Translational Research Programs

At the heart of our mission are three core pillars, Diagnostics, Therapeutics, and Implementation Science, each guided by the essential themes of Foundation, Innovation, and Invention. This structured approach ensures that every child who comes to Sidra Medicine for cancer care benefits from the latest diagnostic advances and innovative treatment options, giving them the best chance for successful outcomes.

1. Precision Diagnostics and Personalized Treatment

Our research is at the forefront of precision medicine, utilizing advanced diagnostic tools to tailor treatments specifically for your child. Understanding the genetics of individual childhood cancers is crucial for identifying molecular targets that can be exploited for therapeutic benefits.

Advanced Molecular Profiling

Every child with a suspected or diagnosed cancer can benefit from molecular profiling at the point of diagnosis, providing the most accurate diagnostic methodology available today.

• Comprehensive Characterization: We use advanced genetic testing methods called Next-Generation Sequencing (NGS) and multi-omics analyses to deeply study each child’s cancer. This means we examine the entire DNA (whole genome sequencing), all the messages sent by genes (whole transcriptome or RNA sequencing), and chemical changes that control how genes work (epigenetic profiling). Looking at cancer in this much detail helps us make a more accurate diagnosis, understand each patient’s level of risk, and find treatments that are most likely to work for them. We also analyze the child’s non-cancer DNA (germline DNA) to compare with cancer cells and to see if there is an inherited risk for cancer for them or their family.
• Rapid Results for Aggressive Cases:For aggressive or relapsed cases with poor prognoses, our Expedited Genomic Oncology Profiling (eGOP) procedure provides rapid molecular characterization and reporting, typically within 2–3 weeks, to guide urgent diagnosis and treatment decisions.
• Cutting-Edge Technology: We are always searching for and using the latest technologies to improve how we care for children with cancer and to advance our research. For example, we use Oxford Nano Pore Technology long reads sequencing to look closely at big changes in a person’s DNA that might be missed by other tests. We also use spatial single cell techniques (such as Visium or Curio) and special microscopes like Phenocycler, which help us study individual cancer cells and their surroundings in great detail. On top of this, we plan to use machine learning and AI algorithms with digital slides of tissue samples. This helps us spot tiny differences in how cells look and find links between these changes and gene mutations, making it easier to understand and treat each child’s cancer more precisely.
• Non-Invasive Monitoring (Liquid Biopsy): Our researchers are investigating a method called liquid biopsy, a simple, noninvasive way to check how a child’s cancer is changing or responding to treatment over time. Instead of needing a traditional tissue biopsy, this process uses a regular blood sample to look for tiny bits of cancer cells or genetic material from the tumor (like DNA or RNA) that are floating in the blood. By studying these fragments, doctors can spot signs that the cancer might be becoming resistant to treatment and can also analyze patterns in the patient’s proteins and other small molecules. This helps us better understand what’s happening in the body and make more informed decisions about care, all without the need for surgery or other invasive procedures.

2. Building National Resources and Research Cohorts

We continuously build national resources like the Sidra Medicine Pediatric Cancer Biorepository and our Pediatric Cancer Registry to gather crucial clinical data and biological samples. These resources form the foundation for personalized medicine tailored specifically to our patient population.

• Cohort Focus: Each year, our program expects to include about 35 to 50 children, which is roughly half of all young cancer patients in Qatar. For every child who joins, we carefully collect important samples and information to help us learn as much as possible. Right now, we’re especially focused on two main groups: kids with Acute Lymphoblastic Leukemia (which makes up 24% of the cases we see), and kids with Central Nervous System tumors (which account for 21% of the cases). This helps us gather detailed knowledge about these common cancers.
• Collaborative Strategy: We are bringing these repositories under an Institutional organizational structure and build larger regional cohorts for selected cancer types (such as brain tumors and Neuroblastoma) through synergy and collaboration with regional institutions (Pakistan, Jordan, Turkey).

3. Therapeutic Innovation

The SPOQ-TRP is focused on high-impact therapeutic interventions for our gold cohorts of cancer patients.

