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    The U.S. Food and Drug Administration (FDA) has approved — with conditions — Xpovio (selinexor) tablets, in combination with the corticosteroid dexamethasone, for the treatment of adults with relapsed or refractory multiple myeloma who received at least four prior therapies and failed to respond to treatment. The combo is designed for patients whose disease is resistant to several other forms of treatment, including at least two proteasome inhibitors, two immunomodulatory agents, and an anti-CD38 monoclonal antibody.

    The endorsement — called accelerated approval by the FDA — allows a medication addressing an unmet need in a serious condition to be used if it shows benefit on surrogate or interim measures made in a clinical trial. But its continued use, and full approval, requires a further showing of efficacy in a confirmatory study.

    Xpovio, Karyopharm Therapeutics‘ lead compound, is a first-in-class oral inhibitor of the XPO1 protein that prevents tumor suppressor proteins from exiting the cell nucleus. This leads to cancer cell death while leaving healthy cells unharmed.

    After designating Xpovio an orphan drug, the FDA granted it fast track status, and now has given the therapy accelerated approval.

    “Today we approved a treatment under our accelerated approval program that provides a treatment option for patients with multiple myeloma with no available therapy,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, said in a press release.

    “While there is no cure for multiple myeloma, there are FDA-approved treatments to target the cancer and slow down the spread of the disease. Sadly, often over time, patients can exhaust all available treatments and still see their disease progress,” said Pazdur, the acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research (CDER).

    “With today’s accelerated approval of Xpovio by the FDA, patients with heavily pretreated multiple myeloma will now have a new therapeutic option to treat their disease,” Sharon Shacham, PhD, MBA, founder, president, and chief scientific officer of Karyopharm, said in a company-issued press release.

    “Discovering, developing and securing FDA approval for Xpovio with its novel mechanism of action over the past decade required the dedication of many people, including the patients, caregivers and physicians involved in our clinical trials, along with the many employees at Karyopharm. We are tremendously grateful for everyone’s contributions to this important milestone, and we look forward to the next stage in our pursuit of improving the lives of patients with cancer,” Shacham added.

    The FDA’s accelerated approval was based on findings from the multicenter, single-arm, open-label, STORM Phase 2b trial (NCT02336815).

    The study assessed the safety and efficacy of Xpovio (80mg), in combination with dexamethasone (20mg), in relapsed or refractory multiple myeloma patients who had received at least three prior therapies. The treatment was given twice per week until disease progression, death, or unacceptable toxicity.

    In Part 2 of STORM, treatment efficacy and safety were assessed in a group of 83 people who failed to respond to therapy with two proteasome inhibitors, Velcade (bortezomib) and Kyprolis (carfilzomib), two immunomodulatory agents, Revlimid (lenalidomide) and Pomalyst (pomalidomide), and the anti-CD38 monoclonal antibody Darzalex (daratumumab).

    Study findings showed that 25.3% of patients responded to the Xpovio-dexamethasone combination, with a median duration of response of 3.8 months and a median time to first response of 4 weeks.

    The most common side effects of treatment reported in the 202 patients who participated in Part 1 and 2 of STORM were low platelet count (thrombocytopenia), low white blood cell count (neutropenia), and low red blood cell counts (anemia). Other common side effects included fatigue, nausea, vomiting, diarrhea, decreased appetite, weight loss, constipation, upper respiratory tract infections, and low blood sodium levels (hyponatremia).

    Treatment was discontinued due to side effects in 27% of the participants. More than half of the patients (53%) required a reduction in the dose of Xpovio during the trial, and 65.3% had to interrupt treatment with Xpovio. Fatal adverse reactions were reported in 8.9% of patients.

    Xpovio’s effectiveness, in combination with Velcade and low-dose dexamethasone, will be confirmed in the ongoing, randomized, BOSTON Phase 3 trial (NCT03110562). If proven effective, Xpovio will receive FDA’s full approval to be used together with dexamethasone as a therapy for heavily treated patients.

    According to Karyopharm, Xpovio should become available to patients living in the U.S. no later than July 10, 2019. The full prescribing information, including dosing, scheduling and side effects, can be found here.

    The company, meanwhile, is waiting for feedback from the European Medicines Agency (EMA) regarding its marketing authorization application (MAA). That MAA seeks the approval of Xpovio for the same indication in the E.U.

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    FDA Approves Xpovio Combo for Heavily Treated RRMM Patients

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    Joliet cancer survivor has 4 reasons to live

    'If you keep praying to God, everything will turn out all right.'
    Carolyn Tucker is a five-year cancer survivor and she's extremely thankful.

