Dr. Douglas Kerr painstakingly collected spinal fluid from hundreds of patients with a mysterious disease that can paralyze within hours of attacking — and thinks he may have found a way to fight back.
Transverse myelitis, which is inflammation of the spinal cord, is rare. But, importantly, it may share common triggers and treatment approaches with a dozen illnesses, such as Guillain-Barre, myasthenia gravis, multiple sclerosis, that all attack the nerves, spinal cord or brain.
So Kerr is bringing international specialists together in Baltimore this week to pool their resources in the fight against these diseases. And furiously taking notes will be dozens of patients desperate to learn the latest research, an unusual meeting largely due to the relentless campaigning of a teenager.
"This is a really big dream of mine," says Cody Unser, who is paralyzed from the chest down by transverse myelitis. "All the doctors, now they're sharing data and talking, and that never was before." She is the daughter of race car driver Al Unser Jr.
Seeing patients in the audience, the Albuquerque, N.M., 17-year-old believes, is an important motivator for researchers more used to a lab: "There's this understanding of what science means to a person."
Neuroimmunologic diseases can have very different symptoms: Some kill or paralyze; others weaken or blind. Some patients recover, although there's no good way to predict. But the underlying mystery is why the immune system suddenly malfunctions and attacks the central nervous system.
Now research is pointing to a host of potential culprits these illnesses may share, disease triggers and damage-spurring abnormalities that in turn suggest new treatment strategies.
"We think we're on the verge of better, more targeted therapies for the immune system gone amok," says Kerr, who heads the transverse myelitis center at Johns Hopkins University and is coordinating the neuroimmunology meeting, which begins Thursday.
On the agenda:
- Scientists recently discovered that an anemia drug called erythropoietin, or EPO, seems to protect central nervous system cells from inflammatory damage. A Hopkins researcher, Sanjay Keswani, is beginning a study of whether EPO can protect patients' spinal cords from transverse myelitis' inflammatory assault.
- Kerr's own study of spinal fluid found that transverse myelitis patients harbor the chemical interleukin-6 at "levels never before seen in human disease." At high levels, the normally protective chemical turns deadly, triggering a cascade of cellular aberrations that ultimately chews up spinal cord cells, he explains.
Thalidomide, the notorious birth defect-causing drug now used to treat leprosy, can block IL-6, Kerr says. If upcoming animal tests pan out, he hopes to begin studies in transverse myelitis patients early next year.
- "Molecular mimics" may explain why these diseases sometimes strike after simple bacterial or viral illnesses. For example, Guillain-Barre syndrome sometimes strikes after the diarrhea-causing food-poisoning bug campylobacter. It turns out that a certain campylobacter strain's molecular shape looks, to the immune system's attacking antibodies, just like certain nerve cells.
- Then there are chemokines, proteins that guide infection-fighting white blood cells to their target. But chemokines act in the development and functioning of the nervous system, too, in ways that can either protect or further harm, says Dr. Richard Ransohoff of the Cleveland Clinic. Early-stage studies of chemokine-blocking drugs are under way for multiple sclerosis.
- For restoring function, scientists also are trying to manipulate the body's master stem cells to fix damaged spinal cords. But it will require years more work.
This is pretty in-depth science for laymen. But when transverse myelitis struck Cody Unser in 1999, she and her mother, Shelley, discovered the difficulty in getting information about this rare disease, which afflicts 34,000 Americans.
They started a research-advocacy foundation that pushed for patient-scientist meetings, and Kerr urged including related diseases. "We could become much more powerful in studying this together than individually," he explains.
Hearing the science, patients "feel kind of intimidated at first," Cody acknowledges. But the opportunity to question specialists is invaluable, and learning about even early-stage research "makes me feel there is hope."