CIRM-funded kidney transplant procedure eyeing faster approval

Kidney transplant surgery.

Medeor Therapeutics, which is running a CIRM-funded clinical trial to help people getting kidney transplants, just got some really good news. The US Food and Drug Administration (FDA) has just granted their product Regenerative Medicine Advanced Therapy (RMAT) designation. That’s a big deal because it means they may be able to apply for faster review and approval and get their therapy to more patients faster.

Here’s why that RMAT designation matters.

Over 650,000 Americans suffer from end-stage kidney disease – a life-threatening condition caused by the loss of kidney function. The best available treatment for these patients is a kidney transplant from a genetically matched living donor. However, patients who receive a transplant must take life-long immunosuppressive drugs to prevent their immune system from rejecting the transplanted organ. Over time, these drugs are toxic and can increase a patient’s risk of infection, heart disease, cancer and diabetes.  Despite these drugs, many patients still lose transplanted organs due to rejection.

To tackle this problem Medeor is developing a stem cell-based therapy called MDR-101. This is being tested in a Phase 3 clinical trial and it’s hoped it will eliminate the need for immunosuppressive drugs in genetically matched kidney transplant patients.

The company takes blood-forming stem cells and immune cells from the organ donor and infuses them into the patient receiving the donor’s kidney. Introducing the donor’s immune cells into the patient creates a condition called “mixed chimerism” where immune cells from the patient and the donor are able to co-exist. In this way, the patient’s immune system is able to adapt to and tolerate the donor’s kidney, potentially eliminating the need for the immunosuppressive drugs that are normally necessary to prevent transplant rejection.

So how does getting RMAT designation help that? Well, the FDA created the RMAT program to help speed up the development and review of regenerative medicine therapies that can treat, modify, reverse, or cure a serious condition. If MDR-101shows it is both safe and effective RMAT could help it get faster approval for wider use.

In a news release Giovanni Ferrara, President and CEO of Medeor, welcomed the news.

“This important designation underscores the tremendous unmet medical need for alternatives to today’s immunosuppressive therapies for transplantation. We have the potential to help people live longer, healthier lives without the need for high dose and chronic immunosuppression and we thank the FDA for this designation that will assist us progressing as efficiently as possible toward a commercially available product.”

This is the seventh CIRM-supported project that has been granted RMAT designation. The others are jCyte, Lineage, Humacyte, St. Jude’s/UCSF X-linked SCID, Poseida, Capricor

CIRM Invests in Medeor Therapeutics’ Phase 3 Clinical Trial to Help Kidney Transplant Patients

Steven Deitcher, President and CEO of Medeor Therapeutics, receives $18.8 million clinical award from CIRM to fund Phase 3 trial to help kidney transplant patients. (Photo: Todd Dubnicoff/CIRM)

Last week, CIRM’s governing Board approved funding for a Phase 3 clinical trial testing a stem cell-based treatment that could eliminate the need for immunosuppressive drugs in some patients receiving kidney transplants.

Over 650,000 Americans suffer from end-stage kidney disease – a life-threatening condition caused by the loss of kidney function. The best available treatment for these patients is a kidney transplant from a genetically matched, living donor. However, patients who receive a transplant must take life-long immunosuppressive drugs to prevent their immune system from rejecting the transplanted organ. Over time, these drugs are toxic and can also increase a patient’s risk of infection, heart disease, cancer and diabetes.  Despite these drugs, many patients still lose transplanted organs due to rejection.

Reducing or eliminating the need for immunosuppressive drugs in kidney transplant patients is an unmet medical need that our Agency is well aware of. That’s why on Friday at our January ICOC meeting, the CIRM Board voted to invest $18.8 million dollars in a Phase III clinical trial sponsored by Medeor Therapeutics that will address this need head on.

Medeor, a biotechnology company located in San Mateo, California, is developing a stem cell-based therapy, called MDR-101, that they hope will eliminate the need for immunosuppressive drugs in genetically matched kidney transplant patients.

The company takes blood-forming stem cells and immune cells from the organ donor and infuses them into the patient receiving the donor’s kidney. Introducing the donor’s immune cells into the patient creates a condition called “mixed chimerism” where immune cells from the patient and the donor are able to co-exist. In this way, the patient’s immune system is able to adapt to and tolerate the donor’s kidney, potentially eliminating the need for the immunosuppressive drugs that are normally necessary to prevent transplant rejection.

CIRM President and CEO, Dr. Maria Millan, commented in a CIRM news release:

Maria Millan

“These immunosuppressive drugs not only can cause harmful side effects, but they are also expensive and some patients lose their transplant either because they can’t afford to pay for the drugs, or because their effectiveness is not adequate. Medeor’s stem cell-based therapy aims to prevent transplant rejection and eliminate the need for immunosuppression in these kidney transplant patients. If they are successful, this approach could be developed for other organs including heart, liver, and lung transplants.”

CIRM funding will enable Medeor to test their stem cell-based treatment in a Phase 3 clinical trial. If the trial meets its objective in allowing patients to eliminate immunosuppressive drug use without rejection, Medeor may apply to the US Food and Drug Administration (FDA) for permission to market their therapy to patients in the United States.

Dr. Steven Deitcher, co-founder, President and CEO of Medeor, touched on the impact that this CIRM award will have on the advancement of their trial:

“We are very grateful for the financial support and validation from CIRM for the MDR-101 program. CIRM funding accelerates our timelines, and these timelines are what stand between needy patients and potential transformative therapies. This CIRM award combined with investor support represent a public-private collaboration that we hope will make a difference in the lives of organ transplant recipients in California, the entire U.S., and beyond.”

This is the fourth clinical trial targeting kidney disease that CIRM’s Board has funded. CIRM is also funding a Phase I trial testing a different stem cell-based therapy for end-stage kidney disease patients out of Stanford University led by Dr. Samuel Strober.

To learn more about the research CIRM is funding targeting kidney disease, check out our kidney disease fact sheet on our website.