Stem Cell Profiles in Courage: Brenden Whittaker


Brenden Whittaker: Photo Colin McGuire

It’s not often you meet someone who says one of their favorite things in the world is mowing the lawn. But then, there aren’t many people in the world like Brenden Whittaker. In fact, as of this writing, he may be unique.

Brenden was born with severe chronic granulomatous disease (x-CGD), a rare genetic disorder that left him with an impaired immune system that was vulnerable to repeated bacterial and fungal infections. Over 22 years Brenden was in and out of the hospital hundreds of times, he almost died a couple of times, and lost parts of his lungs and liver.

Then he became the first person to take part in a clinical trial to treat x-CGD. UCLA researcher Don Kohn had developed a technique that removed Brenden’s blood stem cells, genetically re-engineered them to correct the mutation that caused the disease, and then returned those stem cells to Brenden. Over time they created a new blood system, and restored Brenden’s immune system.

He was cured.

We profiled Brenden for our 2016 Annual Report. Here’s an extended version of the interview we did with him, talking about his life before and after he was cured.


Brenden with a CIRM Game Ball – signed by everyone at CIRM

Brenden’s story:

I still think about it, my disease, every few days or so and it’s weird because in the past I was sick so often; before this year, I was sick consistently for about 5 years and going to doctor’s appointments 2 or 3 times a week and being in the hospital. So, it’s weird having a cough and not having to be rushed to the ER, not having to call someone every time the smallest thing pops up, and not having to worry about what it means.

It’s been good but it’s been weird to not have to do that.  It’s a nice problem to have.

What are you doing now that you didn’t do before?

Cutting the grass is something I couldn’t do before, that I’ve taken up now. Most people look at me as if I’m crazy when I say it, but I love cutting grass, and I wasn’t able to do it for 22 years of my life.

People will complain about having to pick up after their dog goes to the bathroom and now I can follow my dog outside and can pick up after her. It really is just the little things that people don’t think of. I find enjoyment in the small things, things I couldn’t do before but now I can and not have to worry about them.

The future

I was in the boy scouts growing up so I love camping, building fires, just being outdoors. I hiked on the Appalachian Trail. Now I’ll be able to do more of that.

I have a part time job at a golf course and I’m actually getting ready to go back to school full time in January. I want to get into pre-med, go to medical school and become a doctor. All the experience I’ve had has just made me more interested in being a doctor, I just want to be in a position where I can help people going through similar things, and going through all this just made me more interested in it.

Before the last few months I couldn’t schedule my work more than a week in advance because I didn’t know if I was going to be in the hospital or what was going on. Now my boss jokes that I’m giving him plans for the next month or two. It’s amazing how far ahead you can plan when you aren’t worried about being sick or having to go to the hospital.

I’d love to do some traveling. Right now most of my traveling consists of going to and from Boston (for medical check-ups), but I would love to go to Europe, go through France and Italy. That would be a real cool trip. I don’t need to see everything in the world but just going to other countries, seeing cities like London, Paris and Rome, seeing how people live in other cultures, that would be great.

Advice for others

I do think about the fact that when I was born one in a million kids were diagnosed with this disease and there weren’t any treatments. Many people only lived a few years. But to be diagnosed now you can have a normal life. That’s something all on its own. It’s almost impossible for me to fathom it’s happening, after all the years and doctor’s appointments and illnesses.

So, for people going through anything like this, I’d say just don’t give up. There are new advances being made every day and you have to keep fighting and keep getting through it, and some day it will all work out.

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From Stem Cells to Cures with Shinya Yamanaka and Google Ventures

How do you go from basic stem cell research to cures for patients? We ask this question everyday at CIRM, and we’re not alone in our tireless pursuit to find answers to this challenging question.

In fact, two leaders on different sides of the stem cell arena – research and investment – came together last week at the Gladstone Institutes’ Fall Symposium to discuss how stem cell research can be translated into effective cures.

Nobel prize winner, Dr. Shinya Yamanaka, and Google Ventures partner and Stanford PhD, Dr. Blake Byers, shared their thoughts on where stem cell research is now and the future of stem cell therapy for treating and curing disease.

iPS Cells and the Stem Cell Revolution

Gladstone President, Sandy Williams

Gladstone President, Sandy Williams

President of the Gladstone Institutes, Dr. Sandy Williams, laid the groundwork for the symposium by outlining ways that stem cell research, especially Dr. Yamanaka’s discovery of cellular reprogramming and induced pluripotent stem (iPS) cells, will lead to cures.

