Heart disease, blindness, Alzheimer’s and muscular dystrophy research get multi-million dollar funding boost from Stem Cell Agency
San Francisco, CA –Researchers working on using stem cells to treat heart disease, muscular dystrophy, Alzheimer’s and retinitis pigmentosa have been awarded $63 million in funding by California’s stem cell agency, the California Institute for Regenerative Medicine (CIRM) at a meeting of its governing board on September 5th and 6th.
One of the projects, using stem cells to try and reduce scarring and regenerate heart muscle after a heart attack, has already been approved by the Food and Drug Administration to move into clinical trials.
“There are more than 6 million people in the US living with heart disease so this approach has the potential to make an enormous impact on public health,” says Jonathan Thomas, PhD, JD, Chair of the Independent Citizens Oversight Committee (ICOC), CIRM’s governing body. “Our goal is to fund and support the best science, and the best scientists to produce therapies for a wide range of diseases. What they learn, what they discover, will ultimately benefit all of us.”
The heart disease project was previously funded by CIRM as a Disease Team project using the patient’s own cells. The researchers have since changed their methodology, using donor cells to develop the therapy. With that in mind the ICOC voted to fund this for phase II clinical trials following NIH funding of the phase I safety approach. The retinitis pigmentosa research project has moved rapidly forward from a CIRM translational project to one nearly ready for a phase I/II clinical study.
“These are excellent examples of CIRM moving projects efficiently and effectively through the translational pipeline and into clinical trials,” says Alan Trounson, PhD, President of CIRM. “We expect many other projects to evolve to first-in-human therapeutics that hopefully will deliver significant benefit to patients”
The muscular dystrophy application was awarded funding as an Early Translation project, which focuses on proof of concept of a new therapy.
The ICOC also awarded more than $38 million to 28 other projects as part of its Basic Biology awards program, focusing on basic research, to gain a better understanding of the different kinds of stem cells and how to work with them.
“This kind of basic research is essential to helping us answer some essential questions about stem cells,” says Pat Olson, PhD, Executive Director of Scientific Studies at CIRM. “The knowledge we gain from these studies will ultimately inform other work and advance our understanding of the fundamental mechanism of stem cell biology, and move us ever closer to knowing how best to use stem cells to help patients.”
In addition the ICOC approved $6.7 million in funding for a Research Leadership Award. The Leadership Awards are intended to help draw talented faculty members to California institution. In this case it will bring Dennis Steindler to the Parkinson’s Institute in Sunnyvale to do research that will help better understand the importance and uses of stem cells for the treatment of Parkinson’s disease, and at the same time get a better insight into their role in disease repair and causation.
The meeting celebrated the tenure of two ICOC members and welcomed a new member to the board. Ted Love, MD and David Serrano Sewell, JD were thanked for their contributions to the stem cell agency, and praised for their dedication and compassion; the ICOC said the skills and expertise they brought played an important role in helping shape CIRM. The Board also welcomed its newest member, Anne-Marie Duliege, MD. She is currently the Chief Medical Officer for biotech company Affymax and brings with her considerable clinical trial expertise to the Board and hands-on experience with the FDA approval process.
Disease Team II applications funded
Disease Team II Award project summaries
| Application | Researcher | Institution | Total Funds Requested |
|---|---|---|---|
| DR2A-05735 | Rachel Smith | Capricor, Inc | $19,782,136 |
| DR2A-05739 | Henry Klassen | University of California, Irvine | $17,306,668 |
| DR2A-05426 | Stanley Nelson | University of California, Los Angeles | $6,000,000 |
| DR2A-05416 | Alexandra Capela | StemCells, Inc. | $20,000,000 |
| Total | $63,088,804 |
Research Leadership application funded
Research Leadership award project summaries
| Application | Researcher | Institution | Total Funds Requested |
|---|---|---|---|
| LA1-06535 | Dennis Steindler | The Parkinson’s Institute | $6,718,471 |
Basic Biology IV applications funded
Basic Biology IV award project summaries
| Application | Researcher | Institution | Total Funds Requested |
| RB4-05763 | Howard Chang | Stanford University | $1,386,627 |
| RB4-05779 | George Sen | University of California, San Diego | $1,080,000 |
| RB4-05785 | Deepak Lamba | Buck Institute for Age Research | $1,526,319 |
| RB4-05825 | Roel Nusse | Stanford University | $1,195,811 |
| RB4-05855 | Jeremy Reiter | University of California, San Francisco | $1,387,800 |
