The Division of Urology at Children’s National has developed a first-of-its-kind orientation program for pediatric urology advanced practice providers (APPs). This program was created based on real feedback from pediatric urology APPs in the United States and Canada.
The primary purpose of the orientation program is to increase the knowledge and confidence of pediatric APP’s in transition to independent practice. The program prepares the next group of advanced practice providers in the Division of Urology to achieve the same high quality of care that the current group provides to our patients.
“I think what makes our orientation program unique is how involved leadership is in training the orientee. We are with our orientee every step of the way every week. We are there making sure that they are attaining their skills and their knowledge. We’re checking in on them very regularly, and we also are trying to make it very individualized to that person.” said Katie Scarpaci, nurse practitioner and creator of the APP orientation program.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2024/03/AAP-still-feature.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2024-03-20 12:50:252024-03-20 12:51:33The division of urology creates tailored orientation program
The 2023 International Congress on Early Onset Scoliosis and the Growing Spine (ICEOS) was a three-day conference for surgeons and health professionals from around the world. The sessions focused on solutions for pediatric spine deformities. Matthew Oetgen, M.D., chief of Orthopaedic Surgery and Sports Medicine at Children’s National Hospital, was a member of the faculty.
Session highlights included:
Pre-Course: Pediatric Spine Study Group (PSSG) Mini-Research Symposium – Considerations for your biosketch, regulatory and IRB
How Do You Propel Your Research Team
“Overall this was a great meeting with valuable content,” says Dr. Oetgen. “Learning and teaching alongside fellow orthopaedic leaders is a fantastic opportunity for everyone involved.”
Dr. Oetgen participated in the following sessions:
Timing of Hemivertebra Excision
Let’s Debate! nView Workshop – 3D imaging and guidance, available technologies for pediatric spine and when to use them
Hip preservation treats a wide variety of conditions such as hip dysplasia, hip impingement, hip torsion/rotation abnormalities, hip synovitis, snapping hip and hip labrum tears.
The Hip Preservation Program at Children’s National Hospital offers advanced care to children and adults with hip pain, providing a continuum of care that extends well into adulthood. Jeffrey Peck, M.D., an orthopaedic surgeon with subspecialty interests in pediatric and young adult hip preservation, discusses the program and what Children’s National is doing to move the needle forward in patient care.
Q: What is hip preservation and what are common conditions it addresses?
A: The field of hip preservation is dedicated to diagnosing and treating hip conditions that result in hip pain and can potentially result in hip degeneration.
There are several potential conditions that can lead to hip pain and eventual hip joint degeneration. These include atypical anatomy in and around the hip joint, which can be congenital, developmental or traumatic in origin. Strains and sprains of the muscles and ligaments around the hip may also cause hip joint pain. Hip preservation treats a wide variety of conditions such as hip dysplasia, hip impingement, hip torsion/rotation abnormalities, hip synovitis, snapping hip and hip labrum tears.
Q: Who is affected and when should a patient seek out an orthopaedic expert?
A: The people most often affected by conditions treated through hip preservation are adolescents and young adults up to age 45 years old who develop hip pain. When hip pain does not subside, it can be due to a condition that has resulted in hip joint inflammation that may eventually begin to cause hip joint degeneration leading to arthritis if the condition is not treated. If a patient has hip pain that lasts for multiple months without improving, it is prudent to seek evaluation with a hip preservation orthopaedic surgeon specialist.
Q: What is Children’s National doing to move the needle for hip preservation patients?
A: Children’s National cares for patients with hip pain using an integrated care team, featuring a Hip Preservation fellowship trained surgeon, physiatry experts, sports psychologists, radiologists and physical therapists with a focus on adolescents and young adults. We work in a collaborative environment with the shared goal of helping patients with hip pain return to living life without the pain that has held them back.
The Children’s National team also collects patient reported outcome scores to better understand the severity of patient symptoms and, later, how surgery has improved those symptoms. Additionally, Children’s National is engaged in research endeavors evaluating hip conditions and how patients have responded to treatment. By pursuing these efforts, we not only assist the patients we see today, but also contribute to advancing care to better help future patients.
Q: What excites you about the future for the field of hip preservation?
A: The field of Hip Preservation is a young specialty within orthopaedic surgery that has demonstrated consistently positive outcomes for patients who previously had very limited treatment options for their hip pain. With this comparative newness comes continual innovation and advancement as we learn more about how hip pain can occur and what we can do to best treat that pain. Procedures that we perform at Children’s National such as the Ganz Periacetabular Osteotomy (PAO) and the use of hip arthroscopy to treat hip impingement are relatively new innovations in the field of orthopaedics that can provide immense relief to patients having hip pain.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2024/01/hip-x-ray-feature.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2024-01-09 16:30:492024-01-16 16:07:52Hip preservation: Moving the needle in patient care
“With each kidney transplant, we’re not just restoring the health of children, we’re pioneering progress and setting new benchmarks for the field of pediatric nephrology,” says Marva Moxey-Mims, M.D., chief of Nephrology at Children’s National Hospital.
