Talaris is developing an investigational, single-dose, cell therapy to induce immune tolerance to the donated kidney, potentially eliminating the need for chronic immunosuppression and restoring long-term quality of life to transplant recipients.
Immune tolerance has long been considered to be the “Holy Grail” of organ transplantation. It refers to the ability to train a recipient’s immune system to naturally recognize the donated organ as “self”. Normally, a transplant recipient’s immune system sees a donated organ as foreign and will attack it. To avoid organ rejection, drugs are used to suppress the transplant recipient’s immune system. These drugs must be taken for life and have significant risks and complications. Inducing tolerance in a transplant recipient eliminates the need to chronically suppress their immune system to prevent rejection of the donated organ, and avoids the many undesirable side effects associated with these drugs.
Talaris’ FCR001 is an investigational cell therapy that is derived from the kidney donor’s stem cells and immune cells, processed at our specially equipped facility, and then infused into the kidney transplant recipient the day after the kidney transplant. The goal of our therapy is to create a “dual immune system” (part-donor and part-recipient) in the transplant recipient. These two immune systems coexist, recognizing both the recipient’s own body as well as the donated organ as “self”. This is also referred to as achieving “chimerism” in the recipient’s immune system. Achieving durable chimerism in a recipient’s immune system potentially enables long-term acceptance of the organ transplanted from the same donor. Ultimately, this could allow the transplant recipient to live free of the daily pill burden typically required to suppress the immune system and prevent organ rejection, and the many complications associated with chronic immunosuppression.
Kidney donor donates stem cells via standard mobilization and apheresis procedure.
Talaris’ proprietary manufacturing process removes certain undesirable cells and enriches product for stem cells and facilitating cells.
Recipient undergoes three days of moderate outpatient conditioning to make space in the bone marrow for the donor’s cells to engraft.
Surgeon transplants the donor’s kidney into the recipient.
Physician infuses FCR001 therapy into the recipient.
Patient remains on standard immunosuppression and returns to clinic at regular intervals for routine
Simple blood test looks to confirm that donor and recipient immune cells are coexisting in recipient’s bone marrow. If so, physician gradually lowers immunosuppression.
Recipient free of all chronic immunosuppression drugs.
Talaris’ FCR001 therapy begins with the donor’s blood cells. Several weeks prior to the scheduled kidney transplant, the kidney donor receives some injections of specific medicines over a five-day period to mobilize the donor’s stem cells and immune cells and allow these cells to circulate out of bone marrow and into the bloodstream. These cells are then collected from the donor through a routine procedure called apheresis. The donated cells are refrigerated and shipped to the Talaris cell processing facility, where our proprietary process removes certain types of undesirable cells and enriches the product for blood stem cells and Facilitating Cells. The cell doses in our FCR001 therapy are customized for each patient. The final product is frozen, shipped back to the transplant center, and stored until the transplant date.
In our Phase 2 study, nearly every patient who demonstrated “chimerism” at six months post-transplant was able to be completely weaned off all immunosuppression, and every patient who was weaned off all immunosuppression at twelve months has been able to stay off chronic immunosuppression, without any sign of organ rejection. We have followed these patients for up to nine years.
Our Phase 3 Clinical Trial
Talaris is now enrolling living donor kidney transplant patients for its Phase 3 FREEDOM-1 clinical trial. The purpose of this study is to learn more about whether FCR001 can prevent the rejection of living donor kidney transplants without the need for lifelong anti-rejection drugs. Learn more about FREEDOM-1 at https://freedom1study.com/