Dr. Thomas Griffith makes his living causing cancer cells to commit suicide. Without a foundation like Alliance for Cancer Gene Therapy (ACGT), his progress might be impossible. Founded by Barbara and Edward Netter and based in Stamford, Connecticut, ACGT is the only foundation solely dedicated to cancer gene therapy research.
For the uninitiated, cancer gene therapy is a ground-breaking approach to repair defective or missing genes that may cause cancerous cells. It involves bold attempts to reprogram cells by altering their genetic structure. Through gene therapy, researchers may bolster the body’s ability to fight cancer, or they may alter the cancer itself by cutting off its blood supply or may cause the cell to implode. Other methods make the cancer cells increasingly susceptible to chemotherapy or radiation.
Gene therapy is vastly different from traditional cancer treatments. Any time cancer is discovered, it’s a race to kill a tumor before it spreads throughout the body. They may surgically remove the tumor or poison it with chemotherapy and radiation. But those treatments can be as debilitating as the cancer itself.
Dr. Griffith’s genetic method is of the Trojan horse variety. He injects a virus that’s designed to carry a special molecule to cancer cells in the prostate. When it reaches its target the molecule commandeers cancer cells, causing them to self-destruct. Griffith, an associate professor at the University of Iowa, perfected the process on laboratory mice, and he is now in the earliest stages of human testing. So far, patients have suffered no side effects.
Griffith said “it sounds kind of futuristic,” but gene therapy could become a standard cancer treatment.
Everyone has a stake in the success of Griffith and other cancer researchers. The deadly disease is increasing and tapping our resources, costing about $210 billion in 2005, according to the National Institutes of Health (NIH). About half of men, and a third of women, have a chance of contracting the disease sometime in their lives. About 1.4 million Americans will be diagnosed with cancer in 2006, and it will kill 565,000 people. That’s like losing the population of Boston, Massachusetts, or Seattle, Washington, every year to cancer.
The success of Griffith and other researchers is dependent on private funding. Dozens of them are funded by Alliance for Cancer Gene Therapy.
Co-founder Edward Netter, 71, formerly a principal in an investment banking firm, currently operates life and health insurance companies as well as an educational mail order company. His wife Barbara is a therapist with an insatiable appetite and curiosity for scientific knowledge. Knowing the pain of losing a loved one to cancer inspires each of them.
The Netters spent 11 years watching their daughter-in-law fight and eventually lose her battle against breast cancer. The experience was devastating to their family and a fundamental motivation for starting ACGT. Watching a loved one fight cancer brings on “a feeling of helplessness,” says Barbara. “And to be able to do something about it is healing.”
Cancer gene therapy has been successful on humans in clinical trials, but no drug has been approved by the Food and Drug Administration (FDA) for human patients. Some consider gene therapy controversial, as it involves such powerful technology and has had some high-profile setbacks. According to the NIH, gene therapy research currently focuses on correcting genetic flaws and curing disease. In the future, though, it could be possible to alter human eggs or sperm, enhance memory or intelligence, and permanently alter the genetic lines for generations. The FDA forbids scientists to pursue these controversial methods, and they are not endorsed or funded by ACGT or practiced by grantees.
ACGT strives to combine sound science with solid business principles and accountability to ensure a sound return on donor dollars. Grants are given to individual scientists, not institutions, out of a belief that it’s the researchers themselves who are the visionaries, according to Margaret Cianci, ACGT Executive Director.
Grantees are required to publicize discoveries immediately, and their research is monitored to ensure it conforms to the grant’s stated goals.
“Whether it’s in the business world or in the pursuit of scientific research,” says Netter, “it’s important to critically examine the status quo and relentlessly pursue a better way. Even if everyone in science takes the same approach to treating cancer, no one should assume that’s the only approach to solving the problem.”
Eric Rothfeld, an ACGT donor and board member, appreciates how the foundation incorporates these accountability measures into its operation. The most donor-friendly aspect of ACGT, he says, is that 100 percent of foundation funds go to research. Separate funding covers all operating expenses, estimated at about $250,000 annually.
“This is not an organization that’s building an administrative bureaucracy,” Rothfeld says. “As more administration is needed it hasn’t reduced what the funds are used for.”
Additionally Rothfeld notes that ACGT multiplies donor dollars by claiming intellectual property rights to any breakthroughs, essentially making donations seed money for long-term research.
During its five years of operation, ACGT has given $14.2 million to 24 grantees, selected from 259 applications from 105 different institutions. Dr. Savio Woo, Professor and Chairman of the Department of Gene and Cell Medicine at Mt. Sinai School of Medicine in New York City, is integral to the foundation’s success. He created and currently leads ACGT’s scientific council, a group of 18 nationally recognized physicans and researchers, who assess and make recommendations about grant applications. The Council maps ACGT’s direction and ensures funding of only the most innovative proposals.
ACGT grants are some of the largest of their kind—$500,000 for Young Investigators and $1 million to Senior Investigators over a three to five year period—and provide vital funding at a time when money is scarce, due to increased competition for research funds.
Stephen Solender, a consultant and donor to ACGT, says the funding problem became painfully clear at a recent conference where he met a national leader in gene therapy research who spends about 60 percent of his time writing grant proposals to keep his research going.
Iowa’s Griffith launched his project in 2002 with a $500,000 Young Investigators grant. It was a huge sum for him, and he used it to complete studies that led to FDA approval of his clinical trial. “You can’t imagine how beneficial something like this was at that particular time,” Griffith says.
Now, Griffith feels the excitement of moving from the laboratory to the clinic, and seeing a potential breakthrough for cancer patients. He’s realizing a dream that may never happen for some researchers.
“ACGT’s long-term goal is to develop cures for cancer,” says Woo. But the immediate objective is to prolong the lives of patients without pain and suffering and to develop gene drugs that are safe for patients and deadly for cancer.
ACGT grantees have already spent years in the laboratory and have demonstrated success. They’re ready to test their techniques on people, which increases both the stakes and the financial costs. The earliest phase of clinical research is designed to demonstrate a procedure that is safe for human patients. The cost for such a project? $3 to $5 million, says Woo. The hope is that after Phase I, a pharmaceutical company will take interest in the research and fund it to completion.
John Adler, one of the original ACGT board members and donors, believes it’s important to focus on research that’s closer to the clinical trial phases. He adds that funding research at this stage means it may be only five years—instead of 15—until breakthroughs turn into life-saving drugs. Barbara and Edward Netter are expanding their pool of donors and plan to increase ACGT’s growth. Meanwhile, the alliance’s growing expertise in cancer treatment and its network of contacts in the medical field has made it an invaluable resource to friends and family members stricken with cancer.
The personal knowledge of so many people with cancer lends urgency to the Netters’ cause. While a cure for cancer is out of the immediate picture, Edward Netter believes that even extending someone’s life by months and turning cancer into a manageable disease are important victories.
“It’s important to uncover treatments to better people’s lives immediately,” says Netter.
His wife agrees. “It’s not soon enough if you’re suffering from this devastating disease. It can’t be soon enough.”