Drug Therapy for Pleural Mesothelioma Care

Combination Drug Therapy Approval for Lung Cancer Opens Door For Targeted Care for Pleural Mesothelioma

Lung cancer patients with a specific genetic mutation just received good news with the approval of a combination drug treatment. The U.S. Food and Drug Administration announced in June that it approved dabrafenib and trametinib as a combination therapy for use in non-small cell lung cancer patients expressing the BRAF V600E mutation. Certain pleural mesothelioma patients may also benefit from this recent approval.

The FDA approved the two drugs from Novartis Pharmaceuticals Inc., Tafinlar (dabrafenib) and Mekinist (trametinib), based on success in a clinical trial of 93 patients with a median duration of response that ranged from six months to 12.6 months (depending on previous treatment status), according to a June 22 press release. The FDA also approved the Oncomine Dx Target Test to select those patients with NSCLC with the BRAF V600E mutation for treatment.

Novartis reports that “there is an urgency to treat people with this mutation, as BRAF V600E mutation-positive tumors have been shown to be more aggressive and may lead to a poorer prognosis.” According to Novartis, BRAF mutations are in just one to three percent of NSCLC cases worldwide. One study shows that BRAF is also a rare type of mutation in pleural mesothelioma, however, for an already rare disease with few treatment options, this approval brings a glimmer of hope to patients.

“Patients with BRAF V600E mutation-positive metastatic NSCLC have responded less favorably to standard chemotherapy, suggesting that there is a critical need for a targeted therapy,” said Bruno Strigini, CEO, Novartis Oncology, in a June 22 press release.

Treatment for pleural mesothelioma, an asbestos-caused cancer affecting the lining of the lungs, and non-small cell lung cancer is often similar, with chemotherapy being one of the primary treatment modes. Chemotherapy is used to manage symptoms and to slow the growth of the disease, but both cancers are notably aggressive and often develop resistance to the drugs, and, eventually, the treatment fails.

This double therapy oral treatment is another step towards personalized cancer care that could benefit those battling mesothelioma. Personalized care targeted to a patient’s unique characteristics and genetic makeup, such as the BRAF biomarker, optimizes the potential for success of the treatment.

“This is an important milestone for the lung cancer community as we are continuing to better understand the genomic drivers of cancer and develop effective treatments targeted for these biomarkers,” said Bruce Johnson, MD, Professor of Medicine, Chief Clinical Research Officer, Dana-Farber Cancer Institute and Harvard Medical School at Dana-Farber Cancer Institute.

This is the first approval for targeted treatment in the U.S. specifically for BRAF V600E mutation-positive metastatic NSCLC.

Talk to your mesothelioma care team to find out if this combination therapy will work for you.

Daughter of Mesothelioma Victim Holds Memories

A song. A simple tune that other passers-by don’t even notice. A smell, wafting on the breeze. A place, so full of meaning, that sharing it with other people seems like you’re telling a secret. These are all triggers for me; triggers of beautiful memories of my father. Knowing that I don’t have the chance to make new ones with him, makes me protective and possessive of the ones I hold so close to my heart.

Dad loved music and he was a beautiful singer. Even though he played with his bluegrass band, most of my memories of him singing were a bit different. Yes, he always sang me “You Are My Sunshine,” but there was more to it than that. I remember during our infamous Saturday morning adventures when he would sing along with me to the radio when REM or No Doubt was playing. I remember the time he went to an NSYNC concert with me, just because.

Along with music, my father was a lover of food. It didn’t have to be a delicacy, in fact, he would prefer it not to be. The smell of no-bake cookies reminds me of the first time we “baked” together, unsuccessfully, may I add. How we managed to mess them up, I’ll never know. The fun was in the experience.

Every time I smell a beautiful fall breeze, it takes me back to nights spent with him in the yard at our home, playing outside. Volleyball became a specialty, and during the last set of the night, we would both agree that we would go inside once the ball hit the ground. This led to bouts of hilarious laughter as we would run around, doing everything we could to make the evening last just a bit longer.

Dad and I shared so many memories in various places. Some of the memories I treasure the most are ones that I choose not to share. They are embedded deep within me; the stories that usually ended with “Don’t tell Mom!” are ones that only we knew. Now, I am left to reminisce about these alone. They were our secrets, and I intend to continue keeping them.

Now, my memories of my beautiful father are all that I have left of him. This harsh truth of my new reality reminds me to treasure each day. As you walk through life, stop and think that you might be creating memories that your loved ones will carry with them for years. Live each second with that thought; if you do, you’ll do a lot more living, and create a lot more unforgettable moments.

Know more about mesothelioma and how to deal with it.

Two Gene Therapys Brings Hope to Mesothelioma

Two Gene Therapy Approaches Pending Approval from FDA Bring Hope to Mesothelioma Community

Nearly five years ago, MesotheliomaHelp reported about a breakthrough treatment called gene therapy. At the time, it was touted as the “next frontier” in medicine, and cancer patients from around the world watched closely in the hopes that the treatment could bring a cure to even the rarest of cancers, such as mesothelioma. Now, all eyes are on the U.S. Food and Drug Administration as it is poised to approve two types of gene therapy.

