• Researchers have found success in leveraging AI to quickly identify new uses for existing approved medicines, providing hope for millions with rare diseases that currently lack treatment options.
• An experimental treatment, adapted from blood cancer therapies, has shown promise in treating pediatric solid tumors, offering hope to patients who have exhausted conventional therapies.
• UCLA researchers are developing a promising off-the-shelf CAR-NKT cell therapy to eliminate ovarian cancer and prevent its return, potentially transforming treatment for this challenging disease.

Recent News

• Doctors Told Him He Was Going to Die. Then A.I. Saved His Life
  The New York Times – March 20, 2025
  A little over a year ago, Joseph Coates was told there was only one thing left to decide. Did he want to die at home, or in the hospital? Coates, then 37 and living in Renton, Wash., was barely conscious. For months, he had been battling a rare blood disorder called POEMS syndrome, which had left him with numb hands and feet, an enlarged heart and failing kidneys. Every few days, doctors needed to drain liters of fluid from his abdomen. He became too sick to receive a stem cell transplant — one of the only treatments that could have put him into remission. “I gave up,” he said. “I just thought the end was inevitable.” But Coates’s girlfriend, Tara Theobald, wasn’t ready to quit.
• Small study hints anti-amyloid therapy may keep Alzheimer’s symptoms at bay in certain patients
  CNN Health – March 20, 2025
  For the first time, scientists say, they have evidence that using a biologic drug to remove sticky beta amyloid plaques from the brains of people destined to develop Alzheimer’s dementia can delay the disease. The researchers have been testing amyloid-removing therapies in a group of people who have rare genetic mutations that make it almost certain they’ll develop Alzheimer’s. The study – which is small, including just a few dozen participants – is a follow-up to a randomized-controlled trial that found no significant benefits for people who were taking one of two amyloid-lowering therapies, compared with a placebo.
• Alnylam drug gets long-awaited FDA approval in deadly heart disease
  BioPharmaDive – March 20, 2025
  The Food and Drug Administration approved an Alnylam Pharmaceuticals medicine for a serious heart condition, a decision that should help the biotechnology firm secure its position as one of the sector’s most valuable companies. The agency cleared the drug, called Amvuttra, for people with a cardiac form of the rare disease transthyretin amyloidosis. Alnylam already sells the therapy for individuals with a genetic type of the disease that causes progressive nerve damage. Transthyretin amyloidosis with cardiomyopathy is considered deadlier than the nerve type, and often leads to hospitalizations and heart failure. It’s also thought to be more common, making it a target for drugmakers seeking a lucrative market opportunity.
• Experimental treatment borrowed from blood cancer shows promise for pediatric brain tumors
  NBC News – March 19, 2025
  Gavin Nielsen was 2 years old when he was diagnosed with a rare and aggressive brain cancer. The smiley toddler had diffuse intrinsic pontine glioma, or DIPG, a cancer that occurs in the part of the brain stem that controls vital functions including breathing, blood pressure and heart rate. Very few treatment options exist, and the prognosis for children diagnosed with the disease is, on average, less than one year. “When we have a child newly diagnosed with this disease, we walk into the room and tell the child’s parents their child has a terminal disease and the only option is palliative radiation,” said Dr. Robbie Majzner, director of the pediatric and young adult cancer cell therapy program at Dana-Farber Cancer Institute and Boston Children’s Hospital.
• Measles in California: Do you need to check your vaccination status?
  San Francisco Chronicle – March 12, 2025
  Two new confirmed cases of measles in California highlight the risk for the unvaccinated, and the need for all Californians to check their vaccination status, infectious disease experts said. The cases, reported in Los Angeles and Fresno counties, bring California’s total measles count to five for 2025, according to state data. They follow — but appear unrelated to — a major measles outbreak in West Texas that has resulted in more than 250 cases and two deaths in unvaccinated individuals. The Fresno County case was in an unvaccinated adult who recently traveled abroad. The department did not specify where the person traveled from. He is recovering and quarantining at home.
• A once-yearly PrEP? Gilead’s lenacapavir shows promise as company plots phase 3
  Fierce Pharma – March 11, 2025
  After Gilead Sciences’ lenacapavir made waves with stellar clinical results as a twice-yearly HIV prevention candidate, the drug has shown promise being dosed even further apart at only once a year. Two different once-yearly formulations of lenacapavir achieved blood concentrations that exceeded those associated with the strong HIV prevention efficacy that twice-yearly lenacapavir showed in phase 3 preexposure prophylaxis (PrEP) studies. The pharmacokinetic results came from a small phase 1 trial presented at the Conference on Retroviruses and Opportunistic Infections and simultaneously published in The Lancet.
• As measles cases climb in 2025, here’s what to know
  UCLA Health – March 11, 2025
  As of March 13, 2025, the Centers for Disease Control and Prevention had reported 301 measles cases across 15 states. Most of these cases are associated with three outbreaks, the largest being in Texas. Of all cases, 76% have been reported among children and young adults up to age 19. On Tuesday, March 11, the Los Angeles County Department of Public Health reported the county’s first measles case of the year. Here are some frequently asked questions about measles: What is measles? Measles is a very contagious infectious disease spread through coughing, sneezing, talking and breathing. It can remain in the air for up to two hours after an infected person leaves the area, according to the CDC.
• Neurotech nabs FDA approval to treat rare eye disorder with cell therapy implant Encelto
  Fierce Pharma – March 10, 2025
  With a U.S. green light for its encapsulated cell therapy technology in the bag, privately held biotech Neurotech Pharmaceuticals is on the verge of making its commercial debut. The FDA approved (PDF) Neurotech’s cell therapy implant revakinagene taroretcel to treat the vision-robbing neurodegenerative disease macular telangiectasia (MacTel) type 2. The drug, which will adopt the brand name Encelto, is the first to win the FDA’s blessing in the rare vision disorder, Neurotech said in a release. In MacTel type 2, small blood vessels at the center of the retina dilate and, in some cases, new blood vessels form under the retina in a process known as neovascularization, according to the American Academy of Ophthalmology.
• AstraZeneca touts disease recurrence win, ‘strong trend’ in survival benefit for Imfinzi in early stomach cancer
  Fierce Pharma – March 7, 2025
  AstraZeneca is preparing to talk to the FDA about a potential new use of its immunotherapy Imfinzi after a pivotal study in early-stage stomach cancer met its main goal. When added to a chemotherapy combination called FLOT, Imfinzi significantly reduced the risk of disease recurrence, worsening or death in patients with resectable, early-stage gastric and gastroesophageal junction cancers who got the regimen before and after surgery, the company said. The event-free survival readout vaults the phase 3 Matterhorn trial to the positive category, following a previous interim analysis that showed Imfinzi could help more patients achieve no detectable cancer cells in resected tumors, known as a pathological complete response (pCR).
• UCLA researchers unite to develop immunotherapy they hope will transform ovarian cancer treatment
  UCLA Health – March 5, 2025
  For decades, the story of ovarian cancer treatment has often followed the same pattern: surgery, chemotherapy, recurrence, repeat. This was the grim reality Sanaz Memarzadeh, MD, PhD, encountered as a second-year UCLA resident watching young women cycle through treatments only to face the disease’s inevitable return. “Some of these women were relatively young and had already undergone multiple surgeries, but their cancer had come back, and they were dying,” she recalled. “I remember thinking, ‘There has to be something more we can do for these patients.’” Her search for answers — through medical textbooks, research conferences and conversations with experts — revealed a sobering fact: treatment for ovarian cancer hadn’t meaningfully changed in 40 years.