“With each kidney transplant, we’re not just restoring the health of children, we’re pioneering progress and setting new benchmarks for the field of pediatric nephrology,” says Marva Moxey-Mims, M.D., chief of Nephrology at Children’s National Hospital. “We offer not just treatment, but a chance for our patients to flourish and live their best life.”
Big picture
The Kidney Transplantation Program at Children’s National is the only one of its kind in the Washington, D.C., region focused on the needs of children and teens with kidney disease.
Performing an average of 15-20 kidney transplants per year, the program’s latest one- and three-year graft and patient survival data from the Scientific Registry for Transplant Recipients is 100%.
“Our program surpasses national numbers, reflecting a commitment to exceptional care, superior patient outcomes and a focus on setting a new standard for excellence in transplant support,” says Dr. Moxey-Mims.
Children’s National leads the way
At the forefront of groundbreaking treatment, the nephrology team is pioneering progress by providing innovative care and resources. One example is the hospital’s collaboration with the National Kidney Registry and MedStar Georgetown Transplant Institute’s Living Donor Program. This provides a greater chance of finding more suitable donors for difficult-to-match children and has resulted in altruistic donations.
“Through living kidney donations, we redefine the timeline for pediatric transplants. Children experience shorter waits and swifter paths to renewed health compared to the national wait list,” says Asha Moudgil, M.D., medical director of the Kidney Transplantation Program at Children’s National. “It’s a testament to our commitment to making a difference in every child’s life.”
That commitment is also seen in the hospital’s focus on making sure families have the resources they need during their medical journey.
“Caring for a child with renal disease is an immense challenge, not only for the young patients but also for their families. The demanding schedule of hospital visits, often three times a week, adds an extra layer of stress and disruption to daily life,” says Dr. Moudgil.
Through grant funding from donors and national programs, families with children undergoing kidney transplants and dialysis can receive financial assistance. In some cases, families receive up to $2,000 of mortgage or rental payment assistance. Families can also receive funds for medication co-pays and other expenses incurred around the time of the transplant.
“In the face of medical adversity, these funds become a source of support,” says Dr. Moudgil. “We make sure every family, regardless of financial constraints, can access the care and resources their child needs.”
Bottom line
Children’s National is at the forefront of pediatric kidney transplantation. “Our unique approach ensures every facet of a patient’s journey is considered,” says Dr. Moxey-Mims. “We’ve created an environment where success is not just a medical outcome but a comprehensive and sustained improvement in a child’s overall quality of life.”
Learn more about our latest advances in pediatric Nephrology.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/Marva-Moxey-Mims-in-the-lab-feature.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2023-12-15 12:14:532023-12-26 13:15:08Revolutionizing pediatric nephrology one patient at a time
Leaders in medicine, academia, industry and state and local government came together for the first annual Cell and Gene Therapy in the DMV Symposium, hosted at the Children’s National Research & Innovation Campus. The mission: Connect the local scientific community – bursting with expertise and collaboration potential – to develop these cutting-edge therapies for cancers, sickle cell disease and immune-mediated disorders.
The daylong event drew over 100 experts from a range of organizations in the D.C, Maryland and Virginia region, sometimes called the DMV: Children’s National Hospital, the Food and Drug Administration, the National Institute of Standards and Technology, the National Institutes of Health, the General Accounting Office, Virginia Tech, MaxCyte, AstraZeneca, Kite Pharma, Montgomery College, the Maryland State Department of Commerce and more. Together, they unraveled a host of topics including the regulatory environment, workforce development and training, research standards and the promise of these therapies.
“Our Cell & Gene Therapy Symposium brings together our current collaborators and future partners in the D.C., Maryland and Virginia space, which is an incredibly rich area. We see tremendous opportunity and breakthroughs in our future,” said Catherine Bollard, M.D., M.B.Ch.B., interim chief academic officer and chief of Pediatrics at Children’s National Hospital. “Many different diseases rely on the immune system to either be ramped up or to be controlled, and we can seize on these biological processes. Cell and gene therapies are at the heart of where medicine is going.”
The big picture
For decades, oncologists largely have turned to the same menu of treatments to fight cancer, including surgery, chemotherapy and radiation. Cell and gene therapies offer the promise of training the immune system to fight diseases with fewer side effects and potentially higher success rates. Early work has shown progress in liquid cancers, like leukemia, raising the possibility that the therapies could be used on solid tumors and other disorders, such as lupus and sickle cell disease. However, many disciplines must come together to yield discoveries.
“Nobel Prize-winning work doesn’t necessarily translate into available therapies for patients. It takes a whole community like this to make it happen,” said Cenk Sumen, chief scientific officer at MaxCyte Inc., an international cell engineering company based in Rockville, Md. “It has been exciting to see this diverse group of stakeholders come together, which is probably unmatched anywhere on the planet.”
