Misconceptions About Clinical Trials Dispelled

Having never made such a monumental decision, many are concerned about enrolling in a clinical trial.  Some worry that the patient will be treated like a guinea pig or will receive the placebo or sugar pills.  Worse yet, some think that clinical trials should be considered only when there is no hope.  These misconceptions about clinical trials are dispelled at the International Mesothelioma Program (IMP) at Brigham and Women’s Hospital in Boston, Massachusetts.

Dr. David Sugarbaker, the founder and director of the International Mesothelioma Program and the Chief of Thoracic Surgery at Brigham and Women’s Hospital, has been treating mesothelioma patients along with his colleagues at the Dana-Farber Cancer Institute since the 1980s.  In fact, the clinical trials being conducted at the IMP are at the forefront of state-of-the-art mesothelioma treatment and care in the United States.  Each year approximately 160 mesothelioma patients are treated at the International Mesothelioma Program by Dr. Sugarbaker and his team of medical experts.  Twice that number of patients are seen for consultation.

Surgical Clinical Trials Leading to Increased Survivability

In a lecture given in late 2009, Dr. Sugarbaker noted that through his surgical clinical trials, the IMP is seeing a 17 percent survival rate for patients who are four years post-surgery.  This is remarkable when you consider these patients’ original prognosis, which was a mere six to nine months to live.  Dr. Sugarbaker is seeing survivors who are eight to ten years post-surgery and a few others who are living as long as 13 years with no recurrence.

Attorney Gary Galiher, who has represented mesothelioma clients for 32 years, is very hopeful when he see the results Dr. Sugarbaker has had from his clinical trials.

“I have seen my clients suffer and die from this devastating cancer.  Dr. Sugarbaker’s clinical trials portend much hope and promise that one day in the not so distant future mesothelioma will be a curable disease.”

Clinical Trials at the International Mesothelioma Program

One important mesothelioma clinical trial is Protocol 07-091 for patients with resectable pleural malignant mesothelioma; that is, a tumor that can be removed surgically.  The surgery is followed by heated chemotherapy drugs applied directly into the chest cavity during the surgical procedure.  The drug combination used is Cisplatin along with Gemcitabine.  Amifostine is also used to protect the normal cells.  The patient is then followed weekly.

Of 304 patients screened, Dr. Sugarbaker says 64 were enrolled in this clinical trial.  The study is still open and accepting patients.  The clinical trial requires that certain criteria are met in order for a patient to qualify for enrollment in Protocol 07-091.  Since time is of the essence, it is important that patients discuss this and other possible courses of treatment with their physician right away.   They may also consider calling the IMP directly at phone:  617-732-5922 to get immediate information.

It is clear that through a number of clinical trials, medical advances are taking place at the International Mesothelioma Program under Dr. Sugarbaker’s leadership.  Virtually all advances in cancer treatment involve clinical trials.  These advances translate to hope and a prolonged and enhanced quality of life for mesothelioma patients.

New Clinical Trial Underway

The International Mesothelioma Program will be conducting a new clinical trial for patients with advanced stage mesothelioma.  This new Phase I trial would use the neoadjuvant mTOR inhibitor followed by administering Cisplatin in combination with pemetrexed or Alimta.  After the chemotherapy regimen, depending on the patient’s response, an extrapleural pneumonectomy could then be performed.

Over the years through the clinical trials, the IMP has been able to increase the dose of chemotherapy drugs to the maximum tolerable amount.  Additionally, because of Dr. Sugarbaker’s experience treating so many mesothelioma patients, he and the IMP have learned the site of recurrence of the cancer is usually in the chest or abdomen where the original tumor appeared.  Dr. Sugarbaker explained that he and his medical team have observed over the last 15 years that, even after surgery, microscopic cells are present and thus he developed the in vivo model, as he calls it, of administering the heated chemotherapy in the maximum tolerable dose into the open chest cavity.

His patients have words of praise and gratitude as they speak of Dr. Sugarbaker being a hero and a pioneer in mesothelioma research with the clinical trials which the International Mesothelioma Program is conducting.

Surgery to Remove the Tumor

Cytoreductive surgery falls into two categories:  (1) extrapleural pneumonectomy that involves the removal of the affected lung, and (2) pleurectomy and decortication which is the removal of the pleura and as much tumor tissue as possible.  The type of surgery that a patient receives at the IMP depends upon how early the mesothelioma was diagnosed and the extent to which the disease has spread.  It is part of the preoperative protocol at the International Mesothelioma Program to perform a procedure called a mediastinoscopy, to biopsy the patient’s lymph nodes in the  mid-chest area or mediastinum.  This procedure tells the physicians at the IMP the extent to which the malignant mesothelioma has spread.

