Marlo Thomas carries on her father's legacy of fighting pediatric cancer.

By John Morgan, Spotlight Health
With medical adviser Stephen A. Shoop, M.D.

Marlo Thomas gained national fame in the late 60's playing the single and vivacious Ann Marie on the hit show That Girl. Now she is helping bring her famous father's legacy, St. Jude Children's Research Hospital, to even greater star status in the world of pediatric cancer.

"St. Jude was my father's greatest gift to children and families desperate for hope," says Thomas, the National Outreach Director for St. Jude. "St. Jude is the patron saint of hopeless causes. Many parents come to us having been given a death sentence for their child. But our doctors have at excelled at fulfilling our promise of hope -- so many of these parents ultimately end up saying, 'You saved my child.'"

And the 'other miracle' is St. Jude does it free of charge.

That's because the Danny Thomas legacy assures that St. Jude will never turn a sick child away because of money.

"That's a promise we have kept for four decades," says the seemingly ageless Thomas.  "And this Saturday April 26 we are celebrating our 40^th anniversary and this tremendous achievement because we need to do even more now since so many families don't have health insurance."

Not only does St. Jude pay for treatment, which can cost from $250,000 to $600,000 depending on the illness, they also pay for children and their families to travel to their Memphis-based medical center as well as all accommodations and meals.

Celebrating life

"This anniversary weekend we really wanted to have people come down and see the hospital and what we do," Thomas explains. "This is an amazing place and every time I visit I am reinvigorated because we've been practicing 21^st century medicine for 30 years."

To pay for St. Jude's cutting edge research and treatments, St. Jude's fundraising team needs to fill their medical war chest with over $300 million annually because St. Jude operates at a daily cost of nearly one million dollars a day.

Fortunately, St. Jude's substantial investment has already created enormous breakthroughs in the field of pediatric cancers and other catastrophic diseases, like HIV.

According to the American Cancer Society, cancer remains the chief cause of mortality for American children aged 1 through 15, even though death rates have declined 50% since 1973. The ACS estimates there will be 9,100 new pediatric cancer cases this year. Of those, approximately 1,400 children will die, and one-third of those from leukemia.

But not if Thomas and St. Jude get their way.

"In 1962 when my father first opened the hospital, only 4% of kids with acute lymphoblastic leukemia (ALL) survived," Thomas says. "And now because of St. Jude, over 80% survive today."

The many St. Jude treatment success stories include:

• A 90% survival rate for Hodgkin's disease (only 50% in 1962)
• A 90% survival rate for retinoblastoma (75% in 1962)
• An 80% survival rate for non-Hodgkin's lymphoma (only 7% in 1962)
• A 59% survival rate for neuroblastoma (only 10% in 1962)

"While these are amazing numbers, in the case of ALL, which is the most common childhood leukemia, it still means 20% of the children are still dying. And that's not good enough. So we are working very hard to help the 20% we haven't been able to cure."

And with a new field of medicine called pharmacogenomics, St. Jude is poised to cut into that frustrating 20% of cases that don't respond to treatment.

Roadmap to success

Pharmacogenomics studies how populations of specific genes influence a person's response to drug therapy. Using a technique called gene expression profiling, scientists can study which of the genes responsible for activating or breaking down drugs are active in a patient before the treatment is undertaken.

And that's a critical breakthrough because time is of the essence with cancer.

"If you have high blood pressure, we can use trial and error approach and probably be fine whether it takes a month to six months to find the right anti-hypertensive drug for you," says William E. Evans, scientific director of St. Jude.  "But if you have cancer you don't want to wait six months and moreover cancer drugs can be very toxic and potentially life-threatening.  So we'd like to avoid toxicity if at all possible."

According to Evans, a child's blood is analyzed and a 'genetic map' is created for the particular gene being focused on in order to determine whether a particular drug is compatible with a patient's genetics.

"Some people have very fast systems that eliminate drugs very quickly," Evans explains. "Other people may inherit a deficiency in their ability to get rid of a drug because they don't make the protein needed for that pathway and therefore can't metabolize the drug. So if these people get the standard dose of the drug, they will get very high blood levels of the drug and possibly develop toxicity."

For instance, with one of the anti-leukemic agents if a patient has inherited a bad copy of this gene from each parent and can't make the enzyme needed to get rid of the drug at the normal rate, he or she should be treated with only 5 or 10 percent of the normal dose, which produces the same blood levels of the drug as the people getting a full dose.

Better medicine

"Because the empirical approach in the clinic is if you're giving three leukemia drugs to a child and they're having toxicity, you reduce the dose for all three by 25% and try again and again if needed," Evans explains. "This paradigm has two things wrong with it. One is you're not reducing the dose of the poorly tolerated drug nearly enough - this drug should be reduced 90%.  The second mistake is you're reducing the other two drugs and you shouldn't be doing that at all."

Genetics can also be used in advance of treatment to determine that a child needs more aggressive therapy, like a bone marrow transplant, because they fall into the non-responsive group.

"This can spare children and families the heartbreak of spending 2 ½ years on something that isn't going to work for them and spare the patient unnecessary and debilitating side effects," Thomas says.

Evans believes that using pharmacogenomics, St. Jude's team of doctors and researchers will chip away at the stubborn 20% of cases where treatment fails.

And so does Thomas - with her father's same passion.

"My father was a visionary," Thomas says. "He insisted that everything be kept under one roof - the children, their families, the doctors and nurses all eat in the same dining room. When a doctor has lunch with a little girl and then goes back to his lab, he's no longer working on disease X. He's now working on 'Amy's' disease."

"So many of our doctors have told me this is the most motivating force in the world for their work," Thomas adds. "We call it the St. Jude fever."

Let's hope there's no cure.

• St. Jude Children's Research Hospital

Spotlight Health is the leading creator of celebrity-featured health-issue awareness campaigns, connecting consumers with impassioned celebrities whose personal health battles can open eyes, dispel myths and change lives. Spotlight Health helps sufferers and caregivers meet the challenges of difficult health circumstances with understandable, in-depth medical information, compassionate support and the inspiration needed to make informed healthcare choices.

Created: 5/10/2003  -  John Morgan & Stephen A. Shoop, M.D.