Why we’re excited
Symposium host Patrick Hanley, Ph.D., chief and director of the Cellular Therapy Program at Children’s National, said the goal was to cement the region as the No. 1 location for this highly technical research and development. He believes Children’s National can offer essential elements to this success, given its clinical and research expertise, workforce training opportunities and geographic proximity to the scientific leadership of the federal government. “What makes us unique is our proximity to all the players who can help create new treatment options for patients. We truly are the biomedical capital of the world,” he said.
Michael Friedlander, vice president for health sciences at Virginia Tech, notes that the earliest stages of invention will emanate from academic labs including those at Virginia Tech and Children’s National. “You have basic scientists who are doing fundamental research on properties and procedures that will lead to the new therapies of tomorrow,” he said. “We are putting in place the fundamental pieces to advance children’s health in all dimensions.”
What’s ahead
One challenge is developing a workforce to help prepare cell therapies for patients, following precise standards to ensure the therapy works as designed. Children’s National does this training, as do others in the region. Lori Kelman, Ph.D., M.B.A., biotechnology coordinator and professor at Montgomery College, said that the area is full of people who want to help people and who like science.
“The thing that people might not know is that you don’t need a Ph.D. to work in cell and gene therapy,” she said. “There are opportunities at all levels, including the entry level, which is where a great career often starts.”
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“By the time a baby is diagnosed with bronchopulmonary dysplasia, families have already had a long journey with prematurity in the neonatal intensive care unit (NICU),” says Hallie Morris, M.D., neonatologist and lead of the Complex Bronchopulmonary Dysplasia (BPD) Program at Children’s National Hospital. “To be able to have a team that is focused on the holistic health of their child in the context of this diagnosis makes a world of difference to these families.”
The big picture
Some premature infants with BPD experience more severe respiratory disease with comorbidities associated with their underlying disease processes, but also factors related to their lengthened intensive care unit (ICU) stay. This includes delayed development with neurodevelopmental impairment, ICU delirium, pulmonary hypertension, airway disease, gastroesophageal reflux disease, feeding difficulties, retinopathy of prematurity and more.
The Complex BPD Program at Children’s National encompasses a group of specialists dedicated to improving the care of infants with BPD and other chronic lung disease of infancy. BPD places extreme demands on families. Education is a critical component for families and our team works to make sure they are well informed, have realistic expectations and understand their care plan.
What they’re saying
“Our program is unique in that it has the ability to follow the patient for several months in the NICU as well as after discharge,” says Maria Arroyo, M.D., pulmonologist and co-lead of the Complex BPD Program at Children’s National. “This includes a subacute facility where some of our NICU patients transfer to for continued respiratory weaning and rehabilitation with parent education and outpatient visits once families are home.”
“Since this program was created, we have improved patient care and outcomes with this interdisciplinary approach,” says John Berger, M., medical director for the Pulmonary Hypertension Program at Children’s National. “We expect that with our consistent and personalized care, patients will continue have better overall outcomes, less readmissions and improved neurodevelopmental outcomes.
Q: In your opinion, how would you classify the state of Celiac Disease among children in the United States as it compares to 10 years ago?
A: Celiac disease in the United States is increasingly recognized as a common disorder of childhood. While this can partly be attributed to improved recognition and more testing by primary care providers, “true incidence” of this immune-mediated disease is also said to be rising, independent of the screening practices.
While there is a larger variety of gluten-free food options available today, these options are often more costly than their gluten-counterparts. Additionally, gluten-free food options are not covered by insurance companies. With inflation and rising food prices, there is a real concern for increased economic burden and food insecurity for the families of children with celiac.
Q: Are there any misnomers about Celiac Disease that you think are important to address?
A: Classic symptoms of celiac disease, including diarrhea and malnutrition, now occur in a minority of celiac patients. There is a rising proportion of milder or asymptomatic presentation at the time of diagnosis. Paired with the fact that most celiac patients identified through mass-screening did not have a family history of celiac disease (~90%) or gastrointestinal symptoms (70%), one may need to have a high index of suspicion for children with atypical presentation, such as neurologic symptoms, iron and vitamin D deficiency.
Q: What excites you about the future of treatment for Celiac Disease?
A: I am encouraged that researchers across the globe are testing new, pharmacologic therapies for celiac disease. Some of the tested compounds, such as latiglutenase or TAK-062 , involve breaking down gluten-using enzymes, while others, such as TAK-101 aim to induce immune tolerance. These compounds are still at the early stages of the long and arduous process of drug development and approval. Current randomized trials typically include adults, although some recently opened enrollment to adolescents. While there are no guarantees, I am cautiously optimistic that in the future we will have pharmacologic, non-dietary treatment options for celiac disease.
Q: How is Children’s National a leader in the field of pediatric Celiac Disease?
