Doctor awarded grant to study cancer drugs
Dr. Nancy Gordon seeks solutions to rare problems, even when the revenue stream is not easy to come by.
Gordon is an associate professor in the division of pediatrics at MD Anderson’s Graduate School of Biomedical Sciences. And she wants to make life better for patients diagnosed with osteosarcoma, a cancer that develops in the bone and affects about 700 to 1,000 new people a year, including 400 adolescent and young adult patients.
Chemotherapy and surgery are typically successful at controlling the tumor in the bone, but 80 percent of adolescents and young adults who are diagnosed with the cancer will relapse, with the cancer metastasizing in their lungs, Gordon said.
“What we discovered was that one of the chemotherapy agents used in patients who relapse induces a resistant mechanism in the tumor, which puts the cells in a state of inanimation,” Gordon said.
Gordon and her team at MD Anderson hypothesized that adding a third drug, hydroxychloroquine, to the original regimen of gemcitabine and docetaxel — could make life better for young patients who have relapsed.
Hydroxychloroquine, a radioactive drug that has seen national prominence as an unproven treatment for COVID-19, inhibits the tumor’s resistance to chemotherapy in a laboratory setting, Gordon said. In 2019, a study found that gemcitabine and docetaxel work together to stop
tumor growth in relapsed highgrade osteosarcoma when administered in chemotherapy.
Because osteosarcoma is so rare, there is not a large enough
pool of patients to participate in a clinical trial. But Gordon and her team needed to conduct a trial that incorporated biopsies to know whether these three drugs would work to stop tumor growth in humans.
Gordon received a $50,000 grant from Cures Within Reach, a Chicago-based nonprofit orga
nization that funds clinical trials in humans and some late-stage animal studies, for the means to conduct a multiphase trial. Cures Within Reach funds several studies on drugs and therapies that are already approved by the U.S. Food and Drug Administration to treat other medical ailments, usually enrolling between 50 and 100 patients, said Barbara Goodman, president and CEO.
Researchers need a small sample of success to secure future funding from larger institutions, Goodman said.
“The fastest way to patient impact is testing approved therapies,” she said. “Our goal is to do the critical, first in-human study that is successful. Once all the other funding comes in, a follow-up study can be done.”
Projects funded by Cures Within Reach range from $50,000 to $1 million, Goodman said. As a nonprofit, Goodman said Cures Within Reach is indifferent to the commercial value of a clinical trial. In Gordon’s research, the three drugs that needed testing are FDA approved and commercially available, but there was little incentive for major pharmaceutical companies to provide funding.
That’s where the organization steps in and covers small trials, Goodman said. Since 2005, Cures Within Reach has funded more than 30 U.S. projects and seven in other countries.
Gordon said the grant will go toward conducting biopsies on osteosarcoma patients who receive the three-drug chemotherapy combination. Hopefully, the biopsies will help identify biological markers for the disease that will allow the MD Anderson researchers to see how patients respond to the therapy.
“It is helping at complementing with studies that could not be done otherwise,” Gordon said.