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

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

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.

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.

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”.