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.
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.
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:
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.
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.
Explore all our free resources for patients here:
Sources:
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.
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.
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:
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.
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.
Explore all our free resources for patients here:
Sources:
about the author
Audrey Burton-Bethke
Audrey joined the HealthTree Foundation as a Community Manager in 2020 after previously working in the nonprofit field for 4 years as a director of Fundraising and Development. She graduated from BYU with a major in Spanish and Nonprofit Management. Audrey is passionate about serving others, loves learning, and enjoys a nice mug of hot chocolate no matter the weather.
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