Month: January 2019

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

/ Kevin McCormack / Leave a comment

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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.

 

By the numbers – a look at how the field of Regenerative Medicine is growing

/ Kevin McCormack / 6 Comments
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ARM State of the Industry briefing

The Golden State Warriors, the current US basketball champions – and your favorite Stem Cell Agency’s neighbors in Oakland – have a slogan, “Strength in Numbers”. That could well apply to the field of Regenerative Medicine because the field is growing in numbers, growing in strength, and growing in influence.

Yesterday, the Alliance for Regenerative Medicine (ARM), the organization that represents the field, held its annual State of the Industry briefing in San Francisco, detailing what happened in 2018. It was pretty impressive.

In fact, just the number of people in the room was impressive. More than 800 RSVP’d for the event, more than for any previous meeting, but even then the room was filled over capacity with many standing around the edges because there were no seats left.

ARM itself is growing, 32 percent last year, and now has more than 300 members. Other impressive numbers include:

  • 906 gene and cell therapy companies worldwide
  • 484 gene and cell therapy companies in the US alone
  • 1,028 clinical trials taking place worldwide
  • 598 of those clinical trials (58 percent of the total) are targeting cancer
  • 59,575 patients are slated to be enrolled in those trials

All those numbers are up dramatically on last year. You can see all the details on the ARM website.

Another sign the industry is growing comes in the amount of money being invested. When people are willing to pony up hard cash you know it’s a sign they believe in you. Last year the field raised $13.8 billion worldwide, that’s up a whopping 73 percent on 2017. That represented a strong year across all fronts from corporate partnerships to Initial Public Offerings (several CIRM-supported companies such as Orchard Therapeutics and Forty Seven Inc. are in that number) and venture capital investments.

Clearly there are still challenges ahead, such as figuring out ways to pay for these therapies when they are approved so that they are available to the people who need them, the patients.

One of the issues that is going to be front and center in 2019 is reimbursement and developing new payment models. But that in itself is a sign of a maturing field. In past years the emphasis was on developing new treatments. Now that those are in the pipeline, we’re working on ways to pay for them.

That’s progress.

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

/ Kevin McCormack / Leave a comment
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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.