Greenwich resident Barbara Netter is no stranger to the extraordinary breakthroughs that gene therapy research has led to in cancer treatment. Her nonprofit foundation, the Alliance for Cancer Gene Therapy (ACGT), has funded some of the cutting-edge research that has allowed the word "cure" to become a part of the cancer conversation.
But when she's able to stand next to Emily Whitehead, a child of seven, free of cancer for nearly a year after being treated with gene therapy that came after ACGT-funded research -- the reality of what those breakthroughs can mean can be quite moving.
"It's really very exciting," says Netter. "At the time of her treatment it was such a breakthrough. She had lymphoblastic leukemia at age 5. When she was 6 she was on a ventilator. They gave her a day to live."
Netter met both Emily and her parents at a celebratory meeting held last Tuesday at the University of Pennsylvania's Perelman School of Medicine, where Dr. Carl June developed ground-breaking treatment of Emily's leukemia that involved genetically engineering her T cells to attack her cancer with a disabled AIDS virus acting as a delivery system. June treated Emily last April when she was near death. After surviving a severe reaction to the treatment, Emily emerged cancer- free.
Now, she is happy -- and healthy.
"We did provide the initial seed money to fund Dr. June's T cell research," said Netter. It was ACGT's initial support of June in 2004 and again in 2008 -- nearly $2 million worth of funding -- that helped his clinical trials get off the ground and lead to his breakthrough treatment.
Emily recently has been joined by another 7-year-old, Maddie Major, who also has been declared cancer-free after receiving June's gene therapy treatment. She is one of four other children with advanced leukemia reported to be in similar treatment.
June has had other successes, as well, with his gene therapy treatment. Eight of 10 adults with chronic leukemia he has treated are in full remission, and that treatment is now being adapted to target solid tumors: prostate, pancreatic, ovarian and breast cancers.
Also present at the UPenn meeting was Dr. Robert Vonderheide, a senior researcher at the university. Vonderheide received funding from Netter's nonprofit in 2003 when he was chosen as an ACGT Young Investigator. The Young Investigator Award funds assistant professors who are conducting independent and innovative cell and gene therapy for cancer research in their own labs. Vonderheide shared with those at Tuesday's meeting of a clinical trial for pancreatic cancer that he hopes will soon take place.
"This is what can happen with these Young Investigators," said Netter. "They're pretty far ahead in their research."
Netter went on to announce that ACGT has two new 2012 Young Investigators who are conducting promising research. They will each receive half a million dollars.
Canadian Dr. Douglas Mahoney, ACGT's first international Young Investigator, is an assistant professor in the Department of Microbiology, Immunology and Infectious Disease at the University of Calgary. Like June, Mahoney is working on the use of viruses to treat cancer. With his initial focus on breast cancer, he reportedly will work with a team using oncolytic virus therapy to help reprogram cancer cells to self-destruct in various forms of cancer.
ACGT's Scientific Advisory Council chose as its second Young Investigator Dr. Alexander Stegh, assistant professor of neurology, at Northwestern University's Feinberg School of Medicine. Stegh is working on gene therapy as a possible treatment for brain cancer. "Despite significant advances in neurosurgical techniques, radiation oncology and numerous clinical trials," Stegh says, "high-grade brain tumors, especially glioblastoma, remain incurable diseases." Stegh's research is targeting certain proteins to be used towards fighting glioblastoma using glioma cell and mouse models.
Netter's optimism was apparent as she spoke of advances she learned about at the University of Pennsylvania meeting.
"They hope to have a blood test that can pick up circulating cancer cells before they evolve into a tumor," she says.
Netter, however, did express some disappointment. Despite the momentum in cancer treatment research, she says, the federal government is cutting back on cancer research funding
But that just makes ACGT's efforts all the more important.
ACGT is stepping into the breach," Netter said, "to boost funding for promising research that can deliver cancer cell and gene therapy discoveries."
To that end she said ACGT will give out additional grants this June.