Table 2. Update on 2009 CIRM grants. Status of research grants awarded in 2009 by the California Institute for Regenerative Medicine (CIRM) to develop stem cell-based therapies.

Source: CIRM

Lead institution

Lead principal investigator

CIRM funding ($M)

Proposal summary

Status

Cedars-Sinai Medical Center

Eduardo Marbán

5.6

Treat advanced heart failure using autologous cardiac-derived cardiospheres or cardiosphere-derived cells

Team is developing allogeneic cardiac-derived cells and on track to submit an IND within four-year award period

City of Hope

Karen Aboody

18.0

Treat recurrent glioblastoma using neural stem cells modified to carry a tumor-killing drug

Team is on track to complete preclinical proof of concept by end of 2012

City of Hope

John Zaia

14.6

Treat HIV using genetically modified autologous hematopoietic stem cells that give rise to HIV-resistant T cells

Team is progressing toward IND-enabling studies

Stanford University

Alfred Lane

11.7

Treat epidermolysis bullosa using genetically modified induced pluripotent stem (iPS) cells derived from patient's skin cells

Team has generated iPS cell lines from several patients

Stanford University

Gary Steinberg

20.0

Treat stroke using implanted neural stem cells derived from human embryonic stem cells (hESCs)

Team has shown functional recovery in three preclinical models of stroke

Stanford University

Irving Weissman

20.0

Develop a mAb that targets leukemia stem cells

Team is conducting preclinical studies of a humanized anti-CD47 mAb

University of California, Los Angeles

Irvin ChenA

20.0

Treat HIV using RNAi-modified autologous hematopoietic stem cells that give rise to HIV-resistant T cells

Team has identified small hairpin RNAs that prevent HIV cell entry and replication

UCLA

Donald Kohn

9.2

Treat sickle cell disease using genetically modified hematopoietic stem cells that become normal red blood cells

Team has identified a lead therapeutic and had a pre-IND meeting with FDA

UCLA

Dennis Slamon

20.0

Develop compounds that target cancer stem cells in gliomal, colon and ovarian tumors

Team is conducting IND-enabling studies of a lead inhibitor targeting an undisclosed kinase

University of California, San Diego

Dennis Carson

20.0

Develop mAbs and small molecules that destroy leukemia stem cells to treat acute myelogenous leukemia (AML), chronic lymphocytic leukemia (CLL) and three other hematological malignancies

Grant revised in 2012 to focus on two mAbs to treat AML and CLL; budgets and timelines being reassessed

UCSD

Larry GoldsteinB

15.6

Treat amyotrophic lateral sclerosis (ALS) by implanting precursor astrocyte cells derived from hESCs

Team anticipates selecting a single cell line by mid-2012

University of California, San Francisco

Mitchel Berger

19.2

Treat brain tumors using neural stem cells modified to carry a tumor-killing drug

Grant discontinued in March 2012 for failure to meet an undisclosed milestone

University of Southern California

Mark Humayun

15.9

Treat age-related macular degeneration (AMD) using transplanted retinal pigment epithelium (RPE) cells derived from hESCs

Team on track to file IND within four-year award period

ViaCyte Inc.C

Allan RobinsD

20.0

Treat type 1 diabetes by implanting islet stem cells generated from hESCs

Team is preparing for IND-enabling studies

Total

 

229.8

 

 

AGeoff Symonds at biotech company Calimmune Inc. is also a principal investigator. BThe original principal investigator was Samuel Pfaff of the Salk Institute for Biological Studies. CFormerly Novocell Inc.; the company changed its name to ViaCyte in 2010. DThe original principal investigator was Emmanuel Baetge of Novocell (now ViaCyte).