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									    ADVENTUR ES I N


    philanthropy
This article appeared in the November/December issue of Philanthropy, a publication of The Philanthropy Roundtable.


Challenging the status quo in cancer research
Alliance for Cancer Gene Therapy is creating breakthroughs
in cancer treatment




D
BY MARSHALL ALLEN



     R.THOMAS GRIFFITH MAKES HIS               Stamford, Connecticut, ACGT is the            attempts to reprogram cells by altering
    living causing cancer cells to             only foundation solely dedicated to           their genetic structure. Through gene
    commit suicide. Without a foun-            cancer gene therapy research.                 therapy, researchers may bolster the
    dation like Alliance for Cancer                 For the uninitiated, cancer gene         body’s ability to fight cancer, or they
Gene Therapy (ACGT), his progress              therapy is a ground-breaking approach         may alter the cancer itself by cutting
might be impossible. Founded by Bar-           to repair defective or missing genes that     off its blood supply or may cause the
bara and Edward Netter and based in            may cause cancerous cells. It involves bold   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 sur-
                                                                                             gically 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 pro-
                                                                                             fessor at the University of Iowa, per-
                                                                                             fected 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 suc-
ACGT Senior Investigator Dr. Eckhard Podack works in his lab at the University of Miami’s    cess of Griffith and other cancer
                         Leonard M. Miller School of Medicine.                               researchers. The deadly disease is
9   Philanthropy • November/December 2006
     A D V E N T U R E S                      I N   P H I L A N T H R O P Y


