Ground-breaking Parkinson's Drug Trial Hailed

Friday 26 May 2006

Ground-breaking Parkinson’s Drug Trial Hailed

- Reflects University’s thorough approach to commercialisation -

News that an innovative Parkinson’s disease-fighting compound invented at Otago University will go to Phase II clinical trials is being warmly welcomed by the University’s commercialisation arm, Otago Innovation Limited, (OIL).

OIL CEO Colin Dawson says the new drug, MitoQ, is a prime example of the kind of innovative laboratory science the University is striving to see translated into products that significantly benefit health and society.

“We are very excited and gratified that this innovative Otago research - which could potentially make a huge difference to the lives of millions - is now well into the long and difficult process of being approved as a medicine,” says Mr Dawson.

Worldwide, many highly promising medical discoveries fail to make even the earliest stages of this transition, because the vital patent protection and commercial development processes were not in place from the outset, he says.

“Early on the University and OIL took care to ensure that the intellectual property relating to this research was protected. Antipodean Biotechnology was engaged as the commercial partner that would help take Professor Robin Smith’s and former Otago colleague Dr Michael Murphy’s discovery from the laboratory bench top to the world,” says Mr Dawson.

OIL has been very impressed with how capable Antipodean has proved in commercially developing the research in New Zealand, he says.

“Antipodean, under Dr Ken Taylor’s leadership, has strongly progressed the project to a point where OIL was quite confident to transfer the intellectual property to the new company charged with international development, Antipodean Pharmaceuticals Inc.,” he says.

Any resulting benefits from the shareholding will be shared by the researchers, their departments and the University, he says.

“Getting to this important point in commercialising this brilliant Otago research has required years of very thorough behind-the-scenes work by OIL, and we are very proud of the excellent work of all involved.”

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Notes to Editor:

About MitoQ

What is MitoQ, and why is it special in seeking to treat Parkinson’s disease?

- Parkinson's disease is caused when the nerve cells in the part of the brain that produces dopamine (called the substantia nigra) begin to die, leading to the lack of coordination and other difficulties such as tremors, stiff muscles and joints, associated with the disease.

- There is no treatment for the disease. Existing therapies focus on relieving the symptoms only. They can provide relief for a few years, but eventually the drugs lose their potency and can bring on unwelcome side effects. If this drug is effective it will be the first drug to slow the progression of Parkinson’s disease.

- Research in recent years has increasingly linked the death of these dopamine-producing nerve cells to oxidative damage caused by mitochondrial dysfunction.

- The mitochondria are the cells’ power sources. They are “batteries” within cells that metabolise food and oxygen to supply the body with energy. In doing this the mitochondria also produce reactive oxidant by-products which are normally defused by healthy cells. But when that control process breaks down, oxidation causes cell damage leading to progressive neurological disorders such as Parkinson’s.

- Otago University scientists were the first to target drugs to the mitochondria that are potent blockers of oxidative damage.

- Over ten years they developed a new drug, MitoQ, that involves two parts:

o A “targeting component” that directs the drug to the mitochondria, and

o An antioxidant component which prevents oxidative damage and nerve cell death.

- The antioxidant part was modeled on a naturally occurring antioxidant found in the body, Coenzyme Q. Laboratory tests have shown that MitoQ is 1000 times more effective in preventing oxidative damage than Coenzyme Q.

- The “targeting component” is a positive electrical charge that delivers MitoQ by electrical attraction to the negatively charged mitochondria.

- As a result MitoQ is uniquely able to travel through the cell membrane and position itself in the mitochondria to prevent the chemical oxidation that leads to damage to the mitochondria and dopamine nerve cell death.

- Additionally, MitoQ can accumulate and recycle in the mitochondria, extending its therapeutic effect.

Has this approach been tried by others before now?

- No. This is a completely new and patented technology.

Who discovered this technology, and how did it come about?

- Timing and talent. A top medicinal chemist, Professor Robin Smith, and a world expert on mitochondria dysfunction, Dr Michael Murphy, led the research at the University of Otago.

