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Clinical trials present huge risks and potential rewards

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When you or a loved one is in critical need of medical care, the words "clinical trial" may, at first, seem downright scary.

It may seem like a last resort for some, but for others, a clinical trial offers hope. 

According to the Society for Women's Health Research, 94 percent of Americans say their doctor has never talked to them about participating in a clinical trial.

There are both risks and benefits of participating in this type of medical research.

Jeanie Huelsman was upset when she learned her breast cancer had come back and that it was spreading throughout her body.

"Any woman when they hear - breast cancer - their life flashes in front of their eyes, they view it as a death sentence," said Dr. Steven Limentani, Director of Clinical Trials at the Levine Cancer Institute in Charlotte, N.C.

Huelsman took a deep breath when her doctor suggested she take part in a clinical trial, or a research study, for a new experimental pharmaceutical drug treatment.  

"You think to yourself - Oh, does this mean it's near the end?" Huelsman said.

With a little boy to care for, she didn't want to play any games with her health.

Her biggest concern was feeling like a human guinea pig for the pursuit of science.

Many medical experts, however, say that's one of the biggest myths about clinical treatment trials.

Clinical trials are not always a last resort. They are offered to people with all types and stages of disease.

Sometimes, a clinical trial is suggested by an individual's physician. In other cases, patients must do research on their own to determine if a medical trial is offered for the health condition they have. The website www.clinicaltrials.gov is a great resource for many.

Taking part in any clinical trial is a big decision. All the risks and potential rewards are outlined in the study's informed consent paperwork.

By agreeing to participate in the HER2 Clinical trial, Huelsman was volunteering to receive treatment that wasn't available yet to the public.

She understood the new treatment may work better or worse than prior treatments she received, and that, maybe, the new treatment could cause unknown side effects.

But what if she received a placebo?

In a blind study, you don't know if you're receiving the new treatment or not.

With Stage 4 breast cancer, Huelsman was concerned that by taking part in the study she might only be given a 'sugar' pill or no treatment at all.

"The only time there would be a no-treatment arm is if there was no treatment," Dr. Limentani said. 

With most clinical treatment trials, every treatment option is called an arm of the study. The standard of care is the control, or placebo arm, and the new treatment is the other arm.

So, if the standard treatment is to do nothing, then that would be the placebo arm.

But the standard of care for cancer is chemotherapy. So, at the very least, Huelsman was assured she would still be getting treatment to fight the spreading cancer in her body. She just wouldn't know if she was also receiving the new drug.

"Oh gosh, this is really scary, to not know what you're going to get, but it's also hopeful, too, because it could be a good thing," Huelsman said.

If Huelsman's condition worsened, she knew she could refuse to continue being part of the clinical trial. 

That's because in all clinical trials, at any point, participation is always in the patient's hands.

"It's my decision to get off of this," Huelsman said.

Several weeks into the trial, Huelsman's new scans came back and her cancer had stopped spreading. The final results on the clinical treatment trial also came back.  

"The benefit was double. One, patients did better. Two, treatment was less toxic," Dr. Limentani said referring to the HER2 clinical trial. "It's the one-two punch for trying to kill breast cancer cells."

Now this new targeted breast cancer therapy call Pertuzumab is FDA approved and women across the country will have access to what Huelsman describes a medical miracle.

"Knowing this is not necessarily a cure [for cancer], it's sure making me feel good," and Huelsman. "Knowing that I can go on and just keep going, makes me pretty darn happy!"

One of the other benefits of participating in a clinical trial is that it can, sometimes, significantly reduce your treatment costs.

Many of the tests and check-ups required while participating in a clinical trial are paid for by the trial's sponsor and health insurance often covers the rest.
 

Additional Information: 

The following information is from Dr. Steven Limentani, Director of Clinical Trials for Levine Cancer Institute located in Charlotte, NC.

  • Check with your insurance. Sometimes the trial sponsor pays for everything, but not always.
  • The trial referenced in this story is for HER2 Over-Amplified breast cancer, which has been considered high-risk for earlier relapse and it more commonly goes to the brain.
  • The trial was for a pharmaceutical drug called Pertuzumab and was done on "late-stage" or Stage 4 breast cancer.
  • Pertuzumab is unique in that it doesn't come with the toxicity effects (feeling sick, losing hair). It is a "targeted" treatment.
  • All cells have receptors on their surface. HER2 is a growth receptor on a cell. Every breast cancer cell has a HER2 receptor on it.
  • Twenty percent of breast cancer cells have an extra copy of the HER2 receptor.
  • Pertuzumab inhibits the ability of HER2 to bind to other receptors causing it to die.
  • Almost always the patient does not end up with a financial burden from going on a clinical trial.
  • If you decide to quit participating in a study, you cannot rejoin at a later date.

