Cell Therapy & Immunobiology Department

The Cell Therapy & Immunobiology (CT&I) Department at KFSH&RC aims to work towards achieving excellence in Cell Therapy using advanced technologies based on scientific discovery and Precision Medicine with the ultimate goal of curing human diseases that are presently considered incurable.

We focus on investigating the molecular and cellular mechanisms of stem and immune biology, with direct therapeutic application in cell therapy and management of cancer and hematopoietic stem cell transplantation.

We employ state-of-the-art technology platforms including transcriptomics and proteomics for a better understanding of stem and immune cell biology and its relation to human diseases and works towards achieving the goal of excellence in immune and cell therapy in the Middle East and worldwide. We aim to expand the production of therapeutic cells e.g. CAR T cells, CAR NK cells, Mesenchymal stem cells (MSc), Natural Killer (NK) cells and Regulatory T-cells (T-regs) for different therapeutic applications with all needed modifications that make them suitable for human use. We have developed proof of concept studies from bench to clinic translation and initiation of Phase I/II clinical trials with advance cell and gene therapy products for KFSH&RC patients.

The departmental members actively conduct a range of translational and research activities in collaboration with KFSHRC’s laboratories and clinical departments, while equally pursuing pre-clinical research. In addition, they are building a dynamic collaboration with local and international institutions to develop platforms within the scientific and clinical community as well as the industry at large.

Apart from research and development, the Department is committed to education and training, and the advancement of Saudi scientists, physicians, technicians and students.

 
Focus areas
•Conduct translational research that has the potential for scientific discovery to promote the management of disease cellular and immune therapies.
•Production of therapeutic cells for therapy like: CAR T cells, CAR NK cells, Mesenchymal stem cells (MSc), Natural Killer (NK) cells and Regulatory T-cells (T-regs) for different therapeutic applications with all needed modifications that make them suitable for human use.
•Proof of concept studies from bench to clinic translation and initiation of Phase I/II clinical trials with advance cell and gene therapy products for KFSH&RC patients.
•Obtain SFDA approval for production and utilization of proven new live medicines at KFSH&RC and to other hospitals in the kingdom.
•Provide advanced biological tools to tract, characterize and study therapeutic cells using techniques like flow cytometry, bio-imaging and sorting cells from a variety of tissues, including bone marrow, dental tissue, and umbilical cord cells.
•Provides functional protein analysis platforms for research and clinical diagnostic
•Provide training and education with student and junior technical staff supervision.
•Conduct courses and workshops for different ranges of audiences.

Therapeutics & Biomarker Discovery for Clinical Applications
We have well established life science proteomics facility with long-standing impact in training, analysis, and development of cutting-edge translational research in the areas of clinical proteomics, stem cell research, and cancer biomarkers. We have many on-going translation research projects involving multiple clinical departments including but not limited to, oncology center, heart center, pediatric urology, DPLM, pediatric dentistry, department of Medicine, and orthopedics department as well as several RC departments.
Our specific research focus are towards development of novel biomarker panels and molecular biological signatures of various disease processes using quantitative expression profiling of proteins and peptides present in tissue specimens, blood and other bodily fluids under different clinical states. Furthermore, we strive to identify and validate potential biomarkers for their clinical usefulness for different disease entities.

The use of a “Multi-OMICs” analysis represents a novel approach especially for Saudi Arabia and the region, and may help to develop and patent novel:
•Diagnostic protein markers
•Prognostic protein markers
•Therapeutic protein markers
Here at the KFSHRC, the existing Proteomics Unit has the real potential to engage in basic and applied clinical research and development. We are well equipped to develop new projects and hopefully generating new information and testing the real value of the technology in clinical practice.

Stem Cell Biology
Tumor composed of heterogeneous population, which pose great challenges for development of efficient targeted therapeutics. Within the tumor bulk, only a small cell subset is able to initiate tumor when transplanted into immunodeficient mice, demonstrating their stem cell-like properties. This scarce cell subset is thus referred to as cancer stem cells (CSCs) to distinguish them from the more differentiated cancer cells, which constitute the majority of the tumor bulk. CSCs are considered the cancer seeds that contribute to therapy resistance, increased metastasis and tumor recurrence through several mechanisms. Various biomarkers and functional assays have facilitated the Identification of CSCs in hematological and solid tumor and stimulated their therapeutic targeting. Our group is working toward understanding the molecular and cellular signatures that regulate CSC biology in order to design anti-CSC targeted therapies that could eliminate the tumor from the root and minimize the risk of recurrence. Another interest of the group is to test the effect of using combination of cytotoxic drug, which kills majority of the highly proliferative non-CSCs and shrink the tumor, along with selective therapies, which target the slow cycling CSCs. The ultimate goal of trying these different treatment modalities is to improve the clinical outcome.

Immunotherapy
The immune system has a major role in the surveillance and elimination of cancer cells. However, cancer cells can evade immune recognition by exploiting molecules/pathways that are physiologically designed to promote tolerance to normal tissues. Indeed, immunotherapy using the immune checkpoint inhibitors which target PD-1/or its ligand PD-L1 became an essential part of therapy for several types of cancer.
The immunotherapy section focuses on studying the immunobiology of immune checkpoints and their signaling pathways in order to design a better immunotherapy for cancer using approaches like:
Screening for novel immunotherapy combinations composed of an immune checkpoint inhibitor and other therapies like chemotherapy, targeted therapies or food supplements.
Identifying biomarkers that respond to immunotherapy in the labto select patients who are likely to respond to this immunotherapy and give them a chance to use other treatment modalities.
Advance Cell & Gene Therapy (CGT) and Precision Medicine by Genome Editing.
Advance cell and gene therapy (CGT) is an upcoming wave of therapeutic innovation. Hematology and oncology are the leading areas of research and development in the CGT arena. With over 1200 ongoing clinical trials (2021-2022), the field is becoming more promising with time. Global CGT market is projected to reach over $50 billion by the end of 2027. We are utilizing various advance CGT/genome editing modalities to build a broad and diverse portfolio as described below:
•Autologous/allogenic chimeric antigen receptor (CAR) immunotherapies
•Gene therapy/Genome editing approaches for the treatment of genetic disorders
•Gene therapy for Aging
•Umbilical cord/ Adipose Tissue derived mesenchymal stem/stromal cells (UC/Adipose MSCs) for clinical applications
•Viral and non-viral vector platform designing and testing for cell and gene therapy purposes.

Career Opportunities
The Cell Therapy & Immunobiology Department is under the hands of a well-talented and specialized collaborative team. This includes scientists and research technical staff.