Division of Hematology/Oncology

Phase 1 Trials & Experimental Therapeutics Program

Penn is one of a number of institutions nationally with an outstanding reputation for offering patients high-quality, high-impact clinical trials for cancer.

The Cancer Therapeutics Program at the Abramson Cancer Center is dedicated to the study of promising new drugs, or combinations of drugs in treating cancer. It is through these studies that innovative drugs discovered in the laboratory, become useful cancer treatments.

Clinical trials in the Cancer Therapeutics Program may take several forms, depending on the experience already obtained for a given treatment. Many of the studies are testing new drugs in Phase 1 and 2 clinical trials. Clinical trials are patient studies in which highly experienced physicians determine the side effects, dose, schedule and benefits of new cancer treatments or new drugs to prevent the development of cancer.

About Phase 1 and Phase 2 Clinical Trials

Phase 1 clinical trials are the first step in testing a new drug treatment in humans. Phase 1 studies seek to discover the best dose and means of administration of a new treatment, and to detect any early evidence of possible effectiveness against the cancer.

Phase 1 studies usually include a number of patients with well-defined characteristics for whom entry on a novel therapy has a favorable risk/benefit profile. As part of these studies, the response of the patient's normal and tumor tissues may be monitored using technologically sophisticated analyses.

Phase 2 clinical trials are the second step in testing the safety of a drug or treatment. Phase 2 clinical trials begin to evaluate how well the new drug actually works in a specific type of cancer once the dose and schedule have been determined in phase 1.

Phase 2 studies seek to answer specific questions such as:

  • Does this treatment shrink a tumor?
  • Does this treatment stabilize the growth of tumors?
  • What are the typical side effects of this drug?

How Clinical Trials are Carried Out

In any clinical trial, the individual patient's well-being and safety are paramount. Research in people is carried out according to strict scientific and ethical principles. These include the following:

  • The person in charge of the study is called the principal investigator. This is usually a doctor.
  • Each clinical trial has an action plan, or protocol, explaining in great detail how the study will be carried out. The investigator prepares the protocol for the study. This protocol states why the study is being done, how many people will take part in the study, what medical tests they will receive and how often, as well as the overall treatment plan. The same protocol is used by each doctor participating in the study.
  • For patient safety, each protocol is approved by:
    • The organization that sponsors the study (such as the National Cancer Institute or a pharmaceutical company).
    • Penn's Institutional Review Board, (IRB). This board, which includes citizens from the community, clergy and health professionals, reviews the protocol to ensure that the research will not expose patients to unacceptable or unethical risks.
    • The Cancer Center's Clinical Trials and Scientific Review and Monitoring Committee, which reviews the scientific merit of a clinical trial before it begins.
  • Each study's protocol describes the required characteristics for all patients in the study. These key characteristics are called eligibility criteria. Eligibility criteria differ from study to study, depending on the research purpose. They may include such factors as age, gender, and the type and stage of cancer.

The Penn Difference

  • At the University of Pennsylvania, clinical research in cancer is an area of focus and expertise.
  • Faculty members in Cancer Therapeutics are leaders nationally in directing clinical research in cancer, and at Penn, and perform ground-breaking trials that often lead to new medicines for resistant diseases.
  • In the last five years, Penn faculty-led trials have resulted in Food and Drug Administration-approval of several new targeted drugs for cancer, and to ongoing large-scale trials of other agents that if successful may change medical treatment for cancer.

For more information about Penn's Cancer Therapeutics program including how to enroll in a Phase 1 or Phase 2 clinical trial, refer to Phase 1 and Phase 2 clinical trials on the Abramson Cancer Center website.