Joel Wood has just one goal for his foundation—to shutter it. Wood heads the Foundation to Eradicate Duchenne, a public charity that funds clinical trials for Duchenne disease. A form of muscular dystrophy, Duchenne disease strikes only boys and is always fatal—usually by the victim’s late teens. Joel’s 6-year-old son, James, suffers from it.
“Shutting our doors is the only measure of success,” he says over lunch with Philanthropy. “If I’m doing this fifteen years from now,” Wood continues, it will mean Duchenne is still taking lives and James is still a victim.
Wood is one of a growing number of philanthropists who, unhappy with the pace of development for new drug therapies, are taking matters into their own hands. They’re bypassing traditional, large disease-oriented charities—such as Muscular Dystrophy Association or the American Cancer Society—and directly funding those researchers who are translating rapid medical advances in genetics and other fields into practical treatments that patients and their physicians can use.
Fresh Market Approaches
The philanthropists who are breaking with tradition and funding research and clinical trials directly are very often involved with so-called “orphan” diseases. Orphan diseases are ones that afflict a very small percentage of a given population, a fact that discourages traditional funders of drug research and development, pharmaceutical companies, from funding drug development programs. The small population all but guarantees the company will never recoup the money it puts into research and development.
This is the case with Multiple Myeloma, a rare form of cancer that affects less that 1 percent of all cancer patients. Prior to 1998, there was little research on the disease, and no new therapies had been developed in over a decade despite significant advances in our understanding and treatment of other cancers.
So when Kathy Giusti, a former marketing executive with GD Searle pharmaceutical company, was diagnosed in 1996 with the disease, she understood the odds she was up against. But instead of waiting for the pharmaceutical companies to get involved, she found a way to use the market and motivate them to do drug development for the disease.
Her effort to find new therapies began in 1998, when she founded the Multiple Myeloma Research Foundation, a public charity. Giusti figured out that drug companies would be more likely to move products to market if they could be sure the products they developed would prove effective. So she took away the risks drug developers face by funding the early, risky stages of research that are necessary to prove or disprove the efficacy of a particular drug.
Velcade, the first new drug in over a decade approved for treating Multiple Myeloma, owes its existence to MMRF. A Boston company, Millennium, brought the drug to market. But MMRF funded the initial research through the Dana-Farber Cancer Institute at Harvard University. The research Giusti’s foundation helped fund established the drug’s efficacy, thereby making it economically attractive for Millennium to take on the project.
Over the past four years, MMRF has given an average of $4 million per year in research grants. The amount seems small when one considers that the United States government earmarked $20 million for Myeloma research alone in 2001. Yet it’s the work funded by MMRF that led to Velcade.
The foundation attributes its success to its approach to grantmaking. “We aren’t your father’s nonprofit,” Scott Santarella, executive director of MMRF, tells Philanthropy. We “target dollars to specific research projects, and hold the researchers to doing the work.” By doing this, he continues, it’s possible to “almost force the issue of progress.”
The foundation also holds itself to the fire. “We have a 24/7 mentality at the foundation,” says Santarella. The organization is lean, with a staff of 10, and every staff member is involved with either fundraising or cost-cutting. The board also keeps the staff to the grindstone. Santarella says while board members leave the day-to-day activities to him and his people, they provide “strategic direction and leverage their financial ties to keep us focused on the mission.” The board also sets guidelines for fundraising that ensure the foundation doesn’t lay out a disproportionate amount of dollars to raise funds.
Research Ex Nihilo
The Theodore and Vada Stanley Foundation, like MMRF, is also tackling a problem that traditionally hasn’t received significant research attention. But unlike Multiple Myeloma, the two related illnesses the Stanley Medical Research Institute funds research on could hardly be considered orphan. Bipolar disorder and schizophrenia affect some 4 million Americans. Jonathan Stanley, the son of SMRI founders Theodore and Vada Stanley, is one of those victims and the inspiration for the foundation.
Dr. E. Fuller Torrey was asked in 1989 by the Stanleys to help them spend a modest amount of funds on research. With the Stanleys, Dr. Torrey launched SMRI, which today has a budget of some $45 million. He has recently stepped down as executive director to spend more time in the lab doing research.
Under Dr. Torrey’s leadership, SMRI became the largest private supporter of research into bipolar disorder and schizophrenia, funding approximately half of all U.S. research on bipolar disorder, and about one-quarter of all U.S. research on schizophrenia. To date, SMRI has invested about $160 million.
Among the institute’s greatest achievements is its collection of brains from deceased individuals who suffered from bipolar disorder and schizophrenia. “We have 575 specimens,” says Dr. Torrey, evenly divided between the two disorders. When the institute was founded in 1989, there was little brain tissue available for those doing research on these disorders. The institute now has sent out over 160,000 sections for study to researchers around the world.
The institute is also funding fundamental research into the possibility that there are infectious agents that may cause these disorders in some people. “If we’re right,” Dr. Torrey says, “this leads to the possibility of vaccines.”
MDA and Duchenne
Dr. Eric Hoffman, a researcher at Children’s National Medical Center in Washington, D.C., has been working with Joel Wood on Duchenne disease since 2000. He’s also a strong advocate of philanthropists in disease research. The federal government spends billions each year for scientists who are unlocking the basic biology of a disease, he explains, but “once they discover what’s wrong the researchers are off to the next disease.”
Hoffman could have gone on to the “next disease” in 1987. That year, he discovered that Duchenne victims all lack the same protein, dystrophin. But Hoffman wanted to be part of the cure, not just the team that discovered what was broken. He received an offer to bring his research to Children’s Hospital in 1999.
At Children’s, Hoffman recruited Dr. Diana Escolar, and together they established the Cooperative International Neuromuscular Research Group, or CINRG, to launch advanced research on Duchenne and conduct the clinical trials necessary to bring new treatments to the public. Currently, this 15-site, eight country network is conducting trials on five agents, including Creatine, a steroid they hope will slow the disease’s progression.
When defending its funding of Duchenne research, the Muscular Dystrophy Association points to the CINRG study as evidence of its work in finding a cure. Hoffman acknowledges the MDA support, particularly through parent-directed donations, and appreciates the contributions, but he’s quick to add that there would be no CINRG program to fund were it not for Joel Wood and other parents.
The money Wood has raised has been “absolutely critical” to CINRG’s success, says Hoffman. Right now, of CINRG’s three major sources of revenue, Wood’s contribution and that of other private donors account for half of CINRG’s funds. The National Institute of Health provides about 23 percent of funds, and MDA provides about 27 percent.
Further, Hoffman adds, private donations accounted for “probably 100 percent . . . for the first two years; then MDA and NIH kicked in.” Without the help that Wood has provided, this clinical network and the these clinical trials “would not have happened.”
Hoffman and Wood both acknowledge that MDA does some outstanding work. MDA clinics, according to Hoffman, are excellent and provide quality care. But these clinics don’t have clinical trials as part of their funding base. Wood points out that MDA’s palliative care is also excellent and necessary. Currently, 58 percent of MDA’s budget goes for “patient and community services.”
But for both Hoffman and Wood—as for Giusti, the Stanleys, and others like them—the urgent need is to find a cure. James Wood at age six is still a relatively healthy young boy. “If I were at a different stage in my son’s illness, I would probably feel different,” says Joel. “Do I know we’ll find a cure?” Wood asks. “Absolutely. Do I know it will come in time to save my son? No.”
Hoffman and his team of researchers are providing Wood hope, and Wood is providing the funds that will one day lead to a cure, and the shuttering of his organization.