ADVANCING NEW THERAPEUTICS
RESEARCH & CLINICAL TRIALS
RESEARCH & CLINICAL TRIALS
ADVANCING NEW THERAPEUTICS
While xenotransplantation eventually could resolve the shortage of human donor organs , the UAB Comprehensive Transplant Institute ( CTI ) is also focused on overcoming the incompatibility that ’ s often seen in human-to-human organ transplantation . Supported by both industry and federal funding , UAB currently is participating in 33 active research studies or projects in this area and hopes to double that number within the next several years .
As one example , the UAB CTI is participating in a national , multi-center clinical trial of a medication called imlifidase ( Idefirix ), which is designed to reduce patient sensitivity by breaking up , or “ cleaving ”, certain antibodies . The drug specifically targets IgG and inhibits IgG-mediated immune response . This Phase 3 clinical study is called the ConfIdeS trial and is sponsored by the drug ’ s manufacturer , Hansa Biopharma . Imlifidase already is being used in some European countries .
UAB is one of 13 participating study sites , which demonstrates our commitment to such patients and to improving access for those who are disadvantaged due to immunologic barriers . In the most extreme cases , highly sensitized patients might only match with less than 0.5 % of kidneys that come available . This is often due to a prior transplant , blood transfusion , or pregnancy , which creates pre-formed antibodies that increase the risk of rejection and early graft failure . Currently at UAB , there are over 120 candidates on the kidney waitlist who have human leukocyte antigen ( HLA ) antibody profiles that make it very hard to match them with a donor organ .
Other desensitization therapies do exist , but typically they require weeks of pre-treatment , which is possible with living-donor organs but tends to rule out deceased-donor organs because of the short notice . Pre-treatment of patients with imlifidase can be completed in as little as 24-48 hours . The medication is discontinued upon transplantation , but as with all organ transplants , recipients will need to continue taking standard anti-rejection medications for life . The efficacy of imlifidase will be compared against the present standard of care , which for most patients is to remain on the waiting list hoping for a suitably matched organ . Enrollment is ongoing , and trial-related follow-up evaluation of the patients will occur after one year of participation .
As part of the trial , the organ-matching criteria will be adjusted for participants , so that they are considered for a wider range of kidneys , according to Douglas Anderson , M . D ., an assistant professor of Surgery in the UAB Division of Transplantation and director of the Pancreas Transplant Program . “ The trial is still in progress , so there are no results that can be shared as of yet ,” Dr . Anderson said . “ However , we are very excited about the potential of this drug , should it be shown to be successful , in assisting sensitized patients achieve transplantation .”
Expanding research infrastructure Among the 33 active studies , the UAB CTI is also testing a new type of solution used to perfuse extracted donor kidneys while awaiting transplant , with the goal of improving long-term outcomes for recipients . UAB is expected to participate in many other studies aimed at improving outcomes and reducing incompatibility as it expands its research capacity and ability .
“ We are investing a lot of time and resources into building infrastructure to support the aggressive growth in research and tempo that we want to see , because we have to have the team and the knowledge necessary to advance these therapeutics ,” said Paige Porrett , M . D ., Ph . D ., the inaugural director for Vascularized Composite Allotransplantation in the UAB CTI . “ This includes people like research coordinators and data analysts but also regulatory specialists who can help prepare and submit protocols to the Institutional Review Board . There are extensive rules governing human research subjects , and this requires a lot of knowledge and paperwork . They make sure that our investigators are all compliant with those federal rules and that we are conducting human subject research both safely and adequately .”
6 UAB CTI Report