If the patient’s malignant mesothelioma is discovered early enough, Dr. Sugarbaker may perform an extrapleural pneumonectomy.  Not only is the affected lung removed, but the diaphragm, pleura, and pericardium are also surgically removed.  The diaphram and other structures in the chest are re-built using Gortex, which is a synthetic gas-permeable membrane.   The other surgery, a pleurectomy/decortication is used to removed the diseased pleura and as much of the patient’s mesothelioma tumor as possible.

Chemotherapy Drugs Administered Immediately

At the International Mesothelioma Program, following either the extrapleural pneumonectomy or the pleurectomy/decortication, the patient’s chest cavity is infused with a lavage (washing out the organs) of heated chemotherapy drugs.  Researchers and physicians at the IMP have determined that there is much greater chemotherapy penetration when the drugs are heated and washed directly over the affected area compared to being delivered intravenously.  The purpose of the intracavitary heated chemotherapy lavage is to kill the malignant mesothelioma cells that might be left behind after surgery and that cannot be seen by the surgeon.

Success With Dr. Sugarbaker’s Techniques

Dr. Sugarbaker and his team have been very successful in using cytoreductive surgery and intracavitary heated chemotherapy in prolonging the lives of malignant pleural mesothelioma patients.  Dr. Sugarbaker reports some patients surviving five to 10 years using this combination of surgery and heated chemotherapy.

Clinic Trials Recruiting Pleural Mesothelioma Patients

Brigham and Women’s Hospital, in collaboration with the Dana-Farber Cancer Institute in Boston, are currently recruiting patients with malignant pleural mesothelioma for two clinical trials.  The first is a Phase I clinical trial that will study the effects of extrapleural pneumonectomy/pleurectomy decortication followed by a combination of heated chemotherapy drugs Cisplatin and Gemcitabine administered during surgery.  The second is a Phase II clinical trial that will study the effects of pleurectomy/decortication combined with intraoperative heated Cisplatin, then followed by an intravenous administration of sodium thiosulfate to reduce the effects of the Cisplatin.

Dr. Michele Carbone

Dr. Giovanni Gaudino began his collaboration with Dr. Michele Carbone at the Cancer Research Center of Hawaii (CRCH) in early 2008.  Since then, he has been to Hawaii three times, each time taking what he calls “short sabbaticals” from his teaching and research duties in Italy.  While in Hawaii, he has had the opportunity to appreciate the good work of the scientists and the wonderful progress that has been made at the Center.  He and Dr. Carbone are working on finding ways in which they can collaborate even more.  Today Dr. Gaudino is now on the faculty of the Cancer Center as a Professor of Research.

In Dr. Gaudino’s view, it is not easy to run a top-level cancer center in the United States.  It is much more competitive in the United States than in Europe.  Here the universities and the cancer centers are very open to foreigners, and they hire the best scientists from all over the world.  For this reason, the best work and publications and clinical trial results are coming from this country.

Dr. Gaudino also enjoys Hawaii.  It is a beautiful place, of course, but the most important part of his experience here has been what he calls “the human environment,” the friendliness of the people, the Aloha spirit, which he says reminds him of the Mediterranean area.

Mesothelioma Research Requires Collaboration

“I have to say that for any kind of cancer, this collaboration, which is going [on] here, …is, … the only way to proceed, and so I’m very, very glad that it’s here.  The Cancer Center is doing so well.” – Giovanni Gaudino

In Dr. Gaudino’s experience, there is very good collaboration between the scientists at the Cancer Research Center of Hawaii and the clinicians and hospitals in Honolulu.  He feels that the Cancer Center has the potential to develop a network even better than that which they have developed in Italy.  His colleagues, the basic scientists, are working hard to develop new therapies, and their communication with physicians is very good.  The work is very promising and offers much hope to mesothelioma patients.  Dr. Gaudino believes that this collaborative approach is the only way to proceed, whether it concerns research for mesothelioma or for any type of cancer.