The following information is from Jeanie Huelsman, the patient featured in our story.

  • Huelsman was diagnosed with Stage 1 Breast cancer in August, 2004.
  • In October 2011, she had a recurrence of cancer (Stage 4) in her breast, lungs, breast bone and three of her lymph nodes.

The following information is from the website ClinicalTrials.gov (http://clinicaltrials.gov/ct2/info/understand).

  • There are two types of trials: Interventional studies involving a treatment or other intervention with measured outcomes, and Observational studies involving patient observation.
  • Participation in a trial is based on inclusion and exclusion criteria such as age, gender, type and stage of disease, previous treatment history, and other medical conditions.
  • Participants receive an informed consent document which gives details about the study (purpose, duration, required procedures, contacts, risks, potential benefits). Participants must decide whether or not to sign. The participant may withdraw from the trial at any time.
  • What are the benefits and risks of participating in a clinical trial?
    • Risks include: serious perhaps life-threatening side effects to treatment, ineffective treatment, added time and attention to treatment.
    • Most clinical research is federally regulated with built in safeguards to protect the participants. As a clinical trial progresses, researchers report the results of the trial at scientific meetings, to medical journals, and to various government agencies. Individual participants' names will remain secret and will not be mentioned in these reports.
  • Questions to ask about your trial:
    • What is the purpose of the study?
    • Who is going to be in the study?
    • Why do researchers believe the experimental treatment being tested may be effective? Has it been tested before?
    • What kind of tests and experimental treatments are involved?
    • How do the possible risks, side effects, and benefits in the study compare with my current treatment?
    • How might this trial affect my daily life?
    • How long will the trial last?
    • Will hospitalization be required?
    • Who will pay for the experimental treatment?
    • Will I be reimbursed for other expenses?
    • What type of long-term follow up care is part of this study?
    • How will I know that the experimental treatment is working? Will results of the trials be provided to me?
    • Who will be in charge of my care?
  • Bring a tape recorder to your clinical trial meeting to record the discussion to replay later so you can remember the details about the trial you will be participating in.
  • Every clinical trial in the U.S. must be approved and monitored by an Institutional Review Board (IRB) to make sure the risks are as low as possible and are worth any potential benefits. An IRB is an independent committee of physicians, statisticians, community advocates, and others that ensures that a clinical trial is ethical and the rights of study participants are protected. All institutions that conduct or support biomedical research involving people must, by federal regulation, have an IRB that initially approves and periodically reviews the research.
  • Participants can leave a clinical trial, at any time. When withdrawing from the trial, the participant should let the research team know about it, and the reasons for leaving the study.
  • The control group in a study is the standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.
  • Different types of 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, vaccines, vitamins, 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.
  • Phases of trials:
    • Phase One: researchers test an 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.
    • Phase Two: 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.
    • Phase Three: 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.
    • Phase Four: post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
  • Expanded access means a manufacturer is making an investigational new drug available to treat a patient who cannot participate in a clinical trial due to other health issues, age, distance, etc. FDA regulations enable manufacturers to offer the drug to those who may benefit.

The following information is from the National Cancer Institute.

  • Patient costs include routine care (doctor visits, lab tests, x-rays, scans, etc) which are often covered by insurance. As well as extra care costs (extra tests needed for the trial).
  • Patients are not responsible for research costs (doctor's time, analysis of results, clinical tests purely for research).
  • This map: http://www.cancer.gov/clinicaltrials/payingfor/laws shows which states have passed legislation requiring health plans to pay the cost of routine medical care you receive as a participant in a clinical trial.

The following information is from the U.S. Food and Drug Administration's website in an article entitled, Basic Questions about Clinical Trials (http://www.fda.gov/forconsumers/byaudience/forpatientadvocates/hivandaidsactivities/ucm121345.htm).

