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Month: July 2017

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/
Hope in Patient_s Mesothelioma Journey

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

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!

Biomarker Drug for MesotheliomaTreatment

Drug Targeting Biomarker Could Bring New, Personalized Treatment to Mesothelioma Patients

Last month, MesotheliomaHelp reported on the U.S. Food and Drug Administration’s first-ever approval of an immunotherapy drug that targets a particular biomarker, regardless of cancer type. Now, researchers report they have developed another anti-cancer drug that targets just one biomarker and that it has shown “dramatic antitumor effect” across all 17 cancer types tested.

In a report presented by David Hyman, M.D., Chief of the Early Drug Development Service at Memorial Sloan Kettering Cancer Center, at the American Society of Clinical Oncology’s annual meeting, researchers found that when the tropomyosin receptor kinase (TRK) biomarker was targeted by the experimental drug larotrectinib (LOXO-101) “the vast majority of both adults and children whose tumors have this [TRK] mutation” responded to the treatment.

Across three pivotal clinical trials covering 17 tumor types, larotrectinib resulted in a 76% overall response rate in 55 adult and pediatric cancer patients with the TRK mutation. According to the report, of those who responded, 89% were progression-free at the time the data were analyzed, indicating that the cancer had not gotten worse in those patients since starting treatment. These positive results “are potentially practice changing,” said the researchers.

“We are seeing the true potential of precision medicine come to life,” says Dr. Hyman, in a June 3 press release announcing the findings. “It’s groundbreaking to have such a consistent response across multiple cancer types.”

Dr. Hyman said that this is the first cancer therapy to be developed simultaneously in adults and children. He also added that in patients with a cancer carrying the TRK fusion, “it’s found in every cancer cell in that patient.”

Personalized medicine is considered one of the best ways to combat mesothelioma, an asbestos-caused cancer. Tailored care ensures a mesothelioma patient’s unique disease characteristics, including the cancer’s genetic makeup, are targeted when being treated. Research has shown that with targeted therapy success of the treatment is higher.

According to a 2014 article in ScienceDaily, the TRK gene was first identified in 1982, but researchers are only now focusing on the family of biomarkers as a target to treat cancers. The family of TRK genes, including NTRK1, NTRK2 and NTRK3, have shown up in many cancers, including lung, breast, and melanoma.

Dr. Hyman notes that the TRK fusions are rare within most individual cancers. Mesothelioma patients should work with their oncologist to determine if they are TRK positive and if there is a clinical trial that could work for them.

Both the FDA and European Medicines Agency have granted orphan drug designation for larotrectinib (LOXO-101) for treatment of patients with soft tissue sarcoma.

For more information about the LOX-101 clinical trials visit Loxo Oncology’s website, or read more about the NAVIGATE clinical trial at ClinicalTrials.gov.

 

Sources

  • ClinicalTrials.gov
    https://clinicaltrials.gov/ct2/results?term=%22malignant+mesothelioma%22&recr=Open&pg=1
  • Loxo Oncology’s website
    https://www.loxooncology.com/patients-caregivers/our-clinical-trials
  • Memorial Sloan Kettering Cancer Center
    https://www.mskcc.org/blog/asco17-drug-targeting-genetic-mutation-works-across-all-tumor-types
Osteoporosis Drug Can Help Mesothelioma Patients

Mesothelioma-Fighting Benefits Found in Osteoporosis Drug

In April, MesotheliomaHelp reported on the potential benefits of using a drug for the treatment of multiple sclerosis in the fight against mesothelioma. Now, researchers report a drug used in the treatment of osteoporosis may also help mesothelioma patients.

Researchers from the Comprehensive Cancer Center at the University of Alabama at Birmingham conducted a pilot study to evaluate the antitumor activity, duration of response, and progression-free survival of malignant pleural mesothelioma patients treated with zoledronic acid. Given as an intravenous infusion, zoledronic acid is used to treat osteoporosis, a condition characterized by low bone density, decreased bone strength and increased susceptibility to fractures.

The drug is also sometimes used with cancer chemotherapy treatments to treat bone problems that may occur with various cancers, including lung cancer, that have spread to the bones, according to WebMD. The drug lowers high blood calcium levels by reducing the amount of calcium released from the bones into the blood, as well as slowing the breakdown of bones by cancer, aiding in the prevention of bone fractures.

Preclinical studies showed that zoledronic acid could inhibit mesothelioma growth through apoptosis (cell death), and by inhibiting the growth of new blood vessels, as well as through other means. To assess these results, the researchers used zoledronic acid to treat eight patients with unresectable pleural mesothelioma who had progressed on prior treatments.

The team reported positive results including median progression-free survival of two months and median overall survival of seven months, with no toxicities in the patients. In addition, the team found that vascular endothelial growth factor (VEGF) amounts “were predictive of favorable response.” VEGF is an autocrine growth factor released by mesothelioma cells, causing new blood vessels to form, and is shown to be higher in mesothelioma patients.

“Our pilot study suggests modest activity of zoledronic acid as a single agent in the treatment of mesothelioma and warrants further investigation in combination with other agents,” the researchers concluded.

Mesothelioma is an incurable cancer that invades the lining of the organs after exposure to asbestos. The disease can take decades to develop, but once diagnosed, patients often receive a prognosis of less than 18 months. However, effective treatment can improve survival and quality of life.

Using a drug already approved by the U.S. Food and Drug Administration lessens the time and costs for approval of the same drug for other diseases.

To find out more, read the full study in Lung Cancer: Targets and Therapy.

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