SpecialKids

Cancer Survival Rates Soar; Research Intensifies

by Emily Lambert

Survival rates for childhood cancer have increased in the last 50 years from less than 10 percent to almost 80 percent, according to a leading research organization, CureSearch Children’s Oncology Group. Clinical trials in place today are bringing additional hope to children with cancer and their families.

Alex’s Lemonade Funds Research, 1 Cup at a Time

Alex’s Lemonade Stand has become a significant source of pediatric cancer research funding. Its founder, Alex Scott of Wynnewood, PA, started selling lemonade at age 4 to raise money for her hospital “because all kids want their tumors to go away.”

By the time she died at the age 8 from neuroblastoma, Alex’s cause had spread throughout the country.

To date, Alex’s Lemonade Stand has raised more than $5 million. The charity gives pediatric cancer research grants to children’s hospitals across the U.S., “so that sicker kids will get new and better treatments faster,” says Elizabeth Scott, Alex’s mother and the fund’s development director.

“The most important thing for people to know is that speed and progress are directly related to the amount of funding for cancer research,” she says.

Raising money for the fund by hosting a lemonade stand provides an opportunity to involve your children; they can paint stand banners, make lemonade and even collect donations.

You can hold an Alex’s lemonade stand any day of the year, but most participating families will set up stands during Alex’s annual Lemonade Days, June 9-11.

If you register with Alex’s by mid-May, the fund will send support materials to your home. 610-649-3034, www.alexslemonade.com

These research studies test new treatments. Each trial proceeds throughthree phases: Phase One tests for safety, such as what dosage is best. Phase Two tests for effectiveness. Phase Three tests for safety, effectiveness and superiority to treatments already in place. Trials can take from one to five years.

Promising trials are underway for leukemia, the most common form of pediatric cancer, as well as for neuroblastoma and retinoblastoma.

Leukemia (Lymphoblastic)
The most common childhood cancer is acute lymphoblastic leukemia (ALL), a cancer of the lymphoid cells in the bone marrow and the lymphoid organs of the body. The cure rate for ALL was four percent in 1962. Today, William Evans, PharmD, director and CEO for St. Jude Children’s Research Hospital in Memphis, TN, says, “We’re pushing cure rates, optimally, to 90 percent for ALL in the next 3-5 years.”

Subdividing this disease by genetic differences and other attributes has allowed oncologists to individualize a child’s therapy. “This has played a huge role in increasing the cure rate. We’re using the same drugs we’ve had for 20 years, just in a different fashion,” says Dr. Evans.

At The Children’s Hospital of Philadelphia (CHOP), Stephan Grupp, MD, PhD, director of stem cell biology, studies how a specific targeted therapy, temsirolimus, works against ALL.

Targeted therapies, often referred to as “smart drugs,” target and kill cancer cells, but keep healthy ones intact. “The current model of chemotherapy worked well for 40 years but it is toxic and we are trying to get away from that,” says Dr. Grupp.

He is completing Phase One testing of temsirolimus, a drug made by Wyeth that attacks cancer cells by turning off growth signals in them.
Beverly J. Lange, MD, medical director of Pediatric Oncology at CHOP, says a pending study of infant acute lymphoblastic leukemia could produce “a major breakthrough.” The study uses CEP-701, a chemical, to target a molecule on the leukemia cell surface.

Leukemia (Acute Myeloid)
Acute Myeloid Leukemia (AML) is a cancer of the myeloid blood cells, which help combat bacterial infections. A child diagnosed with AML 50 years ago had a life expectancy of one month and little chance for survival.

Today, about half of children with AML survive. Mylotarg, a targeted therapy drug, is slated for a national Phase Three trial with 1,000 patients. “When used in patients with a relapse, we could get remission. We are now adding it to our standard therapy,” says Dr. Lange.

St. Jude’s is conducting a pilot AML study using cellular therapy. So far, the approach seems “very safe, there are few side effects and it may, theoretically, be effective for all types of AML,” says Jeffrey E. Rubnitz, MD, PhD, director at St. Jude’s.

In acute promyelocytic leukemias (a subtype of AML), a form of vitamin A has replaced some chemotherapy. In AML, leukemic cells have a mutation that suppresses the normal maturation of white blood cells.

“If they do not mature, the white blood cells continue to multiply indefinitely. (The vitamin) counteracts the effect of the mutation and allows the cells to mature and eventually die,” says Dr. Lange.

Neuroblastoma
At CHOP, John M. Maris, MD, studies neuroblastoma, the most common cancer in infants. “What we’re trying to do is develop a standardized approach to children who have relapsed cancer,” says Dr. Maris.

MIBG, a compound that selectively focuses on neuroblastoma cells, is in Phase Two of clinical trials. “MIBG is not curative but it results in long remissions that improve the quality of life,” says Dr. Maris.

“This medicine, usually pretty quickly, gets children out of pain. It can stay that way for weeks or months. The key is to figure out how to make that longer,” he says.

Retinoblastoma
A cancer of the retina, the innermost layer of the eye, retinoblastoma is most common in children under five.

“We’ve known for 50 years we could save children’s lives 95 percent of the time, but for the 25 percent of children with tumors in both eyes, we haven’t been able to save their vision with chemotherapy as well until the last ten years,” says Anna Meadows, MD, who directs a series of national studies at CHOP.

This treatment first shrinks the tumors with chemotherapy, then uses heat and cold to destroy the blood vessels that feed the tumors. If the tumors are small, eyesight can be saved in 90 percent of the cases. If the tumors are big and can be shrunk, sight can still be saved in 50-70 percent of cases. Use of this process is spreading throughout the U.S., Australia and England.

As for the future, CureSearch believes what once seemed an impossible dream, that of curing childhood cancer, is now an achievable goal in our lifetime.

Emily Lambert is local freelance writer.