- The collaboration began when Dr Murphy came to Otago as Associate Dean for Research and the two men began looking at ways to combine their respective areas of expertise to tackle the challenges posed by treating mitochondrial damage.

- Dr Murphy is a world leader in mitochondrial studies. Professor Smith is a widely acknowledged leader in the field of chemistry. MitoQ is a consequence of bringing their areas of knowledge together to create a compound that has the potential to exploit natural cell behaviour to both enter mitochondria and deliver a powerful anti-oxidant.

- Dr Murphy is now Head of the Mitochondrial Dysfunction Laboratory, at the MRC Dunn Human Nutrition Unit, Cambridge, UK which is headed by the Nobel Prize winner, Sir John Walker.

- Professor Ian Tucker, Dean of the School of Pharmacy at Otago, heads the team assessing the compound for its formulation into tablets.

Is this a New Zealand project?

- Absolutely. The invention and clinical development of this exciting drug candidate were carried out using New Zealand centres of research excellence.

- To date the drug has been manufactured totally in New Zealand. The initial clinical studies to establish safety and determine dose levels were completed in New Zealand. Antipodean maintains a research contract with the University of Otago Chemistry Department.

The study

What is this study seeking to establish?

- This study will test the efficacy of MitoQ as a treatment to delay the progression of Parkinson’s.

- This study will recruit patients who have early onset symptoms of Parkinson’s disease.

How long will the trial run for, when will we know if it has been a success?

- Trial participants will be recruited over 6 months and the trial is then expected to run for 12 months.

- Results are expected to be reported in early 2008.

How will we know if it is a success?

- The trial is a double-blind, placebo controlled trial. Patients will be randomly allocated to take either 40 mg of MitoQ, 80 mg of MitoQ or placebo. Neither the patient, their doctor or Antipodean will know what the patient is taking until the end of the study when the results are available.

- This trial will evaluate the progression of Parkinson’s disease. Trial participants will have regular clinical examinations to rate the severity of the symptoms they experience due to Parkinson’s. The hypothesis is that trial participants who take MitoQ will experience less of a deterioration in their Parkinson’s disease symptoms than those that take placebo.

Where is it being run?

- The principal investigator is Dr Barry Snow, Clinical Director of Auckland Hospital’s Neurology Department.

- The trial is being run by consultant neurologists in 10 hospitals in the main centres, and is being supported by Parkinsons New Zealand.

Who will be involved?

- The trial will involve 120 participants in total with the majority coming from New Zealand, and smaller number from Australia.

- It will involve people who have early-onset symptoms of Parkinson’s and have not received any other treatment for symptom relief.

If the trial is a success when can we expect to see a new drug?

- If the outcome of this Phase 2 trial is positive then Phase 3 clinical trials will be run. These trials typically involve much larger patient numbers than Phase 2 trials and aim to provide more detailed data on the safety and efficacy of the drug. The Phase 2 programme will take approximately two years to conduct. Following the Phase 3 trial programme (which can take several years), the regulatory processes involved in gaining approval to market a new drug typically take about two years.

Will it only work for early onset patients or for everyone with Parkinson’s?

- If the drug is shown to benefit people with early Parkinson’s then it is likely that it will also benefit people with more advanced symptoms. This trial is being conducted in people with early Parkinsons as these kinds of patients are not taking other medications for Parkinson’s disease which could obscure the true effect of MitoQ.

Organisations involved in MitoQ

Who has been involved in developing MitoQ?

- Antipodean Pharmaceuticals is the company developing MitoQ. Founded by New Zealand investors and based in New Zealand, Antipodean has offices in Auckland and San Francisco.

- Further information on Antipodean is available at www.antipodeanpharma.com

- MitoQ was discovered at the University of Otago.

- The University of Otago, through its commercialisation company, Otago Innovation Ltd, is a foundation shareholder in Antipodean. Organisations involved in manufacturing and testing the drug in New Zealand include Industrial Research Limited, Douglas Pharmaceuticals, the Institute of Environmental Science and Research and the Christchurch Clinical Studies Trust.

- The research was supported by investment of $1.7 million from the Foundation for Research, Science and Technology, through its Technology for Business Growth scheme.

ENDS

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