Medeor Therapeutics has just received the FDA go-ahead to start a clinical trial of its cell-based immunotherapy, MDR-101. The personalized therapeutic could replace immune-suppressing drugs in patients who have received an organ transplant.
Currently, patients who are given a donor organ must take drugs designed to dampen the immune system for the rest of their lives. These drugs can help mitigate the risk of organ rejection, however they carry their own risks.
Over 30,000 people in the US undergo organ transplant surgery each year, however as many as 33 percent of these transplants will be rejected within five years of the procedure. Heart, liver and kidney transplants remain some of the most common procedures, and the need for these vital organs often outweighs the supply.
Immunosuppressive drugs – including cyclosporine and tacrolimus – are effective, however they have also been linked to an increased risk of serious infections, heart disease, diabetes and cancer. For kidney transplant recipients, these drugs can even have damaging effects on the kidneys, thereby potentially harming the transplanted organ and putting the patient back on the waiting list.
“The current standard of care to prevent rejection of organ transplants is far from optimal due to unacceptably high long-term failure rates and treatment-associated complications, and new approaches are greatly needed to relieve organ transplant recipients from the litany of undesirable effects associated with anti-rejection drug regimens,” said Dr. Scott Batty, Jr., chief medical officer of Medeor. “MDR-101 has the potential to address the two most critical transplant patient needs: preventing organ rejection and mitigating anti-rejection treatment-associated toxicities.”
Not only will the FDA allow Medeor to start trials of their investigational new drug, but the company will also be permitted to jump directly into Phase III with living donor kidney transplants. This is thanks to a Special Protocol Assessment, which means the FDA has signed off on the company’s protocol design and endpoints.
MDR-101 is a cellular immunotherapy designed to promote the existence of blood and immune cells from both the donor and the recipient in the transplant patient. The drug is designed to stimulate a donor-specific immune tolerance so that the body recognizes the biological signature of the transplant organ, thereby reducing the risk of rejection.
A Phase I clinical trial was used to test MDR-101 in HLA-matched donor organs, however this case only occurs when a close relative – such as a sibling – is able to donate an organ. The immunotherapy may improve outcomes for patients who receive organ transplants that aren’t an exact HLA match, potentially increasing the number of people who could effectively be donors.
The clinical trial is set to begin in the second half of this year, and FDA approval of the immunotherapy will be contingent upon the safety and efficacy data generated during this study. The technology was developed by researchers at Stanford University, for which Medeor holds exclusive worldwide licenses.