Solutions Magazine, Summer 2006 - Baylor College of Medicine, Houston, Texas

With the birth of her first and only child, Kori Spikes considered her life complete. All that changed 10 months later, however, when she was told her daughter Jada had acute lymphocytic leukemia (ALL).

"I was in denial. Complete shock," recounted Spikes. "It was the worst feeling in my life, something I would never wish on anybody."

Under the counsel of Dr. Zoann Dreyer, associate professor of pediatrics at BCM, Jada was enrolled in a clinical trial at Texas Children's Cancer Center, which is designed to bring cutting-edge cancer treatment to the infant's bedside. She underwent the latest in chemotherapy for six months, followed by another six months of less intensive maintenance therapy.

The fact that Jada had a 50 percent chance of survival—as scary as it sounded—actually represented a quantum leap in predicting pediatric cancer outcomes. As recently as 10 years ago, a child diagnosed with ALL, the most common form of infantile leukemia, had a 20 percent chance of beating the disease. Forty years ago, the survival rate was zero.

"The patients who were treated in clinical trials in the past have really benefited the kids today, because we were able to advance outcomes and cures based on how those children did," said Dreyer.

Dreyer estimates that she is involved in more than 20 different clinical trials at any given moment, many of them nationwide and collaborative. One study might test whether a particular drug is more potent when administered over an entire day versus just a few hours. Another might assess whether the outcome improves if one type of steroid is used over another.

Not Just Another Phase

In Phase I trials, researchers test a experimental drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.

In Phase II trials, the experimental study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.

In Phase III trials, the experimental study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely.

In Phase IV trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.

What are the different types of clinical trials?

Treatment trials test experimental treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.

Prevention trials look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes.

Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular disease or condition.

Screening trials test the best way to detect certain diseases or health conditions.

Quality of Life trials (or Supportive Care trials) explore ways to improve comfort and the quality of life for individuals with a chronic illness.

Source: ClinicalTrials.gov

"Clinical trials allow us to share patients' outcome data anonymously so that, statistically, we can evaluate different arms of treatment," said Dreyer. "Then we can ask and answer research questions to find the best way to treat patients."

Literally thousands of clinical studies of all shapes, sizes, and durations are conducted at Baylor and its affiliated hospitals each year with the common goal of advancing novel therapies. In addition to leukemia, physicians and researchers with diverse medical and scientific backgrounds hunt for cures to virtually every other form of cancer, not to mention diabetes, heart disease, AIDS, autism, liver disease, and asthma, just to name a few.

Earlier this year, BCM joined 32 other organizations in the country to be accredited by the Association for the Accreditation of Human Research Protection Programs Inc. Although not required for conducting clinical trials, the designation illustrates the College's commitment to upholding the highest possible standards in all of its various research endeavors.

Safe Guards

For every clinical trial looking to advance science and medicine, patient safety remains priority one. Participation in clinical trials is strictly voluntary and is decided entirely by the patient (assuming he or she meets the study's inclusion criteria). Study procedures are tightly regulated and monitored by federal and state agencies and institutional groups; and data are frequently analyzed so that new therapies continue to build on lessons learned from the past.

The value of clinical trials to modern health care cannot be underscored enough. The United States' global preeminence in science and medicine owes heavily to its prolonged investment in biomedical research over the 20th century.

In the 1960s, it took 8.1 years to develop a new drug; in the 1990s, it took researchers 15.3 years, or nearly double the time.
—Center for Information & Study on Clinical Research Participation, July 2005

Since World War II, a series of state and federal laws have been implemented to ensure patients' rights and safety. Informed consent requirements, for example, state that study objectives and procedures must be clearly spelled out to clinical trial participants.

Whether testing a new drug or conducting a behavioral questionnaire, each study plan, or protocol, must also be approved by the research institution's institutional review boards (IRBs) before patient enrollment can begin. These boards make sure each protocol protects patient confidentiality and work alongside external data safety monitoring boards (DSMBs) to keep apprised of each study's progress.