A: Concerns about gluten exposure can be quite stressful for our patients and families, which can lead to hypervigilance, anxiety and even depression. We are fortunate to have a full-time psychologist, Shayna Coburn, Ph.D., as part of our Multidisciplinary Celiac Disease Program team. This allows us to treat not only physiological symptoms and concerns associated with celiac disease, but to also evaluate the psychological problems that may arise as a result of the condition.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2023/03/USA-line-art-map-with-a-wheat-plant-icon-feature.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2023-03-01 11:09:492023-03-01 11:11:16State of Celiac Disease in the United States
Dr. Dome’s mission is to come up with other therapies to treat Wilms tumor.
Conducting Wilms tumor research has placed Children’s National Hospital as the leader in this space. Jeffrey Dome, M.D., Ph.D., talks about the future of Wilms tumor therapies and what excites him about being able to offer different treatment options to this group of patients.
Q: What’s next for Wilms tumor therapy treatments?
A: Something we’ve learned is that we’re reaching the maximum benefit that we can achieve with conventional chemotherapy and have hit a plateau with some of the high-risk Wilms tumors.
If you look at the history of Wilms tumor, we’ve ratcheted up the cure rate by adding more and more chemotherapy agents. We’re now at a point in which patients can’t tolerate having more toxic chemotherapy added to their regimen. Our mission is to come up with other therapies that work differently.
Q: What have you learned that can lead to new therapy options?
A: I was a co-investigator on a National Cancer Institute-funded project called the TARGET study, which entailed sequencing several hundred Wilms tumors to identify genetic mutations that could be druggable. While a wealth of knowledge was gained on the nature of the biology of Wilms tumor, only a minority of Wilms tumors have targetable mutations. We understand what the mutations are, but most are not mutations that lend themselves to drug therapy.
Therefore, we must think about other forms of therapy for Wilms tumor, such as immunotherapy. That’s where the work with the T cells targeting the WT1 protein Wilms tumor cells come into play.
Q: What do the new therapies look like?
A: I think the future of Wilms tumor therapy will be combining the classic chemotherapy and radiation therapy that we’ve used for many years but also adding another component for the patients with the highest risk of relapse, such as immunotherapy.
Up until now, the studies that we’ve conducted using immunotherapy have introduced this treatment very late as a last resort after everything else has failed. We have seen early signals of efficacy and if we can corroborate this in current studies, I believe the future will be to introduce immunotherapy earlier in the treatment course.
There has been success using immunotherapy in other pediatric solid tumors such as neuroblastoma. I believe the prospects for Wilms tumor are also exciting.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2023/02/diseased-kidneys-feature.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2023-02-22 11:43:522023-02-22 11:52:30The future of Wilms tumor therapies: Q&A with Jeffrey Dome, M.D., Ph.D.
Children’s National has become a resource for patients and families with Wilms tumor.
During the past year, Children’s National Hospital saw nearly 100 patients with Wilms tumor and other less common kidney cancers of childhood, far more than most centers in the country. This is largely due to the reputation the hospital has established for specializing in these diseases. While most patients with Wilms tumor have excellent outcomes, a significant minority of children with kidney cancer do not fare well. Children’s National has become a resource for patients and families with these challenging cancers.
Behind this reputation is Jeffrey Dome, M.D., Ph.D., senior vice president of the Center for Cancer and Blood Disorders and division chief of Oncology at Children’s National, and the team of researchers he leads. For over a decade, he chaired the Children’s Oncology Group Renal Tumor Committee, an opportunity that gave him and his work great exposure.
Dr. Dome shares more on how Children’s National is leading in this space and what the future holds for new, exciting Wilms tumor treatment options.
Q: How is Children’s National leading in this space?
A: The good news is that for the most common type of childhood kidney cancer, Wilms tumor with “favorable histology,” the survival rate is more than 90%, which is an incredible success story. But approximately 25% of children and teens with other types of Wilms tumor and other kidney cancers do not fare as well. We specialize in kidney cancers that are harder to treat, such as anaplastic Wilms tumor, relapsed favorable histology Wilms tumor, bilateral Wilms tumor, clear cell sarcoma of the kidney, malignant rhabdoid tumor and renal cell carcinoma. Because we see a relatively large number of patients, we can draw on our prior experience and observations to recommend the best treatment options.
Q: What’s unique about this research?
A: We have several early-phase clinical trials that are of interest for children with relapsed kidney tumors. Some of these trials are part of research consortia, such as the National Cancer Institute-funded Pediatric Early Phase Clinical Trials Network (PEP-CTN). Other studies have been developed in-house at Children’s National, including a couple of studies using T cells to target pediatric solid tumors. The T cells that have been engineered by the Children’s National Cellular Therapy Laboratory are of particular interest for Wilms tumor because they target a protein called WT1, which is expressed in most Wilms tumors. In fact, WT1 was named after Wilms tumor. We have now had more than 25 patients with relapsed Wilms tumor come from around the country to participate in these studies. Based on early successes, we are continuing this line of research and trying to improve the technology in the current generation of studies.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2023/02/Wilms-tumor-CNRI-feature.png385685Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2023-02-22 11:17:572023-02-22 11:33:50Leading Wilms tumor research nationwide: Q&A with Jeffrey Dome, M.D., Ph.D.