The Winning Mesothelioma Research Team

“… every scientist is convinced that his own work is the most important…And this is understandable.  But it’s not true…So,…the winning team is the team made of many different persons sitting around the table and trying to argue, and trying to demonstrate that his own point is the best.  But this is the way things can be done.” – Giovanni Gaudino

Dr. Michele Carbone, the director of the Cancer Center and a well-funded mesothelioma researcher, believes that the multidisciplinary approach to cancer research is the only way to win the battle, and Dr. Gaudino agrees with him 100%.  Only by scientists from multiple disciplines – molecular scientists, immunologists, epidemiologists, and physicians – coming together to share their views will the goal be reached in a fast, efficient way.  This is the only way to get things done.  These scientists, from their varied disciplines, make up the winning team to improve treatment of mesothelioma patients with the goal of prolonging lives.

Working Toward A Cure for Mesothelioma

We hope you have enjoyed your visit with Dr. Giovanni Gaudino, our Visiting Scholar at the Cancer Research Center of Hawaii, as much as we enjoyed the time we spent with him.  We hope this presentation has informed, inspired and encouraged you, and that you have been reassured that you are not alone in this battle.  Scientists and researchers from around the world and from multiple disciplines are collaborating with each other and with clinicians and other physicians to develop new therapies for mesothelioma patients, and in “working towards a cure,” to improve the quality of life for those who suffer from this disease as a result of exposure to asbestos in their work or other environments.

Oxaliplatin

In the search for a cure for malignant mesothelioma, researchers are looking at various other chemotherapy drugs that might be used as a secondary treatment protocol to follow the first-line treatment with Pemetrexed and Cisplatin.  One such drug that is being considered is Oxaliplatin, which goes by the trade name Eloxatin.  Oxaliplatin (Eloxatin) was first developed by Dr. Yoshinori Kidani at Nagoya City University in 1976.  Oxaliplatin (Eloxatin) is an alkylating agent that works by causing damage to the DNA of cancer cells.  Other alkylating agents that are used in the treatment of malignant mesothelioma are Carboplatin and Cisplatin.

Oxaliplatin (Eloxatin) and Gemcitabine Tested as a Second-Line Treatment of Malignant Pleural Mesothelioma in a Clinical Trial

On December 18, 2008,  the Journal of Occupational Medicine & Toxicology, published the article entitled Gemcitabine Combined with Oxaliplatin in Pretreated Patients with Malignant Pleural Mesothelioma: An Observational Study.  This paper was based on a clinical trial of 29 patients who were diagnosed with malignant pleural mesothelioma and had been previously treated with Cisplatin and Pemetrexed.  These patients were given a combination of Gemcitabine and Oxaliplatin as a second-line treatment for their malignant pleural mesothelioma.  It was found that when at least three cycles of Gemcitabine and Oxaliplatin were given to these patients in this clinical trial, 6.9% obtained partial remission and another 37.9% achieved stable disease.  This amounted to a malignant pleural mesothelioma control rate of 44.8%.  Just as important, the patients tolerated the treatment well with no significant toxicities or side effects reported in any of the patients.  This drug combination provides great hope and promise in mesothelioma treatment.

New Clinical Trial Using Oxaliplatin (Eloxatin) and Bortezomib (Velcade) in Patients with Malignant Pleural Or Peritoneal Mesothelioma

Columbia University opened a new clinical trial that began recruiting patients with malignant pleural mesothelioma or peritoneal mesothelioma in October 2009 that will use Bortezomib (Velcade) and Oxaliplatin (Eloxatin) for treatment.  To be included in this clinical trial, patients must have received only one prior form of chemotherapy.  Patients who received a combination of Pemetrexed plus Cisplatin or Pemetrexed plus Carboplatin will qualify for this clinical trial.

Many of us know that malignant mesothelioma is very difficult to treat.  Dr. Giovanni Gaudino, the prominent Italian medical researcher, talked to Galiher DeRobertis Ono about mesothelioma treatment and the hope his research offers.  He said:

“… since this cancer is so aggressive, since the incidence of this cancer is increasing . . . we have to go fast to find new therapies, effective therapies.  And the only way to do that is to foster as much as possible clinical trials.  I know that clinical trials . . . can be seen as an experiment on humans . . . And for individuals this could be hard to accept . . .  to be part of an experiment.  But at this point, clinical trials, I think, are the best way to try something new to get rid of this disease.”