The average American spends almost $5,000 a year on health care. Less than $20 of that amount is invested in research to prevent, treat and cure diseases and disabilities.
—Center for Information & Study on Clinical Research Participation, July 2005

The most important criterion of any protocol, said Dr. Baruch Brody, director of the Center for Medical Ethics and Health Policy at BCM, is the minimization of risk to the patient.

"The goal of all clinical trials is to get valuable information," said Brody, who has published extensively on clinical trials and has served on more DSMBs than anyone in the United States. "At the same time, we have to be careful to protect research participants."

It can take the better part of a year between when a clinical trial is conceived to the time it is cleared to begin admitting patients. To facilitate this process, BCM recently created a Clinical and Translational Research Support Service unit, headed by Dr. Placido Grino, associate dean in the office of research.

Grino's team of research nurses and coordinators, regulatory affairs coordinators, data managers, and financial analysts develop strategic infrastructure programs that streamline the College's clinical research enterprise. Together, they work directly with sponsors of studies and help shoulder researchers' loads on matters such as liability concerns, volunteer recruitment, informed consent, and budget components.

"The things that used to take forever no longer take as long," said Grino. "Now, more people may consider doing clinical research."

Out of danger

Clinical trials' symbiosis of doctors and patients continues to redefine the status quo and, as evidenced in the Spikes' case, make inroads against some of the most elusive diseases of our time. Jada, now almost seven, fully recovered from ALL one year after being diagnosed, and she subsequently entered Dreyer's Long-Term Survivor Program, a compressive follow-up treatment regimen. "You would never know by looking at her that her life had been in danger," said Kori. "She's a healthy, active, beautiful, wonderful child."

For physicians and clinical investigators like Dreyer, the value of clinical trials becomes increasingly evident with the passage of time.

"For any of the kids we treat and cure, it's phenomenal to see them grow up and, for those old enough, start their own families," said Dreyer. "In some cases, their parents go from being completely distraught that their child has leukemia to counseling families of new patients."

Jada's recovery inspired her mother, who had not previously considered working in a hospital, to reinvent herself as a medical assistant. After getting her license, Kori wound up at the place where her daughter's life was saved.

"They took such good care of her that I prayed to God and said, 'I'll do whatever you want me to do to give back,' and he just kept guiding me here," said Kori, who has assisted cancer specialists at TCH for the last three years. "I finally said, 'OK, I hear you.'

People, Protocols and Promise

Not Just Another Phase

What are the different types of clinical trials?

