The Heywood Brothers: Jamie, Ben, and Stephen

The story of the ALS Therapy Development Institute (ALS TDI) begins with a family. Originally named the ALS Therapy Development Foundation, ALS TDI was founded in 1999 by Jamie Heywood for his brother Stephen, who was diagnosed with ALS at the age of 29. When Jamie realized there were no treatments available for Stephen, he set out to change that by creating the world’s first nonprofit biotech company.

As the founder and first leader of ALS TDI, Jamie oversaw its early growth from the basement of his parents’ Newton, MA home to one of the most trusted and respected institutions in ALS research. Stephen passed away in 2006, but Jamie and other members of the Heywood family are still involved as members of ALS TDI’s board.

To help celebrate ALS TDI’s 25th anniversary, Jamie Heywood joined us to talk about the organization’s early days and why ALS TDI’s work and mission to end ALS remains vital today. 

Q) When your brother Stephen was diagnosed with ALS, why did you decide to start your own organization – a nonprofit biotech – to search for treatments?

A) When Stephen was first diagnosed, I worked at a San Diego neuroscience institute. I spent about three months studying ALS, trying to understand what the options were for him, organizing all the trials at the time and all the available research data.

Eventually, there came a moment when I settled on a gene therapy that seemed, given the evidence of the time, to be the most promising idea. I switched modes from learning to execution. I wrote the business plan for ALS TDI in a truck in Oklahoma, in the passenger seat while I was moving back across the country to be with my parents and Stephen.

The idea was to create an institution that was only dedicated to the objective of serving patients – specifically Stephen. That meant that you had to organize it to be independent of all the other constraints like grants, profit, and investment returns. I used to say, “I don't work for a journal. I don't work for a university. I don't work for the FDA. I work for my brother.”

Q) What were the early years at ALS TDI like? How was our research approach different?

A) The focus was very directly on Stephen. Our first 18 months were about executing two therapeutic ideas: the gene therapy above and a stem cell transplant that Stephen and two other early-involved patients received. We learned so much doing those first clinical programs, going through IRBs and the FDA, doing animal safety studies, manufacturing clinical material, and working with clinicians. The stem cell transplant was safe, but we did not see any improvement. We were the first to publish on using stem cells in ALS patients. Very rapidly, we found that the problem was not how do we get something into patients or Stephen, but how do we know what to put into patients or Stephen.  

That was when ALS TDI as an idea truly emerged - we decided we wanted to test everything. In hindsight, there were probably more bad ideas than good ideas, but what was important is they were new ideas. Those early years were hyper-inventive, hyper-kinetic and very, very fast. We would change course quickly if we felt we didn't like what was going on or as new data emerged. We made a lot of mistakes, and then we figured out ways of preventing not just that one mistake but all mistakes like it in the futureThis was more engineering than science – things like scaling, quality control, automation – so that you learn from each mistake and make sure you don't make them again and again.  

We made more mistakes than anyone else faster. We just learned from each one.

When you want to do something different, sometimes naiveté or even ignorance of the scale of the challenge is actually helpful. It creates an opportunity to create something new.

Early on, I called up Jackson Labs and said, “We're going to order 50 mice a week.”

They said, “You want 50 mice?”

I said, “No, I want 50 mice a week.”

They said, “Until when?”

We've never stopped.

That was such an insane idea to them, that someone would commit to testing a drug every week forever. No one had ever done it before. And that insanity forced us to come up with [new standards for testing ALS treatments in mice]. All of that was driven by a naive, uninformed idea that then, through intelligent, thoughtful, caring people, became the standard in the field. But it would not have happened if we had just tried to just change the way the system worked. We had to build something completely new to do it.

Q) In its early years, ALS TDI established standards for testing treatments in animal models that revolutionized preclinical ALS research. Why did we choose to focus on this approach? What did we do differently?

A) The question I asked myself was “what would make Stephen want to take a drug?”  And at the time, the best answer was “a drug that made the mouse live longer.” And so, the next question was “How do we test every drug in that way?” That became the pipeline tool, where anyone could suggest any ideas for us to test. In five years, we evaluated about 750 ideas. We conducted about 250 studies in about 120 drugs out of those 750 ideas. It all came from the idea of giving choices for Stephen to try to live longer. But Stephen was also a representation of everyone with ALS.

Q) There are many people at ALS TDI today that have been here for 10 years, 20 years, or more. What do you think inspires people to dedicate their careers to ALS TDI’s mission?

A) I think that all of us, in the end, seek meaning in our lives. I think the people who are happiest and most fulfilled are the ones who choose to spend their lives figuring out how to have an impact on others. Since the beginning, and continuing through [former ALS TDI board chair] Augie [Nieto], through all the growth, ALS TDI has had a deep relationship with its community and commitment to truth for our patients, and therefore in science, over all other purposes. I believe this has meant that there is more opportunity to have an impact at ALS TDI than there is almost anywhere else.

I’m incredibly proud of ALS TDI. I'm incredibly proud of [ALS TDI’s current CEO and Chief Scientific Officer Dr.] Fernando [Viera, M.D.].  Fernando understands and embodies those values. Today, ALS TDI is coming into its mature strength. All these ideas that took so long to formalize and learn are now pillars that we can build upon and scale and have more impact.

The organization has a heart that is represented by the fundraising team and the board. It has muscle in terms of people who have been trained here, technicians who have been there more than 20 years now – people that know that doing it right is more important than anything else. And there’s leadership in Fernando, who believes in never shying away from truth in science and following it where it leads us for patients. That's what makes ALS TDI utterly unique. It’s a living being.

Q) What do you think ALS TDI’s impact has been over the past 25 years?  Why do you think it’s still essential to the fight against ALS moving forward?

A) I think that ALS TDI single handedly accelerated the effective use of animal models for research across all neurology, in fact across all disease.  I think that the implications of our early work were profound. I think the early work we did – studying longitudinal biology, the RNA profiling that led to [tegoprubart], and the [ARC Data Commons] databases that are now going public and being shared with everyone – are going to profoundly change and improve the way we we study health and disease.  The standards for using animal models, the fact that all this data is tracked and comparable, has already changed the world.That these were all invented in one tiny little institution is incredible.

I think ALS TDI is unique in its ability to survey, identify, operationalize, and execute whatever it takes to turn an idea into something for patients. Not for money, not for grants, but for patients –  and there's nothing else like it..

So, you know, I regret that I did not find something for my brother. We were not pollyannaish, we knew the odds were against us for him.  But Steven knew, I knew, my family knew,  that the effort we made would make it more likely we would find the answer for someone else's brother. And that person might be just getting diagnosed now.

To learn more about how you can support ALS TDI’s research to end ALS, click here

What to do Next: