Fight for Untested Treatment Spawns Debate

May 25, 2009— -- Few stories of desperation could match that of a parent fighting a bureaucracy to help their dying loved one.

In the last couple years, some families have become real professionals at getting the latest untested treatment that holds promise, triggering a debate that pits medical ethics against a mother's love for a suffering child.

When Penny Lopez, 25,learned that two of her three young children had Sanfilippo Syndrome -- a rare genetic disease that would lead to mental decline and certain death before adulthood, doctors told Lopez that "there is no treatment, no cure for it."

"They gave us no hope," said Lopez, of Lakewood, Wash.

But Lopez refused to listen. First she found a doctor at Duke University who was developing stem cell treatments.

Her 5-year-old daughter Hannah could not be helped, but Duke researchers were hopeful that the treatment could slow the disease in her 1-year-old son, Aiden.

Although her military health care, TriWest HeathCare, twice denied the $700,000 treatment plan, Lopez did not give up. She called up local news stations with her story and got a congressman to write a letter of appeal.

Finally, TRICARE agreed to use a part of the contract allotted for rare diseases to approve the stem cell transplant. The Lopez family and TRICARE were willing to risk the 30 percent chance that Aiden would not make it though the entire treatment.

Lopez might seem alone in her work, but several families this year have used savvy public relations campaigns, appeals from powerful people and old fashioned negotiation to get expensive, untested treatments for their dying loved ones despite debates from doctors and regulators about whether this trend is a good idea.

Last week, the Thompson family of Virginia Beach gained national attention in The New York Times for their fight to win access to a $100,000 a year drug called Iplex for Lou Gehrig's disease.

Chris Hempel of Reno, Nev., waged a two-year, full-time public relations, networking and research campaign at finding a new drug for her 5-year-old twins Addison and Cassidy.

The Hempel twins suffer from a rare, degenerative and genetic disorder called Niemann-Pick Type C. Like Lopez, Hempel was faced with few options to treat her children. So, like Lopez, she looked up the latest research and found a treatment that was neither covered by her insurance nor tested by the U.S. Food and Drug administration.

After appearances on national television shows, and several written appeals to the government, Hempel won a compassionate use designation from the FDA to try infusions of a compound used in cholesterol free foods called cyclodextrin.

"In our disease we don't have the time to wait for a new drug. We have to find things that are sitting on a shelf," Hempel told ABCNews.com in April.

For people like Lopez and Hempel, there is no question about the importance of fighting for new treatments that might help their children.

But this same trend stirs debate among doctors and drug regulation experts whether an ethical choice for one family may actually do more good or harm for others in the future.