Personalized Pharmacogenomics (PGx)

We apply Pharmacogenomic (PGx) approaches to understand how your child’s unique genetic profile influences their response to medication. This personalized strategy helps us to optimize drug dosages, reduce potential toxicities, reduce morbidity, and improve long-term survival. We plan to develop and uniformly apply PGx at diagnosis for all cancer patients. We have an ongoing PGx trial for cancer patients.

Cell Therapy

For patients with advanced tumors who have developed recurrence or resistance to standard treatments, our Advanced Cell Therapy Core (ACTC) team is actively engaged in investigating and developing Chimeric Antigen Receptor T-Cells (CAR-T) manufacturing. This includes developing adoptive cell therapy products like “off-the-shelf” living drugs.

• Local Manufacturing and Infrastructure:This manufacturing occurs within our newly established Good Manufacturing Practice (GMP) facility. The manufacturing process will utilize vectors provided by Caring Cross, USA, to develop CAR-T cells (e.g., anti-CD19 CAR-T cells) designed to redirect T cells, a subset of cells from the immune system specialized in fighting cancer, towards pediatric tumors.
• Regional Hub:Our goal is to make these advanced “living drugs” available to cancer patients in the Gulf Region through collaborations with biotech and pharmaceutical partners.

4. Discovery and Invention

Our research is dedicated to discovering new ways to understand and treat cancer. We focus on several key areas: the immune environment around tumors. This means we study how the body’s natural defense system [the immune system] interacts with cancer cells, which could help us find new treatments that use the immune system to fight cancer (immunotherapy). We are also interested in how cancer and the microbiome interact. The microbiome is the community of tiny living things (like bacteria) in our bodies. We plan to study how these microbes, along with diet and nutrition, might affect children with certain types of cancer. Another important area is classifying brain tumors using methylomics and artificial intelligence (AI). Methylomics is the study of small chemical changes to DNA that can influence how genes work. By using AI to analyze these changes, we hope to better identify and understand different types of brain tumors.

5. Collaborative Science and Research Leadership

Our program is structured to ensure alignment between clinical care and research objectives, led by a dedicated team. Our collaborations with international organizations like The Children’s Oncology Group (COG) and the International Society of Paediatric Oncology (SIOP) ensure our patients benefit from global expertise and access to the latest advancements in cancer treatment.

6. Annual report pediatric oncology in Qatar

The Sidra Medicine Pediatric Oncology Report (released every year in September, highlighting Pediatric Cancer awareness month) is a key annual publication representing a joint effort by multiple divisions, including the Translational Research Program: Sidra Pediatric Oncology Qatar. The report’s primary purpose is to share precision oncology data, highlighting how clinical and research efforts help detect actionable somatic mutations and drive the identification of emerging biomarkers and innovative therapeutic approaches. Content includes detailed insights into the most prevalent pediatric cancers in Qatar (Leukemia, CNS malignancies, Lymphoma, Neuroblastoma, and Germ cell tumors), which constitute over 70 percent of cases, and offers an overview of foundational resources like the Sidra Medicine Pediatric Cancer Biorepository and Registry. It details the Expedited Genomic Oncology Profiling (eGOP) procedure and provides snapshots of advanced diagnostic reports such as the Personal Cancer Genome Reporter (PCGR), Fusion reports, Cancer Predisposition Sequencing Reporter (CPSR), and Methylation Classification Reports. Additionally, the report contains a section on Cancer Grant Awards and the Cancer Publication List. The report, alongside previous years’ editions, can be found and downloaded from the Publications section.

7. Program Leadership Team

The SPOQ-TRP is led by a dedicated team responsible for overseeing operations and ensuring alignment with key objectives.

• Dr. Wouter Hendrickx: Program Director, PI Tumor Biology and Immunology Lab.
• Dr. Chiara Cugno: Program Clinical Co-Director, Acting Division Chief of Oncology and Hematology.
• Dr. Ata Maaz: Research Co-Lead, Senior Attending Physician Pediatric Oncology and Hematology
• Dr. Erdener Ozer: Clinical Co-Lead, Chief of Anatomical Pathology.
• Dr. Mohammed Yousuf Karim: Chief of Hematopathology.
• Dr. Ian Pople: Chief of Neurosurgery.Bottom of Form