Ensconced in their labs for years or even decades to pursue discoveries including new drugs, idealistic basic scientists have had little time and few skills for getting them commercialized for treating diseases. Unfortunately, some of their ideas have thus fallen at the wayside and never reached the patients’ bedsides.
A chemical and systems biologist, Prof. Daria Mochly-Rosen of California’s Stanford University – one of the world’s leading research and teaching institutions, which was founded in 1885 and has produced 85 Nobel laureates – has done much to fill this gap.
Born in Haifa, raised in Moshav Shavei Zion and educated at Tel Aviv University and the Weizmann Institute of Science in Rehovot, she moved to the US and 16 years ago founded SPARK, a model that has been adopted in more than 70 institutes and on all continents. SPARK is a unique partnership between university and industry experts to generate evidence of discoveries’ benefits using academic approaches combined with pharmaceutical industry standards.
Mochly-Rosen, who is SPARK’s president and the George D. Smith Professor of Translational Medicine at Stanford, is the founder of Mitoconix Bio, a start-up company whose goal is to produce drugs that treat Huntington’s disease and other neurodegenerative illnesses. She has also founded two other biotech companies.
“I left Stanford for a year for this so there wouldn’t be a conflict of interest, in 2003,” recalled Mochly-Rosen. “I learned more about drug development and commercialization in that short time than I learned in the previous 20 years. I felt there were a lot of ideas on campus that might not end up to benefit patients. Students aren’t prepared to get involved in industry.”
She leads multi-disciplinary research at Stanford on the molecular mechanisms of diseases, has published more than 280 journal papers and holds dozens of patents. Mochly-Rosen also was chairman of her university’s department of chemical and systems and senior associate dean for research at its medical school.
“I have a lab and teach students on a regular basis, but SPARK is my passion. We stick only to health issues, because we don’t want to spread too thin.”
Although she is an expert in chemical and systems biology and a member of Bio-X, Stanford’s pioneering interdisciplinary biosciences institute, her university also made her a member of its cardiovascular, maternal and child health research, cancer and neurosciences institutes.
Mochly-Rosen's background
She is married to Emanuel (Manu) Rosen, an Israeli-born writer of books about business, and they have four adult children – a psychologist, a geographic information systems specialist, a neuroscience professor at Smith College in Massachusetts and a businesswoman.
A regular visitor to Israel to visit family and for professional work, she was a guest at a recent two-day international conference at Haifa’s Rambam Medical Center, where she spoke about translational medicine, medical innovations and SPARK’s global success.
“My experience in translating basic research to drug development led me to found SPARK in 2006,” she told The Jerusalem Post. “SPARK – which is not an acronym but reflects the idea that it ignites ideas – has a 60% success rate each year, as measured by licensed projects to existing companies or start-ups and/or conducting clinical studies addressing local and global health care needs. Since 2006, 48 projects funded by SPARK have been licensed to either existing biopharmaceutical companies or to new start-ups. We want to ensure that good ideas don’t end up just as good scientific papers in medical journals but that they benefit patients.”
If a project it advises becomes a start-up or joins with an existing company, SPARK regards it as a success.
“We don’t judge it according to how much money it makes. When industry initiates projects alone, without SPARK, its success rate is only 5% to 10%.”
It set up companies that are doing clinical trials, many of them in phase-3 clinical trials.
“We hope some will soon be on the market. Some have been repurposed existing drugs for other diseases,” Mochly-Rosen said.
For example, one team identified two human host proteins that are essential for viral life cycle. With SPARK support, the team discovered that a combination of two FDA-approved anti-cancer drugs were effective against hepatitis C, dengue, West Nile, and Ebola viral infections. During the Ebola outbreak in West Africa several years ago, the drug combination therapy was selected for a clinical study in Sierra Leone that was sponsored by the World Health Organization and the Gates Foundation.
The SPARK program proved that it could be implemented anywhere and be an effective tool.
“Last year, I was asked to show our economic impact,” she said. “Two companies that were established as a result forged a partnership with industry and three went public, with more than $1.3 billion gained.”
SPARK, on the campus near Palo Alto, has more than 200 volunteer mentors who are professionals in their fields, she said. One is even a regulator for the US Food and Drug Administration.