“I just want to hammer home the fact that, if you have a child with a pediatric solid tumor who relapses, most likely the chemotherapy that will be treating that child will be the same chemotherapy that a child diagnosed 20 years ago would have received. This is how little progress has been made…. This is what we are trying to change.”
Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research at Children’s National Hospital, pulled the curtain back on her work fighting pediatric brain tumors at The Atlantic’s People V. Cancer summit. This annual event brings together leading voices from the front lines for in-depth conversations about how to stop this complex and lethal disease. Dr. Bollard discussed the unique importance of collaboration among pediatric oncologists and the optimism she has for using a patient’s immune system to go after solid tumors with CAR T therapies.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2022/11/Catherine-Bollard-at-cancer-summit-CNRI-featured.png385685Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2022-11-15 16:03:362022-11-16 08:50:03In the news: People v. pediatric cancer
The division of urology creates tailored orientation program
The Division of Urology at Children’s National has developed a first-of-its-kind orientation program for pediatric urology advanced practice providers (APPs). This program was created based on real feedback from pediatric urology APPs in the United States and Canada.
The primary purpose of the orientation program is to increase the knowledge and confidence of pediatric APP’s in transition to independent practice. The program prepares the next group of advanced practice providers in the Division of Urology to achieve the same high quality of care that the current group provides to our patients.
“I think what makes our orientation program unique is how involved leadership is in training the orientee. We are with our orientee every step of the way every week. We are there making sure that they are attaining their skills and their knowledge. We’re checking in on them very regularly, and we also are trying to make it very individualized to that person.” said Katie Scarpaci, nurse practitioner and creator of the APP orientation program.
Global summit to address complex pediatric scoliosis
Session highlights included:
“Overall this was a great meeting with valuable content,” says Dr. Oetgen. “Learning and teaching alongside fellow orthopaedic leaders is a fantastic opportunity for everyone involved.”
Dr. Oetgen participated in the following sessions:
The 2024 ICEOS Meeting will be November 13-15 in Phoenix, AZ.
Hip preservation: Moving the needle in patient care
Hip preservation treats a wide variety of conditions such as hip dysplasia, hip impingement, hip torsion/rotation abnormalities, hip synovitis, snapping hip and hip labrum tears.
The Hip Preservation Program at Children’s National Hospital offers advanced care to children and adults with hip pain, providing a continuum of care that extends well into adulthood. Jeffrey Peck, M.D., an orthopaedic surgeon with subspecialty interests in pediatric and young adult hip preservation, discusses the program and what Children’s National is doing to move the needle forward in patient care.
Q: What is hip preservation and what are common conditions it addresses?
A: The field of hip preservation is dedicated to diagnosing and treating hip conditions that result in hip pain and can potentially result in hip degeneration.
There are several potential conditions that can lead to hip pain and eventual hip joint degeneration. These include atypical anatomy in and around the hip joint, which can be congenital, developmental or traumatic in origin. Strains and sprains of the muscles and ligaments around the hip may also cause hip joint pain. Hip preservation treats a wide variety of conditions such as hip dysplasia, hip impingement, hip torsion/rotation abnormalities, hip synovitis, snapping hip and hip labrum tears.
Q: Who is affected and when should a patient seek out an orthopaedic expert?
A: The people most often affected by conditions treated through hip preservation are adolescents and young adults up to age 45 years old who develop hip pain. When hip pain does not subside, it can be due to a condition that has resulted in hip joint inflammation that may eventually begin to cause hip joint degeneration leading to arthritis if the condition is not treated. If a patient has hip pain that lasts for multiple months without improving, it is prudent to seek evaluation with a hip preservation orthopaedic surgeon specialist.
Q: What is Children’s National doing to move the needle for hip preservation patients?
A: Children’s National cares for patients with hip pain using an integrated care team, featuring a Hip Preservation fellowship trained surgeon, physiatry experts, sports psychologists, radiologists and physical therapists with a focus on adolescents and young adults. We work in a collaborative environment with the shared goal of helping patients with hip pain return to living life without the pain that has held them back.
The Children’s National team also collects patient reported outcome scores to better understand the severity of patient symptoms and, later, how surgery has improved those symptoms. Additionally, Children’s National is engaged in research endeavors evaluating hip conditions and how patients have responded to treatment. By pursuing these efforts, we not only assist the patients we see today, but also contribute to advancing care to better help future patients.
Q: What excites you about the future for the field of hip preservation?
A: The field of Hip Preservation is a young specialty within orthopaedic surgery that has demonstrated consistently positive outcomes for patients who previously had very limited treatment options for their hip pain. With this comparative newness comes continual innovation and advancement as we learn more about how hip pain can occur and what we can do to best treat that pain. Procedures that we perform at Children’s National such as the Ganz Periacetabular Osteotomy (PAO) and the use of hip arthroscopy to treat hip impingement are relatively new innovations in the field of orthopaedics that can provide immense relief to patients having hip pain.
