2018 Annual Report

Frustration, failure and finally hope in the search for treatments for spina bifida

/ Kevin McCormack / 1 Comment
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Dr. Diana Farmer and her team at UC. Davis

By any standards Dr. Diana Farmer is a determined woman who doesn’t let setbacks and failure deter her. As a fetal and neonatal surgeon, and the chair of the Department of Surgery at UC Davis Health, Dr. Farmer has spent years trying to develop a cure for spina bifida. She’s getting closer.

Dr. Farmer and her partner in this research, Dr. Aijun Wang, have already shown they can repair the damage spina bifida causes to the spinal cord, in the womb, in sheep and bulldogs. Last year the CIRM Board voted to fund her research to get the data needed to apply to the US Food and Drug Administration for permission to start a clinical trial in people.

That work is so promising that we decided to profile Dr. Farmer in our 2018 Annual Report.

Here’s excerpts from an interview we conducted with her as part of the Annual Report.

I have been working on this since 2008. We have been thinking about how to help kids with spina bifida walk. It’s not fatal disease but it is a miserable disease.

It’s horrible for parents who think they are about to have a healthy child suddenly be faced with a baby who faces a life long struggle with their health, everything from difficulty or inability to walk to bowel and bladder problems and life-threatening infections.

As a fetal surgeon we used to only focus on fatal diseases because otherwise kids would die. But as we made progress in the field, we had the opportunity to help others who didn’t have a fatal condition, in ways we couldn’t have done in the past.

I’ve always been fascinated by the placenta, it has lots of protective properties. So, we asked the question if we were able to sample fetal cells from the placenta, could we augment those cells, and use them to tissue engineer spinal injuries, in the womb, to improve the outcome for kids with spina bifida?

Dr. Aijun Wang and I have been working on this project for the last decade.  Ten years of work has taken us to this point where we are now ready to move this to the next level.

It’s amazing to me how long this process takes and that’s why we are so grateful to CIRM because this is a rare disease and finding funding for those is hard. A lot of people are scared about funding fetal surgery and CIRM has been a perfect partner in helping bring this approach, blending stem cell therapy and tissue engineering, together.

If this therapy is successful it will have a huge economic impact on California, and on the rest of the world. Because spina bifida is a lifelong condition involving many operations, many stays in the hospital, in some cases lifelong use of a wheelchair. This has a huge financial burden on the family. And because this doesn’t just affect the child but the whole family, it has a huge psychological burden on families. It affects them in so many ways; parents having to miss work or take time off work to care for their child, other children in the family feeling neglected because their brother or sister needs so much attention.

In the MOMS Trial (a study that looked at prenatal – before birth – and postnatal – after birth – surgery to repair a defect in the spinal cord and showed that prenatal surgery had strong, long-term benefits and some risks) we showed that we could operate on the fetus before birth and help them. The fact that there was any improvement – doubling the number of kids who could walk from 20 to 40% showed this spinal cord injury is not a permanent situation and also showed there was some plasticity in the spinal cord, some potential for improvement. And so, the next question was can we do more. And that’s why we are trying this.

It’s pretty amazing. We are pretty excited.

The thing that makes surgeon-scientists feel so passionate is that we don’t just ask the fundamental questions, we ask questions in order to cure a problem in patients. I grew up in an environment where people were always asking “how can we do it better, how can we improve?”

There were many times of frustration, many times when cell types we explored and worked with didn’t work. But it’s the patients, seeing them, that keeps me motivated to do the science, to keep persevering. That’s the beauty of being a clinician-scientist. We can ask questions in a different way and look at data in a different way because we are driven by patient outcomes. So, whenever we get stuck in the rabbit hole of theoretical problems, we look to the patients for inspiration to keep going.

I am very cognizant of stirring up false hope, knowing that what occurs in animal models doesn’t always translate into humans. But we are optimistic, and I am anxious to get going.

 

Performance, Passion and Progress: and that’s just page one of our 2018 Annual Report

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It’s hard to sum up the activities and achievements of a year in a single document, let alone one that’s just 24 pages. But that’s what we have done in putting together our 2018 Annual Report.

