Courtesy of NorthJersey.com
Dr. Burton Appel remembers when he wrote a college letter of recommendation for a patient. Not an uncommon request for most physicians. But this one is particularly noteworthy for Dr. Appel.
When the now college sophomore was three years old, Dr. Appel treated him for leukemia. The boy, Peter Bernhard, 19, of Waldwick, has been in remission for years and is considered cured of the disease.
It is such a pleasure to see him grow and develop into the young man he is,” said Dr. Appel, attending physician in the Division of Pediatric Hematology/Oncology (Children’s Cancer Institute), Joseph M. Sanzari Children’s Hospital at Hackensack University Medical Center, Hackensack.
Bernhard, who sees Dr. Appel once a year for follow ups, said the treatment process was upbeat thanks to Dr. Appel.
“He even helped make it fun. He’d race me down the halls,” Bernhard, who attends Stockton University said. “He’s not just my doctor. He’s also my friend.”
A diagnosis of pediatric leukemia, or other childhood blood disorders, can be devastating news to a family. However, hospitals, such as HackensackUMC, offer advanced methods of treatment in an area of the facility designed specifically for children and their families. Hospitals, including HackensackUMC and Englewood Hospital and Medical Center, in Englewood, also help children deal with the emotional impact of various types of medical testing, exams and treatments.
The research team at HackensackUMC’s Children’s Cancer Institute has access to more than 100 clinical trials. The Joseph M. Sanzari Children’s Hospital provides medical and surgical pediatric care in more than 30 specialties, including blood and marrow transplantation, cardiology and pediatric cancer and blood disorders. Dr. Appel said he helps children from newborn to the young adult age of 22.
Blood Disorders in Children - Progress and Hope
Blood disorders, such as leukemia, starts in the blood forming tissue. The cancer cells overcome and replace normal blood and marrow cells (www.cancercenter.com). Blood carries oxygen, vitamins and other necessities to our tissues. When compromised, patients can experience such symptoms as easy bruising, fatigue, fever, chills and frequent infections.
Leukemia is considered the most common cancer in children and teens, according to the American Cancer Society. Other blood disorders in children include various types of anemia, when patients have fewer healthy red blood cells than normal.
In general, the treatment for acute lymphocytic leukemia (ALL) in children has advanced and changed enormously since the 1960s, Dr. Appel said. Chemotherapy medicines can now cure more than 80 percent of children with ALL, compared to about four percent in the 1960s. About three out of four types of childhood leukemia are acute lymphocytic leukemia, with the remaining being acute myelogenous leukemia (AML).
Basic treatments for leukemia include various types of chemotherapy. One procedure, called photophoresis, can filter a patient's blood to help reduce the side effects of a bone marrow treatment, Dr. Appel said.
The high cure rates can be attributed to the many clinical trials performed nation-wide, Dr. Appel said. New drug combinations are tested on patients, where they can be refined and perfected. Dr. Appel said parents often find it reassuring that treatments have been tested on thousands of children in similar situations.
"That's really how progress is made. They are building on the successes of the past," Dr. Appel said. "Childhood leukemia is a treatable and curable disease."
Keeping Life Normal for Pediatric Patients
Hospital visits can be overwhelming for children, their siblings and their families. Creating an atmosphere that is supportive, while child friendly, is key.
Englewood Hospital and Medical Center, for example, features a Child Life Program. The program helps ease anxiety and stress before medical procedures or hospitalization for children and their families. A certified child life specialist works with children to explain all the procedures he or she may undergo at the hospital.
Courtesy of City Of Hope Hospital
Most people know that the U.S. Food and Drug Administration must approve a drug before it reaches the market or a hospital room. But very few people outside of the medical field know what happens before the drug gets to the FDA.
Clinical trials (or clinical studies) are medical studies that focus on establishing the safety and effectiveness of new, potentially beneficial drugs by testing them on increasingly large groups of human subjects. These trials can involve new medications, treatments, devices and even psychological therapies. It’s a years-long process that involves constant study and research before the new drug is ever presented to the FDA.
At City of Hope, at least 400 clinical trials are going on at any given time. To help illuminate the clinical trial process for patients, here are five facts you may not have known.
1. Clinical trials require approval before commencing.
Clinical trials are only approved once an institutional review board, or independent ethics committee, has weighed the potential benefits against the possible risks and determined the testing worthy. An institutional review board or independent ethics committee pores over all materials related to the study before and during the trials.
Often years of preclinical work has gone into researching and experimenting on isolated living cells, while zeroing in on potential benefits, appropriate dosage ranges, problems and side effects, and the most effective ways of applying the drug, before human subjects can become involved.
2. There are different types of trial subjects.
Often, existing patients with a specific condition or particular set of characteristics, such as age, gender, or stage of disease, will volunteer to participate in a clinical trial. In other instances, trials will make use of volunteers who are otherwise healthy, depending on the type of drug or device being tested.
Patients already under medical care may choose to participate because the trial gives them access to new treatments not available to others. Many volunteers sign on because they want to help advance science and medicine toward new cures. All participants must go through a process of informed consent with the trial’s principal investigators before it starts.
3. Clinical trials are run by special investigators.
Medical professionals who are trained in the “Good Clinical Practice” guidelines designed to safeguard subjects and assure proper collection of data can become investigators on clinical trials. By participating, investigators can position themselves to discover new developments and stay on the front edge of emerging medical discoveries, especially in their fields of expertise.
They can have an impact on advancements, while authoring papers and articles that push their field forward. Investigators are often paid for their work, unlike the subjects of clinical trials.
4. There are four distinct phases of a clinical trial.
Phase 1 focuses on safety by keeping the sample group small, from 20 to 80 people. It’s an initial evaluation of how the human body handles a new drug, while investigators take note of safe dosage ranges and any side effects.
In phase 2, the experiment group grows to 100 to 300 subjects, with more of a focus on effectiveness, though safety is always being evaluated.
During phase 3, investigators increase the sample size to 300 to 3,000 subjects and lengthen the amount of time the drug is taken, while the same safety and efficacy parameters are studied.
Phase 4 requires an even larger sampling group, with a focus on long-term effectiveness and safety, as well as how the new drug compares with other similar drugs and their costs, before approval is sought from the FDA.
5. Only a small percentage of new drugs ever hit the market.
The review process takes anywhere from six to 12 months, depending on whether the new drug is only a minor improvement on an existing therapy, a major advancement or a completely new treatment. Right now, the National Institutes of Health service ClinicalTrials.gov lists more than 215,000 clinical studies currently in progress around the world.
Ultimately, the FDA only approves about 10 percent of the drugs that went through clinical trials in the U.S.