Revolutionizing pediatric nephrology one patient at a time
“With each kidney transplant, we’re not just restoring the health of children, we’re pioneering progress and setting new benchmarks for the field of pediatric nephrology,” says Marva Moxey-Mims, M.D., chief of Nephrology at Children’s National Hospital.
“With each kidney transplant, we’re not just restoring the health of children, we’re pioneering progress and setting new benchmarks for the field of pediatric nephrology,” says Marva Moxey-Mims, M.D., chief of Nephrology at Children’s National Hospital. “We offer not just treatment, but a chance for our patients to flourish and live their best life.”
Big picture
The Kidney Transplantation Program at Children’s National is the only one of its kind in the Washington, D.C., region focused on the needs of children and teens with kidney disease.
Performing an average of 15-20 kidney transplants per year, the program’s latest one- and three-year graft and patient survival data from the Scientific Registry for Transplant Recipients is 100%.
“Our program surpasses national numbers, reflecting a commitment to exceptional care, superior patient outcomes and a focus on setting a new standard for excellence in transplant support,” says Dr. Moxey-Mims.
Children’s National leads the way
At the forefront of groundbreaking treatment, the nephrology team is pioneering progress by providing innovative care and resources. One example is the hospital’s collaboration with the National Kidney Registry and MedStar Georgetown Transplant Institute’s Living Donor Program. This provides a greater chance of finding more suitable donors for difficult-to-match children and has resulted in altruistic donations.
“Through living kidney donations, we redefine the timeline for pediatric transplants. Children experience shorter waits and swifter paths to renewed health compared to the national wait list,” says Asha Moudgil, M.D., medical director of the Kidney Transplantation Program at Children’s National. “It’s a testament to our commitment to making a difference in every child’s life.”
That commitment is also seen in the hospital’s focus on making sure families have the resources they need during their medical journey.
“Caring for a child with renal disease is an immense challenge, not only for the young patients but also for their families. The demanding schedule of hospital visits, often three times a week, adds an extra layer of stress and disruption to daily life,” says Dr. Moudgil.
Through grant funding from donors and national programs, families with children undergoing kidney transplants and dialysis can receive financial assistance. In some cases, families receive up to $2,000 of mortgage or rental payment assistance. Families can also receive funds for medication co-pays and other expenses incurred around the time of the transplant.
“In the face of medical adversity, these funds become a source of support,” says Dr. Moudgil. “We make sure every family, regardless of financial constraints, can access the care and resources their child needs.”
Bottom line
Children’s National is at the forefront of pediatric kidney transplantation. “Our unique approach ensures every facet of a patient’s journey is considered,” says Dr. Moxey-Mims. “We’ve created an environment where success is not just a medical outcome but a comprehensive and sustained improvement in a child’s overall quality of life.”
Learn more about our latest advances in pediatric Nephrology.
Cell & Gene Therapy in the DMV: Experts collaborate for cures
Leaders in medicine, academia, industry and state and local government came together for the first annual Cell and Gene Therapy in the DMV Symposium, hosted at the Children’s National Research & Innovation Campus. The mission: Connect the local scientific community – bursting with expertise and collaboration potential – to develop these cutting-edge therapies for cancers, sickle cell disease and immune-mediated disorders.
The daylong event drew over 100 experts from a range of organizations in the D.C, Maryland and Virginia region, sometimes called the DMV: Children’s National Hospital, the Food and Drug Administration, the National Institute of Standards and Technology, the National Institutes of Health, the General Accounting Office, Virginia Tech, MaxCyte, AstraZeneca, Kite Pharma, Montgomery College, the Maryland State Department of Commerce and more. Together, they unraveled a host of topics including the regulatory environment, workforce development and training, research standards and the promise of these therapies.
“Our Cell & Gene Therapy Symposium brings together our current collaborators and future partners in the D.C., Maryland and Virginia space, which is an incredibly rich area. We see tremendous opportunity and breakthroughs in our future,” said Catherine Bollard, M.D., M.B.Ch.B., interim chief academic officer and chief of Pediatrics at Children’s National Hospital. “Many different diseases rely on the immune system to either be ramped up or to be controlled, and we can seize on these biological processes. Cell and gene therapies are at the heart of where medicine is going.”
The big picture
For decades, oncologists largely have turned to the same menu of treatments to fight cancer, including surgery, chemotherapy and radiation. Cell and gene therapies offer the promise of training the immune system to fight diseases with fewer side effects and potentially higher success rates. Early work has shown progress in liquid cancers, like leukemia, raising the possibility that the therapies could be used on solid tumors and other disorders, such as lupus and sickle cell disease. However, many disciplines must come together to yield discoveries.
“Nobel Prize-winning work doesn’t necessarily translate into available therapies for patients. It takes a whole community like this to make it happen,” said Cenk Sumen, chief scientific officer at MaxCyte Inc., an international cell engineering company based in Rockville, Md. “It has been exciting to see this diverse group of stakeholders come together, which is probably unmatched anywhere on the planet.”