  • Who should participate in a trial?
    • Those who have exhausted standard (approved) treatments (did not work, unable to tolerate side effects).
    • Those interested in contributing to the advancement of medical knowledge.
    • The federal government has regulations and guidelines for clinical research to protect participants from unreasonable risks, although some risks may be unavoidable.
    • The government requires researchers to give participants an "informed consent" document to sign before joining the study indicating they understand that the trial is research, and that they can leave the clinical trial at any time.
  • Benefits to a trial include:
    • Access to potentially new research treatments.
    • Access to expert medical care.
    • Contribution to medical research.
  •  Trial advantages:
    • Potentially receiving treatment before it is widely available to the public.
    • The treatment may be more effective than the standard treatment.
    • Treatment costs may be decreased since many of the tests and check-ups related to the trial are paid for by the sponsoring company or agency.
  •  Disadvantages:
    • All risks and side effects are usually not known at the beginning of a trial.
    • If you are in a randomized trial, you may not receive the new treatment.
    • The new treatment may not work.
    • Insurers do not always cover all costs when taking part in a trial. Talk to your insurer before joining.
    • Inconveniences like frequent testing, time and travel are often required.

The following information is from the Mayo Clinic's website in an article entitled, Clinical trials: A chance to try new therapies (http://www.mayoclinic.com/health/clinical-trials/DI00033/).

  • Some participants in clinical trials get a placebo — a pill or liquid that looks like the new treatment, but has no active ingredients. Using placebos give researchers something to compare with the drug being tested. You can't control whether you receive the placebo or the new treatment. Not all clinical trials have a placebo component, as some clinical trials compare two different active treatments. Ask your doctor if there is a possibility you will get a placebo.
  • You can find a clinical trial on the National Library of Medicine clinical trials website or www.clinicaltrials.gov.
  • Once you have selected a trial:
    • Talk to your doctor about the drug or therapy involved, risks, benefits and eligibility.
    • Contact the clinical trial coordinator (listed on the announcement) and ask about your eligibility.
    • Schedule a pretrial screening to determine if you qualify.

The following information is from the Seattle Cancer Care Alliance in an article entitled, Clinical Studies – Myths vs. Facts (http://www.seattlecca.org/clinical-trial-myths.cfm).

  • MYTH: Patients in clinical studies are little more than human guinea pigs.
    FACT: Some people balk at participating in clinical studies because they fear that doctors and researchers will treat them as a set of symptoms rather than as a human being. But the overwhelming majority of cancer patients who have participated in clinical trials say the experience was positive and that they were treated with respect.
  • MYTH: Clinical studies are not safe, I'd be gambling with my health if I signed up.
    FACT: New treatments are tested on human subjects only after there is valid scientific evidence that the treatments are likely to be effective and safe. If you take part in a Phase III clinical trial, the drug or treatment has already been tested on small groups of patients for both safety and effectiveness.
  • MYTH: You should only agree to take part in a clinical study if your cancer is terminal or there are no other treatment options available outside a study.
    FACT: Some clinical studies are reserved for cancer patients who have exhausted all the treatment options for their disease. But clinical studies of various types are open to patients at all stages of treatment. There are clinical studies that test ways of preventing a recurrence in breast cancer survivors, studies that look at cancer prevention in high-risk populations, and trials that compare different doses of chemotherapy drugs already in use.
  • MYTH: My health insurance won't pay for treatment provided through a clinical study.
    FACT: Many people don't even consider a clinical study because they are afraid that their health insurance company will not cover the treatment. In fact, your health insurance may pay for treatment. The majority of patients in clinical trials receive reimbursement from their health insurance, but health insurance may not pay all the costs. To learn more, click here.
  • MYTH: Cancer patients may be given a placebo in a clinical study.
    FACT: Placebos are not used in clinical studies that test cancer treatments. Patients either receive the new treatment or the best standard of care. A placebo may be used in a trial to test drugs that may prevent cancer, but the people enrolled in that trial would not have cancer.
  • MYTH: I will receive poorer medical treatment in a clinical study.
    FACT: Patients in clinical trials receive either the best standard treatment or a new treatment that researchers believe will be at least as good as the standard treatment.
  • MYTH: I will get better medical treatment in a clinical study.
    FACT: This may overstate the case. Researchers cannot guarantee outcomes. However, patients who participate in clinical studies sometimes do have higher survival rates than patients who receive standard treatment. And they are receiving treatment from doctors who are leaders in their fields.
  • MYTH: If I join a clinical study I won't be able to drop out without jeopardizing my treatment.
    FACT: This is not true. You may quit a clinical study at any time.

The following information relates Jeanie Huelsman's clinical trial.

