The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Nammi Therapeutics’ lead immunotherapy candidate QXL138AM for multiple myeloma. The first-in-class therapy is designed to kill targeted cancer cells without the “significant toxicities” of other treatments also based on the interferon-alpha (INF-alpha) protein, Nammi said in a press release. “By reducing systemic…
Category: Cancer
CYT-338 Shows Anticancer Activity in Preclinical Tests
CYT-338, an investigational myeloma therapy designed to simultaneously bind to cancer cells and activate immune cells called natural killer cells, has shown promising anti-cancer activity in preclinical studies. A team led by scientists at Cytovia Therapeutics, the company developing CYT-338, will present the findings in a poster this month at the European Hematology Association’s 2022…
CAR T-cell Therapy Safe, Effective in Treatment-resistant Myeloma: Study
A CAR T-cell therapy called CART-ddBCMA that targets the protein BCMA — produced at high levels in myeloma cells — safely led to lasting responses in relapsed and refractory multiple myeloma (RRMM) patients, a Phase 1 study showed. Given the small study size, further trials in larger groups of patients are needed to confirm these…
Ninlaro as Oral Combo Therapy Fails at Trial in Heavily Treated Patients
Adding Ninlaro (ixazomib) to cyclophosphamide and dexamethasone does not improve progression-free survival for multiple myeloma patients who relapsed after several lines of treatment, a Phase 2 trial reported. The study, “Ixazomib with cyclophosphamide and dexamethasone in relapsed or refractory myeloma: MUKeight phase II randomised controlled trial results,” was published in the journal Blood Cancer Journal.…
Agreement Reached to Develop MMP-13 Inhibitors to Treat Myeloma
SteroTherapeutics has entered into an exclusive license agreement with Moffitt Cancer Center and Florida Atlantic University Research Corporation to develop matrix metalloproteinase 13 (MMP-13) inhibitors for treating multiple myeloma. “This partnership with Moffitt Cancer Center and the Research Institute of Florida Atlantic University will allow us to further develop this novel line of highly selective MMP-13…
Organizations Rally to Help Ukrainian Rare Disease Patients
A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult, Zamorska recently told BioNews, Inc.,…
Europe Awaits Proposed New Framework for Sharing Health Data
The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to help encourage evidence-based healthcare, spur…
Researchers Identify Potential Antibody-based Myeloma Therapy
Researchers in Japan have identified an antibody that binds to a myeloma-specific form of a common cell surface protein, called CD98 heavy chain, and that prolongs survival when given to a mouse model of multiple myeloma. The findings were detailed in the study, “Selective targeting of multiple myeloma cells with a monoclonal antibody recognizing the…
Telehealth Added to Latest Digital-friendly NORD State Report Card
The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven years ago with the goal of evaluating…
Myeloma Action Month Reaches More Than 30M Worldwide, Foundation Says
More than 30 million people in 46 countries joined together to participate in Myeloma Action Month (MAM), a campaign in March to raise awareness of the rare blood cancer. The International Myeloma Foundation (IMF), which organized the month-long campaign, says the awareness efforts were a success and helped to inspire “a culture of hope and…