| RB4-05886 | Gerald Crabtree | Stanford University | $1,392,426 |
| RB4-05901 | Benoit Bruneau | The J. David Gladstone Institutes | $1,708,560 |
| RB4-05990 | Barbara Panning | University of California, San Francisco | $1,285,214 |
| RB4-06016 | Robert Tjian | University of California, Berkeley | $1,220,968 |
| RB4-06028 | Miguel Ramalho-Santos | University of California, San Francisco | $1,184,883 |
| RB4-06035 | Sheng Ding | The J. David Gladstone Institutes | $1,708,560 |
| RB4-06036 | Michael Cleary | Stanford University | $1,244,455 |
| RB4-06041 | Ben Barres | Stanford University | $1,385,517 |
| RB4-06045 | Gene Yeo | University of California, San Diego | $1,393,200 |
| RB4-06079 | Steven Finkbeiner | The J. David Gladstone Institutes | $1,506,420 |
| RB4-06087 | Anne Brunet | Stanford University | $1,414,044 |
| RB4-06093 | Michelle Monje | Stanford University | $1,264,248 |
| RB4-06102 | Alexander Dunn | Stanford University | $1,064,224 |
| RB4-06133 | Kathrin Plath | University of California, Los Angeles | $1,382,400 |
| RB4-06144 | Maike Sander | University of California, San Diego | $1,391,400 |
| RB4-06209 | Owen Witte | University of California, Los Angeles | $1,382,400 |
| RB4-06215 | Eduardo Marbíçn | Cedars-Sinai Medical Center | $1,367,604 |
| RB4-06244 | Ananda Goldrath | University of California, San Diego | $1,161,000 |
| RB4-06276 | Huei-sheng Chen | Sanford-Burnham Medical Research Institute | $1,582,606 |
| RB4-06345 | Miles Wilkinson | University of California, San Diego | $1,360,450 |
| RB4-06158 | David Traver | University of California, San Diego | $1,363,698 |
| RB4-06277 | Yanhong Shi | Beckman Research Institute of City of Hope | $1,367,172 |
| RB4-05764 | Deborah Lieu | University of California, Davis | $1,334,880 |
| Total | $38,042,886 |
Disease Team and Early Translational Award brief descriptions
Capricor
This company worked with the team from Cedars-Sinai to make a version of its Cardiospheres in which the cells are separated, which they call Cardiosphere Derived Cells. Using donor cells makes the therapy easier than using a patient’s own cells and makes it accessible to more patients – an important factor in treating heart attack patients where speed is important. In June 2012 they received FDA permission to begin a clinical trial in patients who had a heart attack relatively recently, and who already had clear signs of reduced heart function. This CIRM award will fund a clinical trial to test the safety and effectiveness of this therapy.
More information about CIRM funding for heart disease
University of California, Irvine
For retinitis pigmentosa the team plans to use donor tissue to isolate cells that are part way down the path from neural stem cells to adult eye tissue. These retinal progenitor cells would be grown in large quantities in the lab and then injected into the eye. The team suggests the cells could help in two ways. They may be able to protect the photoreceptors not yet damaged by the disease, and they may be able to form new photoreceptors to replace those already lost. They plan to complete testing needed to apply to the FDA to begin an early phase trial of the therapy.
More information about CIRM funding for blindness
University of California, Los Angeles
The team plans a therapy that can give Duchenne Muscular Dystrophy (DMD) patients a correct version of dystrophin, a protein that plays a key role in muscle cell health, but is defective in DMD. They plan to alter genes in the muscle cells so as to result in normal production of dystrophin. The initial goal of this project was to complete preclinical studies looking at safety and effectiveness, and to file an Investigational New Drug application (IND) within four years. However, the ICOC felt these goals were too ambitious and instead recommended this be re-categorized as an Early Translational study, one more focused on proof of concept rather than drug development.
More information about CIRM funding for muscular dystrophy
StemCells, Inc.
This team proposes to inject adult brain stem cells into the front section of the brain. They have shown in animal models that these cells produce chemicals that appear to help protect existing nerve cells. They plan to use this award to produce cells that meet the high standards needed to get FDA approval and to complete the testing required before they can begin clinical trials in people. More information about Alzheimer’s disease.
More information about CIRM funding for Alzheimer’s disease
About CIRM: CIRM was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure, which provided $3 billion in funding for stem cell research at California universities and research institutions, was overwhelmingly approved by voters, and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities, and other vital research opportunities. A list of grants and loans awarded to date may be seen here: https://www.cirm.ca.gov/grants.
Contact:
Kevin McCormack – 415-361-2903 or kmccormack@cirm.ca.gov
or Don Gibbons – 415-740-5855 or dgibbons@cirm.ca.gov