It’s a look back at the year just gone, the highlights, the low lights (spoiler alert – there weren’t any) and the impact we had on the field of stem cell research. But it’s far more than that. It’s also a look ahead. A look at the challenges we face, and profiles of the people who are going to help us overcome those challenges and maintain our progress.

And people are truly at the heart of this report, from UC San Francisco’s Dr. Tippi MacKenzie who is on the front cover for her work in developing an in-utero treatment for the almost always fatal disorder alpha thalassemia major (and the photo of the baby and mom whose lives were changed by that therapy) to Rich Lajara on the back cover, the first person ever treated in a CIRM-funded clinical trial.

Inside are an array of simple images designed to reflect how we as a state agency have performed this year. The numbers themselves tell a powerful story:

  • 50 clinical trials funded to date, 7 this year alone
  • $2.6 billion in CIRM grants has been leveraged to bring in an additional $3.2 billion in matching funds and investments from other sources.
  • 1,180 patients have been involved in CIRM clinical trials

We know people don’t have a lot of time to read Annual Reports so we have made this as visually engaging and informative as possible. We want you to get a real sense of who we are, what we have done and who has helped us do that without you having to wade through a document the size of War and Peace (great book by the way – the Russians beat Napoleon).

We think we have a great story to tell. This Annual Report is one chapter in that story. We hope you like it.

 

The power of one voice: David Higgins’ role in advancing stem cell research

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David Higgins: Photo courtesy Nancy Ramos @ Silver Eye Photography

As we start a new year, we are fine tuning our soon-to-be-published 2018 Annual Report, summarizing our work over the past 12 months. The report is far more than just a collection of statistics about how many clinical trials we are funding (50 – not too shabby eh!) or that our support has generated an additional $3.2 billion in leveraged funding. It’s also a look at the people who have made this year so memorable – from patients and researchers to patient advocates. We start with our Board member David Higgins, Ph.D.  David is the patient advocate on our Board for Parkinson’s disease. He has a family history of Parkinson’s and has also been diagnosed with the disease himself.

How he sees his role

As a patient advocate my role is not to support any Parkinson’s program that comes in the door and get it funded. We have to judge the science at the same level for every disease and if you bring me a good Parkinson’s project, I will fight tooth and nail to support it. But if you bring me a bad one, I will not support it. I see my role as more of a consultant for the staff and Board, to help advise but not to impose my views on them.

I think what CIRM has done is to create a new way of funding the best science in the world. The involvement of the community in making these decisions is critical in making sure there is an abundance of oversight, that there is not a political decision made about funding. It’s all about the science. This is the most science-based organization that you could imagine.

The Board plays a big role in all this. We don’t do research or come up with the ideas, but we nurture the research and support the scientists, giving them the elements they need to succeed.

And, of course the taxpayers play a huge role in this, creating us in the first place and approving all the money to help support and even drive this research. Because of that we should be as conservative as possible in using this money. Being trustees of this funding is a privilege and we have to be mindful of how to best use it.

On the science

I love, love, love having access to the latest, most interesting, cutting edge research in the world, talking to scientists about what they are doing, how we can support them and help them to do it better, how it will change the world. You don’t have access to anything else like this anywhere else.

It’s like ice cream, you just enjoy every morsel of it and there’s no way you can find that level of satisfaction anywhere else. I really feel, as do other Board members, that we are helping people, that we are changing people’s lives.

I also love the learning curve. The amount I have learned about the field that I didn’t know before is amazing. Every meeting is a chance to learn something new and meeting the scientists who have spent years working on a project is so fascinating and rewarding.

 Unexpected pleasure

The other joy, and I hadn’t anticipated this, is the personal interaction I have with other Board members and staff members. They have become friends, people I really like and admire because of what they do and how committed they are.

When I talk about CIRM I tell people if you live in California you should be proud of how your money is being spent and how it’s making a difference in people’s lives. When I give a talk or presentation, I always end with a slide of the California flag and tell people you should be proud to be here.