Publication / Source: Originally published by CIRM (www.cirm.ca.gov)
Authors: California Institute for Regenerative Medicine (CIRM)
Two projects aimed at helping people who have had a stroke, plus a therapy that hopes to correct a fatal blood disorder by operating on a baby in the womb, are among the programs approved today by the governing Board of the California Institute for Regenerative Medicine (CIRM).
The Board also honored C. Randal Mills, Ph.D., President and CEO of CIRM, and James Harrison, CIRM’s General Counsel, both of whom are leaving the agency.
But first, the science. Every year around 800,000 Americans suffer a stroke; 130,000 people die; and for those who survive, strokes are the leading cause of serious, long-term disability in the US, representing an unmet medical need.
“Today the CIRM Board approved two very different methods, using different kinds of stem cells, to address this need,” says Dr. Maria Millan, interim CEO and President of the agency. “By funding “multiple shots on goal” we believe that we have a better chance of finding a way to repair the damage caused by stroke and give people a better quality of life.”
A team at SanBio was awarded almost $20 million to carry out a Phase 2 clinical trial using bone marrow-derived mesenchymal stem cells that have been modified to help people suffering from chronic disability following a stroke.
Stanford University’s Dr. Gary Steinberg received $5.3 million to complete the pre-clinical studies needed to apply to the Food and Drug Administration (FDA) to test his neural cell therapy in a clinical trial.
Dr. Tippi MacKenzie, at the University of California, San Francisco, was awarded $12.1 million to treat babies in the womb who have alpha thalassemia major. This is a blood disorder that is only detected in the last few months of pregnancy and is almost always fatal. MacKenzie is using hematopoietic stem cells (HSCs), taken from the mother’s bone marrow, and transplanting them into the baby before birth. The baby’s immune system is able to tolerate the mother’s cells, increasing the chances of a healthy birth, and improving the chances of having effective treatments after birth.
The Board also awarded Cellerant Therapeutics $6.86 million to conduct the studies needed to get FDA approval for a clinical trial to help people with acute myeloid leukemia (AML). This is a cancer that affects bone marrow and blood. The five-year survival rate for people over 60 with AML is just 26 percent.
Cellerant has developed a novel drug that will target Leukemic stem cells (LSC). It’s believed that during chemotherapy LSCs are able to lie dormant and survive, and then later become active again allowing the cancer to return. It’s hoped that targeting LSCs will reduce relapse rates and prolong survival.
The Board also approved investing more than $20 million in 13 projects in the Discovery Quest Awards Program. These are early stage research awards whose goal is to promote the discovery of promising new stem cell-based technologies that could be translated to enable broad use and ultimately improve patient care.