Expanded Access: Programs and Possibilities

Current Expanded Access Programs

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The FDA requires that all cancer expanded access programs be listed on the government website www.clinicaltrials.gov. To directly access information on Expanded Access programs that are open for enrollment, from the ClinicalTrials.gov home page, click on "Search for Clinical Trials." You will then see a page called "Basic Search" with an open box for you to enter the topic of your interest. Skip this and instead, click on the "Advanced Search" tab at the top of the page. This will open a page with many open boxes. Ignore all of them except the fourth entitled "Study Type" which will have the term "All Studies" filled in. Use the drop-down button to select "Expanded Access Studies." Then go to the fifth open box entitled "Conditions" and type in the word "Cancer." Then push the "Search" button. This will take you to the page listing both older closed and new open Expanded Access Programs for various types of cancer approved by the FDA. Note that if you are interested in an Expanded Access program for a condition other than cancer, you can either leave the box blank to see all programs for all conditions, or fill in the name of the condition you are looking for.

You can also try clicking on this link to go directly to the list of cancer Expanded Access Programs at ClinicalTrials.gov: Cancer Clinical Trials

Currently Open Expanded Access Programs

T-DM1

T-DM1 (more completely known as Trastuzumab-DM1) is an antibody-drug conjugate developed by Genentech in association with ImmunoGen. An antibody-drug conjugate is composed of an antibody linked to a drug in a manner that allows the drug to be released once the antibody binds to its target. In this case, the antibody used is called trastuzumab, also known by the product name Herceptin, which targets HER2-positive breast cancer cells. The drug that is linked to the antibody is called mertansine and it interferes with tubulin, a protein essential for cell division. The idea of an antibody-drug conjugate is to enable potent doses of a highly toxic drug to be targeted precisely to only the cells that need to be killed, in this case HER2-positive breast cancer cells, without doing too much damage to normal cells.

In a Phase II clinical trial, T-DM1 shrank tumors in one-third of women who had received extensive prior treatment for advanced HER2-positive breast cancer. In some cases, T-DM1 was useful even for women whose disease progressed during treatment with Herceptin. Genentech has numerous clinical trials of T-DM1 underway or planned, and has opened a "T-DM1 Patient Access Study" in the United States while T-DM1 is under FDA review for marketing approval.

Go to www.clinicaltrials.gov for more information on this Expanded Access Program, including contact information. The formal title of the program is "A Study of Trastuzumab-MCC-DM1 in Patients With HER2-Positive Locally Advanced or Metastatic Breast Cancer."


BSI-201

BSI-201, also known as Iniparib, is currently in Phase III clinical development. The drug is an encapsulated formulation of the chemical compound 4-iodo-3-nitrobenzamide, first described as an anti-cancer agent by Professor Ernest Kun and his collaborators in the early 1990's. The drug is one of many members of a class of compounds called PARP (Poly ADP Ribose Polymersase) inhibitors. These are generally small chemical compounds, relatively easy to manufacture, under development by various biotechnology and pharmaceutical companies for the treatment of a wide variety of cancer types.

Sanofi-Aventis, through its subsidiary BiPar Sciences, is testing BSI-201 as a treatment for metastatic breast cancer which is estrogen receptor (ER)-negative, progesterone receptor (PR)-negative, and HER2-negative. This is sometimes referred to as "triple negative" breast cancer. In a phase II clinical trial of BSI-201 in which 116 women participated, the experimental drug plus chemotherapy produced a tumor response in 48 percent of patients whereas chemotherapy alone produced a tumor response in only 16 percent of patients. In this study, BSI-201 extended both progression-free survival and overall survival.

Sanofi recently opened an Expanded Access program for BSI-201 for certain women with advanced triple negative breast cancer. The program includes a lottery allocation program administered by the National Organization for Rare Disorders. Go to www.clinicaltrials.gov for more information on this Expanded Access Program, including contact information. The formal title of the program is "An Open-Label, Expanded Access Protocol of Iniparib Breast Cancer."

Experimental Drugs in Phase 3 Clinical Trials

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Remember:

• No cancer drug works for every patient who uses it
• Most cancer drugs have significant side effects
• We don't know if a new treatment will be better than the existing treatment until comparison trials are complete.

There are some new drugs - for example, Gleevec - which offer a dramatic improvement to standard treatments. Unfortunately, most new drugs don't fall into that category.

Experimental drugs in phase 3 clinical trials are generally the drugs closest to FDA review and possible approval. Companies tend to be most open to considering access outside of trials for drugs at this stage of development.

Remember, you cannot force companies to allow access. However, your call may get them thinking about either expanded access or single patient use. Contact us for help.

New Uses for Old Drugs

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Companies may apply to the FDA for approval of their drug in new forms of cancer, based on clinical trial results. This approval means that treatment for the new form of cancer is considered non-experimental and your insurance will cover costs.