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Who gets stem cell funding is the $3 Billion Question

Bay Area scientists studying Alzheimer's, sickle cell anemia among those seeking cash

By Rebecca Vesely, STAFF WRITER

ALONDRA FERNANDEZ, 7, was cured of leukemia after receiving the cord blood of younger brother Bernardo, now 4, at Children's Hospital Oakland. The hospital plans to seek funding from the state's new stem cell research program to finance the sibling cord blood bank, the only one in the world.

04/10/2005 - BERNARDO FERNANDEZ JR. saved his sister's life the day he was born. It's quite a feat for such a little boy, one that gives him great pride four years later.

"He says, 'Alondra! Alondra! I gave Alondra blood, and now she's fine,'" explained his mother, Fabiola Jovinez, 25, of Concord.

Alondra, who is 7 and loves computers and writing, was diagnosed with acute myelogenous leukemia at age 2. She is one of about 50 children cured through Children's Hospital Oakland's sibling cord blood bank the only such program in the world.

The sibling cord blood bank was launched five years ago with a research grant through the National Institutes of Health. It provides families who have children with certain life-threatening diseases the optionof saving the cord blood of any siblings born after the ill child is diagnosed, at no charge. Cord blood is rich in stem cells that have, as in Alondra's case, cured childhood leukemia, sickle cell anemia and some other illnesses.

NIH funding for the bank ends this month. The hospital is hoping it can get long-term funding through California's new $3 billion stem cell research program, which voters approved in November under Proposition 71.

Dr. Bertram Lubin, president and medical director of Children's Hospital Oakland Research Institute, said there is still much to learn about cord blood.

"We'd like to see the state fund the service as well as research exploring the regenerative process of cord blood," Lubin said. "There's no ethical issues here. Parents own the cord blood, and it provides an option for these families."

Researchers lining up

Lubin's pitch is just one of many that officials at the state's stem cell research program will hear in the coming months. Tasked with distributing $3 billion over the next decade, the California Institute for Regenerative Medicine is beginning to formulate grant-making guidelines.

While it could be at least six months before checks are cut, Bay Area researchers are wasting no time forming alliances and reviewing their scientific goals to better position themselves when the dollars do start to flow.

Any slice of the pie would be a huge boon to scientists, who often toil in tiny labs with a patchwork of grants. Scientists working on controversial embryonic stem cell research are even more constrained due to federal limits on funding.

"We've been working on human embryonic stem cell research forever, and we want to work on it some more," said Renee Reijo, co-director of the Human Embryonic Stem Cell Program at the University of California, San Francisco. "We just want it to be easier. It's really hard to piece funding together when you can't use federal dollars."

The 29-member Independent Citizens Oversight Committee, which oversees the new stem cell institute, has ultimate authority over the grants' focus. The committee is made up of top scientists at state universities, patient advocates and biotech leaders.

The ICOC is anticipating the release of ethical and medical standards from the National Academy of Sciences on stem cell research, expected by the end of the month. The committee will use these national standards as guidelines for developing protocols for the institute, said spokeswoman Fiona Hutton.

"We're on pins and needles," Reijo said of the final guidelines.

Enormous potential

Zach Hall, the interim president of the newly created California Institute for Regenerative Medicine, which will issue the grants, said the first round will likely lay the groundwork to support an explosion in biomedicine in the state.

"Our job is not just to be a banker," he said recently. "Hopefully, we will facilitate research."

The institute hired a top official at the National Institutes of Health, Arlene Chiu, last week as director of scientific programs and review. Chiu was associate director of the Office of Research Administration of the National Institute of Biomedical Imaging and Bioengineering, a division of NIH, where she oversaw grants management and policy programs.

Encouraging universities to develop strong stem cell biology programs and laboratories will likely be the first funding priority, Hall said. In addition, training grants for students will be key to reach a critical mass of scientists who can solve the complex riddles of stem cells, he said.

"Our first job is to understand the natural history of stem cells," Hall said. "What makes them differentiate? What makes them develop?"

Stem cells hold enormous potential for medical science because they are undifferentiated, meaning they haven't yet formed into a cell with a specific purpose.

Adult stem cells undifferentiated cells found in tissues have research limitations because they can develop only into cell types of the originating tissue.

Cord blood stem cells are harvested within 15 minutes of birth, while the umbilical cord is attached to the placenta. Researchers have been most successful in turning these stem cells into blood cells hence their ability to treat blood diseases such as leukemia and sickle cell anemia.

Most promising are embryonic stem cells, primitive cells from the embryo. They hold untold potential because they can develop into any cell type in the body. Figuring out what makes an embryonic stem cell become a liver cell, heart cell or brain cell could lead to cures of our most devastating diseases, such as Alzheimer's, heart disease and diabetes.

Researchers across the Bay Area are working on these very basic questions. Reijo, for instance, studies the genes involved in sperm and egg cells with the goal of understanding human development and the problems that can arise along the way, such as infertility and birth defects.