Clinical trials are of paramount importance in the search for a cure for malignant mesothelioma.  There is general agreement that in most cases, conventional therapies are not working.  Time is of the essence, but it takes a very long time for new therapies to be approved by the Food and Drug Administration (FDA) in the United States.  The story is the same in Europe and in India – it takes a long time to approve a new treatment.  Clinical trials are a chance for patients to be exposed to novel therapies.  In some instances, existing, approved drugs are repurposed in new combinations.

To find a list of clinical trials, you can go to the National Cancer Institute’s (NCI) website.  With almost 100 clinical trials listed on this site, all with different drug combinations and approaches, this can be confusing.  How do you decide which therapy is the best?

You may wonder if one clinical trial is better than all others.  This is not the case.  Cancers, and mesothelioma in particular, come in many different forms.  As a result, no one therapy is right for all patients.  One therapy may be effective for one patient, or one population of patients, but not for others who may find other therapies are more effective.

The evaluation and initial determination of whether one qualifies for a particular clinical trial is an important discussion a patient must have with his or her physician.  In general, patients must be carefully selected for these trials.  Patients must meet certain criteria to participate in any given study.  You cannot simply choose a clinical trial.  You must be evaluated by physicians to see  if you have the right “markers” for enrollment in the clinical trial.  While enrollment in clinical trials offers no guarantee that they will work, all patients should be encouraged to consider them.

The International Mesothelioma Program (IMP) at Brigham and Women’s Hospital in Boston, Massachusetts is developing a new treatment aid that may assist physicians in prescribing the most appropriate form of chemotherapy for their mesothelioma patients.  This tool is called the Extreme Drug Resistance (EDR) assay.

Extreme Drug Resistance (EDR) Assay

To do an EDR assay, physicians at Brigham and Women’s Hospital remove a mesothelioma tumor fragment during surgery (extrapleural pneumonectomy or pleurectomy/decortication) and send it to the laboratory of Oncotech, Inc. in Tustin, California.  The cultured tumor is then tested against various chemotherapy drugs that are commonly prescribed for malignant mesothelioma.   Brigham and Women’s Hospital has Oncotech expose the tumor to Cisplatin, Gemcitabine, a combination of Cisplatin and Gemcitabine, and Vinorelbine.  As of now, Alimta, which is a drug frequently used to treat mesothelioma, cannot be used in the EDR assay, but the technical problems associated with Alimta in this test are being worked out, according to Dr. William Richards, Operations Director of the Brigham and Women’s Hospital Tissue and Blood Repository.

Oncotech then determines whether the mesothelioma tumor for that particular patient is resistant to each of the chemotherapy drugs or the combination of drugs.  If the EDR assay shows that a cultured tumor is extremely resistant to a particular drug, then there is a 99 percent probability that the tumor in the patient will also be resistant to that drug or drug combination.

Next Step: Clinical Trial Testing

According to Dr. Gavin Gordon, co-director at the Brigham and Women’s Hospital Thoracic Surgery Oncology Laboratory, the EDR assay for malignant mesothelioma must still be tested in a clinical trial for it to be approved as part of the treatment protocol.  There are case reports where the predictive value of the EDR assay has shown a very high correlation between the in vitro test and actual drug resistance in the patient.

In a published case report from the Kobe University Graduate School of Medicine in Japan, physicians used a test similar to the EDR assay called the collagen gel droplet embedded culture-drug sensitivity test (CD-DST) to identify the correct treatment for malignant pleural mesothelioma in a 63 year old woman.  The CD-DST test showed that the patient’s tumor was particularly sensitive in vitro to Gemcitabine and Vinorelbine.  Based on this result, her physicians selected these chemotherapy agents for her treatment.

The EDR assay test could be an invaluable tool in the treatment of malignant pleural mesothelioma and peritoneal mesothelioma, diseases that have shown a wide variability in response to different chemotherapy agents.

Gleevec: A New Role for a Classic Drug

“So we started in this direction and we used … Gleevec and we observed, of course, Gleevec was blocking all the targets we identified . . . .”
– Giovanni Gaudino

Touted by some doctors as one of the most impressive drugs in 20 years when it first came into use in 2001, Gleevec is well known as a successful drug for the treatment of some forms of leukemia and gastrointestinal tumors.  Gleevec is the brand name; the chemical name is imatinib mesylate.  Imatinib mesylate is a specific inhibitor of the receptor tyrosine kinase (the “big button” described earlier) called PDGFRB.  This receptor is widely expressed in most mesotheliomas and is one of the molecules critical to inducing chemoresistance.  With the discovery of the importance of these receptors, it was thought that Gleevec, a classic drug, could be used to inhibit these receptors and thus overcome the tumor cells’ chemoresistance and allow conventional chemotherapeutic drugs to work better.  Gleevec blocked all the identified targets. In lay terms, Gleevec kills abnormal cells and has very little effect on the normal cells.  When it was first used in the treatment of chronic myeloid leukemia (CML), the use of Gleevec was a medical breakthrough.  The primary investigator responsible for the leukemia research at the time dreamed of the day when Gleevec would be effectively used to fight other cancers.  This repurposing of existing, approved drugs such as Gleevec helps to bring new treatment therapies to patients more quickly and efficiently.