Why we’re excited
Symposium host Patrick Hanley, Ph.D., chief and director of the Cellular Therapy Program at Children’s National, said the goal was to cement the region as the No. 1 location for this highly technical research and development. He believes Children’s National can offer essential elements to this success, given its clinical and research expertise, workforce training opportunities and geographic proximity to the scientific leadership of the federal government. “What makes us unique is our proximity to all the players who can help create new treatment options for patients. We truly are the biomedical capital of the world,” he said.
Michael Friedlander, vice president for health sciences at Virginia Tech, notes that the earliest stages of invention will emanate from academic labs including those at Virginia Tech and Children’s National. “You have basic scientists who are doing fundamental research on properties and procedures that will lead to the new therapies of tomorrow,” he said. “We are putting in place the fundamental pieces to advance children’s health in all dimensions.”
What’s ahead
One challenge is developing a workforce to help prepare cell therapies for patients, following precise standards to ensure the therapy works as designed. Children’s National does this training, as do others in the region. Lori Kelman, Ph.D., M.B.A., biotechnology coordinator and professor at Montgomery College, said that the area is full of people who want to help people and who like science.
“The thing that people might not know is that you don’t need a Ph.D. to work in cell and gene therapy,” she said. “There are opportunities at all levels, including the entry level, which is where a great career often starts.”
A team approach to complex bronchopulmonary dysplasia
“By the time a baby is diagnosed with bronchopulmonary dysplasia, families have already had a long journey with prematurity in the neonatal intensive care unit (NICU),” says Hallie Morris, M.D., neonatologist and lead of the Complex Bronchopulmonary Dysplasia (BPD) Program at Children’s National Hospital. “To be able to have a team that is focused on the holistic health of their child in the context of this diagnosis makes a world of difference to these families.”
The big picture
Some premature infants with BPD experience more severe respiratory disease with comorbidities associated with their underlying disease processes, but also factors related to their lengthened intensive care unit (ICU) stay. This includes delayed development with neurodevelopmental impairment, ICU delirium, pulmonary hypertension, airway disease, gastroesophageal reflux disease, feeding difficulties, retinopathy of prematurity and more.
The Complex BPD Program at Children’s National encompasses a group of specialists dedicated to improving the care of infants with BPD and other chronic lung disease of infancy. BPD places extreme demands on families. Education is a critical component for families and our team works to make sure they are well informed, have realistic expectations and understand their care plan.
What they’re saying
Learn more about the Complex BPD Program.
State of Celiac Disease in the United States
Q: In your opinion, how would you classify the state of Celiac Disease among children in the United States as it compares to 10 years ago?
A: Celiac disease in the United States is increasingly recognized as a common disorder of childhood. While this can partly be attributed to improved recognition and more testing by primary care providers, “true incidence” of this immune-mediated disease is also said to be rising, independent of the screening practices.
While there is a larger variety of gluten-free food options available today, these options are often more costly than their gluten-counterparts. Additionally, gluten-free food options are not covered by insurance companies. With inflation and rising food prices, there is a real concern for increased economic burden and food insecurity for the families of children with celiac.
Q: Are there any misnomers about Celiac Disease that you think are important to address?
A: Classic symptoms of celiac disease, including diarrhea and malnutrition, now occur in a minority of celiac patients. There is a rising proportion of milder or asymptomatic presentation at the time of diagnosis. Paired with the fact that most celiac patients identified through mass-screening did not have a family history of celiac disease (~90%) or gastrointestinal symptoms (70%), one may need to have a high index of suspicion for children with atypical presentation, such as neurologic symptoms, iron and vitamin D deficiency.
Q: What excites you about the future of treatment for Celiac Disease?
A: I am encouraged that researchers across the globe are testing new, pharmacologic therapies for celiac disease. Some of the tested compounds, such as latiglutenase or TAK-062 , involve breaking down gluten-using enzymes, while others, such as TAK-101 aim to induce immune tolerance. These compounds are still at the early stages of the long and arduous process of drug development and approval. Current randomized trials typically include adults, although some recently opened enrollment to adolescents. While there are no guarantees, I am cautiously optimistic that in the future we will have pharmacologic, non-dietary treatment options for celiac disease.
Q: How is Children’s National a leader in the field of pediatric Celiac Disease?
A: Concerns about gluten exposure can be quite stressful for our patients and families, which can lead to hypervigilance, anxiety and even depression. We are fortunate to have a full-time psychologist, Shayna Coburn, Ph.D., as part of our Multidisciplinary Celiac Disease Program team. This allows us to treat not only physiological symptoms and concerns associated with celiac disease, but to also evaluate the psychological problems that may arise as a result of the condition.
The future of Wilms tumor therapies: Q&A with Jeffrey Dome, M.D., Ph.D.
Dr. Dome’s mission is to come up with other therapies to treat Wilms tumor.