Answering these fundamental questions of human biology first requires a basic framework to do the research, especially in a new area that has lacked federal support, said Dr. Bruce Conklin, associate investigator at the UCSF-affiliated J. David Gladstone Institute. Conklin studies heart rate control and arrhythmia using heart cells derived from embryonic stem cells in mice. "If you're going to write a book, you would need a basic vocabulary," Conklin said. "We need to think about what are these basic words, or tools, that everyone can use."

Partnership plans

Working collaboratively across institutions will likely be one outcome of Proposition 71.

Researchers at the University of California, Berkeley, are not yet working on human embryonic stem cells, but Dr. Randy Schekman, professor of cell and developmental biology, said the university plans to do so.

Schekman is one of 30 scientists at UC Berkeley who are interested in starting an embryonic stem cell program. Research would focus on gene expression, or how genes turn on and off, as well as immune function and self-renewal in cells, he said.

Schekman's research focuses on how cells go bad and cause Alzheimer's. He is experimenting with undifferentiated stem cells to suppress peptides short protein segments that lead to the brain-wasting disease.

UC Berkeley is considering a partnership with Children's Hospital and Research Center Oakland to develop an embryonic stem cell program.

Lubin said teaming up with UC Berkeley is a natural fit.

"We'd like to be the hospital that would be the research partner to Berkeley," he said. "Working with Randy Schekman would only be a positive for us."

Schekman said the university is looking at sites in West Berkeley and Emeryville for a new embryonic stem cell laboratory. Embryonic stem cell projects underwritten by Proposition 71 must be kept separate from federally funded programs because of federal policies limiting the use of embryos in research.

Just how separate is not yet clear, but Hall, Schekman and others said that building new labs specifically for state-funded research should help alleviate any concerns at the National Institutes of Health that federal funds are being used inappropriately.

UC Berkeley will likely submit Proposition 71 grant applications for new facilities, at a cost of between $5 million and $7 million, Schekman said.

Officials at UCSF and Stanford University also said they would seek facility funding for their own stem cell research projects but declined to say how much that might cost.

Currently, UCSF has a partnership with Geron Corp. in Menlo Park to share space for embryonic stem cell research. But Dr. Arnold Kriegstein, director of UCSF's Developmental Cell Biology program, said he hopes to renovate space on or near campus to allow researchers to work nearby.

Dr. Michael Longaker, director of children's surgery research at Stanford University Medical Center, said in addition to leasing new space, Stanford will recruit about 15 new faculty scientists in cancer and stem cell research. Competition for these faculty members will likely be intense among universities, he said.

Waiting for results

Recruiting top scientists, training students and getting laboratories up and running could take years, raising concerns that taxpayers will grow frustrated with the state's stem cell program, which promised them cures to myriad diseases.

Hall cautioned that late-stage human clinical trials for any practical applications could take at least 10 years. University researchers interviewed agreed.

Continuing his pitch, Lubin of Children's Hospital Oakland said the institute should fund some programs that are further along such as his cord blood bank so voters can see immediate results. The hospital has the cord blood of 1,500 children in the bank, and it costs a mere $1 million a year to run.

Cord blood doesn't have to be a perfect match between patients, so some children have been cured using cord blood banked by nonrelatives.

"The return on investment would be dramatic," Lubin said.

Still, Proposition 71's aim was to support embryonic stem cell research that can't get federal dollars under the Bush administration's restrictions. President Bush, by contrast, has touted the promise of cord blood and adult stem cell research.

"The initial effort would be to fund things that can't be funded by the federal government," Hall said. "We want these projects to have a compelling interest."

Alondra's mother remembers sitting at her daughter's bedside at Children's Hospital Oakland, nine months pregnant with Bernardo Jr. She had already been told that Alondra's younger sister, Yulisa, now 6, was not a match for a bone marrow transplant.

"The doctors told me that it was possible this baby would be a match to Alondra, and it was my only hope," Jovinez said through a Spanish interpreter.

Alondra went into remission after Bernardo Jr. was born, but the hospital saved his cord blood in case she needed it. And she did. When Bernardo was 1, Alondra received both a bone marrow transplant from her brother as well as his banked cord blood.

When Alondra woke up from the procedure, she got out of bed and hugged her little brother and thanked him for saving her life.

For now, at least, Alondra and her family are among the lucky few who can say they were cured by stem cells.

Contact Rebecca Vesely at [email protected].

About LEUKINE

LEUKINE, a man-made form of a naturally occurring growth factor that helps fight infection and disease in appropriate patients by enhancing cells of the immune system, was approved in the United States in 1991, and is marketed by Berlex, Inc.

LEUKINE is the only growth factor approved in the United States for use following induction chemotherapy in older adults with acute myelogenous leukemia (AML) to shorten the time to neutrophil recovery and reduce the incidence of severe and life-threatening infections and infections resulting in death.

LEUKINE has also been approved in the U.S. for use in four additional indications: myeloid reconstitution following allogeneic and autologous bone marrow transplantation (BMT), peripheral blood stem cell (PBSC) mobilization and subsequent myeloid reconstitution in patients undergoing PBSC transplantation, and bone marrow transplantation failure or engraftment delay. LEUKINE has been administered to more than 300,000 patients.

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