Tag Archive for: Nickel

Kendric receives care at Children's National.

Rewriting the script for sickle cell disease

More than 100,000 Americans have sickle cell disease, an inherited blood disorder that can cause excruciating pain crises and shorter life expectancies.

Children’s National has one of the largest sickle cell programs in the United States. We are pioneering treatments and provide specialized care to about 1,500 patients each year. We participate in clinical trials to improve outcomes, shorten treatment time, reduce complications and minimize the need for opioids and chemotherapy.

Kendric receives care at Children's National.

Kendric receives care at Children’s National.

In recognition of our clinical and research excellence, Children’s National was one of a few U.S. pediatric hospitals selected to offer two promising new FDA-approved gene therapies.

Hematologist Robert Sheppard Nickel, M.D., leads a study to reduce toxicities in bone marrow transplants. “Years of development led to these curative therapies,” Dr. Nickel says. “I hope in the future we can safely cure more children with sickle cell disease.”

“The future looks promising to revolutionize the lives of our patients and make these therapies accessible worldwide,” says Andrew Campbell, M.D., director of our Comprehensive Sickle Cell Disease Program.

Kendric and Nasir find hope

In May 2024 at Children’s National, 12-year-old Kendric of Clinton, Maryland, became the world’s first patient with sickle cell disease to begin a commercially approved gene therapy that could dramatically reduce or even eliminate his pain. It involved extracting his bone marrow stem cells; genetically modifying them in a specialized lab to reduce the risk of sickling; and then, after chemotherapy, infusing them back into his bloodstream.

Expert, compassionate care empowered Kendric to understand the science behind his treatment and chart a path to recovery. “My care team taught me how to deal with my disease and everything that I need to know for the future,” he says. “They gave me hope that I could be cured.”

Nasir and his care team

Nasir and his care team.

Nasir, age 20, spent his childhood waiting to find a match for a stem cell transplant to address his sickle cell disease. Finally, in 2023, at Children’s National, he found an answer in gene therapy to alter his own cells.

Due to painful episodes and the need for frequent blood transfusions, both Kendric and Nasir missed out on a lot of school, important moments with friends and simply being kids. Now, they can explore a world in which patients like themselves can overcome this disease and reclaim their health.

“I have all of this oxygen and energy that came out of nowhere,” Nasir says. “It’s really a new life. I feel reborn.”

“The network of doctors at Children’s National gave us reassurance and lots of hope,” says Kendric’s mom, Deborah. “They made us feel like family. We are in awe of how quickly things moved and how much compassion they have shown us.”

Read more stories like this one in the latest issue of Believe magazine.

researchers in a lab at Children's National

Pioneering gene therapy as a treatment for sickle cell disease

Gene therapy is a new and exciting treatment option available for patients with sickle cell disease (SCD). Children’s National Hospital is one of the few pediatric hospitals in the country that offers both FDA-approved sickle cell disease gene therapies: CASGEVY™ (exagamglogene autotemcel) and LYFGENIA™ (lovotibeglogene autotemcel).

What this means

Gene therapy involves an autologous transplant, taking the patient’s own stem cells, genetically changing those stem cells and then, after chemotherapy, infusing those stem cells back into the patient to make healthy blood.

“I’m excited about gene therapy for sickle cell disease. I think it has the potential to be a curative option for every single child with sickle cell disease,” said Robert Nickel, M.D., hematologist at Children’s National.

Currently both treatments are only approved for patients 12 years and older with severe disease. Children’s National was the first hospital in the world to collect stem cells for the LYFGENIA™ treatment.

Moving the field forward

Clinical trials hold incredible promise to advance the care of SCD. Children’s National continues to pioneer transplant therapies to cure SCD and is one of the leading centers participating in clinical trials of new treatments for this condition.

Experts at Children’s National are leading a multi-site clinical trial of a chemotherapy-free transplant approach for SCD using a matched sibling donor. This chemotherapy-free approach has less toxicity and side effects for children undergoing transplant.

In addition, Children’s National has been leading the way with innovative approaches to support sickle cell patients. “We’re providing alternative approaches to pain such as healing touch, acupuncture, massage VR technology, physical therapy and exploring other ways of treating pain in an integrated manner,” said Andrew Campbell, M.D., director of the Comprehensive Sickle Cell Disease Program at Children’s National.

The team is also exploring non-opioid treatments, such as intravenous citrulline, a naturally occurring amino acid that has been proven to enhance blood flow and potentially alleviate pain in treated patients in preliminary studies under the direction of Suvankar Majumdar, M.D., chief of Hematology at Children’s National.

Microscopic view of thalassemia

What it means to be a designated treatment center for beta thalassemia

Microscopic view of thalassemia

ZYNTEGLO® (betibeglogene autotemcel) is an FDA-approved gene therapy for transfusion-dependent beta thalassemia, which is an inherited blood disorder that causes the body to make less hemoglobin, resulting in anemia.

Children’s National Hospital is a designated qualified treatment center for Beta Thalassemia Gene Therapy. ZYNTEGLO® (betibeglogene autotemcel) is an FDA-approved gene therapy for transfusion-dependent beta thalassemia, which is an inherited blood disorder that causes the body to make less hemoglobin, resulting in anemia.

