The Center for Inherited Blood Disorders (CIBD), a nonprofit clinic, specializes in treating rare blood disorders such as hemophilia, sickle cell disease and other uncommon bleeding disorders.
“There’s always going to be the drive to treat the largest number of people with the most common disorder, so that leaves people with rare disorders out in the cold,” Founder Diane Nugent told the Business Journal.
“I hope that we can be a resource for patients with rare disorders that are looking for a place to have their gene therapy done.”
This month, the clinic unveiled a new clean room at its headquarters in Orange.
The clean room took two years to complete and joins the recently opened infusion center, which offers services for gene therapy, mast cell infusion and iron infusion, among others.
Hemophilia Treatment
Last December, CIBD administered a recently approved gene therapy to a patient with hemophilia A, marking the first time it was commercially administered within the United States.
The patient is being closely monitored and so far, has been “doing well,” according to CIBD Medical Director Amit Soni.
“I’m in contact with the patient on a weekly basis and they are able to do normal activities without requiring any additional infusions,” Soni told the Business Journal.
Roctavian, developed by global biotech company BioMarin Pharmaceutical Inc., received Food and Drug Administration approval last June. It’s advertised as the first gene therapy treatment for hemophilia A to be approved in the U.S.
Hemophilia A is one of the most common types of hemophilia, a rare blood disorder that prevents blood from clotting properly and can lead to spontaneous bleeding.
Federal Funding
CIBD has all its services available on one floor for patients, including six patient rooms, an ultrasound room, a physical therapy room and an in-house pharmacy.
The clinic receives federal funding through the 340B Program that requires pharmaceutical companies participating in Medicaid to provide outpatients drugs at reduced prices.
The savings from the program are then reinvested back in the clinic and its patients, according to Nugent.
Nugent is the chief of hematology at Children’s Hospital of Orange County, and says she often saw adult patients at the pediatric facility.
She noticed a need in the community for a clinic to serve patients regardless of their age or ability to pay, which led her to start CIBD in 2009.
“Our biggest problem is as kids transition to adult care, the majority of adult hematologists don’t handle malignant hematology, so leukemias, myeloma and other cancers,” Nugent said.
CIBD currently has six ongoing clinical trials. One study is in partnership with the Sickle Cell Disease Foundation to develop an artificial intelligence platform that can predict a health crisis before it occurs.
