A Phase 3 clinical trial is recruiting adults with amyotrophic lateral sclerosis(ALS) to evaluate the safety and effectiveness of NeuroNata-R (lenzumestrocel), a stem cell therapy approved to treat ALS in South Korea.

Enrollment is currently ongoing in South Korea, with additional sites expected to open in the U.S.,Corestem, the therapys developer, stated in an email to ALS News Today.

With U.S. Food and Drug Administration (FDA) approval, one patient was treated with NeuroNata-R atMassachusetts General Hospital in November 2020 under an expanded access program, the company also stated. Such programs make investigational therapies available to people with serious conditions and few or no treatments, when the therapys benefits are thought to outweigh potential risks.

ALS is a progressive neurodegenerative disordercausedby the gradual loss of motor neurons the specialized nerve cells responsible for controlling voluntary movements in the spinal cord and brain, leading to muscle weakness and shrinkage.

Stem cell therapy is emerging as a potential way of treating ALS. Taking advantage of stem cells unique ability to give rise to many types of specialized cells, the approach is expected to help regenerate damaged or dying neurons and key cells that support them.

NeuroNata-R, Corestems lead product, makes use of a patients own mesenchymal stem cells (MSCs), which are capable of maturing into many other cell types that may help to promote nerve cell repair.

The treatment involves isolating MSCs from a persons bone marrow, growing them in the lab, and returning them to the patient, mixed with their cerebrospinal fluid (CSF), via an injection directly into the spinal canal. CSF is the liquid that surrounds the brain and spinal cord.

The first NeuroNata-R injection typically takes place one month after bone marrow extraction, followed by a second injection about a month later.

Prior studies highlighted the immunosuppressive, anti-inflammatory, and neuroprotective properties of NeuroNata-R. As such, the therapy is expected to promote motor neuron growth and prevent the death of these cells, slowing ALS progression.

The therapy was previously tested as an add-on to riluzole (marketed asRilutek, Tiglutik, and Exservan) in a Phase 1/2 clinical trial (NCT01363401) conducted in South Korea.

Results showed that NeuroNata-R, given as two intrathecal (into the spinal canal) injections, safely and effectively slowed disease progression as assessed with the ALS Functional Rating Scale(ALSFRS-R) in 32 ALS patients for at least six months.

These findings supported the conditional approval of NeuroNata-R in South Korea in 2014 as the first stem cell therapy for ALS. Its continued use, and full approval, requires further verification and description of clinical benefit in a larger, longer-term confirmatory study.

It was designated an orphan drug as a potential ALS treatment in the U.S. in 2018, followed by a similar designation in Europe about six months later. Both designations are meant to accelerate its development and review.

Corestem also reports that the FDA has approved its request to open a Phase 3 clinical trial of NeuroNata-R in the U.S.

This study, called ALSummit (NCT04745299) and already underway in Korea, is designed to evaluate the long-term safety and effectiveness of NeuroNata-R in up to 115 adults, ages 2575, diagnosed with ALS within the previous two years.

Participants will be randomly assigned to either NeuroNata-R followed or not by three injections, given three months apart or to a placebo injection (CSF without stem cells). All enrolled will also receive riluzole.

The trials main goal is to assess changes in a combined measure of function, based on the ALSFRS-R score, and survival benefits. Secondary goals include other measures of function and quality of life.

These assessments will be conducted at six months for patients receiving one treatment cycle, and at one year for those given three additional injections.

Eligible patients may also be asked to enter a supplementary study that will continue to week 48 (nearly a year), and long-term efficacy and safety will be evaluated during an observation period of 36 months from first NeuroNata-R injection.

According to Corestem,more than 300 ALS patients have been treated with NeuroNata-R in South Korea as of December, including at least 49 people who traveled from their home countries for the treatment.

Not many patients are aware of the existence of Neuronata-R, so we are eager to let the community know its existence, Eunsan Cho,the assistant manager of Corestems marketing team, said in the email.

Marta Figueiredo holds a masters in evolutionary and developmental biology and a PhD in biomedical sciences from the University of Lisbon, Portugal. Her research is focused on the role of several signaling pathways in thymus and parathyroid glands embryonic development.

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Ins holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Cincias e Tecnologias and Instituto Gulbenkian de Cincia. Ins currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.

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Phase 3 Trial of Stem Cell Therapy NeuroNata-R Likely to Expand to US - ALS News Today

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