Developing the treatments of tomorrow

Drugs work in a variety of ways. They fight germs, destroy abnormal cells that cause cancer, replace substances that we’re lacking (such as vitamins), or change the way that cells work in the body.

Most of the drug treatments we currently have for Parkinson’s work by replacing the chemical dopamine inside the brain. But they cannot stop the loss of brain cells, which means symptoms get worse over time.

We now have a much better understanding of how processes inside these precious brain cells go awry in Parkinson’s — causing these cells to stop working properly and die. And this means we are ready to develop treatments that can tackle the underlying causes of Parkinson’s by changing the way the brain cells work.

The key is finding small molecules that are exactly the right shape and size — the way a key fits a lock — and that target the process that has gone wrong in the cell. And crucially, they need to do this without causing problems in other organs and tissues that could produce serious side effects.

The actual process of developing these new treatments is shown in the cartoon on the next page.

It’s a long and difficult journey. Each step in the process is vital, and a drug can fail at any stage along the way. But the rewards for success are compelling — treatments that can slow, stop or even reverse Parkinson’s.

Introducing the Parkinson’s Virtual Biotech

We speak to our Director of Research, Dr Arthur Roach, about how Parkinson’s UK is planning to fast-track the most promising scientific discoveries into life-changing treatments for people with Parkinson’s.

What are we planning to do?

The crucial early stages of developing new drugs and treatments are often led by companies called biotechs. They have special expertise in this vital stage in the journey from a scientific discovery to a new treatment.

“But opportunities could be lost because there’s not enough investment from industry to drive scientific discoveries forwards.”

This is a major roadblock in our mission to develop better treatments and a cure. We believe we can step in here to bring new treatments forward faster.

We’re calling this major new programme of work the Parkinson’s Virtual Biotech because we’ll be acting in the same way as a small biotech company. But unlike commercial biotechs, we will be dedicated to developing new treatments for one condition — Parkinson’s.

“And instead of making money, our goal is delivering better treatments that improve life as quickly as possible.”

How will the Parkinson’s Virtual Biotech work?

This is the clever bit! We want to do the same type of drug development work as a regular biotech company, but we don’t want to build our own labs, employ a huge team of scientists or buy expensive equipment.

Instead, we will work in partnership with a range of other organisations — these may be companies, universities or other charities — who have the facilities and staff to carry out the scientific work for us. We will carefully manage these projects with guidance from a team of industry and scientific experts who are volunteering their time.

We will bring these elements together to create our own portfolio of projects — all at different stages of the drug development pipeline — in a manner similar to a commercial biotech company.

“This means we can deliver new treatments at a fraction of the cost.”

And we can stay agile — rapidly investing in the most promising projects, and cutting off projects that turn into dead ends so that we make maximum use of every pound we spend.

Do other medical research charities do this?

We are not alone as there are a few other charities taking a similar approach, but our virtual model seems to be fairly unique in the charity sector.

Alzheimer’s Research UK is actually setting up three Drug Discovery Institutes which will employ teams of research scientists at state-of-the-art facilities, so they could be a potential partner for some of our projects.

When will the Parkinson’s Virtual Biotech start developing new treatments?

I believe it’s the perfect time to be launching our Virtual Biotech. The discoveries in the past ten years, particularly in the genetics, molecular biology and biochemistry, provide us with a wealth of promising ideas for new treatments that have the potential to tackle the underlying biology of Parkinson’s.

“And that means treatments that can actually slow, stop or reverse the development of the condition are within touching distance.”

We hope to have new projects to tell you about in the next few months, and to build an exciting portfolio of projects over the next few years.

Find out more about the Parkinson’s Virtual Biotech

You can read more about the Parkinson’s Virtual Biotech and the projects that have now started on our website.