Ongoing clinical trials offer hope for most common early form of dementia - MedCity News

Frontotemporal dementia (FTD) or frontotemporal lobar degeneration (FTLD) is a rare, rapidly progressive neurodegenerative disease that is the most common form of dementia in people under the age of 60. FTD affects the frontal and temporal lobes of the brain—areas typically associated with personality, behavior, and language. Thus, people with FTD often develop symptoms such as behavioral changes, lapses in judgment, and severely reduced language skills by the time they are in their 40s and 50s. Unlike Alzheimer’s disease, which is characterized mainly by memory problems but generally not major changes in personality or language, FTD strikes at the core of what makes us who we are.

Given that FTD is a rare disorder, estimated to affect <0.02% of the population in the United States and the European Union, it is easy for people who have symptoms of FTD to be misdiagnosed as having a mental illness. disorder or another more common type of dementia such as Alzheimer's disease. In addition, some of the cognitive, behavioral, and language difficulties in FTD can either go unrecognized or be stigmatizing and uncomfortable for individuals and their families to discuss. As a result, the road to receiving an accurate diagnosis is often long and complicated for FTD patients and their families. To make matters worse, once a diagnosis of FTD is received, there are currently no approved treatment options available for any form of FTD.

The good news is that there are now numerous interventional clinical trials evaluating potential investigational therapies for the treatment of various types of FTD. Some cases of FTD are genetic or hereditary in nature, meaning that a parent can pass a genetic variant associated with FTD to their child. The 3 most common genes that are associated with the development of FTD are GRN (progranulin gene), C9orf72and MAPT. In the past, genetic testing for variants of these genes was less common; however, now that potential therapies are being developed to address the underlying problems associated with each of these different gene variants, genetic testing is becoming more common and more important than ever to help carriers of these variants understand which type of therapy to investigate is best suited for them. There are several options for individuals to obtain free genetic counseling and genetic testing. The Frontotemporal Dementia Association (AFTD) is a great resource for people interested in genetic testing.

Ongoing clinical trials in FTD

Once a person is aware of their genetic status, they may be eligible to participate in a clinical trial of an investigational therapy that is designed to slow or stop disease progression in that specific genetic subtype of FTD. There are several ongoing interventional trials, which are listed by genetic subtype below.

FTD-GRN:

    • We in voter conducted a phase 3, double-blind, placebo-controlled study evaluating the efficacy and safety of a monoclonal antibody (AL001) administered intravenously in participants who were at risk for or had been diagnosed with FTD due to the presence of a mutation in the progranulin gene
    • Prevail Therapeutics is conducting a phase 1/2, open-label, dose-escalating, first-in-human study that will evaluate the safety and effect of intracisternal administration of an investigational gene therapy (PR006) on progranulin levels in FTD patients with a mutation in the progranulin gene
    • Passage Bio conducted a phase 1/2 open-label, single-arm, dose-escalating study of the safety, tolerability and efficacy of intra-cisternal administration of an investigational gene therapy (PBFT02) on progranulin levels in FTD patients with a mutation in the progranulin gene
    • Denali Therapeutics conducted a Phase 1/2, randomized, placebo-controlled, double-blind study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of DNL593 in healthy volunteers and patients with FTD with a progranulin gene mutation

FTD-C9orf72:

      • Transposon therapy conducted a Phase 2a randomized, double-blind, placebo-controlled trial to evaluate the safety and tolerability of a LINE-1 reverse transcriptase inhibitor (TPN-101) in patients with amyotrophic lateral sclerosis (ALS) and/or hexanucleotide-associated FTD Repeat expansion in the C9orf72 gene (C9ORF72 ALS/FTD)
      • Wave Sciences for Life conducted a Phase 1b/2a randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of intrathecal administration of an antisense oligonucleotide (WVE-004) in adult patients with C9orf72-associated ALS or FTD

There is a huge unmet medical need to develop effective therapies for FTD, and this is an exciting time with multiple studies of new potential treatments. Participation in interventional clinical trials is critical to bringing new therapies to market. Anyone who has been diagnosed with FTD or who has a potential family history of FTD and is interested in genetic testing options and clinical trials can learn more at the links above.

Photo: Andreus, Getty Images

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