Conducting Wilms tumor research has placed Children’s National Hospital as the leader in this space. Jeffrey Dome, M.D., Ph.D., talks about the future of Wilms tumor therapies and what excites him about being able to offer different treatment options to this group of patients.
Q: What’s next for Wilms tumor therapy treatments?
A: Something we’ve learned is that we’re reaching the maximum benefit that we can achieve with conventional chemotherapy and have hit a plateau with some of the high-risk Wilms tumors.
If you look at the history of Wilms tumor, we’ve ratcheted up the cure rate by adding more and more chemotherapy agents. We’re now at a point in which patients can’t tolerate having more toxic chemotherapy added to their regimen. Our mission is to come up with other therapies that work differently.
Q: What have you learned that can lead to new therapy options?
A: I was a co-investigator on a National Cancer Institute-funded project called the TARGET study, which entailed sequencing several hundred Wilms tumors to identify genetic mutations that could be druggable. While a wealth of knowledge was gained on the nature of the biology of Wilms tumor, only a minority of Wilms tumors have targetable mutations. We understand what the mutations are, but most are not mutations that lend themselves to drug therapy.
Therefore, we must think about other forms of therapy for Wilms tumor, such as immunotherapy. That’s where the work with the T cells targeting the WT1 protein Wilms tumor cells come into play.
Q: What do the new therapies look like?
A: I think the future of Wilms tumor therapy will be combining the classic chemotherapy and radiation therapy that we’ve used for many years but also adding another component for the patients with the highest risk of relapse, such as immunotherapy.
Up until now, the studies that we’ve conducted using immunotherapy have introduced this treatment very late as a last resort after everything else has failed. We have seen early signals of efficacy and if we can corroborate this in current studies, I believe the future will be to introduce immunotherapy earlier in the treatment course.
There has been success using immunotherapy in other pediatric solid tumors such as neuroblastoma. I believe the prospects for Wilms tumor are also exciting.
Leading Wilms tumor research nationwide: Q&A with Jeffrey Dome, M.D., Ph.D.
Children’s National has become a resource for patients and families with Wilms tumor.
During the past year, Children’s National Hospital saw nearly 100 patients with Wilms tumor and other less common kidney cancers of childhood, far more than most centers in the country. This is largely due to the reputation the hospital has established for specializing in these diseases. While most patients with Wilms tumor have excellent outcomes, a significant minority of children with kidney cancer do not fare well. Children’s National has become a resource for patients and families with these challenging cancers.
Behind this reputation is Jeffrey Dome, M.D., Ph.D., senior vice president of the Center for Cancer and Blood Disorders and division chief of Oncology at Children’s National, and the team of researchers he leads. For over a decade, he chaired the Children’s Oncology Group Renal Tumor Committee, an opportunity that gave him and his work great exposure.
Dr. Dome shares more on how Children’s National is leading in this space and what the future holds for new, exciting Wilms tumor treatment options.
Q: How is Children’s National leading in this space?
A: The good news is that for the most common type of childhood kidney cancer, Wilms tumor with “favorable histology,” the survival rate is more than 90%, which is an incredible success story. But approximately 25% of children and teens with other types of Wilms tumor and other kidney cancers do not fare as well. We specialize in kidney cancers that are harder to treat, such as anaplastic Wilms tumor, relapsed favorable histology Wilms tumor, bilateral Wilms tumor, clear cell sarcoma of the kidney, malignant rhabdoid tumor and renal cell carcinoma. Because we see a relatively large number of patients, we can draw on our prior experience and observations to recommend the best treatment options.
Q: What’s unique about this research?
A: We have several early-phase clinical trials that are of interest for children with relapsed kidney tumors. Some of these trials are part of research consortia, such as the National Cancer Institute-funded Pediatric Early Phase Clinical Trials Network (PEP-CTN). Other studies have been developed in-house at Children’s National, including a couple of studies using T cells to target pediatric solid tumors. The T cells that have been engineered by the Children’s National Cellular Therapy Laboratory are of particular interest for Wilms tumor because they target a protein called WT1, which is expressed in most Wilms tumors. In fact, WT1 was named after Wilms tumor. We have now had more than 25 patients with relapsed Wilms tumor come from around the country to participate in these studies. Based on early successes, we are continuing this line of research and trying to improve the technology in the current generation of studies.
In the news: People v. pediatric cancer
“I just want to hammer home the fact that, if you have a child with a pediatric solid tumor who relapses, most likely the chemotherapy that will be treating that child will be the same chemotherapy that a child diagnosed 20 years ago would have received. This is how little progress has been made…. This is what we are trying to change.”
Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research at Children’s National Hospital, pulled the curtain back on her work fighting pediatric brain tumors at The Atlantic’s People V. Cancer summit. This annual event brings together leading voices from the front lines for in-depth conversations about how to stop this complex and lethal disease. Dr. Bollard discussed the unique importance of collaboration among pediatric oncologists and the optimism she has for using a patient’s immune system to go after solid tumors with CAR T therapies.