New Clinical Study for Living Donor Kidney Transplant Recipients

Today, kidney transplant recipients must take a lifelong, daily regimen of anti-rejection drugs. Although anti-rejection drugs are very effective at preventing rejection of kidneys, they can have multiple side effects and can degrade kidney function over time. 

To eliminate the need for these medications, so that transplant recipients could avoid their side effects and spare the kidney being harmed by them, was a challenge that Dr. Suzanne Ildstad set out to solve early in her career. 

“As a transplant surgeon, I saw firsthand the life-saving benefits of organ transplantation. However, I also saw the impact on patients’ lives due to their lifelong dependence on anti-rejection drugs,” Dr. Ildstad explains. 

Long-term use of anti-rejection medications may cause or worsen conditions such as high blood pressure, diabetes, and high cholesterol, and is associated with increased risk for certain cancers. The medications are also toxic to the kidneys, often leading to a decline in kidney function over time. Roughly one-third of living donor kidney transplants and roughly half of deceased donor kidney transplants fail within 10 years. Thus, most kidney transplant recipients will require more than one transplant over the course of their lives. 

As an alternative to taking lifelong anti-rejection medications, Dr. Ildstad began studying how to create what’s known as “immune tolerance” in transplant recipients. That research, begun over 30 years ago, has paved the way for the investigational cell therapy (FCR001) that is now being evaluated in a Phase 3 registration clinical study called FREEDOM-1. 

About Immune Tolerance

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 a donated organ as “self.” 

Normally, a transplant recipient’s immune system sees a donated organ as “foreign” and will attack it, leading to rejection of the organ. To avoid this, anti-rejection drugs are necessary to suppress the transplant recipient’s immune system from attacking the organ. 

However, immune tolerance offers a potential alternative. Durable immune tolerance would allow the transplant recipient’s immune system to accept the transplanted organ without the need for lifelong anti-rejection drugs.

About FCR001 Cell Therapy

The investigational therapy that resulted from Dr. Ildstad’s research is called FCR001. It is a cell therapy administered once which is designed to create immune tolerance to a transplanted organ. 

FCR001 is comprised of living immune cells, including stem cells, obtained from the kidney donor. Stem cells are a unique kind of immature cell that can generate many types of mature immune cells in the body. Prior to kidney transplantation, the recipient undergoes a moderate outpatient conditioning regimen which prepares the bone marrow to accept the new cells, and FCR001 is infused once into the recipient one day after surgery. The cells in FCR001 then take up residence in the recipient’s bone marrow and generate new immune cells. The goal of FCR001 treatment is to create a dual immune system (part-donor and part-recipient) in the transplant recipient. This allows for immune tolerance to develop and anti-rejection medications to be gradually withdrawn.

In a Phase 2 clinical study conducted between 2009 – 2016 at two leading U.S. transplant centers, 26 of 37 participating kidney transplant recipients (70%) treated with FCR001 were able to discontinue all anti-rejection drugs within one year after their transplant. All 26 have remained off anti-rejection medications since then. At five years of follow-up, these patients showed stable kidney function with only 18% requiring medication for hypertension and 9% for high cholesterol respectively.

FREEDOM-1 Clinical Study Underway

FCR001 is now being evaluated in a Phase 3 registration clinical study of living donor kidney transplant at sites across the U.S. A registration trial is used by the FDA to decide if the therapy can be approved for use in all patients. This study, called FREEDOM-1, will evaluate the efficacy and safety of FCR001 treatment and standard anti-rejection treatment. 

You can learn more about FREEDOM-1 by visiting https://freedom1study.com/ or ClinicalTrials.gov (identifier NCT03995901).  

CALLOUT BOX: Quick Facts About FREEDOM-1 Study

·      Now open at sites across the U.S. – view the latest map at https://freedom1study.com/

o   Omaha, Neb.

o   Chicago, Ill. 

o   Minneapolis, Minn.

o   Durham, N.C.

o   Washington, D.C.

o   La Jolla, Calif. 

·      Phase 3 clinical trial 

·      Enrolling adults who plan to undergo a living donor kidney transplant

CALLOUT BOX: You may qualify for the FREEDOM-1 Study if…

• You are at least 18 years old

• Your doctor has recommended that you should have a kidney transplant

• This would be your first kidney transplant

• A suitable living kidney donor between ages 18-60 has been identified

• You have not been diagnosed with or treated for any type of cancer