March 2000
Chelation

The McGourthy family of Middleboro, Mass., started chelation (pronounced KEY-lay-shun) therapy for son Michael in November, 1999, as a treatment for HSS. It is too soon to judge whether the treatments are helping, the family said, nor is there consensus on whether the therapy is a good one for HSS patients. But the McGourthys thought it was worth a try to reduce the iron in Michael’s brain—a key feature of HSS.

Chelation therapy involves administering drugs intravenously to reduce the metal or minerals that have built up and are causing damage in the body. Some of those metals and minerals are lead, iron, calcium, copper and aluminum. Drugs, called chelators, act like sponges sopping up the over-abundant metals or minerals and making them water soluble. That way, they can be excreted by the kidneys in the urine. For HSS patients, chelation is seen as a way to soak up excess iron in the brain.

Chelation therapy is not new. The British used it during World War II to treat victims of poison gas exposure. Today, chelation therapy remains the undisputed treatment-of-choice for acute lead poisoning, even in children. It has been used as an alternative to heart bypass surgery for patients with atherosclerosis, a condition in which fatty substances build up in the inner walls of the arteries and clog them.

Chelation also is used to treat patients who have accumulated too much iron in their bodies, either from frequent blood transfusions or other causes.

But a problem with using chelation for HSS is that the drug must cross the blood-brain barrier to make it beneficial. The blood-brain barrier is like an efficient filter that prevents some drugs and other substances in the blood from having free access to the all-important brain. Many drugs can’t get across the barrier.

In a March, 1997, paper published in the Annals of Neurology, researchers found an example in which chelation therapy did cross the blood-brain barrier and decreased iron in the brain. The patient in that case had aceruloplasminemia, a genetic disorder of iron metabolism. Doctors used the drug desferrioxamine, which is another name for desferal, the same drug the McGourthys are using. Patients who have aceruloplasminemia can experience diabetes, retinal degeneration and neurologic symptoms. The pathologic findings of iron accumulation in the brain are similar to those observed in HSS. They also exhibit such clinical symptoms as dystonia and dysarthria - similar to HSS patients.

Dr. Jonathan Gitlin of St. Louis Children’s Hospital, one of the authors of the 1997 paper, says he is involved in clinical trials on a larger group of aceruloplasminemia patients in Japan and hopes to publish results in a year. He indicated, anecdotally, that other patients appear to have responded well to chelation therapy as a way to remove iron from the brain.

Even so, iron chelation can pose serious problems for HSS patients, according to Dr. Susan Hayflick of the Oregon Health & Science University. Because the iron overload is only in the brain for HSS, differing from aceruloplasminemia in that respect, the therapy could cause an iron deficiency in other parts of the body. When iron chelation was tried on HSS individuals in the past, there was no documented evidence that the treatment worked, and iron deficiencies outside of the brain were evident, Hayflick said.

UPDATE: December 2000

As some of you know, we are trying chelation therapy on our son, Michael, who has HSS. It has been a year now, and Michael is doing well with the treatment. We feel the progression of the disease has slowed, and our doctor at the New England Medical Center in Boston is interested in testing chelation on other HSS patients.

For a chelation trial to be possible, we need to know who else might be interested in taking part. Please contact us directly and let us know. In no way would you be committing to the trial at that point. The trial would be conducted by Dr. Lawrence Wolfe at the New England Medical Center in Boston. We are willing to raise money to help finance the project.

For information, contact us by e-mail at Rural9@aol.com, or by mail at The McGourthys, 286 Wareham Street, Middleboro, MA, 02346.