Safe Guards

Out of danger

Patient Care

Research

Education

Community Service

Alumni & Development

College News

Glossary

Volume 2, Issue 2, Summer 2006


	People, Protocols and Promise by Ross Tomlin The Spikes family With the birth of her first and only child, Kori Spikes considered her life complete. All that changed 10 months later, however, when she was told her daughter Jada had acute lymphocytic leukemia (ALL). "I was in denial. Complete shock," recounted Spikes. "It was the worst feeling in my life, something I would never wish on anybody." Under the counsel of Dr. Zoann Dreyer, associate professor of pediatrics at BCM, Jada was enrolled in a clinical trial at Texas Children's Cancer Center, which is designed to bring cutting-edge cancer treatment to the infant's bedside. She underwent the latest in chemotherapy for six months, followed by another six months of less intensive maintenance therapy. The fact that Jada had a 50 percent chance of survival—as scary as it sounded—actually represented a quantum leap in predicting pediatric cancer outcomes. As recently as 10 years ago, a child diagnosed with ALL, the most common form of infantile leukemia, had a 20 percent chance of beating the disease. Forty years ago, the survival rate was zero. "The patients who were treated in clinical trials in the past have really benefited the kids today, because we were able to advance outcomes and cures based on how those children did," said Dreyer. Dreyer estimates that she is involved in more than 20 different clinical trials at any given moment, many of them nationwide and collaborative. One study might test whether a particular drug is more potent when administered over an entire day versus just a few hours. Another might assess whether the outcome improves if one type of steroid is used over another. Not Just Another Phase In Phase I trials, researchers test a experimental drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects. In Phase II trials, the experimental study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety. In Phase III trials, the experimental study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely. In Phase IV trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use. What are the different types of clinical trials? Treatment trials test experimental treatments, new combinations of drugs, or new approaches to surgery or radiation therapy. Prevention trials look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes. Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular disease or condition. Screening trials test the best way to detect certain diseases or health conditions. Quality of Life trials (or Supportive Care trials) explore ways to improve comfort and the quality of life for individuals with a chronic illness. Source: ClinicalTrials.gov "Clinical trials allow us to share patients' outcome data anonymously so that, statistically, we can evaluate different arms of treatment," said Dreyer. "Then we can ask and answer research questions to find the best way to treat patients." Literally thousands of clinical studies of all shapes, sizes, and durations are conducted at Baylor and its affiliated hospitals each year with the common goal of advancing novel therapies. In addition to leukemia, physicians and researchers with diverse medical and scientific backgrounds hunt for cures to virtually every other form of cancer, not to mention diabetes, heart disease, AIDS, autism, liver disease, and asthma, just to name a few. Earlier this year, BCM joined 32 other organizations in the country to be accredited by the Association for the Accreditation of Human Research Protection Programs Inc. Although not required for conducting clinical trials, the designation illustrates the College's commitment to upholding the highest possible standards in all of its various research endeavors. Safe Guards For every clinical trial looking to advance science and medicine, patient safety remains priority one. Participation in clinical trials is strictly voluntary and is decided entirely by the patient (assuming he or she meets the study's inclusion criteria). Study procedures are tightly regulated and monitored by federal and state agencies and institutional groups; and data are frequently analyzed so that new therapies continue to build on lessons learned from the past. The value of clinical trials to modern health care cannot be underscored enough. The United States' global preeminence in science and medicine owes heavily to its prolonged investment in biomedical research over the 20th century. In the 1960s, it took 8.1 years to develop a new drug; in the 1990s, it took researchers 15.3 years, or nearly double the time. —Center for Information & Study on Clinical Research Participation, July 2005 Since World War II, a series of state and federal laws have been implemented to ensure patients' rights and safety. Informed consent requirements, for example, state that study objectives and procedures must be clearly spelled out to clinical trial participants. Whether testing a new drug or conducting a behavioral questionnaire, each study plan, or protocol, must also be approved by the research institution's institutional review boards (IRBs) before patient enrollment can begin. These boards make sure each protocol protects patient confidentiality and work alongside external data safety monitoring boards (DSMBs) to keep apprised of each study's progress. The average American spends almost $5,000 a year on health care. Less than $20 of that amount is invested in research to prevent, treat and cure diseases and disabilities. —Center for Information & Study on Clinical Research Participation, July 2005 The most important criterion of any protocol, said Dr. Baruch Brody, director of the Center for Medical Ethics and Health Policy at BCM, is the minimization of risk to the patient. "The goal of