Grants support doctors innovative cancer research

Some battles are so difficult, you have to take a gamble to have any hope of winning.

That's what leaders of Stand Up To Cancer are doing.

Since May 2008, the group has raised funds for groundbreaking cancer research. Earlier this month, it awarded nearly $10 million to a group of young research scientists who are thinking outside the box to find cures for cancer.

The Innovative Research Grants, overseen by the American Association for Cancer Research, will provide each of these projects with up to $750,000 over three years.

In all, 13 researchers will receive grants, out of about 450 who submitted proposals.

Among them are a pair of doctors at Childrens Hospital Los Angeles - Dr. Elizabeth Lawlor, an assistant professor of pediatrics and pathology at USC's Keck School of Medicine; and Dr. Markus Muschen, director of the Leukemia and Lymphoma Program at USC's Norris Comprehensive Cancer Center.

"We were looking for people who had good ideas - good research ideas - that had the potential for clinical application," said grant review committee chairman Richard Kolodner, professor of medicine at UC San Diego's Ludwig Institute for Cancer Research.

"We were particularly interested in innovative ideas that weren't necessarily being funded," he said. "In a lot of the grant world, those that issue the funding systems like to fund things that are guaranteed. That's OK, but it doesn't necessarily do a good job of identifying early-stage projects for which a lot hasn't been done."

Stand Up To Cancer, however, is more inclined to take a risk on unproven research, if there's a chance that success could lead to a major breakthrough.

Kolodner asked. "We were looking for early-stage, good, justifiable ideas that can have an impact. They could fail - but they could succeed."

"What do you want, the conservative thing that will always do a little bit, or something that has the chance to do a lot?"

Dr. Elizabeth Lawlor

Dr. Elizabeth Lawlor was a clinician, working with pediatric cancer patients, when she encountered one particular 12-year-old boy.

"He said, `It's the little kids I feel sorry for' - he was worried about his 5-year-old neighbor," Lawlor recalled. "Kids are phenomenal. Something that people always say to me is that it must be so sad (working with pediatric cancer patients). Yes, it's profoundly sad, but it's also profoundly inspiring. These children have a will to live and a perspective that we could all learn from."

Lawlor's experience with these young patients is a primary reason the Los Feliz resident is working to find a cure for Ewing's sarcoma.

"It's a rare disease," Lawlor said. "This is a problem with pediatric cancers in general. The numbers are very small, so it doesn't get the attention, so it doesn't get the funds. But if we save one 10-year-old and he lives to be 70, that's 60 years of life. That's a lot of life."

Lawlor said it is her belief that Ewing's sarcoma originates in the stem cells - that it's these cells that undergo the transformation that leads to the disease.

"You have to take a leap of faith," she said. "The cells we are using (in the research), we are saying we believe it is these cells in the body that give rise to the tumor - it's a controversial hypothesis. I still think that even if these are not the cells that give rise to the tumor in the body, we are still going to understand the changes in the body that happen when cancer evolves."

If the stem cells can be directly targeted, she said, spreading of the disease could be stopped. Finding a new, less-harmful treatment would be the next step.

"Since the 1960s, (treatment of) childhood cancer has been a huge success - some leukemias have a 90 percent survival rate," Lawlor said. "But it comes at a price - developmental delays, long-term heart problems - they are lifelong patients because of the side-effects of the drugs. My goal is that they've suffered as a child, let's let them be healthy as an adult."

Dr. Markus Muschen

Dr. Markus Muschen, a native of Cologne, Germany, compares his approach to attacking leukemia - finding a drug that goes directly to the root cell - as being similar to attacking an ant colony.

"If you have a state of ants and you kill the queen, then the entire organization falls apart, the state of ants can no longer function and the colony would die out," he said. "Our main hypothesis is that if we can kill the queen of ants at the root of the leukemia, then reproduction and generation of new leukemia cells is impaired and there's no way for leukemia to recur."

Through a collaboration with other scientists, Muschen said he has developed a drug that halts leukemia in mice. His grant proposal involves further development of a drug that can be used on human patients as part of a combination therapy to rid the body of leukemia.

"In our experiments, to cure 10 mice costs $18,000 ... and the mice have to be treated repeatedly," he said. "And of course, humans have more body to cover. We have to overcome this obstacle by finding a different formulation so we can produce (the drug) in larger amounts and it can be readily available for treatment."

Muschen expects to begin working with the U.S. Food and Drug Administration in 2010, and for Phase 1 clinical trials on a new leukemia drug to begin in 2011.

"Based on the results, we would modify the compound or go ahead and expand the scope," he said.

"What we have proposed to do right now, is for leukemia that already has relapsed once," Muschen said. "From an ethical standpoint, there is a very good rationale to offer this treatment regimen for patients for whom no established regimen is available."

Dr. Elizabeth Lawlor
Project proposal: Modeling Ewing Tumor Initiation in Human Neural Crest Stem Cells

In layman's terms: Studying the stem cells believed to be responsible for the development of the bone cancer Ewing's sarcoma, and developing a treatment that can be more effective and less toxic than what is currently available. According to the American Cancer Society, Ewing's sarcoma, of which there are three types, is most common in early teenagers. University of San Francisco Children's Hospital reports that about 1 in 50,000 teenagers develops Ewing's sarcoma, and that the disease accounts for about 30 percent of bone cancers in children. It most often starts in the arms or legs, often in the thigh bone.

Dr. Markus Muschen
Project proposal: Targeted Inhibition of BCL6 for Leukemia Stem Cell Eradication

In layman's terms: Development of a drug that targets the stem cells that are at the root of acute leukemia. Acute lymphocytic leukemia is the most common form of leukemia among children, with about 3,400 contracting the disease annually in the United States, Muschen said. About 80 percent of cases are considered cured right away through chemotherapy, but the other 20 percent relapse. Of those 20 percent, only a small portion can be cured. Once leukemia starts to relapse - some as quickly as within a few months - the initial treatment is no longer effective, Muschen said. A drug targeting the root cell would hopefully prevent this recurrence.

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