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Frontotemporal dementia: current state of therapeutic development
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Frontotemporal dementia (FTD) has turned many people’s attention to the fact that famed action star Bruce Willis’ family announced his diagnosis last week. , academics and companies are pushing for research that can help patients and their families.
Various approaches being investigated include antisense oligonucleotides (ASOs) and gene therapy, which is in early clinical trials. Other studies have explored the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used to develop treatments for both conditions.
Accurately diagnosing FTD in its early stages is difficult, writes Dr. Sami Barmada, an associate professor of neurology at the University of Michigan School of Medicine in Ann Arbor, in an email. pharmaceutical technologyThis is also one of the major challenges slowing the development of effective treatments for FTD, he adds. He is also focused on researching blood marker tests for early detection of FTD.
This comes almost a year after Willis diagnosed with aphasia, a brain disorder that affects language and speech. In a statement, Willis’ family wrote: advanced actor statusled to a new announcement.
According to the National Institutes of Health (NIH) Institute on Aging, Approximately 60% of patients with FTD 45 to 64 years old.there is Up to 60,000 FTD patients in the United States.
Different approaches for different states
Barmada said the lack of known mutations is one reason FTD is so difficult to develop treatments for. His 40% of FTD patients have a family history of the condition, but the majority do not carry the mutation, he explains.
However, multiple companies are developing therapies that target specific mutations, which may cause this condition. Includes Denali Therapeutics in exam. This therapy is specifically aimed at treating her FTD patients who have mutations in the granulin gene, which encodes the protein progranulin (PGRN). This protein promotes lysosomal function.
Mutations here result in loss of PGRN activity and are described as one of the most common genetic causes of FTD, based on the company’s website. TAK-594/DNL593 acts as a PGRN replacement therapy.
In November 2022, the biopharmaceutical company shared interim data from the first part of the study in healthy volunteers. There, a single dose of DNL593 was broadly well tolerated and demonstrated a dose-dependent increase in progranulin levels measured in cerebrospinal fluid. Supporting the transition to Part 2 of a human subjects study.company expect final data From the first part of the study in mid-2023. Denali aims to continue recruiting FTD patients for Phase 2 of the trial this year.
Denali isn’t the only company targeting this mutation. August 2022 Passage Bio first subject dosed in a phase I/II trial of their FTD gene therapy PBFT02.
Prevail Therapeutics, Subsidiary of pharmaceutical giant Eli Lillyis conducting a Phase I/II study of the gene therapy PR006.company administered to the first patient In this survey in December 2020.
UK-based AviadoBio is working on a potential gene therapy called AVB-001 that targets this form of FTD.therapy designated as an orphan From both the FDA and the European Commission last year. It was expected to enter clinical development last year, but no announcements were made.
In addition to gene therapy, gene silencing, which can be done using ASOs, is also an interesting approach, Barmada said. But these treatments need to be genetically targeted, which is difficult because many FTD patients lack mutations, he said. Overall, FTD is a heterogeneous disorder, he adds.
Examples of ASO approaches include the treatment WWE-4 from Wave Life Sciences. WWE-4 is being investigated in Phase Ib/IIa trials as a potential treatment for both ALS and FTD. Specifically, WWE-4 targets a transcriptional variant characterized by a hexanucleotide repeat expansion (HRE) associated with her C9orf72 gene in ALS and FTD. This trial will include four cohorts.
In April 2022, the company shared a positive update that treatment reduced levels of poly (GP) dipeptide repeat proteins in the CSF. Wave plans to release data from all four of his cohorts in the first half of this year.
The HRE of the C9orf72 gene has been described as a common genetic factor behind ALS and FTD.
proteins and biomarkers
Pathological accumulation of proteins is also a potential drug target. Here, ALS and FTD also overlap. Companies such as AcuraStem are now keen to explore this as a potential therapeutic route. This biotechnology addresses ASOs that silence the PIKFYVE gene and target two proteins: TDP-43 and tau. Both proteins are associated with his ALS and FTD.
Yet this approach also has its own set of questions.
“Just under half of FTDs are associated with accumulation of TDP-43 in the brain. [the] The same percentage indicates the accumulation of another protein called tau. The rest he binds to a third protein, he FUS,” Barmada says. “Are we going to develop a single therapy for all three, or individualized therapies for each protein?”
The development of biomarkers to detect these proteins should soon answer this question, says Barmada.
Biomarker testing is also necessary for early detection of disease.Last April, a team of researchers funded in part by the NIH Institute on Aging shared their findings About potential blood markers of FTD. Targeted at the neurofilament light chain (NfL) protein, this test is used for early detection of the condition. The FTD patient had higher blood levels of Nfl in her than those with other conditions, according to the researchers. Also NfL detection being studied for Alzheimer’s disease.
Early detection of FTD is critical for potential treatments.
“The loss of brain cells in FTD is potentially irreversible. In this regard, effective therapy should be implemented as early in the course of the disease as possible,” Barmada said.
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