all clinical trials is to get valuable information," said Brody, who has published extensively on clinical trials and has served on more DSMBs than anyone in the United States. "At the same time, we have to be careful to protect research participants." It can take the better part of a year between when a clinical trial is conceived to the time it is cleared to begin admitting patients. To facilitate this process, BCM recently created a Clinical and Translational Research Support Service unit, headed by Dr. Placido Grino, associate dean in the office of research. Grino's team of research nurses and coordinators, regulatory affairs coordinators, data managers, and financial analysts develop strategic infrastructure programs that streamline the College's clinical research enterprise. Together, they work directly with sponsors of studies and help shoulder researchers' loads on matters such as liability concerns, volunteer recruitment, informed consent, and budget components. "The things that used to take forever no longer take as long," said Grino. "Now, more people may consider doing clinical research." Out of danger Clinical trials' symbiosis of doctors and patients continues to redefine the status quo and, as evidenced in the Spikes' case, make inroads against some of the most elusive diseases of our time. Jada, now almost seven, fully recovered from ALL one year after being diagnosed, and she subsequently entered Dreyer's Long-Term Survivor Program, a compressive follow-up treatment regimen. "You would never know by looking at her that her life had been in danger," said Kori. "She's a healthy, active, beautiful, wonderful child." For physicians and clinical investigators like Dreyer, the value of clinical trials becomes increasingly evident with the passage of time. "For any of the kids we treat and cure, it's phenomenal to see them grow up and, for those old enough, start their own families," said Dreyer. "In some cases, their parents go from being completely distraught that their child has leukemia to counseling families of new patients." Stella Montya and Jada Spikes at Texas Children's Hospital Jada's recovery inspired her mother, who had not previously considered working in a hospital, to reinvent herself as a medical assistant. After getting her license, Kori wound up at the place where her daughter's life was saved. "They took such good care of her that I prayed to God and said, 'I'll do whatever you want me to do to give back,' and he just kept guiding me here," said Kori, who has assisted cancer specialists at TCH for the last three years. "I finally said, 'OK, I hear you.' "And here we are."		Search all issues: Patient Care People, Protocols and Promise An Infectious Enthusiasm Canvas for Creativity Research Seeing the Invisible Trekking Into New Territory: Translational Biology and Molecular Medicine Closing the Gap Between Lab and Clinic Education Tulane's Journey Back to New Orleans Community Service PUSHing for a Skate Park Alumni & Development Making Sense of Antisense Million Dollar Treatment Artist, M.D. Moving a Medical School College News A TEN-dency Toward Excellence Building Baylor Seamless Science Glossary An alphabet soup of organizations, regulations, and tools on the federal, state, and institutional levels ensure that patient safety remains the top priority of every clinical trial. BRAIN (Biomedical Research and Assurance Information Network) – An on-line tool developed at BCM that facilitates studies' procedural requirements between clinical investigators, research analysts, internal review boards, study coordinators, and department heads. DSHS (Department of State Health Services) – State agency that improves services, increases efficiencies, and enhances accountability among the state's health and human service agencies. DSMB (Data Safety Monitoring Boards) – An independent committee set up specifically to monitor data throughout the duration of a study to determine if continuation of the study is appropriate scientifically and ethically. HIPAA (Health Insurance Portability and Accountability Act) – Congressional legislation passed in 1996 that establishes standards for protecting the confidentiality and security of patients' health information. ICH (International Conference on Harmonization) – Brings together the regulatory authorities and health experts of Europe, Japan and the United States to streamline the development of new therapies and issue guidelines on clinical and laboratory practice, which must be observed to satisfy the Food and Drug Administration. IRB (Institutional Review Board) – Group of experts that ensure that clinical trials are ethical and that the rights of study participants are protected. The six IRBs at Baylor comprise scientists, health experts, legal counselors, ethicists, and members of the community. All clinical trials in the United States must be approved by an IRB before they begin. NIH (National Institutes of Health) – The primary federal agency for conducting and supporting medical research in the United States. Composed of 27 institutes and centers within the U.S. Department of Health and Human Services, the NIH provides leadership and financial support to researchers around the world. OHRP (Office for Human Research Protections) – Federal agency within the United States Department of Health and Human Services that sets ethical and regulatory guidelines regarding participation in clinical trials. OOR (Office of Research) – Department at BCM that develops and maintains strategic infrastructure programs to streamline and support the College's clinical and basic research process while assuring adherence to ethical and regulatory standards. It facilitates communication between investigators and research sponsors as well as collaborating organizations.

	Volume 2, Issue 2, Summer 2006



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