This unique therapy is made specifically for each child or adult, by adding functional copies of the beta-globin gene to their own blood stem cells. Most patients with beta thalassemia who have received a one-time ZYNTEGLO® treatment have been able to produce sufficient hemoglobin because of the treatment, freeing them from regular blood transfusions.

Evelio Perez, M.D., and Robert Nickel, M.D., lead the gene therapy program and discuss the importance of offering this gene therapy to patients with beta thalassemia.

Q: What has been the hold-up in this field and how does this work move the field forward?

A: Stem cell transplant using a donor’s cells (called allogeneic transplant) has been a curative treatment option for patients with beta thalassemia for many years. Unfortunately, many patients do not have a suitable donor. And, even for patients who have a donor, allogenic transplants have serious risks including a problem called graft versus host disease (GVHD) in which the new donor cells attack the patient’s body. Gene therapy like ZYNTEGLO® has no risk of GVHD because we use the patient’s own cells.

Q: How will this benefit patients? What excites you most about this advancement?

A: This treatment will give almost every patient with beta thalassemia the option of undergoing curative therapy. This is obviously exciting for patients because it means they no longer need to come to the hospital every 3-4 weeks for transfusions as well as take medications to treat the dangerous accumulation of iron in their body. It is also good for the health system because it will allow donated blood to go to other patients in need.

Q: How is Children’s National leading in this space?

A: This therapy really requires a multi-disciplinary team including members of the transplant, hematology, apheresis, stem cell lab and others! At Children’s National we have the experts on these teams and experience working together. As one of the largest sickle cell disease centers in the country, we are participating in research to hopefully help bring gene therapy to patients with sickle cell disease in the near future too.

pregnant woman at fertility consultant

Fertility preservation in sickle cell disease patients

pregnant woman at fertility consultant

Fertility is a long-standing concern for patients with sickle cell disease and their families.

In a recent review in the Journal of Clinical Medicine, researchers from Children’s National Hospital look at the current state of fertility preservation in patients with sickle cell disease and make recommendations for longitudinal post-treatment for these individuals.

Fertility is a long-standing concern for patients with sickle cell disease and their families. Current curative therapy for the disease requires gonadotoxic conditioning, which many patients resist because of the resulting risk of infertility. And, while standard fertility preserving interventions exist for pre- and postpubescent females and males, best practices for integrating these interventions into sickle cell disease care have not yet been established.

In their article, Children’s National hematologist Robert Sheppard Nickel, M.D., and co-authors review current fertility assessments, fertility considerations in pre- and post-transplant patients with sickle cell disease and fertility preserving interventions for patients. The authors conclude that in the future, less toxic curative approaches may make fertility preservation unnecessary, but at present, fertility preservation should be offered to patients with sickle cell disease pursing curative therapy.

Additional authors from Children’s National include Michael Hsieh, M.D., Ph.D., and Jacqueline Maher, M.D.

Read the full review article, Fertility after Curative Therapy for Sickle Cell Disease: a Comprehensive Review to Guide Care, in the Journal of Clinical Medicine.

Children’s National Health System advances sickle cell disease cure through Doris Duke Charitable Foundation grant

Sickle-Cell-Blood-Cells

An innovative Children’s National Health System project aimed at improving the only proven cure for sickle cell disease – hematopoietic cell transplantation – will receive more than $550,000 in funding from the Doris Duke Charitable Foundation’s inaugural Sickle Cell Disease/Advancing Cures Awards, which provides grants to advance curative approaches for sickle cell disease. The study, a three-year, multi-center trial that will study a low intensity, chemotherapy-free transplantation approach to cure children with sickle cell disease using a matched related donor, is led by Allistair Abraham, M.D., blood and marrow transplantation specialist, and Robert Nickel, M.D., hematologist, and is one of seven projects receiving approximately $6 million total through the awards.

While transplantation using a matched sibling donor today has a high cure rate (>90 percent) for sickle cell disease, traditional transplant approaches have many risks and side effects in both the short and long term. The study will examine if a chemotherapy-free approach can lead to a successful transplant without resulting in graft-versus-host disease (GVHD). GVHD is one of the most challenging complications of a transplant, in which the transplant immune cells attack the patient’s body. The researchers anticipate that this new transplant approach will be so well tolerated that patients’ quality of life will be maintained and improved throughout the process, with most of the care administered in a clinic setting.

“This approach has proven to be effective for adults with sickle cell disease, so we are grateful for the opportunity to begin this important trial for children thanks to the Doris Duke Charitable Foundation,” says Dr. Abraham. “Children with sickle cell disease are in need of innovative treatments, and we look forward to finding more solutions that improve the quality of life for these patients.”

“Advancing treatment for sickle cell patients to the point where they can live free of the disease is our top priority,” says Dr. Nickel, who is also an assistant professor of pediatrics at the George Washington University School of Medicine and Health Sciences. “This funding is critical to our study and it will accelerate the timeline to achieve the goal of a well-tolerated and safe cure for children with sickle cell disease.”

Matthew Hsieh, M.D., who helped pioneer this work at the National Institute of Health in adults, and Greg Guilcher, M.D., who has used this transplant approach in children, are key collaborators on the project.

The study is projected to begin in December 2018 and continue for three years. The Comprehensive Sickle Cell Disease Program at Children’s National is among the largest in the country, treating more than 1,400 children and young adults with all types of sickle cell disease. Children’s National also offers the largest, most comprehensive blood disorders team in the Washington, D.C., area.