“Cellular reprogramming has really launched the stem cell revolution. There are three pathways that stem cell biology or cellular reprogramming can be turned into new medicines. Cellular transplantation, reprogramming cells inside the body, and cellular models of human disease created by cellular reprogramming are all different routes to cures.”

He followed with the point that the success of the stem cell revolution cannot rest solely on the shoulders of scientists and clinicians. He said, “the best science will never be a cure unless it passes into the commercial arena. It has to pass through venture investors, biotechnology companies, and pharmaceutical companies, device companies for scientific advances to help human beings.”

Yamanaka on iPS Cell Applications

Dr. Shinya Yamanaka

Dr. Shinya Yamanaka

Yamanaka covered the research side of the discussion and shared a heartwarming story about his father inspiring him to pursue medicine before delving into the applications of his Nobel prize winning technology.

After becoming a doctor, Yamanaka continued his training as a scientist, but not without significant hurdles to overcome before his career-defining success.

I had a clear vision, I wanted to help patients by doing medical research. But of course, it’s easy to say, but very difficult to achieve. I spent many hours, many days, and many years in laboratories without significant success. 20 years later however, I became extremely lucky to have a wonderful group of people. And that group developed a new technology. Our group was able to find a way to make a new type of stem cell, which we designated iPS cells.

He then discussed the power of iPS cell technology and how scientists can turn patient iPS cells into almost any cell type in the body. He also emphasized two major medical applications of iPS cells that will lead to cures.

iPS cells are very powerful. We can use these cells for two major medical applications. We can transplant healthy brain cells [derived from iPS cells] back into the patients brains to obtain functional recovery. This approach is known as regenerative medicine or cell therapy. We’ve been trying to apply this approach of cell therapy to many diseases and injuries, for example, eye diseases such as macular degeneration, brain diseases such as PD, and also spinal cord injury, heart failure, liver failure, and diabetes. Also we’ve been trying to make immune cells, or lymphocytes, that attack cancer cells from iPS cells as a new form of cancer therapy. This is the first medical application of iPS cells. Another yet equally important application of iPS cells is in drug discovery. Instead of transplanting back into patients, we can use iPS cells and brain cells or heart cells derived from iPS cells in laboratories at the universities, Gladstone Institutes, or pharmaceutical companies to make disease models to perform drug screening.

Yamanaka ended his speech with his big picture goal. “We really want to bring iPS cells to patients, and we really want to help patients by using iPS cells. Of course we still have a long long way to go, and we need to overcome many problems.”

Byers on Facing Stem Cell Hurdles Because It’s Worth it

On the investment and capital side, Blake Byers from Google Ventures discussed why stem cell research should be pursued even though the obstacles in our path to cures can be daunting.

Blake Byers, Google Ventures

Blake Byers, Google Ventures

While Byers has been on the “evil capitalist side of the world” for the past five years, he has been “taking soul supplements by continuing to do research at Stanford University.” His most recent scientific publication was published in July on generating dopaminergic neurons from human iPS cells and transplanting them into rats with Parkinson’s disease. Using a cutting-edge technology called optogenetics, Byers was able to manipulate the activity of these transplanted neurons in the rat brain using light and fiber optic cables. He said this experience was his “first foray into the power that stem cells have in a therapeutic capacity.”

He then explained why iPS cells show more promise as cures than other therapeutic avenues.

So why work with these stem cells if they are so much harder to work with than just a small molecule or some chemical that we bake up in the laboratory? The reason is because cells have something that none of these other molecules do. Cells have logic embedded into them. They have the ability to respond to their environment, integrate that response, and come up with their own intervention on our behalf. [With cells] we can start to think about things that biology doesn’t even do yet. So not only can we cure diseases as they arise, but we can start thinking about prevention of disease before it arises.

Byers then gave an example of how stem cells will benefit cancer therapy.

On the cancer side, we can take cells out of the body and train them to look for cancer, and then put them back in. They then go and hunt for those cancer cells and eradicate them. This work is being done by many labs. There’s a number of companies working on this strategy that are public companies that are valued in the billions, which gets capitalists like me very excited. And it’s just the beginning of a new field on the cancer side.

