Posts Tagged ‘cancer trials’

Phase I Trials – Where All Anticancer Drugs Begin

Donald Harvey, MD

R. Donald Harvey, PharmD, FCCP BCOP, director of the Winship Cancer Institute of Emory University’s Phase I Clinical Trials section

Each of the agents we use to treat cancer had a beginning, a first step, in understanding how safe and effective they might be.  As drugs are developed, we ask questions in different ways at each step, or phase, of testing. The National Cancer Institute reminds us that clinical trials are available for all patients at all points in their cancer journey, not just for patients with advanced cancer that is not responding to treatment.

When a drug is first given to patients, it enters testing in a phase I trial, where we ask questions such as:

  • What is the right dose?
  • How should it be given (e.g., by mouth, by vein, under the skin)?
  • What is the right schedule of treatment?
  • What side effects are there and how severe are they?
  • How often do we see side effects?
  • Where did the drug go in the patient? How well was it absorbed? How was it metabolized and/or eliminated? (Pharmacokinetics)
  • What did the drug do to the patient, both in blood and at the site of the cancer? (Pharmacodynamics)

Patients courageous enough to enter phase I trials are asked to do many time-consuming but important things during the trial. Frequently, patients are asked to spend 10-12 hours in our clinical trials unit and/or come in daily up to 14 times during the first treatment period, or cycle. During these visits, blood is drawn, tumor or bone marrow biopsies may be performed and safety tests are conducted, all in an effort to get a complete picture of drug effect, disposition and side effects.

Participation in phase I clinical trials:

To participate in a phase I trial, patients typically have cancer that has not been effectively treated with other therapies, and most trials require patients to be otherwise relatively healthy. Phase I trials usually enroll 10-40 patients, but may be larger or smaller depending on the questions being asked. Two types of phase I trials exist: those where the drug is being given for the first time, or first-in-human trials; and those where there is prior experience and the drug is given in combination with another drug or drugs (also called phase IB trials). In each, the investigational agent is given to small groups of patients, and doses are increased in each group. Both types are critical to the next step of development to define the dose, frequency, and understand what cancer types are most likely to benefit.

Phase I trials help to determine the future of drugs in cancer treatment. Right now, the large number of new agents in early testing indicates great potential in the transformation of therapy. People in good health may choose to participate in clinical trials simply to help researchers find better treatments. Participation in clinical trials is completely voluntary, but you should also speak with your physician before deciding to enroll.

Related Resources

 

 

 

About Dr. Harvey

R. Donald Harvey, PharmD, FCCP BCOP is director of the Winship Cancer Institute’s Phase I Clinical Trials section, and Associate Professor of Hematology and Medical Oncology at the Emory University School of Medicine. He is a Fellow of the American College of Clinical Pharmacy and a board certified oncology pharmacist. Widely published in peer-reviewed journals, Dr. Harvey’s research interests include the clinical application of pharmacokinetic, pharmacodynamic, and pharmacogenomic data to patient care.

The Skinny on Neuroendocrine Cancer

Neuroendocrine tumors develop in the cells of the neuroendocrine system, a network of glands throughout the body that produce hormones. Neuroendocrine tumors are rare, typically affecting 1 – 2 people per 100,000 people.

The different types of neuroendocrine tumors depend on the particular cells that are affected.  They usually develop in the digestive system and the lungs.  Approximately 38% of tumors are in the appendix, 23% in the ileum (also known as carcinoids), 13% in the rectum and 11.5% in the bronchi.

Symptoms of Neuroendocrine tumors

Neuroendocrine tumors may be slow growing (low grade or well differentiated) or rapidly growing (high grade or poorly differentiated).  They may be functional (release hormones) or nonfunctional.  When functional neuroendocrine tumors release a variety of hormones including a hormone called serotonin, a condition called carcinoid syndrome may occur. Symptoms most commonly associated with neuroendocrine tumors include flushing, diarrhea, weight loss, abdominal pain and cramping.   Patients with carcinoid syndrome may also experience cardiac changes, which are caused from fibrotic damage to the heart.  Usually a patient with carcinoid syndrome does not experience these symptoms until the tumor has spread within the body.

Other terms used for neuroendocrine tumors include
• Gastroenteropancreatic neuroendocrine tumors – tumors in the gut or pancreas
• Pancreatic neuroendocrine tumors – also known as islet cell cancer, or PNET
• Gastrointestinal neuroendocrine tumors – tumors that develop in the bowel, stomach or esophagus
• Pheochromocytoma of the adrenal gland
• Carcinoid tumors of the lung or intestinal tract
• Carcinoid Syndrome
• Gastrinoma, Insulinoma, Glucagonoma, VIPoma (vasoactive intestinal peptide tumor)

Treatment of neuroendocrine tumors

Treating neuroendocrine tumors can be a challenge due to the various types, locations in the body, the symptoms they produce and the diverse number of treatment modalities. Treatment is individualized to  each patient but can include surgery, liver directed therapy, medications, and/or radiation.

When treating a rare, challenging cancer such as neuroendocrine cancer, it is important to have a team of healthcare professionals at your side who are experts in the  disease and can collaborate to create a treatment plan individualized to each patient.

At Winship Cancer Institute of Emory University, we take this multidisciplinary approach with teams of  expert medical oncologists, surgical oncologists, interventional oncologists, gastroenterologists, pulmonologists, endocrinologists, nurses, and dieticians ( just to name a few) to create a care plan unique to each patient.

About Bonnie Josaphs, RN, BSN, OCN

Ms. Josaphs received her Bachelor of Science in Nursing from the University of Delaware, Newark, Delaware in 2002. She has been practicing within Gastrointestinal (GI) Oncology since 2004.  She specializes in gastrointestinal cancers which include cancers of the esophagus, liver, colon, rectum, pancreas, gallbladder and stomach.

Related Links

More information on neuroendocrine tumors:
Winship Cancer Institute Clinical Trials for Neuroendocrine Tumors
 The Winship Cancer Institute of Emory University is Georgia’s only NCI-designated Cancer Center.