Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

    Accelerated Approval Granted to a Novel Drug Combination That Treats Patients with a Common Type of Non-Hodgkin Lymphoma

      Each year over eighteen thousand people in the United States are diagnosed with diffuse large B-cell lymphoma (DLBCL) a common type of non-Hodgkin lymphoma. A recent FDA news release…

    Continue Reading Accelerated Approval Granted to a Novel Drug Combination That Treats Patients with a Common Type of Non-Hodgkin Lymphoma

    Acute Lymphoblastic Leukemia: Miracles for Millions and the People Who Created Them

      According to a recent article in MedicalXpress, the story begins fifty years ago with the grim statistics that a child with acute lymphoblastic leukemia (ALL) would not survive longer than…

    Continue Reading Acute Lymphoblastic Leukemia: Miracles for Millions and the People Who Created Them
    A New Way to Study Disease: Scientists Have Recreated a Blood-Brain Barrier Outside the Body
    source: pixabay.com

    A New Way to Study Disease: Scientists Have Recreated a Blood-Brain Barrier Outside the Body

      The blood-brain barrier is a “gatekeeper” that prevents toxins and foreign matter in the bloodstream from traveling to the brain. Check Orphan recently carried an article that had been…

    Continue Reading A New Way to Study Disease: Scientists Have Recreated a Blood-Brain Barrier Outside the Body

    The New 5F9 Antibody Looks Encouraging as it Targets MDS and AML Leukemia in a Phase 1b Clinical Trial

      The California Institute for Regenerative Medicine (CIRM) has funded a study by a company called Forty Seven, Inc. The clinical trial is investigating the benefits of a drug therapy for two cancers…

    Continue Reading The New 5F9 Antibody Looks Encouraging as it Targets MDS and AML Leukemia in a Phase 1b Clinical Trial

    Legal and Ethical Questions: What is Pharma’s Obligation to Pursue Development of a Promising but Non-Profitable Drug?

      Dr. Craig Klugman, a bioethics professor at DePaul University, expresses his opinion in this recent article in Columbia University’s Bioethics.com. Dr. Klugman begins with a discussion of Pfizer’s biological drug,…

    Continue Reading Legal and Ethical Questions: What is Pharma’s Obligation to Pursue Development of a Promising but Non-Profitable Drug?

    The FDA’s Director of the Center for Drug Evaluation Discusses Accelerated Approvals

      In the mid-1980s the FDA was criticized for being slow in approving new drugs. In fact, a commission was formed as a result of the mounting criticism. Janet Woodcock,…

    Continue Reading The FDA’s Director of the Center for Drug Evaluation Discusses Accelerated Approvals

    The FDA is Reversing Course on Unapproved Products Sold by Stem Cell Manufacturers

      The FDA’s “untitled letter” to R3 Stem Cell (R3), based in Scottsdale Arizona, cautioned R3 that the product they are marketing is considered a drug. It must, therefore, be…

    Continue Reading The FDA is Reversing Course on Unapproved Products Sold by Stem Cell Manufacturers

    The FDA, Congress, A Young Woman Dying of ALS, Her Physician, and Her Parents Are All Struggling Over Access to an Untested Therapy

    1032 words 6% vs 3711 words 2% The Hermstads of Spencer, Iowa lost Jaci’s twin to ALS and now they must watch Jaci, rapidly descend into the depths of the…

    Continue Reading The FDA, Congress, A Young Woman Dying of ALS, Her Physician, and Her Parents Are All Struggling Over Access to an Untested Therapy

    The Challenge: He is A Father, A Caregiver and A Scientist Trying to Save his Son and Others With ME/CFS or Chronic Fatigue Syndrome

      Ron Davis and his gene-sequencing technologies were mentioned years ago in The Atlantic alongside Elon Musk (SpaceX) and Jeff Bezos of Amazon fame. Since then, Davis has amassed over…

    Continue Reading The Challenge: He is A Father, A Caregiver and A Scientist Trying to Save his Son and Others With ME/CFS or Chronic Fatigue Syndrome

    Transthyretin-Mediated Amyloidosis (ATTR-CM); After Nine Years Pfizer Wins FDA Breakthrough Designation

    It has been nine years since Pfizer acquired the drugs vyndagel and vyndamax. According to a report in Biospace, based on the FDA’s recent approval of the two drugs to treat transthyretin-mediated…

    Continue Reading Transthyretin-Mediated Amyloidosis (ATTR-CM); After Nine Years Pfizer Wins FDA Breakthrough Designation

    CRISPR Uncovers New Gene Fusion as Novel Drug Target for Brain, Ovarian and other Cancers

      CRISPR-Cas9, the genome editing tool, has caused quite a stir in the scientific community. According to a recent article in Science Daily, with results published in Nature Communications, CRISPR has…

    Continue Reading CRISPR Uncovers New Gene Fusion as Novel Drug Target for Brain, Ovarian and other Cancers

    An In-Depth Discussion About FDA Oversight of Diagnostic Tests and the Effect of Inaccurate Tests on Patients

      Newly diagnosed patients may decide to either receive medical treatment or forego treatment based on the results of their diagnostic test.  As recently reported in PEW, although it is…

    Continue Reading An In-Depth Discussion About FDA Oversight of Diagnostic Tests and the Effect of Inaccurate Tests on Patients

    Drug Start Up Founder Says Drugs Will Work For You– But They Don’t Know Why

    Although somewhat of an exaggeration, the title of this article falls in line with the thinking of Insitro’s founder, Daphne Koller. In a recent interview with Bloomberg, Koller said that…

    Continue Reading Drug Start Up Founder Says Drugs Will Work For You– But They Don’t Know Why

    A Look at Progress in Healthcare and a Glimpse into the Future

      The Asco Post recently interviewed Dr. Jean Pierre Bizzari, the Executive Vice President of Celgene, a multinational bioparmaceutical company. The interview covered various aspects of research and development (R&D)…

    Continue Reading A Look at Progress in Healthcare and a Glimpse into the Future

    ALZHEIMER’S:  Scientists are Back to Square One; Will Bryostatin-1 be the Missing Link?

      Pharmaceutical companies have spent billions of dollars on Alzheimer’s research. The target has been the toxic protein, amyloid-beta. According to a recent article in NPR News, this theory was…

    Continue Reading ALZHEIMER’S:  Scientists are Back to Square One; Will Bryostatin-1 be the Missing Link?

    New Research With the Potential to Alleviate Symptoms of Parkinson’s, Alzheimer’s and Charcot-Marie-Tooth Disorders

      A recent article in the neuroscience section of the Medical Express reports the results of a study conducted by researchers in the Charcot-Marie-Tooth/Hereditary Neuropathy Center at internationally acclaimed Cedars-Sinai.…

    Continue Reading New Research With the Potential to Alleviate Symptoms of Parkinson’s, Alzheimer’s and Charcot-Marie-Tooth Disorders