The next step for Dr. Gaudino’s research project was to test Gleevec with different chemotherapeutic drugs to see if this specific inhibitor could be used in combination with standard chemotherapeutic drugs to impair tumor resistance.

The Animal Experimental Model

“We moved to [an] animal experimental model, and we used … quite a peculiar model.  We exploited the so-called genetic engineering.  We modified human mesothelioma cells by introducing a foreign gene – a gene coming from a fish – which induce luminescence on these cells.  So these cells basically were emitting lights.”
– Giovanni Gaudino

Gleevec and its combination with other drugs had to first be tested on animals.  In preparation for animal testing, Dr. Gaudino and his team created a very special model.  By the use of genetic engineering, they modified human mesothelioma cells by introducing a foreign gene, a gene from a fish, which caused the cells to emit light.  This enabled the researchers to follow the growth or reduction of the tumor in the live animals by the use of imaging, in the same way a physician uses CT scans or other imaging techniques to see what is going on inside his patient.  So they injected special mice, called SCID mice, with the modified human mesothelioma cells and were able to observe the formation of the tumors by the use of imaging.

Gleevec and Gemcitabine: A New Combination

Next the cancer researchers treated the animals by infusing the tumors with Gleevec combined with different chemotherapeutic drugs, and they found that Gleevec and Gemcitabine were a successful combination.

“It’s quite clear that the treatment … with the two drugs, Gemcitabine and Gleevec . . . reduce a lot of the size of the tumors . . . .  And also, increase quite remarkably the survival of the mice. … [T]he pictures of the mice, I think, are quite convincing because you can see that the right mouse is doing much better than the left one.”
– Giovanni Gaudino

Gleevec & Gemcitabine (Click image to zoom)

Gemcitabine is an old and very well known drug. Gemcitabine is commonly called by its brand name Gemzar.  Gemcitabine has been approved by the FDA since 1996 and is used to treat some lung cancers and pancreatic and ovarian cancers. Dr. Gaudino’s research team knew that Gemcitabine alone was not effective on mice, neither was it effective on mesothelioma patients. But it became quite clear that the combination of Gemcitabine and Gleevec resulted in the reduction in the size of the tumors, which they could measure through imaging, and it also increased, “quite remarkably,” the survival of the mice.

From Mice to Patients: From Pilot Study to Phase II Clinical Trial

The next step, of course, was to move from animal studies to patients.  According to Dr. Gaudino who is involved in bench research, the network the scientists have established is very useful and important, because the results of their work will be published in scientific journals and this will “catch the interest of our clinician colleagues.”

Pilot Study with Gleevec and Gemcitabine (Click image to zoom)

So they started with what is known as a “pilot study” with the combination of Gleevec and Gemzar.  A pilot study comes before the real clinical trial and is usually done on a limited number of patients.  In this case there were 21 patients who had not responded to first-line conventional therapies.  The results were very encouraging: In six patients there was no progression of the disease (stable disease); in one patient there was a complete disappearance of the cancer (complete response); in eight patients there was incomplete reduction of the tumor (partial response); and in six patients, the disease didn’t stop and unfortunately the cancer continued to grow (progression).  Some patients respond better than others and some do not respond at all, which is common in clinical trials.

“And, you know, you may argue that this is not a big deal, just to stop the cancer – you won’t get rid of the cancer.  But for mesothelioma, which is so aggressive, this is a very good result.  We got also one complete response, which means complete disappearance of the cancer.”
– Giovanni Gaudino

This pilot study was followed by a phase II second line clinical trial, which is ongoing and is being validated by the National Cancer Institute (NCI).   It is expected that the results of this study will be coming out in the next few months.  Dr. Gaudino and his team are very optimistic that this therapy will lead to a longer survival.  For mesothelioma patients, the research being done by Dr. Gaudino offers a great deal of hope and promise for longer survival rates and eventually a cure for mesothelioma.