    Especially since Tucker, 63, of Joliet, is raising four young grandchildren, all boys, ages 3, 4, 5 and 6.

    Tucker recently participated in Celebrate Life, which is hosted by Cancer Treatment Centers of America Chicago (CTCA) and recognizes five-year cancer survivors from across the United States.

    After the red-carpet walk, Tucker, took part in a commemorative tree planting ceremony, according to a news release form the CTCA. The 2019 Celebrate Life event marks the 31st year that a tree has been planted in honor of each five-year survivor in attendance, the release also said.

    “When I received my invitation to Celebrate Life from Cancer Treatment Centers of America Chicago, I began shouting with joy,” Tucker said in the news release. “Over the past five years, I have been able to travel to India, Alaska, Haiti and the Bahamas. I’ve also been blessed to watch my five children and 13 grandchildren grow up.”

    The India trip, Tucker said, was to attend the wedding of one of the nurses at CTCA.

    "Gogog. Gogog. Gogog. Gogog."

    That was the noise Carolyn Tucker's heart started making one day. Tucker, who said she was working as a school secretary at the time, had only one explanation for it.

    "I have gas," Tucker said. "So I went to the pharmacy said said, 'What can I take for gas?'"

    But the remedy didn't work. So Tucker, who wasn't in pain, went to the hospital. She learned her heart was "swimming in fluid" and had the fluid removed. Over the next couple of weeks, Tucker's doctor "ran every test he could" to find the problem.

    In the meantime, Tucker went to work. Tucker figured she had a bad heart, just like her sister.

    She was wrong.

    And she was also not prepared to hear the words "multiple myeloma."

    Tucker said it felt like someone dropped a bomb on her.

    "I hadn't even thought about cancer," Tucker said.

    She would have preferred a heart ailment.

    "You can fix a heart problem," Tucker said. "But you can't fix cancer."

    At first, Tucker questioned the cancer diagnosis since the report had mistakes in her race and age. She went to the Cancer Treatment Centers of America in Zion for a second opinion and is glad she did.

    Tucker still had cancer. But Tucker no longer felt the disease was a "death warrant."

    "It had a friendly feeling, like walking into a five-star hotel," Tucker said. "The doctors know who you are; the nurses know who yo are. Everyone treats you really nice. You become part of a family the way they treat you. They act like your. Mama."

    Tucker was ultimately treated with chemotherapy and two stem cell transplants, she said. But during that time, Tucker also learned about the best nutrition for her situation. She even had access to massages, she said.

    "It makes you want to fight," she said.

    Most of the time, though, Tucker did not want to talk about her cancer, even to the health professionals at CTCA.

    "They did their job and I did not question them," Tucker said. "They said they would take good care of me and I let them do their job. But I knew they were doing a good job because I was getting stronger. My color was good and I was happy."

    (Carolyn Tucker is seen with Pete Govorchin, president and chief executive of Cancer Treatment Centers of America (CTCA), Chicago).

    Tucker said after the transplants she took a year off work because she was still weak. Then two years ago, Tucker became a full-time single grandparent when she accepted the care of those four little boys, age 1, 2, 3 and 4 at the time, she said.

    It wasn't easy.

    "I had terrible twos back to back and I was sick," Tucker said. "I didn't have an immune system and I kept catching colds. I had pneumonia three times in less than six months."

    With a laugh, Tucker recalled how she and the boys received their "baby shots" together, referring to the childhood immunizations she had to receive all over again after the stem cell transplants.

    Tucker still goes to CTCA for her routine blood work, she said, and doesn't dwell on her past cancer experiences.

    "I'm not sad. I'm a happy woman," Tucker said. "I've got a team working with me and they pray with me. What more can I say? I'm thankful for what they've done for me and what they continue to do for me."

    Instead, Tucker focuses on her present responsibilities.

    "The boys are doing good in school," Tucker said. "I had to start from scratch with their manners and I had to buy a van to put them in; you have to have car seats for everything. "

    Tucker said money is tight and she can't afford babysitters. But she's proud of her role and proud of making a difference in her grandsons' lives, right down to starching and ironing their pants, she said.

    "I want to teach them about God and I want to teach them how to pray and keep the faith no matter what," Tucker said. "If you keep praying to God, everything will turn out all right."

    From The Herald News

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    So true, has anyone else had this trouble?

    1 Comment
    • GregP_WN's Avatar

      My new me after my first two diagnoses was the same, [mostly] as before. But the third diagnosis changed lots of things about me. My side effects are numerous, some simple, and some serious and getting worse. So I'm not liking my newest me.

      17 days ago