Sickle cell disease (SCD) continues to be a significant health challenge, affecting an estimated 100,000 people in the United States alone. The Food and Drug Administration (FDA) has recently cleared Actinium Pharmaceuticals’ Investigational New Drug (IND) application to study Iomab-ACT, a targeted radiotherapy conditioning agent developed in partnership with Columbia University, for patients with SCD who are undergoing allogeneic bone marrow transplants (BMT). This groundbreaking study, led by Dr. Markus Mapara, will evaluate the safety and efficacy of Iomab-ACT in this patient population.
“Undergoing chemotherapy- or total body irradiation-based conditioning for curative allogeneic bone marrow transplant or gene therapy often brings severe side effects for patients with sickle cell disease,” said Mapara. “These toxicities include organ damage, infections, infertility, and the potential for secondary malignancies. Leveraging extensive data from CD45 ARC conditioning in allogeneic bone marrow transplants, I am thrilled to lead this pioneering study using Iomab-ACT, a non-chemotherapeutic targeted radiotherapy conditioning, for patients with sickle cell disease. This innovative approach aims to minimize toxicity while ensuring complete donor hematopoiesis engraftment. Success in this trial could revolutionize treatment, enabling the use of a low-toxicity method for the engraftment of genetically engineered autologous stem cells in SCD patients.”
Sickle Cell Disease: Iomab-ACT Targets CD45 to Potentially Revolutionize Treatment with Low-Toxicity Approach
Iomab-ACT targets CD45, a marker on blood cancer and immune cells, aiming to replace traditional chemotherapy and total body irradiation conditioning methods, which typically result in extreme adverse events (AEs). According to Actinium, this trial has the potential to influence broader access to curative therapies for SCD, such as BMT and gene therapy. The company believes this therapeutic approach may revolutionize treatment by providing a low-toxicity method for engrafting genetically engineered autologous stem cells in SCD patients.
The targeted conditioning approach of Iomab-ACT is also being studied for broader applications in cellular therapies, such as chimeric antigen receptor (CAR) T-cell therapies and gene therapies. Actinium cites the rapidly expanding pipeline of these therapies, estimating that the number of patients treated with cellular therapies could reach 93,000 in the United States by 2030, with an estimated compound annual growth rate (CAGR) of 11% over the next five years.
In the United States, SCD predominantly affects non-Hispanic Black or African American individuals, who constitute over 90% of the patient population. The life expectancy for people with SCD in the United States is 20 years lower than the global average. SCD is most common in regions such as Saudi Arabia, India, Sub-Saharan Africa, Spanish-speaking regions in the Western Hemisphere, and several Mediterranean countries.
Sickle Cell Disease: Actinium Pharmaceuticals Collaborates with Dr. Mapara to Address Treatment Barriers
“We are honored to collaborate with Dr. Mapara on this important initiative to address the significant number of patients with sickle cell disease who do not pursue or are not able to access transplant or gene therapies due to the required chemotherapy-based conditioning and its severe toxicities,” said Sandesh Seth, chairman, and CEO of Actinium Pharmaceuticals. “Sickle cell disease represents a large and high unmet need that needs better treatment options and outcomes.
This exciting program in sickle cell disease adds to our recently announced commercial CAR T trial addressing the large patient population with blood cancers that can also benefit from broader access to cellular therapy via targeted conditioning. We look forward to further expanding Iomab-ACT’s already large addressable patient opportunity in transplant, cell therapy, and gene therapy conditioning.”
Actinium Pharmaceuticals’ collaboration with Columbia University and the leadership of Dr. Markus Mapara is a crucial step forward in addressing the unmet medical needs of patients with SCD. The introduction of Iomab-ACT aims to provide a safer, more effective conditioning method that minimizes toxicity while ensuring successful donor hematopoiesis engraftment. If successful, this trial could significantly improve the quality of life for SCD patients and potentially revolutionize the standard of care for this challenging disease.
The FDA’s clearance of the IND application for Iomab-ACT marks an important milestone in the development of innovative treatments for SCD. The upcoming study will provide valuable insights into the safety and efficacy of this targeted radiotherapy conditioning agent, paving the way for broader applications in cellular and gene therapies. As the medical community continues to seek better treatment options for SCD, the success of Iomab-ACT could represent a transformative advancement in the fight against this debilitating disease.
Resource: Actinium Pharmaceuticals, July 25, 2024
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