Rare pediatric growth disorders collectively affect a significant number of children worldwide. While each disorder may individually be rare, together they contribute to a substantial portion of pediatric endocrinology cases. For example, pediatric growth hormone deficiency (PGHD) affects approximately one in 3,500 to one in 10,000 children.
PGHD is typically diagnosed during childhood, with most cases identified before the age of three or four years old. However, some cases may not be diagnosed until later in childhood or adolescence.
Existing therapeutic options for PGHD consist of injectable formulations of recombinant human growth hormone (rhGH), administered subcutaneously either on a daily or weekly basis. Initiation of treatment is recommended promptly upon confirmation of a diagnosis of PGHD. Prolonged treatment spanning seven years on average is often necessary to attain “catch-up” growth and realize maximum height potential. However, the discomfort associated with routine injections may result in missed doses and hinder optimal growth outcomes.
With a steadfast commitment to innovation and ethical excellence, Lumos Pharma aims to revolutionize the treatment of rare pediatric growth disorders. Xtalks spoke with Dr. Pisit “Duke” Pitukcheewanont, Lumos Pharma’s Chief Medical Officer, to unravel the company’s remarkable journey and vision for the future.
With over 25 years of experience, Dr. Duke’s expertise in growth and bone disorders has been instrumental in advancing clinical research and improving patient outcomes. His decision to join Lumos Pharma was driven by the company’s innovative approach to developing the first orally administered small molecule candidate for moderate PGHD, which promotes the secretion of growth hormone. Following positive Phase II results in 2023, the company expects to launch a registrational Phase III trial later this year.
In this article, we explore Dr. Duke’s inspiring journey, Lumos Pharma’s unique approach to rare pediatric disease research and development and their unwavering commitment to ethical standards and patient well-being.
XTALKS CLINICAL EDGE: Issue 2 — Lumos Pharma’s Interview
Xtalks Clinical Edge is a magazine for clinical research professionals and all who want to be informed about the latest trends and happenings in clinical trials. This magazine allows you to dive into a world where industry leaders, patient advocates and top researchers converge to bring you the sharpest insights in clinical trials.
Challenges in Rare Pediatric Disease Drug Development
Rare pediatric diseases often present formidable challenges, from identifying patients to navigating complex regulatory pathways. However, for Dr. Duke, a pediatric endocrinologist by training, these challenges are not insurmountable obstacles but opportunities for transformative change.
One of the foremost challenges is identifying patients. Rare diseases, by definition, affect a small subset of the population, making it difficult to assemble a sufficient cohort for clinical trials. Dr. Duke highlighted the need for robust collaboration among healthcare providers, advocacy groups and research institutions to identify and engage eligible patients.
Establishing research infrastructure in regions with limited resources poses another significant hurdle. While some areas may have a high prevalence of rare diseases, they may lack the necessary facilities and expertise to conduct clinical trials effectively. Overcoming this challenge requires strategic partnerships and investment in building research capacity in underserved communities.
Moreover, rare pediatric diseases often present with diverse manifestations, ranging from mild to severe. Understanding the nuances of disease presentation is essential for designing clinical trials that target specific patient populations effectively. Dr. Duke emphasized the importance of tailoring research strategies to accommodate this heterogeneity and ensure that treatments address a range of disease severity.
Obtaining sufficient data is another critical challenge in rare disease research. Limited patient populations and the rarity of these conditions make it tricky to collect comprehensive clinical data. However, Dr. Duke’s extensive experience and collaboration with organizations like the Human Growth Foundation, where he is the President and CEO, position him well to navigate these obstacles and drive impactful research initiatives.
“To address these challenges, we require extensive collaboration across stakeholders, innovative trial design and a patient-centric approach to drive the advancement of this treatment in pediatric growth hormone deficiency.”
— Dr. Pisit “Duke” Pitukcheewanont
Navigating the clinical trial and regulatory pathway requires meticulous planning and adherence to stringent ethical standards. Lumos Pharma is committed to addressing these challenges through innovative trial design, patient-centric approaches, and transparent communication with regulatory authorities. By leveraging Dr. Duke’s expertise and forging strategic partnerships, Lumos Pharma aims to accelerate the development of life-changing treatments for rare pediatric diseases.
In addition, ethical considerations are paramount in developing treatments for children. Dr. Duke emphasized the importance of informed consent, patient well-being and transparent communication.
“Lumos Pharma always addresses these by prioritizing rigorous ethical standards,” he says.
By fostering transparent communication and collaboration with patients, their caregivers, healthcare providers and regulatory authorities, Lumos Pharma ensures that ethical considerations remain at the forefront of every decision.
Navigating the Regulatory Landscape
The regulatory environment plays a significant role in shaping drug development strategies for rare diseases. Dr. Duke highlighted the importance of leveraging Orphan Drug designation to expedite the approval process.
However, navigating the regulatory landscape for rare diseases requires more than obtaining designations and approvals. It also entails robust collaboration with regulatory agencies to address unique challenges and uncertainties associated with rare pediatric conditions. Dr. Duke emphasized the importance of aligning development plans with regulatory expectations and leveraging innovative trial designs to facilitate efficient regulatory review.
“We believe that our proactive engagement will pave the way for a streamlined and efficient development process, thereby expediting access to innovative treatments for patients with this condition,” he says.
Lumos Pharma’s Future Goals and Impact
Dr. Duke emphasized the importance of prioritizing robust clinical development to help ensure the potential approval and availability of the first oral treatment for moderate PGHD. He underscored the significance of strengthening partnerships, both domestically and globally, when planning for global access.
Furthermore, Dr. Duke emphasized the importance of streamlining organizational capabilities to facilitate a seamless transition from clinical development to commercialization. By optimizing internal processes and resources, Lumos Pharma aims to maximize its efficiency and effectiveness.
Ultimately, Lumos Pharma’s overarching goal is to provide an innovative treatment that improves the lives of children affected by PGHD. “Combining scientific excellence with a patient focus, we aim to make a lasting impact on children’s well-being,” says Dr. Duke.
The Future of Rare Pediatric Disease Treatment
Dr. Duke envisions transformative changes in the treatment landscape of rare pediatric diseases. Advancements in genomic technology, precision medicine and gene therapy offer hope for personalized treatments and improved outcomes. Increased collaboration and advocacy efforts will further support the development of innovative therapies, ensuring a brighter future for affected children.
“The future holds promise for improved and personalized treatment, offering hope for better outcomes in those affected children,” he says.
Moreover, Dr. Duke emphasized the importance of increased collaboration among researchers, industry stakeholders and regulatory bodies in driving innovation and accelerating the development of targeted therapies for rare diseases. By fostering collaborative efforts and sharing knowledge and resources, the pediatric healthcare community can overcome challenges and pave the way for groundbreaking treatments.
In addition to technological advancements, Dr. Duke says “advocacy and awareness efforts are important for creating a supportive ecosystem for rare pediatric diseases.” By raising awareness and advocating for increased funding and research initiatives, advocacy groups can amplify their impact and drive positive change.
By harnessing the power of new technologies, fostering collaboration and raising awareness, the pediatric healthcare community can make significant strides towards improving the lives of children with rare diseases.
Guided by Dr. Duke’s expertise, Lumos Pharma is steadfast in its commitment to rare pediatric diseases, striving to make a profound impact on the lives of children worldwide.
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