A biochemist and molecular biologist, Dr. Gaudino came from Italy as a visiting scholar to join world-renowned mesothelioma researcher Dr. Michele Carbone and his team of scientists at the Cancer Research Center of Hawaii (CRCH).

According to Dr. Gaudino,

“Malignant mesothelioma is a cancer strictly associated with asbestos fiber exposure.  Basically, this is the main cause of this disease . . . .  Unfortunately, the worst part of this cancer is that it is very resistant to common conventional radiotherapy and chemotherapy.”

Asbestos fibers are the main cause of malignant mesothelioma.  Asbestos is a fibrous mineral (magnesium and calcium silicate) used in insulation and fireproofing because it is not affected by heat or minerals and does not conduct electricity.  Asbestos has been used from ancient times.  It has variously been called lithios amiantos,  the “undefiled rock”;  linum vivum, the “living cloth”; and it was the “unquenchable” wick of the perpetual flame lit for the goddess Athena.

There are two major types of asbestos fiber – fibrous serpentine and fibrous amphiboles.  Chrysotile (white asbestos) is the only commercial form of asbestos in the serpentine group.   It comes mainly from Canada and was used in the United States.  The most common forms of amphibole asbestos are amosite (brown asbestos) and crocidolite (blue asbestos), originally found in South Africa and later in Russia.  Less common forms of amphibole asbestos are tremolite, anthophyllite, and actinolite.

All of these fibers enter the lungs through breathing and from the lungs they reach the pleura, the thin membrane covering the lungs and lining the chest cavity.  These fibers cause chronic inflammation by means of what are called “inflammatory cytokines,” which enable the fibers to damage the DNA.  Normal cells are transformed into mesothelioma cells by a process called “cell survival.”  In other words, instead of dying, as they are supposed to, the damaged cells survive and they transform into tumor cells.

Mesothelioma is a very aggressive, invasive tumor of the mesothelial serosal pleural lining.  However, it can also develop in the similar linings found around the heart (pericardium) and the abdominal cavity (peritoneum).  Unfortunately, malignant mesothelioma is very resistant to chemotherapy and to radiation therapy, and the medium survival for patients has been 12 to 18 months.

“Given the difficulty of medical research in this day and age, very few important medical breakthroughs can be accomplished by an individual researcher, no matter how smart.  Those days are gone.  Current medical research requires a team approach and I believe that assembling the best team of scientists I can bring together at the CRCH to work on the difficult issues of malignant mesothelioma is the best way to proceed.”

Dr. Michele Carbone

Dr. Carbone is also the Chair of the Department of Pathology at the John A. Burns School of Medicine at the University of Hawaii.  Dr. Carbone was recruited to the cancer center in 2006 from Chicago and brought with him approximately $12 million in funding from grants and other sources.  As such, he is the most highly funded mesothelioma researcher in our country.

Mesothelioma is a cancer caused by breathing asbestos fibers.  It is a latent disease occurring 10 to 50 years after exposure and at present is incurable.  Dr. Carbone’s life commitment is to understand how mesothelioma cells develop and why and what measures can be taken to stop their growth.  He is one of the editors of a book entitled, “Malignant Mesothelioma: Advances in Pathogenesis, Diagnosis and Translational Therapies”.

Speaking with a charming Italian accent, Dr. Carbone originates from Rome and Calabria, a region located in the toe of the boot-shaped peninsula of Italy.  After graduating from medical school at Universita Degli Studi Di Roma La Sapienza in Rome, Carbone worked as a fellow at the National Institutes of Health in Bethesda, Maryland for eight years.  He then did his residency at the University of Chicago Hospitals in Anatomic and Clinical Pathology.  Prior to coming to CRCH, Carbone was at Loyola University Medical Center in Chicago, Illinois.

Dr. Carbone and Gary Galiher listening to a presentation on the latest mesothelioma research at the CRCH

With his experience, talent and commitment, Carbone dreams of greatly expanding his mesothelioma research at the cancer center, especially in the area of therapeutic care for mesothelioma patients.  As a vital part of this effort, Carbone collaborates with researchers around the world who are working with new mesothelioma treatment drug therapies as well as with clinicians in hospitals.  Dr. Carbone, a leading mesothelioma researcher in the U.S., and Gary Galiher, a leading attorney who has represented mesothelioma clients for over 30 years, are working together to give Hawaii the first center for excellence on asbestos and mesothelioma in the country.

