University of Otago Otago Innovation Ltd

Treatment for Neuro-degenerative Disorders

Summary

The University of Otago is currently developing treatments of the following disorders using Mullerian Inhibitory Substance (MIS):

The initial target for treatment with MIS is Motor Neuron Disease. MIS is currently being trialled, by another institution, for the treatment of certain types of uterine, ovarian and endometrial cancer. The University has the research and commercialisation rights using MIS in the brain. Otago Innovation is looking for a partner with the resources and expertise to guide this technology through pre-clinical and clinical trials.

Motor Neuron Disease

We believe that Motor Neuron Disease or Amyotrophic Lateral Sclerosis (ALS) is an excellent first target for treatment. ALS attacks the motor neurons in the brain and the spinal cord, which controls voluntary movement. With the death of the motor neurons the muscles weaken and shrink. The early symptoms are characterised by fatigue, clumsiness, muscle weakness, slurred speech and difficulty swallowing. As the disease progresses, patients gradually lose the use of hands, feet, arms and legs, leading to paralysis. Speaking and swallowing are difficult, however, patients retain all of their mental faculties, bladder control and sexual function. Over half of patients are expected to die within three to five years of diagnosis. The cause of the disease is unknown. Hypotheses range from an auto-immune disorder that attacks the motor neurons, to toxic levels of glutamate or a build up of anti-oxidants. The life time prevalence of ALS is 1 in 2000 and that 90-95% of cases are labelled sporadic and have no inherited component. At any one time there are 30,000 Americans living with ALS and an additional 5000 Americans are diagnosed with the disease each year. The annual cost of treatment and care in the United States is $6 billion. There is no effective cure or treatment for ALS. Rilozole is used to treat ALS, however, the drug only increases life-expectancy by six months. In January of this year Novartis withdrew TCH 346 from Phase III clinical trials when analysis revealed no difference between the treatment group and the control. Foetal stems cells are also being trialled, but anecdotal evidence suggests that any benefit is short lived. There is one known Phase III competitor, Minocycline, which will be used in conjunction with Riluzole to slow progression, also a number of Phase II trials have been identified. Due to the high mortality rate in a short period of time and the low number of sufferers, we believe the use for treating ALS would receive fast track/orphan drug status from the FDA.

Otago Innovation's Solution - Mullerian Inhibitory Substance

A combination of anatomical reasoning and micro-dissection identified the role of MIS as a neuronal survival factor in motor neurons and which also may have a role the regeneration of motor neurons. MIS or AMH (Anti-Mullerian Hormone) is a growth factor or cytokine involved in intracellular communication. Its role in the formation of male gonads has been long known, it signals the regression of the Mullerian duct in utero. Recently, a group in Boston has been using MIS as a possible treatment for certain reproductive cancers, namely, uterine, ovarian and endometrial cancer. Otago researchers were the first to discover the effects of MIS on the regeneration of motor neurons in vitro and recently identified a similar role within other structures in the brain. It is hypothesized that the brain can survive without MIS as neuronal survival is supported by parallel pathways with redundant capacity. The loss of one pathway is survivable but leaves the brain/neurons with less spare capacity for regeneration. Some of the data that is available to support Otago Innovation's solution has recently been published in the Proceedings of the National Academy of Science, a copy of the article is attached for reference. As a result of these findings, Otago Innovation has filed patents relating to the use of MIS for the treatment of neuro-degeneration and –injury. The patent families stem from US 11/229,415, and cover the use of MIS for the treatment of a number of neuro-degenerative diseases and injuries and the method and route of administration.

Other Therapeutic uses of MIS

Additional research by University of Otago researchers has identified the MIS as a neuronal survival factor in a number of other structures in the brain. The structures identified are those linked with the following diseases:

Otago Innovation believes that MIS has potential as a treatment for all of these disorders.

The Investment Opportunity

Otago Innovation is looking for a partner with the resources and expertise to assist this technology through pre-clinical and clinical trials.

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Contact

Hamish Findlay
Otago Innovation
PO Box 56
Dunedin, New Zealand
+64-3-4793870
hamish.finday@otagoinnovation.com