“Multiple advisers is the reason why our program is so successful, because scientists need to hear multiple opinions,” she continued.
Stanford’s SPARK Scholars program provides funding for product proposals and mentoring for participants. The mentors are advisers from the same field with expertise in product development, clinical care and business, preparing participants for careers that link investigation with important new therapies. The program at her university is open to professors, clinicians, postdoctoral scholars and graduate students who seek mentors’ advice.
Other academic institutions around the world – at Rambam Medical Center and the Technion in Haifa and Tel Aviv University, in Canada, Germany, Australia, Taiwan, Brazil, Jordan and other countries – have successfully replicated the SPARK model at Stanford, which is committed to supporting their work. “In Jordan, they know I’m an Israeli, but it doesn’t bother them,” she added.
“The mentors sign confidentiality agreements ensuring that they do not reveal any information on research discoveries. The mentors meet regularly to ensure that research projects don’t get stuck and that industries can invest in them. Clinical trials are sped up, journal papers are written and published and, eventually, approved drugs can be given to patients. Basic scientists are clueless on how to do this.”
She hopes Israel’s chief scientist in the Innovation Authority will become involved with SPARK.
“We are in a very advanced discussion on doing national work with us, especially in drug development. You have to be determined. If you do basic research using taxpayer funds, this is the way to pay the state back. Academic institutions should do their utmost to show that they are mature enough to develop further and to benefit patients. You have to be very disciplined.”
She advises Israeli academic institutions to “teach content, for their scientists to look for things that will have utility. They don’t necessarily have to set up their own company, but the aim is to improve the treatment for patients.”
It takes an average of 15 years for a scientist’s idea to come to market. Some, like the highly successful drug Copaxone – the Weizmann Institute’s product for slowing multiple sclerosis – was worked on by Prof. Ruth Arnon and the late Prof. Michael Sela, with the late Dr. Dvora Teitelbaum – for three decades.
Asked about drug research in Israel, Mochly-Rosen says the country has “quite a few amazing women scientists. Had I been aware of how women experienced their environments in Israeli research, I would have stayed. I regard myself as an Israeli. At the Rambam event, I was so impressed and proud to be an Israeli.”
Comparing young American and Israeli students, she said Israelis are older because of army service, which gives them a sense of confidence and maturity.
“They come with greater resilience and strength from their strong family bonds. There is a sense of belonging and purpose. It is not surprising that Israel has been described as the Start-Up Nation.”
She recalled that “a very-smart senior scientist in my lab told me once when I was starting a new project that I was undisciplined. I think I am undisciplined in a way, because I like many disciplines and variety. I see problems that I think I can solve and go for it – so I never left for industry permanently. In academia you can follow your nose. I am most interested in human health. One has to dare and change directions if one feels there is a need. I am a true believer that important discoveries are made by people not in the same field. One has to be interdisciplinary.” The SPARK (Nitzotz) Program, administered by Rambam Medical Center’s MedTech and funded in part by donors, was established to bridge the gap between invention and commercialization. It enables Rambam-based projects to achieve early technical milestones leading to follow-on investments.
Founded in 2012, the program’s vision is to nurture promising innovations into successful products for the benefit of patients worldwide. To that end, funds are provided to determine feasibility for very early stage projects with a high probability of success, capable of reaching a pre-defined milestone to facilitate commercialization and continued development.
A committee of established researchers and leaders from the medical industry examines the proposals to decide who will be considered for support. Each selected proposal is additionally supported by appropriate professional staff, providing assistance, counsel and developing a related business proposal whenever appropriate.
Rambam Medical Center’s director-general Prof. Michael Halberthal said the “collaboration between Rambam and Stanford Medicine is growing stronger in a number of areas that all have in common medical innovation. It has tremendous potential to strengthen international cooperation and promote the health of different and diverse populations around the world.”
Prof. Rafael Beyar, who was the hospital’s director when it began collaborating with Stanford, added: “It began in the field of oncology and cardiology and is now deepening in the fields of innovation and trauma. During the summit meeting, we expanded our cooperation in the fields of medical innovation, data analysis and big data and in the fields of health systems’ management in emergency conditions.”