My husband Gary Galiher and I also have a very personal experience with cancer treatment and clinical trials.  In 1997, sixteen years after I began working with mesothelioma clients, our daughter was diagnosed with acute lymphocytic leukemia at the age of four.

Like you, I was shell shocked and devastated when the oncologist explained our daughter’s diagnosis.  It was hard to believe the words coming out of his mouth.  After that initial shock, however, I decided that I would follow every aspect of her treatment each step along the way to ensure her remission and cure.

As you face your or your loved one’s diagnosis of mesothelioma, I would like to share some important lessons our family learned from our daughter’s diagnosis and the 2 1/2 years of treatment that followed.

Clinical Trials Are the Best Hope for a Mesothelioma Cure

Many call it a medical miracle that childhood leukemia, which was almost always fatal only 30 or 40 years ago, now has a cure rate of 85% or more.  These phenomenal cure rates were accomplished through clinical trials.

Unlike adults, the majority of children with cancer participate in a clinical trial for their treatment.  Today, participation in a clinical trial is the standard of care for a child with cancer.  Among adult cancer patients, however, only 3% participate in clinical trials.

Curing childhood leukemia is the story of many committed scientists, including researchers in the laboratory together with physicians treating young patients, working collaboratively by involving the sick and dying children in clinical trials over many years.  A cure for malignant mesothelioma will be found by patients like you participating in clinical trials.

You Must Be Your Own Best Advocate

The first lesson we learned was that you must be your own best advocate.  Because clinical trials have certain criteria for participation, you need to ask your oncologist what is available.  Be proactive.

Ask your doctor to explain to you which study or studies he thinks you might be able to qualify for.  Ask which one he would recommend for you and why.  If there is a cancer research center or a university medical school in your area, they may have information about what is available.

The Protocol and Roadmap

Participation in a clinical trial will mean that a medical professional, sometimes a nurse clinician, will explain to you the treatment protocol, as well as the Roadmap and all of the informed consent forms that require your signature.  The Roadmap is a detailed schedule of when each drug must be administered, in what dosage and which tests and procedures must be done along the way.

All of the paperwork and very technical medical language may seem overwhelming.  It is a good idea to have someone from your family there with you to listen and ask questions as well.  If you don’t understand something, do not be afraid to ask.

Your physician or the nurse clinician will be able to explain things in lay terms that will be easier to understand.  This is about you and your loved ones.  You want to take every possible measure to fight your cancer, so don’t be afraid to ask that things be repeated or re-explained.  There is no such thing as a dumb question.

Have a way to organize all of the papers being given to you.  I used a three ring binder with dividers.  I marked on the Roadmap the date the treatment was actually administered.  Sometimes treatment has to be postponed due to infection or other problems.  By staying organized, you will know that you received all of the treatment set forth for your study.

Higher Standard of Care

Participating in a clinical trial will mean that you will have a higher quality standard of care than the average patient.  This is because the study you are on will require very close monitoring of your condition.  You will have certain tests such as blood tests, x-rays or other procedures that must be administered according to schedule.

Your doctor who is participating with you in the study will be providing information to the principal investigator of the study or somehow forwarding your data so that it can be compared with other study participants. You are essentially taking part in a well-controlled human experiment.

You may wonder why you couldn’t just get the medications being used in a study without the bother of the paperwork and formality of actually participating in a trial.

In fact, many doctors around the country may be administering the very same medications being studied and some are probably experiencing certain successes in helping their patients.  However, if everyone in their own locality acts separately, there is no way for doctors to share their experiences and analyze the scientific data.  Unless this data is collected through a clinical trial, researchers will be unable to identify the most promising treatments, and the cure for mesothelioma will be even further away in the future.

Participation in a clinical trial may not only help to extend your life and boost your quality of life, but will also help medical research to find the best possible treatment and eventual cure for this devastating disease.

A Fighting Spirit and Hope Are Strong Medicine

Lastly (and this is not medical or legal advice) have a fighting spirit!  Having hope and the will to make every day count helps you maintain a healthy, strong mind to face adverse circumstances.

I encourage you to ask your doctor today to tell you about the clinical trials available to you.  Our daughter is now 17 years old and is a senior in high school.  I am still helping mesothelioma clients, and I hope our personal perspective will assist you as you go forward in your treatment.