The Oncologic Drugs Advisory Committee (ODAC) sent its recommendation to the FDA on July 12 for CTL019 (tisagenlecleucel) for the treatment of a form of leukemia. Then, on July 17, the FDA accepted a Biologics Licensing Application from Spark Therapeutics for gene therapy for a rare inherited eye disease that causes blindness, approved the name Luxturna for the treatment, and assigned priority status to the treatment for accelerated review.

To better understand these two pending landmark approvals and the future of gene therapy, MesotheliomaHelp turned to Ricki Lewis, a New York-based geneticist and author.

“It’s not right for every disease,” said Lewis. “But it is an approach that can be considered some day along with drugs, surgery and everything else.”

CTL019: CAR-T Treatment for Leukemia Ramps Up the Immune System

Tisagenlecleucel is an investigational chimeric antigen receptor (CAR) T cell therapy from Novartis, developed by researchers at the University of Pennsylvania. The pharmaceutical company wants to use the therapy to treat a rare form of leukemia, B-cell acute lymphoblastic leukemia affecting children and young adults under the age of 25, according to NPR.

Lewis explains that CAR-T therapy is not “conventional” gene therapy, which has been in clinical trials to treat single-gene diseases since 1990. However, she notes that “CAR T cell technology has had astonishing success in treating a form of leukemia and it’s being tested for multiple myeloma, brain cancer, breast cancer, and soft tissue cancers.”

“Although both approaches deliver DNA in viruses, classical gene therapy adds a working copy of a single mutant gene, restoring a specific protein’s function,” says Lewis. “Revving up a not-naturally-occurring immune response isn’t the same thing as replacing an enzyme,” which is what Luxturna does.

According to the National Cancer Institute, in CAR-T treatment, T cells are removed from the patient’s blood and genetically altered in a lab to have chimeric antigen receptors on their surface. The T cells are then multiplied, into the billions, in the lab and infused back into the patient’s blood, where they seek out the cancer cells and launch a precise immune attack against them.

Lewis offers the following explanation of CAR-T:

“CAR operates like a drone, targeting and obliterating cancer cells. It introduces a gene manufactured to contain instructions for making two immune system components in one, something that doesn’t exist in nature: an antibody and a T cell receptor. When delivered in a virus, the CAR enters the person’s T cells, which then manufacture the hybrid (chimeric) protein.

The engineered receptor portion guides the T cells to a specific target – such as cancerous B cells – where the antibody part binds. The action alerts the immune system to respond and kill the cancer cells.”

Ultimately, CAR-T, also described as a process that genetically alters a patient’s own cells to fight cancer, could be used for many more diseases and cancers, and bring an effective treatment to mesothelioma patients.

In a 2013 article for MesotheliomaHelp, Lewis wrote about CAR-T treatment saying, “An ingenious technique that has vanquished leukemia in a handful of patients is also being applied to mesothelioma.”

Lewis highlighted the CAR-T process being used in a mesothelioma clinical trial from the University of Pennsylvania that uses the “doctored T cells,” known as chimeric immune receptor (CIR) instead of CAR, against mesothelin, a protein that is found to be in excess in mesothelioma and other cancers. The idea is that T cells led to the mesothelioma cells will attract an immune response, said Lewis.

Find out more about the mesothelioma clinical trial from the University of Pennsylvania here.

Luxturna Gene Therapy Treats Blindness, Could Lead to Treatment for Other Inherited Diseases

In her book The Forever Fix, Lewis followed the journey of the use of gene therapy to restore the vision of a young boy who was nearly blind from a hereditary disorder. The doctors added a working copy of a single defective gene in the New York boy’s eyes that prevented his eyes from using vitamin A to send visual signals to his brain. The treatment was a success: the boy’s vision was restored and no further treatments or surgery were required.

“Last week’s FDA advisory committee’s greenlight for CAR technology overshadowed a milestone for what is likely to be the first approval of classic gene therapy – for a form of inherited blindness,” Lewis told MesotheliomaHelp. “That’s the Leber congenital amaurosis type 2 – renamed ‘RPE65-mediated inherited retinal dystrophy’ – that I wrote my book about.”

In an interview with Lewis last week, Dr. Katherine High, MD, President, Chief Scientific Officer, and a founder of Spark Therapeutics, said of the future of gene therapy:

“I hope we will see continued accumulation of successful clinical results in a range of target tissues and continued progress in bringing gene therapy products to licensing. When gene therapy products are licensed, there will be increased interest in the medical community, and that will help to expand opportunities.”

Mesothelioma patients typically show disease symptoms years or even decades after exposure to asbestos, a known carcinogen. The cancer is eventually fatal, but aggressive therapy may prolong the lives of patients who are diagnosed early. Approximately 3,000 Americans are diagnosed with the cancer each year.

