# BMT Core - Pediatric Transplantation and Cellular Therapy Consortium (PTCTC):  Providing Clinical Trial Access For Children With Life Threatening Diseases

> **NIH NIH UG1** · NATIONAL MARROW DONOR PROGRAM · 2024 · $184,489

## Abstract

Abstract:
Allogeneic hematopoietic cell transplantation (HCT) is uniquely curative for a variety of pediatric malignant and
non-malignant conditions. With respect to non-malignant conditions, due to the increasing use of DNA-based
screening and diagnostic testing, many of the genetic diseases that are curable by HCT are now identified early
in life. Accordingly, ~35% of all pediatric HCT procedures are performed for non-malignant diseases, which is
significantly higher than the proportion of non-malignant diseases for which HCT is performed for adults. HCT
approaches differ significantly between patients with malignant and non-malignant diseases. Those with
malignant conditions, in both children and adults, undergo extensive cytotoxic chemotherapy cycles, leading to
a compromised immune system and reduced capacity to reject donor cells. In contrast, patients with non-
malignant diseases, having not undergone prior chemotherapy, exhibit a spectrum of immune responses,
ranging from absence to hyper-activation, depending on the specific hematopoietic cell transplantation indication.
Thus, a more personalized HCT approach may benefit such patients. One of the major areas of unmet need for
personalized HCT arises from the fact that the metabolism of pre-HCT chemotherapy varies significantly across
individuals. This variation, in both weight-based and body surface area-based dosing, can lead to some patients
receiving too much or too little chemotherapy exposure, risking either toxicity or non-engraftment. Because of
this, the ideal situation might be one where pharmacokinetic modeling could be used to more accurately dose
patients, which would better balance immune suppression and toxicity. These and many other variables highlight
the unique needs of pediatric HCT recipients, making it a distinct discipline within the larger field of HCT. Further,
at any single transplant center, the number of children and young adults undergoing HCT is relatively small, and
the diseases treated are diverse, making it essential for centers to work together in a cooperative group setting.
For >30 years the Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) has been a pediatric
clinical trials consortium focused on bringing clinical trials to children and young adults with both malignant and
non-malignant diseases. The PTCTC is now the largest pediatric-focused HCT consortium in the world. In this
proposal we describe our plans and desire to continue to be a core clinical consortium (CCC) of the BMT CTN.
These include (A) Detailing our unique capabilities of the PTCTC within the BMT CTN; (B) Proposing a clinical
trial to optimize the conditioning regimen for children and adults with Inborn Errors of Immunity (IEI); and (C)
Describing the operational and scientific approach of the PTCTC to HCT trials, and how this approach could
benefit the BMT CTN. Together, our unique expertise, creativity, commitment to diversity, mentorship, and
innovative clinical tri...

## Key facts

- **NIH application ID:** 10939222
- **Project number:** 1UG1HL174426-01
- **Recipient organization:** NATIONAL MARROW DONOR PROGRAM
- **Principal Investigator:** Leslie S Kean
- **Activity code:** UG1 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $184,489
- **Award type:** 1
- **Project period:** 2024-08-15 → 2031-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10939222

## Citation

> US National Institutes of Health, RePORTER application 10939222, BMT Core - Pediatric Transplantation and Cellular Therapy Consortium (PTCTC):  Providing Clinical Trial Access For Children With Life Threatening Diseases (1UG1HL174426-01). Retrieved via AI Analytics 2026-06-13 from https://api.ai-analytics.org/grant/nih/10939222. Licensed CC0.

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