Frequently Asked Questions About Research

How is the study immunotherapy made?

Tumor cells are collected from cancer patients. The cells are modified to produce GM-CSF, a biological medication widely used to stimulate the bone marrow production of white blood cells. The tumor cells are grown in special flasks, then given a treatment similar to X-rays to reverse any further cell growth and then frozen. Just before use, the cells are thawed and put in syringes for injection.

How is the study immunotherapy given?

The study immunotherapy is injected into the skin in four places, two places on the upper back and one place on each thigh (left and right).

How often is it given?

The study immunotherapy is given in combination with another immunotherapy every 3 weeks. After 6-8 weeks the participant has repeat scans, such as MRI or CT scans, to see any possible effect. Depending on the tumor response, the participant may then continue with this therapy every 3 weeks for up to one year with repeat scans every 8 weeks.

Do I have to be in the hospital?

No, the immunotherapy is designed to be given in a doctor’s office.

What is GM-CSF and why is it needed?

GM-CSF is a biological medication widely used to stimulate the bone marrow production of white blood cells. There is, however, new data that this agent may also help generate a more effective immune response.

What are the side-effects of the immunotherapy?

Side effects are uncommon from the immunotherapy. There have been no life-threatening events that appear to be caused by the immunotherapy and have only rarely seen allergic reactions or local infections. These have been mild and self-limited. You will not lose your hair or vomit from the immunotherapy. Nausea after the priming medicine (cyclophosphamide, see more in paragraph below), if it occurs, is manageable with some of the newest anti-vomiting medicines. The immunotherapy is checked carefully for contamination, viruses, and infectious microbes. In theory, there is always a slight chance of some undiscovered or unpredicted side-effects.

As part of the study, you will also receive cyclophosphamide, which is a cancer chemotherapy, but at a lower dose than is used in cancer chemotherapy. Cyclophosphamide is being used as a priming medicine, to prepare the immune system. This can cause nausea (manageable with medication), lack of appetite, and/or, less commonly, vomiting, constipation, and/or a decrease in blood count. You will also be treated with a very low dose of a medicine called interferon. This can cause local pain, redness, swelling, itching, and/or flu-like symptoms. You will be monitored closely for any side effects and treated to prevent them if possible.

As part of this study, you will also receive a check point inhibitor, to assist immune system during cancer treatment, Retifanlimab. Retifanlimab has been associated with the development of autoimmune diseases (a disease where the immune system attacks different parts of the body). This includes the lungs, intestines, liver, hormone glands, kidneys, skin, nerves, and other organs. You will be monitored carefully for these side effects and treated if they occur. If you take nutritional supplements or herbs, please disclose that information in case you may be asked to stop the use during the clinical study.

Am I being experimented on?

Yes. As a research participant, you should know that this study was reviewed by a registered Institutional Review Board for compliance with medical ethics, safety, and scientific justification. The programs are then reviewed by the FDA and listed in the National Cancer Institute website. You should understand clearly that this is a research investigation. We are trying to develop a new treatment, learn as much as possible, and hopefully help the patients who come to us; but because this is a research study, we cannot guarantee it will benefit you, and we must always be on the lookout for unpredicted or unexpected effects.

What is “informed consent?”

Before you decide to be in the study or begin any study procedures you will be given a document which explains in detail the study and any known or theoretical side effects as mentioned above. The document must be signed to indicate that you have been told the details of your participation in the study and that you enter into it freely without pressure or deception, that you have been informed you can, at any time, ask further questions, and that you also have been informed about your rights and responsibilities as a research participant.

Signing the consent form does not take away any of your rights, including the right to change your mind. The consent process helps protect your rights and it also protects the ethical intent of our research.

How much does the immunotherapy cost?

We do not charge you or your insurance for the immunotherapy. You should be aware that there will be costs and charges, according to standard fees (standard of care), for most study procedures including medical visits, for the doctor’s visits where the immunotherapy and other study drugs are given, for laboratory and imaging tests, and for the supplies, needles and syringes, medications, etc. used in the study.

Will my insurance or Medicare cover these fees?

Generally, third-party payers routinely cover doctors’ visits and routine charges. Depending on circumstances, some carriers will reimburse for expenses associated with the experimental immunotherapy, but we cannot predict or guarantee you will be covered for all or part of the expenses. The sponsor and your doctors are committed to minimizing costs and to providing care and working with all eligible patients. The immunotherapy study is NOT subsidized by federal funds. If you have special circumstances, please be sure to discuss these with your doctors. Travel, lodging, and other reasonable expenses may be reimbursed by the Sponsor when requested by the Investigator but must be pre-approved by the Sponsor.

I’d like to be part of this study. What do I do next?

Notify our office and information will be provided. We will need medical records, pathology reports, x-rays, and scans, etc. We frequently like to discuss your medical situation with your personal physician. In evaluating your case we will try to make our recommendations as responsibly as possible.