  • On June 8, 2012, Food and Drug Administration approved pertuzumab injection (Perjeta, made by Genentech, Inc.) for use in combination with trastuzumab and docetaxel for the treatment of patients with HER2-positive metastatic breast cancer who have not received prior anti-HER2 therapy or chemotherapy for metastatic disease.
  • The approval is based on a randomized double-blind placebo-controlled multicenter trial in patients with HER2-positive metastatic breast cancer. (of which Carolinas HealthCare System participated in).
  • The trial enrolled 808 patients who were randomly assigned.
  • All but two patients were female (99.8 percent), and the median age was 54 years.
  • The most common (more than 30 percent) adverse reactions observed in patients who received pertuzumab in combination with trastuzumab and docetaxel were diarrhea, alopecia, neutropenia, nausea, fatigue, rash, and peripheral neuropathy.

The following information was from a Yahoo.com article entitled FDA approves Perjeta.

  • The FDA approval of Perjeta (pertuzumab) is based on results from CLEOPATRA (CLinical Evaluation Of Pertuzumab And TRAstuzumab), an international, Phase III, randomized, double-blind, placebo-controlled study. The study evaluated the efficacy and safety profile of Perjeta combined with Herceptin and docetaxel chemotherapy compared to Herceptin and chemotherapy plus placebo in 808 people with previously untreated HER2-positive mBC or that had recurred after prior therapy in the adjuvant or neoadjuvant setting. The study showed people who received Perjeta in combination with Herceptin and chemotherapy experienced a 38 percent reduction in the risk of their disease worsening or death compared to people who received Herceptin and chemotherapy plus placebo.
  • In CLEOPATRA, the most common adverse reactions (rate greater than 30 percent) seen with Perjeta in combination with Herceptin and docetaxel were diarrhea, hair loss, low white blood cell count, nausea, fatigue, rash and peripheral neuropathy (numbness, tingling or burning sensation in the arms or legs).
  • Perjeta is designed specifically to prevent the HER2 receptor from pairing with other HER receptors (EGFR/HER1, HER3 and HER4) on the surface of cells, a process that is believed to play a role in tumor growth and survival. Binding of Perjeta to HER2 may also signal the body's immune system to destroy the cancer cells. The mechanisms of action of Perjeta and Herceptin are believed to complement each other, as both bind to the HER2 receptor, but to different regions. The combination of Perjeta, Herceptin and chemotherapy is thought to provide a more comprehensive blockade of HER signaling pathways.
  • Important Safety Information:
    • Perjeta has been shown to work only in people with HER2-positive breast cancer. Patients must have a HER2 test to know if their breast cancer is HER2-positive before receiving an anti-HER2 treatment, such as Perjeta
    • Because side effects from this treatment are common, it is important to know what side effects may happen and what symptoms patients should watch for.
    • A patient's doctor may stop treatment if serious side effects happen. Patients must contact their healthcare team right away if they have questions or are worried about any side effects
  • Most Common Side Effects
    • The most common side effects of Perjeta when given with trastuzumab and docetaxel are diarrhea, hair loss, low levels of white blood cells with or without a fever, upset stomach, fatigue, rash and damage to the nerves (numbness, tingling, pain in hands/feet).
    • Herceptin is a personalized medicine designed to specifically block the HER2 protein on the surface of some cancer cells. Based on preclinical studies, this biologic antibody is believed to work by attaching to HER2 receptors to stop signals that make the tumor cells grow and divide, and also by signaling the body's immune system to destroy the cancer cells.
  • Important Safety Information
    • Herceptin treatment can result in heart problems, including for those patients without symptoms (such as reduced heart function) and those patients with symptoms (such as congestive heart failure). One patient died in an adjuvant breast cancer trial from significantly weakened heart muscle. The risk and seriousness of these heart problems were highest in patients who received both Herceptin and a certain type of chemotherapy (anthracycline).
    • Some patients have had serious infusion reactions and lung problems; fatal infusion reactions have been reported. These reactions usually occur during or within 24 hours of receiving Herceptin.
    • The patient's doctor may need to completely stop Herceptin treatment if the patient has a severe allergic reaction, swelling, lung problems, inflammation of the lung or severe shortness of breath.
    • The most common side effects associated with Herceptin in patients with breast cancer are fever, nausea, vomiting, infusion reactions, diarrhea, infections, increased cough, headache, fatigue, shortness of breath, rash, low white and red blood cells and muscle pain.

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