Date:
04/07/2008 12:47 PM HEALTHBEAT: Deadly skin cancer illustrates struggle
to research rare diseases
By LAURAN NEERGAARD
AP Medical Writer
WASHINGTON (AP) _ The deadliest form of skin
cancer is a little-studied type you've probably never heard
of. After he died, Martin Whatley's classic rock 'n' roll
guitar became his last weapon against it.
Whatley's frustrated widow carefully pulled
the pristine 1964 Fender Stratocaster from under their bed
and put it up for auction on eBay, pledging half the proceeds
for Merkel cell carcinoma research and the rest to pay his
medical bills. She knew scientists were having little luck
finding money to study a killer so rare that few doctors recognize
it, much less know how to treat it.
"It's one thing to give a donation. But
I was thinking if I did it on eBay, so many people saw eBay,
maybe it would make some people out there think about bumps
that they had," and seek help sooner, says Dana Martin
Whatley of Austin, Texas. (Yes, husband and wife shared the
name Martin).
She wound up donating $15,000 last year to
the University of Washington — not where her husband
was treated, but where Merkel cell authority Dr. Paul Nghiem
is struggling to collect samples of patients' tumors to unravel
genes that fuel the cancer.
Merkel cell carcinoma provides a poignant
glimpse of the desperate intersection of research dollars
and the suffering wrought by rare diseases.
It's a cancer only recognized as distinct
from melanoma — even more aggressive, and needing different
treatment — about 15 years ago. Diagnoses have tripled
to about 1,500 a year.
Here's the rub: The first sign is a painless
bump, often reddish to purple, that can resemble a benign
cyst, confusing even dermatologists about whether a biopsy's
needed. But Merkel cell carcinoma spreads rapidly once that
bump appears. A third of patients die in three years, double
melanoma's mortality.
Yet there have been no well-controlled studies
of the best treatment. Intense radiation is key — unlike
melanoma, Merkel cell carcinoma seems unusually radiation-sensitive,
says Nghiem (pronounced Nee-ahm). There is no evidence that
adding chemotherapy right away helps, and it may even worsen
patients' chances, he adds.
Only last month was the first comprehensive
report on symptoms and risk factors published. It almost exclusively
hits Caucasians, over age 50, on sun-exposed skin. And while
a weak immune system greatly increases risk, most patients,
puzzlingly, have normal immunity.
Then in January, University of Pittsburgh
scientists announced they'd discovered a previously unknown
virus lurking inside Merkel cell tumors, a virus that just
may be the cancer's trigger.
"If we'd had funding, we could have done
this easily five years ago," says lead researcher Dr.
Patrick Moore.
Despite earlier success discovering another
cancer virus, Moore couldn't win government money for the
Merkel cell research and spent years cobbling together dollars
to do it. The next step, also so far unfunded, is to create
a blood test for this "Merkel cell polyomavirus,"
to see how common it is and why it only rarely harms.
Indeed, for two decades, Merkel cell research
has depended on small pilot grants and family fundraising
like Whatley's, says Nghiem. Last week, he won what is believed
the disease's biggest research grant to date, $840,000 from
the American Cancer Society.
Rare diseases always struggle against more
common killers for funding.
"There's no money invested in finding
a cure because there's no profit margin, there's so few patients,"
says George Campbell of Charleston, S.C., who founded a Google-based
support group for Merkel cell patients after his 2005 diagnosis.
But belt-tightening today cuts across all
diseases. The National Institutes of Health's budget has been
essentially flat since 2003, which a recent report found equal
to a 13 percent drop if medical inflation is counted.
One consequence: Last year, the number of
clinical trials run by NIH's premier cancer network dropped
enough that 3,000 fewer patients were enrolled, says Dr. Allen
Lichter of the American Society for Clinical Oncology.
Meanwhile, a small fraternity of Merkel cell
patients has banded together to push education, of doctors
and the newly diagnosed — both to help them navigate
the frightening treatment maze, and to urge them to donate
tumor-tissue samples to Nghiem's lab.
"The time it takes, the research, there's
just not an awareness of this at all," says Keith Boyer,
72, of Burlington, Iowa, whose Merkel cell hasn't returned
since radiation two years ago.
———
EDITOR's NOTE — Lauran Neergaard covers
health and medical issues for The Associated Press in Washington.
On the Net:
University of Washington: http://www.merkelcell.org
Patient support group: http://groups.google.com/group/merkelcell
