New FDA-Designated Cell Therapy for AML, ALL, and MDS

The United States Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy Designation to two T-cell receptor T-cell (TCR-T) therapies, TSC-100 and TSC-101. 

These therapies have the potential to successfully treat several hematological malignancies, such as acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and myelodysplastic syndrome (MDS). 

Patients undergoing allogeneic hematopoietic stem cell transplants with reduced intensity conditioning can receive either of these therapies. 

What are TSC-100 and TSC-101?

TSC-100 and TSC-101 are designed to eliminate all remaining blood cells in the host while ignoring the cells received from the donor after an allogeneic (donor) hemopoietic stem cell transplant. 

Not all blood cancer patients have T-cells in their immune system that can recognize cancer cells. Others might have T-cells that are too exhausted to perform their cancer-killing function. This is where T-cell receptor T-cell (TCR-T) therapy is needed. 

This is done by collecting patient T cells and equipping them with a new T cell receptor that enables them to target specific cancer antigens. 

These therapies aim to increase the cure rate in patients receiving hemopoietic stem cell transplants. 

Therapies like these are needed, especially considering around 40% of patients with AML, ALL, and MDS who undergo this type of transplant relapse within two years and then face limited options for treatment and unfavorable outcomes. 

TSC-100 and TSC-101 are currently being tested in a Phase I (one) clinical trial, NCT05973487. 

In this trial, all eight patient treated with TSC-100 or TSC-101 had not relapsed and had no observable disease after a median follow-up duration of more than ten months. 

What is the FDA’s Regenerative Medicine Advanced Therapy Designation? 

According to the FDA website, the Regenerative Medicine Advanced Therapy designation is a special approval given to certain drugs that fit certain criteria, such as: 

• Cell therapy, therapeutic tissue engineering products, human cell and tissue products, or any combination product using these therapies/products 
• Drugs intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition 
• Preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition 

Being cell therapies, the TSC-100 and TSC-101 qualified for this designation. Other therapies receiving this designation include lisocabtagene maraleucel, a CAR T-cell therapy for large B-cell lymphomas, lovotibeglogene autotemcel for sickle cell disease patients and valoctocogene roxaparvovec-rvox for hemophilia patients. 

Conclusion

Although the NCT05973487 study of TSC-100 and TSC-101 is small, it was enough to convince the FDA of its promise in treating hematological malignancies like MDS, AML, and ALL. 

These patients now have the opportunity to receive a long-lasting, successful stem cell transplant. 

Continue learning about new therapies for blood cancers in clinical trials with HealthTree. 

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Sources: 

• TScan Therapeutics Receives FDA’s Regenerative Medicine Advanced Therapy (RMAT) Designation for its Two Lead TCR-T Therapy Candidates for the Treatment of Heme Malignancies
• TScan’s TCR-Ts Get RMAT Designation for Hematologic Malignancies
• Regenerative Medicine Advanced Therapy Designation
• Initial Results of a Phase 1 Trial of TSC-100 and TSC-101, Engineered T Cell Therapies That Target Minor Histocompatibility Antigens to Prevent Relapse after Allogeneic Hematopoietic Cell Transplantation
• CBER Regenerative Medicine Advanced Therapy (RMAT) Approvals
• Adoptive Cell Therapy | Cancer Research Institute

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