Director of the ACTC, Dr. Chiara Cugno
Doha, Qatar: Children in Qatar with complex diseases who once had to travel abroad for life-saving gene and cell therapies can now receive highly personalised treatment closer to home, thanks to Sidra Medicine’s Advanced Cell Therapy Core (ACTC).
“The Advanced Cell Therapy Core brings together, under one roof, everything needed to deliver cell and gene therapies to children, from cell collection, through GMP-grade manufacturing, to clinical delivery and long-term follow-up,” Director of the ACTC, Dr. Chiara Cugno told The Peninsula.
She explained that this integrated model allows Sidra Medicine to design, manufacture and deliver advanced therapies locally, tailored to each child’s specific condition, instead of relying on overseas treatment options.
“That integration is what makes truly personalised care possible. Instead of sending patients and their cells abroad, we design, manufacture, and deliver these medicines locally, tailored to the individual child,” she said.
According to Dr. Cugno, personalised medicine means no child receives “one-size-fits-all care,” with donor selection, conditioning, graft manipulation and post-treatment immunotherapy all adapted to the patient’s disease and clinical journey.
“The ACTC is the infrastructure that turns personalization from a concept into a therapy the patient actually receives,” she added.
Among the most promising breakthroughs in paediatric care, she highlighted gene editing, CAR-T cell therapy and haploidentical transplantation as major advances transforming outcomes for children with previously untreatable diseases.
“Gene editing, exemplified by Casgevy, is now a clinical reality for hemoglobinopathies, with similar approaches advancing for immunodeficiencies and metabolic diseases,” she said.
She added that CAR-T cell therapy has turned what were once terminal leukemias and lymphomas into curable diseases for many children, while haploidentical transplantation has made curative transplant available to nearly every child, even without a matched donor.
“What excites us most is that these advances are arriving in parallel and reinforce one another, the same platforms and expertise that power gene therapy also power transplantation and CAR-T,” Dr. Cugno said.
The ACTC combines stem cell transplantation, regenerative medicine and gene therapy within a single patient pathway by using the same infrastructure and multidisciplinary team throughout the treatment journey.
“One multidisciplinary team of hematologists, transplant physicians, cell processing and quality specialists, and nurses follows the patient from first evaluation through long-term follow-up, whichever modality is ultimately chosen,” she said.
She said the same apheresis unit can collect stem cells for gene therapy, CAR-T treatment or transplantation, while the same GMP facility processes grafts for all three.
“This lets us choose the right therapy for each child, not simply the therapy that happens to be available,” she said.
Dr. Cugno also shared a recent case that demonstrated the importance of local advanced therapy capabilities. A child with Diamond-Blackfan anemia who had undergone transplantation abroad began losing the graft during the post-transplant period and faced a life-threatening situation.
“At Sidra Medicine, we successfully rescued the graft with an infusion of stem cells manufactured in-house, an intervention that would not have been possible without a local Advanced Cell Therapy Core,” she said.
She added that Sidra is currently the only center in the Gulf region offering the most advanced platform for haploidentical transplantation, known as TCR alpha-beta depletion, which is preferred for patients with non-malignant disorders.
The hemoglobinopathy program has also brought major improvements for children with conditions such as sickle cell disease and transfusion-dependent disorders.
“Children who once lived from transfusion to transfusion, or from crisis to crisis, can now be offered a matched or haploidentical transplant, or Casgevy. Within a single program, each family is offered the option best suited to their child,” Dr. Cugno said.
Ensuring safety and long-term effectiveness remains central to the ACTC’s work. Dr. Cugno said this depends on three pillars: rigorous manufacturing, careful patient selection and monitoring, and structured long-term follow-up.
“Every cellular product we deliver is produced under GMP conditions and must meet strict release criteria before it is ever infused into a child,” she said.
She added that every candidate is reviewed by a multidisciplinary team and patients are monitored in a dedicated unit with expertise in managing complications linked to these therapies.
“The third, and perhaps most important for emerging therapies, is long-term follow-up,” she said. “Gene and cell therapies can have late effects that only become apparent years later.”
Through structured follow-up programmes aligned with international registries, Sidra Medicine tracks patients for years after treatment, helping ensure safety while contributing to global research that will improve future therapies.
