The U.S. Food and Drug Administration (FDA) has approved a new blood test that can help doctors figure out if someone has Alzheimer’s disease. This test, called the Lumipulse G pTau217/ß-Amyloid 1-42 Plasma Ratio, looks for signs of Alzheimer’s by measuring two proteins in the blood. These proteins are linked to amyloid plaques, which are commonly found in the brains of people with Alzheimer’s.

Until now, doctors had to use expensive brain scans or a spinal tap to find these plaques. This new blood test is easier and less invasive.  While not 100% accurate, the new blood test can reliably predict the presence or absence of amyloid plaques associated with Alzheimer’s disease in patients who are cognitively impaired, as shown by a study evaluating the blood test compared to standard imaging and testing using spinal fluid.

This new blood test is not intended for use in patients with FTD, but easily accessible and reliable testing for patients with suspected Alzheimer’s disease due to cognitive impairment may lead to better discrimination between various forms of dementia, more accurate diagnoses, and improved care.

Vesper Bio has reached an enrollment milestone in their clinical trial for an experimental treatment of progranulin-related FTD called VES001. This trial is for people who have a genetic variant in the progranulin gene (GRN) that causes frontotemporal dementia (FTD)—but who don’t yet have symptoms.  So far, six people have joined the trial in the Netherlands and the United Kingdom.

VES001 is an experimental treatment, given as a pill, that may help slow or stop the development of FTD symptoms before symptoms start by raising levels of a brain protein called progranulin. People with changes in the GRN gene usually have lower levels of progranulin, which may lead to brain cell damage. The drug is designed to work by preventing the body from breaking down progranulin too quickly.

Earlier tests in healthy volunteers showed that VES001 was safe and increased the levels of progranulin in both the blood and cerebrospinal fluid. Results from this current study are expected in the second half of 2025.

The study is still open and accepting more participants.

AviadoBio announced that it has now treated its 6th patient in an early clinical trial called ASPIRE-FTD. This means that dosing of the second group of patients in the Phase 1/2 study is now complete. The trial is testing an experimental gene therapy called AVB-101 for people with a type of Frontotemporal Dementia (FTD) caused by changes in the GRN gene. Each group in the study receives a different dose of the therapy to help researchers find the optimal amount to use.

The experimental therapy is given directly to a part of the brain called the thalamus using a special surgery. This helps the experimental therapy reach the areas of the brain most affected by FTD while avoiding other parts of the body. Early research in animals showed that AVB-101 may raise levels of progranulin, which is low in people with FTD-GRN.

The second group of patients will be monitored over time. No significant side effects were seen in the first group of patients who have been monitored for up to 52 weeks. The company is actively recruiting participants for a third group.

A recent article in Nature Biotechnology highlighted six clinical trials currently underway to test experimental treatments for progranulin-related FTD (GRN-FTD). Different companies are trying different approaches to treat it, but all approaches aim to raise progranulin levels. That’s because disease-causing changes in the progranulin gene result in reduced levels of progranulin, so restoring progranulin levels is expected to be therapeutic. Some companies (like Alector and Vesper) are working on stopping the breakdown of progranulin by targeting a protein called sortillin. Another company, Denali, is injecting a man-made version of progranulin. Three companies—Lilly/Prevail, Passage Bio, and AviadoBio—are using gene therapy to introduce a new, healthy copy of the progranulin gene.

Alector’s trial is the furthest along, with results of their pivotal Phase 3 study eagerly expected later this year. Laura Mitic, the acting president and chief scientific officer at Bluefield, is hopeful. She says, “It’s fantastic that there’s such interest in the field because it will translate into knowledge faster, to the benefit of patients.”

Passage Bio shared early results from its ongoing clinical trial of PBFT02, upliFT-D, which is testing progranulin gene replacement as a treatment for frontotemporal dementia (FTD) caused by GRN mutations. The first dose level tested showed a significant and lasting increase in progranulin levels in participants’ cerebrospinal fluid (CSF). It is anticipated, though not proven, that increasing progranulin levels in CSF may slow disease progression. In addition, participants’ aggregate plasma neurofilament light chain (NfL) levels changed less after 12 months than in historical controls. This is notable since published data suggest that plasma NfL levels typically increase in symptomatic individuals.  Passage Bio is now testing a lower dose (50% of the original) to explore its effects and gather more data. Safety monitoring showed that most side effects to date were mild to moderate, with two patients experiencing serious but manageable conditions. The company is also expanding its trial to include patients with FTD caused by C9orf72 mutations, aiming to begin dosing in the first half of 2025. Passage Bio plans to report more data in late 2025 and will seek regulatory guidance on the next steps in 2026.

After their Phase Ia trial to evaluate the investigational drug VES001 in healthy volunteers that showed that VES001 was safe, well-tolerated, and successfully increased progranulin levels, Vesper Bio announced the launch of a Phase Ib/IIa clinical trial, called SORT-IN-2, to test VES001 in patients with genetic variants in progranulin (GRN) that cause frontotemporal dementia (FTD-GRN).

VES001 is an oral treatment designed to increase progranulin levels, a key protein for brain health that is deficient in FTD-GRN patients. This phase will enroll patients with GRN mutations who are currently asymptomatic and will test whether VES001 can aise progranulin levels in individuals with GRN variants.

The study will take place at two medical centers in Europe (University College London and Erasmus University) and aims to complete dosing by mid-2025.