said.
“She didn’t skip a beat.”
Photos and video by Mike Dickbernd, IU Health visual journalist, mdickbernd@iuhealth.org
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“Next-generation therapy” for blood disorders at Riley - A 12-year-old Greenwood girl is the first patient to undergo the gene therapy approved for thalassemia and sickle cell disease.
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