Saneron receives grant to develop safety guidelines for transplanting HUBC cells in Alzheimer's disease

Preclinical trials will establish dosage and safety

The National Institutes of Health has awarded a three-year, $2.6-million grant to the University of South Florida and Tampa-based biotechnology company Saneron-CCEL Therapeutics, Inc., to establish dosing and safety guidelines for transplanting human umbilical cord blood cells (HUBC) into animal models of Alzheimer's disease. The researchers hope to use the pre-clinical data to gain U.S. Food and Drug Administration approval to carry out clinical trials with patients suffering from Alzheimer's disease.

"Our immediate goal is to move our beneficial findings with cord blood cells into clinical trials for patients with mild to moderate Alzheimer's disease," said the grant's principal investigator Dr. Jun Tan, a USF neuroscientist and professor of psychiatry.

The NIH Phase II Small Business Technology Transfer grant is based on the success of an ongoing research partnership between USF and Saneron aimed at determining the therapeutic benefits HUBCs offer when transplanted into animal models of a variety of neurological diseases, including Parkinson's disease, Lou Gehrig's disease (ALS), Alzheimer's disease and stroke.

"Our next stage of research is translational. This study's primary aims are the completion of preclinical safety and validation studies and the submission of an Investigational New Drug application to the FDA for a Phase I clinical trial," said Nicole Kuzmin-Nichols, president and chief operating officer of Saneron. "Saneron is very pleased and excited that our long-standing research partnership with USF has provided this opportunity to further the commercialization of technology that was developed at USF and transferred to Saneron for further development."

Additional funding will be provided by a Florida High Tech Corridor Industry Matching Grant through USF Connect.

Previous research by USF and Saneron has shown that injecting HUBC into laboratory (in vitro) and animal models (in vivo) of neurodegenerative diseases does not promote a strong immune response, so rejection is not a problem, Dr. Tan said. The potential for HUBCs to promote an anti-inflammatory response may provide overall benefits. Inflammation is a biochemical component of Alzheimer's disease.

Saneron CCEL Therapeutics, Inc. worked with USF to develop a HUBC cell technology platform called the U-CORD-CELL - program. The program aims to improve the quality of life for patients by developing and producing innovative, high-quality treatments for neurological and cardiac diseases.

"This grant will afford Saneron the opportunity to bring the U-CORD-CELL- technology to the clinic in the pursuit of a potential therapy for those suffering from Alzheimer's disease," Kuzmin-Nichols said.

According to USF senior vice president for research and innovation, Karen Holbrook, PhD, this collaborative work between USF and Saneron is the "essence of technology transfer."

"The best outcome for this kind of collaboration is when technology developed at the university is taken to the patient's bedside," said Dr. Holbrook. "USF is happy and proud to be working with Saneron on this most important research."