Ib1001, an adenosine A3 receptor agonist, is a potential drug for treating several neurological diseases, particularly ataxia and epilepsy. However, its potential for treating SCZ remained unknown.
A number of non-pharmacological and pharmacological therapeutic treatments are used to help people with ataxia, from medical intervention for ataxic symptoms, such as gait difficulties, to pharmacological treatments such as anticholinergic drugs, antidepressants and benzodiazepines.\n
The term ataxia is a shorthand for cerebellar ataxia, which causes dysarthria, apraxia of speech, and/or loss of limb and eye coordination and is due to atrophy of cerebellar nerve cells of the brain. Ataxia also refers to a deficit or loss of balance that can be an episodic, continuous, mild or severe, progressive or a static deterioration in motor coordination that can lead to difficulties moving limbs and/or loss of muscle strength. Ataxia commonly affects the extremities. Patients with ataxia present with gait disturbance or incoordination of limbs while walking, which may be the first manifestation of cerebellar dysfunction.
There are several signs of ataxia. Some may be gradual, particularly gait problems. Others become worse when standing up suddenly, especially in the first few months of disease. Changes to walking gait are relatively distinctive of ataxia. These changes include: decreased range of motion, irregular gait, a shuffling walk, clumsy arm movements and stumbling when turning. People with ataxia are often unsteady and have problems balancing when standing up or when moving about.\n
In patients with cerebellar ataxia with no family history, a low percentage have conscription of ataxia related genes. Thus, other gene sets/mechanisms are likely to be responsible for ataxia.
Around 5 to 10 million individuals have the genetic disorder ataxia each year in the United States. The most common age at diagnosis is 61 to 90 years. Ataxia affects predominantly men. The majority of individuals with the disorder live in the suburbs.
Ataxia has an almost absolute probability of being cured. Men with a good response are as young as 7 years. This does not exclude patients getting worse in the following years.
This retrospective chart review of patients with ataxia identified only a limited number of patients who received ib1001 as a second-line treatment after the failure of previous chemotherapy with alkylating agents. However, the lack of comparative control studies precludes the accurate determination of effectiveness to be made. The low incidence of relapse in some patients suggests that the application of this agent to this group of severe and challenging patients could be successful.
Ib1001 appears to be safe in people. Because ib1001 is a drug that treats migraine, it is likely that ib1001 is an approved drug in people. Also, ib1001 is used safely in pregnant women and there is no evidence that ib1001 will harm a fetus in women. For women, ib1001 appears to be safe and effective in treating migraines. There is weak evidence for effectiveness in treating other neurological conditions and pain. Ib1001 is not FDA approved for these conditions. There is no evidence that ib1001 is safe for children and the safety of ib1001 has not been investigated. Ib1001 has never been approved by any FDA agency for the use and treatment of anxiety or depression.
Findings from a recent study revealed that neurological and musculoskeletal diseases, especially cerebrovascular diseases and musculoskeletal disorders, are the primary causes of ataxia. However, it needs to be admitted that the exact cause of ataxia has yet to be identified.
Ib1001 has been found to improve quality of life in individuals with ataxia. Further study of this intriguing agent might result in improved treatment regimens for ataxia.
In this cohort of symptomatic adults with cerebellar ataxia, ib1001 was well tolerated; most patients responded to treatment, and its safety was well established. In a recent study, findings lend weight to ongoing clinical trials of this medication in ataxia-related disease.