NYBC, Howard and Abby Milstein Foundation to advance use of specialized stem cell lines in regenerative medicine

In August, NYBC announced a partnership with the University of California, Davis, Health System to manufacture specialized lines of stem cells as potential therapies for the repair and regeneration of retina, kidney, lung and liver tissue, as well as for the treatment of neurodegenerative disorders such as Parkinson’s, Alzheimer’s and Huntington’s disease.

Today, it was announced that this effort will be joined by Drs. Peter Wernet of Dusseldorf University and Hans Schöler of the Max Planck Institute, Münster, working with the Good Manufacturing Practices (GMP) Labs of RheinCell Therapeutics.

This collaboration will further the goal of promoting regenerative medicine, a new frontier that will enable doctors to repair or replace virtually every major organ in the human body.

The collaboration with Wernet, Schöler and RheinCell Therapeutics will seek to develop a collection of induced Pluripotential Stem Cell (iPSC) lines from which differentiated cells and tissues can be generated to provide therapies to repair and regenerate damaged tissues – with a low chance of immune rejection – for clinical use by large segments of the patient population.

This new agreement, through NYBC’s Howard P. Milstein Cord Blood Center, is funded by the Howard and Abby Milstein Foundation and will add the scientific stem cell research experience and GMP manufacturing resources of RheinCell, under the direction of Drs. Wernet and Schöler, to the UC Davis partnership established by Dr. Pablo Rubinstein, VP of NYBC and Program Director of the National Cord Blood Program (NCBP) at the Milstein Cord Blood Center, and Dr. Jan Nolta, Director of the UC Davis Stem Cell Program and California University’s Institute for Regenerative Cures.

"We are pleased to provide the necessary funding to facilitate this collaboration and are excited about its potential contributions to advancements in the field of regenerative medicine," said Howard Milstein, Chair of NYBC and the Howard and Abby Milstein Foundation.

"Through collaborations such as these, we are creating the critical mass needed to drive groundbreaking innovations in regenerative medicine."

NYBC president Christopher Hillyer said: "NYBC’s accomplishments in creating and nurturing cord blood banking were made possible through our long tradition of innovation in medical biotechnology, thanks to the outstanding dedication of Dr. Rubinstein and his staff, and the visionary leadership of Board of Trustees Chairman Howard Milstein."

Under separate agreements, UC Davis and RheinCell will manufacture iPSC lines from selected units of the National Cord Blood Program (NCBP) public cord blood bank at NYBC’s Howard P. Milstein Cord Blood Center.

The Program maintains the first and largest public inventory of neonatal – not embryonic – stem cells from umbilical cord blood donations (recovered from the discarded placenta and umbilical cord, minutes after the birth and safe delivery of a baby).

The inventory includes cord blood units that have two identical sets of HLA genes (these sets are called "haplotypes") from each of the parents (i.e., are HLA homozygous). Cells or tissues derived from HLA homozygous (HLA h) donors, previously reprogrammed into iPSC lines, may be clinically used by the many individuals who inherit the same HLA haplotype from at least one of their parents, with little risk of immune rejection.

Making functional, "partially universal," donor cells for regenerative medicine will require the HLA homozygous cord blood cells to undergo two complex differentiation processes.

First, blood-forming stem cells will be reverted to "induced pluripotency" (iPSC) status (capable of differentiating into many different tissues.) This will be done by RheinCell and UC Davis scientists in their GMP facilities at Langenfeld and Sacramento, respectively. Libraries of iPSC will be stored frozen. In a second process, iPSCs will be further reprogrammed into fully functional cells.

Wernet and Schöler said: "This exciting international collaboration of symbiotic partners towards Advanced Regenerative Medicines from unrelated cord blood donors is a very important step towards our joint objective: the establishment of an HLA homozygous iPS cell library for clinical applications."

The fact that a library of 100 iPSC lines from selected HLA h cord blood units could provide matches for up to 80 percent of patients in need emphasizes the groundbreaking nature of the new regenerative medicine. Such a growing library of cell lines homozygous for different HLA gene complexes will allow for future clinical use by a very high proportion of the patients.

Milstein said: "The reprogramming of HLA h iPSC into clinically useful functional cells and tissues will enable their off-the-shelf use for many unrelated individuals, saving time and money and leading to revolutionary advancements in regenerative medicine."