(For an example of this, see our just-approved clinical trail for glioblastoma)

Finally, Byers admitted that the stem cell field itself is far from putting stem cells and their derivatives into humans routinely, and that “there’s going to be lots of stuff that’s going to be difficult about this process. It’s going to be hard, but it will be worth it. So that means we should try to do this, and that’s the exact reason we are excited to be working in this field and very actively looking at companies in this general field of stem cells attempting to cure diseases.”

From Stem Cells to Cures

After listening to both Yamanaka and Byers, it was clear that both had the same view of the stem cell field. They both believe that we are at a turning point in stem cell research and that our efforts both at the bench and on the commercial side need to remain stalwart in their efforts to push stem cell research forward so we can develop safe and effective therapies for patients.

Blake Byers, Shinya Yamanaka, and Sandy Williams take questions from the audience.

Blake Byers, Shinya Yamanaka, and Sandy Williams take questions from the audience.

One comment from the audience that stood out was that the the main limitation to the success of stem cell research seems to be a reduction in funding at the very time we need to increase funding.

In response, Byers agreed and suggested that to fix the funding issue, there needs to be an objective function in stem cell research. He suggested that the field needs to “measure the output we are having and what the impact of it is.” He said what is currently lacking is an ability to “measure of that return on investment for society”.

Yamanaka followed up by addressing the issue of costs for cures. “The cost of new cures and medicines is extremely challenging but important. We now have many new medicines, but they are too expensive. How to lower those costs, [is a question] we seriously need to consider”.


Putting the promise to the test: a new move to see if stem cell therapies can help injured athletes

One of the toughest questions we get asked, and we get asked this a lot, is a variation on the theme of “I have xxxx disease and want to know where I can get a stem cell therapy for it?” All too often, in fact pretty much all the time, we have to explain that there aren’t any therapies available, at least not yet, and that it might be a couple of years before any of the really promising projects we are funding are enrolling patients in a clinical trial.

Injured knee

But still the questions come in, fueled in part by all the clinics and centers out there claiming they can treat everything from rheumatoid arthritis to type 2 diabetes and Crohn’s disease. The biggest problem of course is that very few, if any, of these centers and physicians back up their claims with any evidence or studies to show that their treatments work. They have patient testimonials plastered all over their websites. They have lots of very reassuring sounding information, but no evidence or proof that anything they are doing will work.

Fortunately there are a growing number of researchers and reporters holding these clinics up to the light to see if what they are claiming could be true. In most cases the answer is a resounding “heck no.”

Just a week ago we told you about a couple of recent reports that looked at all the claims about using stem cells to treat sports injuries and whether there is anything to support claims by some cosmetic practitioners that they can use stem cell-based therapies to reverse the aging process (spoiler alert – there isn’t, otherwise I’d be first in line to try them out).

Cover of article about stem cells in Muscle and Medicine

Cover of article about stem cells in Muscle and Medicine

But now some mainstream media reporters are taking a closer look at claims these therapies are effective, particularly those associated with top-flight athletes. A recent issue of Muscle and Medicine – an online website that is part of the Sports Illustrated stable of publications – carried a really in-depth and thoughtful look at the use of stem cells to treat superstar athletes.

Writer Jenny Vrentas sets the tone in the opening paragraph saying:

“It may be the next big breakthrough in the treatment of sports ailments, but for now the use of such therapy is strictly limited in the U.S. – and questions about effectiveness outweigh the answers.”

Vrentas carries that questioning attitude throughout, highlighting some of the athletes who talk openly about procedures (we can’t really call them “treatments” because we don’t know if they actually treat anything) but also profiling orthopedic surgeon, James Andrews, who is a proponent of stem cells and is trying to do the kind of study necessary to see if these therapies work or not:

Andrews speaks carefully about the potential of stem-cell treatments. He’s hyper-aware of the danger of sensationalizing among his clientele of elite athletes, particularly since many questions remain—not the least of which is how well the treatments actually work. But the early returns have motivated him, as has seeing his top patients go abroad for therapy: “They don’t really know what they are getting,” he says. “Are they getting illegal stuff? We don’t have any control over it, so it’s something we needed to bring back and do in a controlled environment here.”

It’s an excellent example of the kind of reporting that can really help people, weekend warriors or anyone else, who are wondering whether stem cells might help them. It highlights the promise, but also underlines the fact that we need proof to back up that promise before it’s ready for prime time.

kevin mccormack