“Getting at the basis of why one person develops mesothelioma and another person doesn’t, that is going to hold a clue to really fighting it,” Lewis said, referring to a clinical trial conducted at Wake Forest University in 2013 to determine whether some mesothelioma patients are genetically predisposed to developing mesothelioma. “Then we will know what to do the gene therapy on.”

The pending FDA approvals could bring groundbreaking treatment to cancer patients and to patients with genetic diseases. Perhaps someday, mesothelioma patients will enjoy long, productive lives through gene therapy.

The FDA is not bound to follow the ODAC’s recommendations, however, the Agency nearly always follows the recommendation. Approval for CTL019 is expected in November. The FDA will decide on Luxturna in January, 2018.

About Ricki Lewis,PhD

Ricki Lewis is a science writer with a PhD in genetics. The author of several textbooks and thousands of articles in scientific, medical, and consumer publications, Ricki’s first narrative nonfiction book, “The Forever Fix: Gene Therapy and the Boy Who Saved It,” was published by St. Martin’s Press in March 2012. In addition to writing, Ricki provides genetic counseling for parents-to-be at CareNet Medical Group in Schenectady, NY and teaches “Genethics” an online course for master’s degree students at the Alden March Bioethics Institute of Albany Medical Center.

Read more about gene therapy on Ricki Lewis’s DNA Science blog.

Find out more about Ricki Lewis at her website.

Sources:

Spark Therapeutics
http://blogs.plos.org/dnascience/2017/07/20/luxturna-a-giant-step-forward-for-blindness-gene-therapy-a-conversation-with-dr-kathy-high/
DNA Science blog
http://blogs.plos.org/dnascience/

Nurse Shares Patient’s Hope in His Mesothelioma Journey

In this age of instant communication, text, email, cell phone, it is nice to be able to talk face-to-face with mesothelioma patients and their families. It is a privilege to be included in a journey that changes their lives forever. For one couple, the sharing of their story and the approach they are taking to deal with mesothelioma is inspirational. Their journey, up to and including the diagnosis of mesothelioma, is one that is not unfamiliar.

Four to five months prior to getting the official diagnosis of malignant pleural mesothelioma, the man was treated for pneumonia and shortness of breath. Once the diagnosis was made, he was told it was inoperable. They were told to return when the symptoms became problematic because there was nothing else to offer him.

Not satisfied with waiting, they turned to the internet for more information. Knowing that there is not a cure for mesothelioma, they searched for and found hope instead. The patient and his wife traveled to a large medical center for mesothelioma that sees more patients and could offer them other options. With many more tests and doctors, surgery was once again reviewed as an option, but once again, he was deemed inoperable.

They continue to hold out hope that someone will find something to offer him. During this time they have not let the weight of the diagnosis or the course of the disease take over their lives. They plan on continuing to search for treatment options that are suitable to his body and disease.

The patient knew where his asbestos exposure happened. As a young man, he worked in a shipyard to help pay his way through college. He has always been honest and hard-working and wanted to be successful. He achieved success, yet now his hard work seems to have come back to harm him. Moving forward, they are exploring chemotherapy, clinical trials, radiation, and are leaving their options open.

The couple’s journey has been a roller coaster. When they recently met a newly diagnosed patient their hearts went out to them, as they realize the roller coaster ride has just begun for them. They also know their are plenty of people out there to support them during their journey.

Mesothelioma Patients Seeking Treatment for Depression

Being diagnosed with mesothelioma, seeking treatment and living with the cancer, brings out a lot of different emotions. One of the conditions that can take over a mesothelioma journey is depression. Depression can appear at any point in the journey, and it can become a serious medical condition. The diagnosis and treatment, or lack of treatment, can have detrimental health consequences.

According to Cancer.net, some of the risk factors for depression include being previously diagnosed with depression or anxiety; a family history of depression and anxiety; financial burdens; and lack of support from friends and family. When dealing with a cancer diagnosis it is important to be aware of depression, and not to think it is part of the disease process.

Some of the common symptoms of depression are low energy, feelings of sadness that will not go away, frequent crying, fatigue and loss of motivation. The symptoms can be mild to severe. Severe depression can interfere with your personal relationships, your day-to-day activities and responsibilities.

In men, the symptoms of irritability and anger are more common than in women. Women tend to be more sad, and men more angry. Men are also not likely to identify the symptoms and, thus, are less likely to seek treatment for depression.

The treatment of depression can include psychotherapy, medication, exercise, and/or diet changes. It is important to recognize the symptoms of depression and seek help. Depression is treatable, and once treated can improve your quality of life. Seek help.

The place to start to ask for help can be your primary care doctor, or your mesothelioma team. Reach out – there is support available, and the sooner you are screened and diagnosed the sooner you can be on your way to feeling better. When dealing with mesothelioma it is important to have all the help and support needed that puts your physical and emotional health first. Depression is treatable- you can feel better!

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