Significant New Clinical Trial Begins For Roche Alzheimer’s Drug
STAT reports that Roche and partner Genentech have begun a four-year clinical trial without waiting for data from ongoing studies, to find if a new drug can halt Alzheimer's disease early in its progression. HIV preventatives, CRISPR, and detection of kidney damage are also in the news.
Roche And Genentech Launch Ambitious New Study Of Alzheimer's Drug
Partners Roche and Genentech are studying whether their investigational treatment for Alzheimer’s disease can prevent memory loss for patients who are yet to show symptoms, embarking on a four-year clinical trial without waiting for data from key ongoing studies. The new trial will test whether the partners’ treatment, gantenerumab, can prevent the development of Alzheimer’s for patients who have disease-related plaques called amyloid in their brains but no evidence of cognitive or functional decline. (Garde, 3/3)
In other pharmaceutical and biotech news —
Vesalius Therapeutics Launches To Create Treatments For Common Diseases
Flagship Pioneering, the Cambridge, Mass., life sciences investment firm that helped found Moderna over a decade ago, has unveiled a new company backed with $75 million to develop drugs for common conditions such as autoimmune disorders, diabetes, and Alzheimer’s disease. The startup, Vesalius Therapeutics, was founded at Flagship in 2019 on the premise that many common diseases remain underserved by drug companies. Biotech companies in particular often focus on developing therapies for rare genetic diseases, where a well-understood root cause of a condition provides a clear path for drug development. (Cross, 3/2)
HIV Preventive Care Is Supposed To Be Free In The US. So, Why Are Some Patients Still Paying?
Anthony Cantu, 31, counsels patients at a San Antonio health clinic about a daily pill shown to prevent HIV infection. Last summer, he started taking the medication himself, an approach called preexposure prophylaxis, better known as PrEP. The regimen requires laboratory tests every three months to ensure the powerful drug does not harm his kidneys and that he remains HIV-free. But after his insurance company, Blue Cross and Blue Shield of Texas, billed him hundreds of dollars for his PrEP lab test and a related doctor’s visit, Cantu panicked, fearing an avalanche of bills every few months for years to come. (Varney, 3/3)
3 Burning Questions Created By The Big CRISPR Patent Ruling
On Monday, the U.S. Patent and Trademark Office ruled that CRISPR patents key to developing human therapies belong to the Broad Institute of Harvard and MIT, and not the home institutions of Jennifer Doudna and Emmanuelle Charpentier — the two scientists who won the Nobel Prize for creating the revolutionary genome-editing technology. After a bitter seven-year battle between the Broad and Doudna and Charpentier’s side, known as the CVC group, the patent judges finally issued a definitive opinion on who invented the “guide RNA” molecule that allows the genome editor to work in eukaryotic cells. They determined that it was Feng Zhang, of the Broad, who first “reduced to practice” this process, which is a critical step for developing medicines to treat human diseases, the most lucrative application of CRISPR technology. (Molteni, 3/2)
Organoids Reveal The Tipping Point When Kidney Damage Turns Irreversible
Chronic kidney disease is a serious medical problem that changes the lives of about 13% of the world’s population. Some kidney damage is reversible; kidney cells can marshal their repair mechanisms to heal harm caused by high blood pressure, diabetes, or harsh medications like chemotherapy. But some damage can become permanent, limiting people’s lives as their kidneys lose their ability to filter blood and remove the body’s waste products. Just where the tipping point sits between injury that is fixable and damage that’s beyond repair hasn’t been clear. (Cooney, 3/2)