IkT-001Pro is a novel formulation being developed by Inhibikase Therapeutics for the treatment of progressive multifocal leukoencephalopathy (PML), one of the major complications of multiple sclerosis (MS) caused by the use of therapies that alter the immune system.
How ikT-001Pro works
PML is caused by a common virus called the John Cunningham Virus, or JC virus. The virus itself is unrelated to MS, and can be present in anyone but kept dormant by the immune system. In a weakened immune system, however, the virus may become active and migrate to the brain, causing inflammation and damage. This can lead to impaired thinking ability and can potentially become fatal.
MS is caused by the immune system mistakenly attacking the myelin sheath (a protective layer surrounding nerve fibers) in the brain and spinal cord, disrupting the flow of messages among nerves. The disease is often be treated with drugs that weaken or inactivate the immune system, such as Tysabri (natalizumab), raising the risk to a user of infections. Treatment with Tysabri is known to be associated with an increased risk of developing PML.
The active ingredient of itK-001Pro is imatinib, a BCR-ABL tyrosine kinase receptor inhibitor. Imatinib (under the brand Gleevec) is an approved treatment for certain types of cancer, and works by blocking the action of particular tyrosine kinase receptors to stop signals that trigger the growth and proliferation of cancer cells.
In PML, signaling pathways triggered via the tyrosine kinase receptor are necessary for the growth and replication of the JC virus. This signaling is blocked by imatinib, preventing the virus from reproducing. However, imatinib cannot penetrate the blood-brain barrier (BBB) to reach the brain and spinal cord, but such penetration is essential to preventing the JC virus from damaging the brain.
Inhibikase Therapeutics developed IkT-001Pro to deliver imatinib, using a proprietary technology, directly to affected areas, including those in the brain . The company believes that the technology will reduce the required dose of the drug, resulting in fewer side effects than imatinib treatment administered otherwise, while improving its ability to suppress the JC virus.
IkT-001Pro in development
ItK-001Pro was granted Orphan Drug status by the U.S. Food and Drug Administration (FDA) in 2014 for the treatment of PML. On Inhibikase Therapeutics’ website, however, itK-001Pro is now listed as an investigative, or pipeline, treatment of chronic myeloid leukemia (CML). A separate pipeline therapy, IkT-01427, is reported to be in development for PML. Both drugs are still in the preclinical development stage.
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