     increasing and tapping our resources,          enhance memory or intelligence, and           rate funding covers all operating
     costing about $210 billion in 2005,            permanently alter the genetic lines           expenses, estimated at about $250,000
     according to the National Institutes           for generations. The FDA forbids              annually.
     of Health (NIH). About half of men,            scientists to pursue these controver-              “This is not an organization that’s
     and a third of women, have a chance            sial methods, and they are not                building an administrative bureaucra-
     of contracting the disease sometime            endorsed or funded by ACGT or                 cy,” Rothfeld says. “As more adminis-
     in their lives. About 1.4 million              practiced by grantees.                        tration is needed it hasn’t reduced what
     Americans will be diagnosed with                    ACGT strives to combine sound            the funds are used for.”
     cancer in 2006, and it will kill               science with solid business principles             Additionally Rothfeld notes that
     565,000 people. That’s like losing             and accountability to ensure a sound          ACGT multiplies donor dollars by
     the population of Boston, Massa-               return on donor dollars. Grants are           claiming intellectual property rights
     chusetts, or Seattle, Washington,              given to individual scientists, not           to any breakthroughs, essentially
     every year to cancer.                          institutions, out of a belief that it’s the   making donations seed money for
          The success of Griffith and other         researchers themselves who are the            long-term research.
     researchers is dependent on private            visionaries, according to Margaret-                During its five years of operation,
     funding. Dozens of them are funded                                                           ACGT has given $14.2 million to 24
     by Alliance for Cancer Gene Therapy.                                                         grantees, selected from 259 applica-
          Co-founder Edward Netter, 71,                      “It’s important                      tions from 105 different institutions.
     formerly a principal in an invest-                                                           Dr. Savio Woo, professor and chair-
     ment banking firm, currently oper-
     ates life and health insurance com-
                                                        to critically examine                     man of the Department of Gene and
                                                                                                  Cell Medicine at Mt. Sinai School of
     panies as well as an educational mail                                                        Medicine in New York City, is inte-
     order company. His wife Barbara
                                                              the status quo                      gral to the foundation’s success. He
     is a therapist with an insatiable                                                            created and currently leads ACGT’s
     appetite and curiosity for scientific
                                                             and relentlessly                     scientific council, a group of 18
     knowledge. Knowing the pain of                                                               nationally recognized physicans and
     losing a loved one to cancer inspires
                                                                  pursue a                        researchers, who assess and make rec-
     each of them.                                                                                ommendations about grant applica-
          The Netters spent 11 years
                                                               better way.”                       tions. The Council maps ACGT’s
     watching their daughter-in-law fight                                                         direction and ensures funding of only
     and eventually lose her battle against                                                       the most innovative proposals.
     breast cancer. The experience was              Cianci, ACGT executive director.                   ACGT grants are some of the
     devastating to their family and a fun-         Grantees are required to publicize dis-       largest of their kind—$500,000 for
     damental motivation for starting               coveries immediately, and their               Young Investigators and $1 million
     ACGT. Watching a loved one fight               research is monitored to ensure it            for Senior Investigators over a three
     cancer brings on “a feeling of helpless-       conforms to the grant’s stated goals.         to five year period—and provide
     ness,” says Barbara. “And to be able                “Whether it’s in the business            vital funding at a time when money
     to do something about it is healing.”          world or in the pursuit of scientific         is scarce. Woo attributes the recent
          Cancer gene therapy has been              research,” says Netter, “it’s important       funding decline to three factors.
     successful on humans in clinical tri-          to critically examine the status quo               First, money allocated by Con-
     als, but no drug has been approved             and relentlessly pursue a better way.         gress to the NIH, the federal govern-
     by the Food and Drug Administra-               Even if everyone in science takes the         ment’s focal point for research, is
     tion (FDA) for human patients.                 same approach to treating cancer, no          commonly earmarked for projects
     Some consider gene therapy contro-             one should assume that’s the only             like bio-defense.
     versial, as it involves such powerful          approach to solving the problem.”                  Second, while NIH grant funding
     technology and has had some high-                   Eric Rothfeld, an ACGT donor             has been flat in the past two years, the
     profile setbacks. According to the             and board member, appreciates how             cost of research is increasing. “At one
     NIH, gene therapy research current-            the foundation incorporates these             time,” Woo says, “the NIH funded
     ly focuses on correcting genetic               accountability measures into its opera-       the top 25 percent of grant applica-
     flaws and curing disease. In the               tion. The most donor-friendly aspect of       tions, but in the past several years
     future, though, it could be possible           ACGT, he says, is that 100 percent of         researchers who aren’t in the top 10
     to alter human eggs or sperm,                  foundation funds go to research. Sepa-        percent don’t pass muster, making the
10    Philanthropy • November/December 2006
situation very dire for a lot of the big
investigators with very exciting and
potentially useful drug development
projects,” Woo says.
     Third, the NIH budget doubled
during the Clinton years to focus on
biomedical research. “This built up a
base of outstanding young investiga-
tors who are now competing for
fewer research grants,” Woo says.
“Young researchers who fail to find
funding often lose their jobs and
then find other employment. We’re
killing off our young.”
     Stephen Solender, a consultant
and donor to ACGT, says the fund-
ing problem became painfully clear
at a recent conference where he met
a national leader in gene therapy
research who spends about 60 per-
cent of his time writing grant pro-
posals to keep his research going.
     Iowa’s Griffith launched his pro-
ject in 2002 with a $500,000 Young
Investigators grant. It was a huge
sum for him, and he used it to com-
plete studies that led to FDA
approval of his clinical trial. “You
can’t imagine how beneficial some-                Edward and Barbara Netter, co-founders of Alliance for Cancer Gene Therapy
thing like this was at that particular
time,” Griffith says.                      after Phase I, a pharmaceutical com-           Meanwhile, the Alliance’s growing
     Now, Griffith feels the excite-       pany will take interest in the research        expertise in cancer treatment and its
ment of moving from the laboratory         and fund it to completion.                     network of contacts in the medical
to the clinic, and seeing a potential           John Adler, one of the original           field has made it an invaluable
breakthrough for cancer patients.          ACGT board members and donors,                 resource to friends and family mem-
He’s realizing a dream that may            believes it’s important to focus on            bers stricken with cancer.
never happen for some researchers.         research that’s closer to the clinical trial        The personal knowledge of so
     “ACGT’s long-term goal is to          phases. He adds that funding research          many people with cancer lends
develop cures for cancer,” says Woo.                                                      urgency to the Netters’ cause.
But the immediate objective is to                                                         While a cure for cancer is out of
prolong the lives of patients without             ACGT combines                           the immediate picture, Edward
pain and suffering and to develop                                                         Netter believes that even extending
gene drugs that are safe for patients        sound science with solid                     someone’s life by months and turn-
and deadly for cancer.                                                                    ing cancer into a manageable dis-
     ACGT grantees have already               business principles and                     ease are important victories. “It’s
spent years in the laboratory and                                                         important to uncover treatments to
have demonstrated success. They’re                  accountability.                       better people’s lives immediately,”
ready to test their techniques on peo-                                                    says Netter.
ple, which increases both the stakes                                                           His wife agrees. “It’s not soon
and the financial costs. The earliest      at this stage means it may be only five        enough if you’re suffering from
phase of clinical research is designed     years—instead of 15—until break-               this devastating disease. It can’t
to demonstrate a procedure that is         throughs turn into life-saving drugs.          be soon enough.”                   P
safe for human patients. The cost for           Barbara and Edward Netter are
such a project? $3 million to $5 mil-      expanding their pool of donors and             Marshall Allen is a journalist in Las Vegas,
lion, says Woo. The hope is that           plan to increase ACGT’s growth.                Nevada.

                                                                                                Philanthropy • November/December 2006    11

								
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