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Clinical Research: Innovation Drives Pediatric Care At Cook Children's

System emphasizes evidence-based research

From a best-selling book to a movie starring Harrison Ford to President Donald Trump’s first address to a joint session of Congress, Megan Crowley’s life has made for a compelling story.

Megan and her brother Patrick battle Pompe disease, a rare neuromuscular disorder. Her father has spent nearly two decades raising money to fund research for drugs to fight Pompe. A story told in the movie, “Extraordinary Measures.”

The use of an experimental drug isn’t an uncommon practice at most major hospitals, including at Cook Children’s, including for Pompe disease.

For more than a decade, Cook Children’s has been involved with research for Pompe disease. Testing and treatment are available at the medical center. Treatment involves frequent intravenous infusions of enzyme replacement (Lumizyme). Enzyme replacement can slow the progression of the disease, as it has in many Cook Children’s patients.

Alglucosidase alfa was an approved drug indicated for use in patients with Pompe disease and marketed in the United States as Lumizyme and as Myozyme. Myozyme has been shown to improve ventilator-free survival in patients with infantile-onset Pompe disease as compared to an untreated historical control.

The drugs used for treatment for Pompe, like most of experimental medications under research at Cook Children’s, were pursued because our physicians wanted the capability to treat their patients in the best way possible. Most of the time, they weren’t opening a trial and then looking for patients to participate. They sought out the medication that would make a difference for their patients.

What is an experimental drug?

An experimental drug is a substance which may be useful to diagnose or treat disease or preserve or enhance health, but has not yet been approved by the U.S. Food and Drug Administration for the specific purpose of treating a patient.

“The drug might or might not be approved for one or a number of conditions, but it becomes ‘experimental’ when used to systematically investigate treatment for an unapproved condition,” said James Marshall, M.D., Chief Research Officer at Cook Children’s.

How Experimental Drugs Are Used



The importantance of 'informed consent'

The data obtained from the use of experimental drugs can be useful to future patients as doctors can see how beneficial they are the next time they are treating a patient with a similar condition.

At Cook Children’s, children participate voluntarily in all experimental drug investigations. Patients and their families, particularly in the case of minor children, must volunteer to participate in any experimental investigation through a process called informed consent.

Informed consent for children is regulated heavily by the U.S. and international governments. This type of consent is managed by a local board concerned only for the safety of research subjects, and far more comprehensive than the normal treatment consent process. Informed consent for children has several key elements:

  • Education by the investigator and his/her team regarding all aspects of the research.
  • Informed consent or actually signing a legal document by a minor child’s parent or legally authorized representative.
  • Assent or documenting that a minor child understands and agrees to participation, and a continuous, open and welcome ongoing conversation between the participant/family and the investigational team to assure understanding through all phases of the research.

The initial consent/assent process can take several hours, and investigators never really consider the conversation to be closed. Patients and their families often learn more about their condition during the consent/assent process than they would by receiving treatment only. Patients and families can decide to stop participation even before the experimental drug is administered.

However, many patient families see the importance of participating in these experimental drugs and the benefit it may bring to not only their child, but to patients in the future.

“At Cook Children’s, we believe that all children deserve the best, evidence-based treatment strategies available to manage their illnesses and maintain good health,” Dr. Marshall said. “Cook Children’s patients have the opportunity to contribute to the health of other children through safe and ethical clinical research. We treasure the fact that clinical research is the essence of people voluntarily caring for the future of mankind.”

Related Article:

Inspiring story sheds light on Pompe disease

More about Clinical Research at Cook Children's

At Cook Children's, we believe that all children deserve the best treatment strategies available to manage their illness and maintain good health. Our research environment applies leading-edge scientific knowledge, technology and evidence-based practice to improve quality care outcomes for children. Click here to learn more.

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