David Higgins, CIRM Board member and Patient Advocate for Parkinson’s disease; Photo courtesy San Diego Union Tribune
When you have a life-changing, life-threatening disease, medical research never moves as quickly as you want to find a new treatment. Sometimes, as in the case of Parkinson’s disease, it doesn’t seem to move at all.
At our Board meeting last week David Higgins, our Board member and Patient Advocate for Parkinson’s disease, made that point as he championed one project that is taking a new approach to finding treatments for the condition. As he said in a news release:
“I’m a fourth generation Parkinson’s patient and I’m taking the same medicines that my grandmother took. They work but not for everyone and not for long. People with Parkinson’s need new treatment options and we need them now. That’s why this project is worth supporting. It has the potential to identify some promising candidates that might one day lead to new treatments.”
The project is from Zenobia Therapeutics. They were awarded $150,000 as part of our Discovery Inception program, which targets great new ideas that could have a big impact on the field of stem cell research but need some funding to help test those ideas and see if they work.
Zenobia’s idea is to generate induced pluripotent stem cells (iPSCs) that have been turned into dopaminergic neurons – the kind of brain cell that is dysfunctional in Parkinson’s disease. These iPSCs will then be used to screen hundreds of different compounds to see if any hold potential as a therapy for Parkinson’s disease. Being able to test compounds against real human brain cells, as opposed to animal models, could increase the odds of finding something effective.
Discovering a new way
The Zenobia project was one of 14 programs approved for the Discovery Inception award. You can see the others on our news release. They cover a broad array of ideas targeting a wide range of diseases from generating human airway stem cells for new approaches to respiratory disease treatments, to developing a novel drug that targets cancer stem cells.
Dr. Maria Millan, CIRM’s President and CEO, said the Stem Cell Agency supports this kind of work because we never know where the next great idea is going to come from:
“This research is critically important in advancing our knowledge of stem cells and are the foundation for future therapeutic candidates and treatments. Exploring and testing new ideas increases the chances of finding treatments for patients with unmet medical needs. Without CIRM’s support many of these projects might never get off the ground. That’s why our ability to fund research, particularly at the earliest stage, is so important to the field as a whole.”
The CIRM Board also agreed to invest $13.4 million in three projects at the Translation stage. These are programs that have shown promise in early stage research and need funding to do the work to advance to the next level of development.
- $5.56 million to Anthony Oro at Stanford to test a stem cell therapy to help people with a form of Epidermolysis bullosa, a painful, blistering skin disease that leaves patients with wounds that won’t heal.
- $5.15 million to Dan Kaufman at UC San Diego to produce natural killer (NK) cells from embryonic stem cells and see if they can help people with acute myelogenous leukemia (AML) who are not responding to treatment.
- $2.7 million to Catriona Jamieson at UC San Diego to test a novel therapeutic approach targeting cancer stem cells in AML. These cells are believed to be the cause of the high relapse rate in AML and other cancers.
At CIRM we are trying to create a pipeline of projects, ones that hold out the promise of one day being able to help patients in need. That’s why we fund research from the earliest Discovery level, through Translation and ultimately, we hope into clinical trials.
The writer Victor Hugo once said:
“There is one thing stronger than all the armies in the world, and that is an idea whose time has come.”
We are in the business of finding those ideas whose time has come, and then doing all we can to help them get there.
The Stem Cellar 
There has actually been remarkable progress on potentially curative stem cell-based therapy for Parkinson’s disease. Clinical trials for dopamine neuron replacement therapies will begin next year by groups belonging to GForce PD, a partnership to coordinate methods and results in similar studies. NYSTEM, New York state’s stem cell agency, has invested more than $20 million in a project to use dopamine neurons derived from human embryonic stem cells that will start clinical trials next year. The European Union has invested in a similar hESC approach in Sweden and the UK, also providing more than $20 million. The Japanese government has invested at least as much in a similar trial that uses non-matched iPSCs.
There is hope for Californians to also have access to this kind of therapy. The fourth member of GForce is my lab in California, who have used mostly private donations to develop a patient-matching autologous neuron replacement therapy for Californians suffering from the disease. But CIRM has invested only $2.4 million in our project, not nearly enough to do the studies required by the FDA, so we’ve had to do a lot of outside fundraising. But we still plan to start our clinical trial in 2019.
“Victory over Parkinson’s disease” is the mission of our largest private donor, the Summit for Stem Cell Foundation. http://www.summitforstemcell.org