First Do No Harm: FDA Regulations Limit Treatment Options of Terminally Ill

By National Center for Public Policy Research | July 7, 2008 | 8:21pm EDT

(Editor's Note: The following is the 37th of 100 stories regarding government regulation from the book Shattered Dreams, written by the National Center for Public Policy Research. will publish an additional story each day.)

In 1998, 2-year-old Alexander Horwin was diagnosed with medullablastoma, the most common form of pediatric brain cancer and one that nearly always is fatal in children.

After two surgeries, Alexander was tumor-free, but doctors warned that treatment was necessary.

As Alexander's father, Michael Horwin, explained June 7, 2002, to a hearing in the U.S. House of Representatives: "We were warned that without further treatment, this cancer always returns."

The Horwins investigated the treatment options.

"We conducted around-the-clock research to find the cancer treatment that offered Alexander the best chance to survive. After scrutinizing therapies and speaking to parents and patients from throughout the world, we selected the Burzynski Clinic in Houston, Texas." Dr. Burzynski has a 20-year track record of curing or controlling the re-growth of malignant brain tumors in children and adults with an innovative cancer therapy. In addition, his therapy is non-toxic and offers a good quality of life.

The Horwins flew to Houston and met with Dr. Burzynski, who told him that the U.S. Food and Drug Administration would not permit him to treat Alexander because Alexander - at that moment - had no tumor. The U.S. government had tied Burzynski's hands.

Finding no other options, the Horwins accepted the recommendation of doctors at the Los Angeles Childrens Hospital for a chemotherapy treatment called CCG-9921. Possible side-effects included damage to the heart, lungs, liver and kidneys, possible loss of hearing, small stature, infertility, another cancer and intellectual decline - maybe even death.

Alexander began the treatment. Michael Horwin told Congress what happened next: "After the first round of chemo, Alexander began to change. Even after two brain operations, Alexander was still a vibrant, ruddy, strong, energetic child. But as the chemotherapy repeatedly filled his small body, Alexander began to die inside. First, the relentless stomach pains and the horrendous projectile vomiting began. Then, his beautiful curly hair fell out. Next, his dark skin tone turned pale as a ghost. He got sick with fevers and spent weeks in the hospital. Then, there were the blood transfusions to replace the blood cells the chemo had killed, the hearing tests to see if the chemo drug cisplatin had not devastated too much of his hearing, the nuclear medicine tests to check if his kidneys were not giving up under the strain of processing so much poison, the liver function tests to ensure that his liver was not being destroyed, etc.

"During chemotherapy, we had to squeeze an antibiotic into his nose called nystatin several times a day. He hated it and buried his face in a pillow when he saw it coming with all the strength his little body could muster. One of us had to pin Alexander down and keep his head immobile while the other pushed the syringe into each nostril and injected the solution. We were also called upon to give him GCSF injections at home. These injections into his legs were designed to raise his blood cell counts. It was horrific. We felt as if we were actively engaged in the slow but sure torture and destruction of our own child."

The Horwins began to consider stopping the chemo. But they learned that if they did so, they could lose custody of Alexander.

"What do we do? We did not have a choice of therapies. The FDA had taken away our first choice of treatment at the Burzynski Clinic. The oncologists warned us that if we didn't use chemotherapy that the tumor would probably return in three months. These doctors assured us that the chemo they were administering to our son was the current "state of the art." They told us repeatedly that this was Alexander's best choice for a long and healthy life. We continued the chemotherapy. As a result of the drugs, Alexander's balance was lost, his ability to see deteriorated, and he lost hearing in one ear. The whole thing was horrendous."

The Horwins had never stopped looking for alternatives. Then, they found one: a clinic in Switzerland that had a good record of accomplishment treating this type of cancer without chemo. But on January 18, 1999, doctors discovered "leptomeningeal sarcoma" - 30 tumors throughout Alexander's small body. The FDA-approved treatment of Alexander's cancer had failed.

The doctors prescribed painkillers and said Alexander had but a few days to live. Now near death, Alexander was finally legally eligible for Dr. Burzynski's therapy. His parents chartered an air ambulance and took him to Houston. The clinic staff worked hard, but it was too late. On January 31, 1999, Alexander died. The horror was not over. After Alexander's death, the Horwins began investigating leptomeningeal sarcoma. They learned that CCG-9921 is at best an unproven treatment for children with Alexander's form of cancer and that doctors have known this for decades.

The Horwins had no options. They weren't allowed to try an alternative treatment. And even though doctors following the conventional treatment couldn't save Alexander and the treatment caused immense suffering, they weren't allowed to turn it down. Michael Horwin sums it up this way: "We were told that time was running out. We were told that chemo would offer Alexander a good chance of survival. We were told that he would be getting a new chemo protocol with 'state-of-the-art' drugs. And we learned later that if we did not bring Alexander in for chemotherapy, a court order was contemplated so that the oncologists could take him from us and administer these poisons without our consent.

"Alexander spent his last months alive not on the beach that he loved so much, but in a hospital room tied to an IV pole, throwing up and in pain from being poisoned. This never should have happened.

"There is no standard of care for this disease in young children. Chemo does not work, and oncologists know it. They have admitted this in their medical journals. But we were told that this was a 'state-of-the-art' therapy, not a 20-year toxic failure. This is not informed consent.

"My wife and I have to live with the knowledge that we never gave our son a fighting chance to survive his disease. He died five months after his diagnosis, three months after starting chemo, just like the other children that had come before him. The ones that had become statistics in the oncologist's articles. And the numbers of dead children continue to mount as these ineffective drugs continue to be used in cancer hospitals throughout the country.

"When orthodox therapy has nothing to offer, parents must be allowed to exercise their parental, ethical, moral and legal right and responsibility to protect their child and fight for his life when he has a terminal disease. The FDA has no business sentencing young children to death by taking away a therapy option that could save their life."

The FDA still strictly regulates access to alternative medical treatments. In 2002, a Competitive Enterprise Institute survey of oncologists found that 70 percent believe the public doesn't understand that the FDA approval process has a high "human cost" measurable in suffering and death. Sixty-eight percent believed unapproved drugs and medical devices should be "made available to physicians as long as they carried a warning about their unapproved status."

On October 15, 2002, Wired magazine reported that oncologists are recruiting more children with medullablastoma to further test CCG-9921.

Alexander's father, once a health-care administrator, has become a lawyer. He now lobbies to give the public greater control over medical treatments.

In 2001, Rep. Peter DeFazio (D-Ore.) and Senator Tom Daschle (D-S.D.), each with bipartisan cosponsors, introduced the Access to Medical Treatment Act into the 107th Congress to give Americans greater access to alternative treatments under specified guidelines. During the 107th Congress, neither the House nor Senate voted on the legislation.

Sources: Testimony of Raphaele and Michael Horwin before the U. S. House of Representatives Committee on Government Reform, H.R. 1964 and S. 1378 ("The Access to Medical Treatment Act of 2001"), Wired News, Competitive Enterprise Institute

Copyright 2003, National Center for Public Policy Research

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