Strategies to accelerate innovative academic projects

Symposium on drug discovery aims to disseminate culture of innovation and explore ways to facilitate conversion of basic research into drugs and other innovative products.

By Karina Toledo  |  Agência FAPESP – The First International Symposium on Drug Discovery in Academia (ISDDA 2016) was held on November 17 at Butantan Institute in São Paulo, Brazil. This symposium was held to allow academic researchers in the fields of biomedicine and biotechnology to network with representatives of the pharmaceutical industry and to discuss strategies favoring the conversion of basic research into drugs and other innovative products.

The event was organized by the Center of Excellence in New Target Discovery (CENTD) – funded by FAPESP, GlaxoSmithKline (GSK) and Butantan Institute – and the SPARK-Supernova Program, established by researchers at the University of São Paulo’s Biomedical Science Institute (ICB-USP) and the same university’s Engineering School (POLI-USP), with additional participation by Dow Chemical, to “accelerate” innovative academic projects.

"SPARK-Supernova was launched in 2014, and since then we’ve held several events to disseminate the culture of innovation in the academic community. More recently we began partnering with Butantan Institute’s CENTD, whose mission is similar to ours: to prospect for and accelerate new projects, with the aim of converting them into products,” said program head Julio Cesar Batista Ferreira, a professor at ICB-USP.

“The idea of the meeting was to bring together academia and industry for a discussion of drug discovery and development,” said Vanessa Zambelli, a researcher at Butantan Institute and a member of CENTD’s Scientific Diffusion Committee. “The event featured a number of presentations on success cases, offering information for us to absorb so that we can try to emulate these examples under our own conditions.”

One of the success cases was presented by Che-Hong Chen, a senior research scientist at Stanford University School of Medicine in the United States. Some years ago, he discovered the molecule ALDA-1. In preclinical trials, this molecule displayed the potential to treat several health conditions associated with a buildup of aldehyde, including peripheral arterial disease, heart failure and alcohol poisoning, especially in patients suffering from deficient production of aldehyde dehydrogenase-2, an enzyme that contributes significantly to aldehyde metabolism and limits heart attack-associated damage.

Chen founded the startup Aldea Pharmaceuticals with financial support from Stanford University via SPARK, the program that inspired SPARK-Supernova and that is now its partner. SPARK’s mission is to advance new biomedical research discoveries into promising new treatments for patients (“bench to bedside”).

“The program connects researchers at Stanford with physicians in the field and with specialists in the pharmaceutical industry and in biotechnology to partner on projects, with the latter acting as mentors,” Chen said. “There’s strong pressure for results, and there’s a timetable with goals to be achieved during the two-year funding period.”

Ferreira, who worked at Stanford for part of his PhD and did his postdoctoral research there, is currently collaborating with Chen’s team and with Aldea Pharmaceuticals in preclinical testing of ALDA-1 (read more in these stories: Molecule reduces heart function impairment after myocardial infarction, Molecule shows potential for fighting heart failure, and The heart has a new ally against myocardial infarction).

The first round of clinical trials to prove the molecule’s effectiveness in treating alcohol poisoning is complete, and the results will be published soon. “In the case of patients with a deficiency of aldehyde dehydrogenase-2, the treatment can drastically reduce hospital stay length, representing significant savings for hospitals,” Ferreira said.

Seven projects are currently being accelerated at ICB-USP with the support of SPARK-Supernova. Five have already become startups and are being funded by FAPESP’s Small Business Innovative Research Program (PIPE): “Improvement and innovation applied to the newborn screening test for congenital immunodeficiencies”, “Characterization of induced pluripotent stem cells-derived cardiomyocytes and cellular assay standardization”, “Development of human keratinocytes and dermo-epidermal equivalents from pluripotent stem cells”, “New immunotherapeutic approach against HPV-induced tumors”, and “Inexpensive rapid serological detection of Zika virus using IgG/IgM ELISA platform”.

“We offer training and guidance for professors and students interested in converting their ideas into products,” Ferreira said. “To this end, SPARK-Supernova has a team of voluntary academic and non-academic consultants who help accelerate the project development process with the target product in mind. We have partners at the University of São Paulo’s School of Pharmaceutical Sciences [FCF-USP], Medical School [FM-USP], School of Veterinary Medicine [FMVZ-USP] and Center for Innovation, Entrepreneurship & Technology [CIETEC-USP]; at Butantan Institute; at the São Paulo State Technological Research Institute [IPT]; and at the Energy & Nuclear Research Institute [IPEN], as well as collaborators in the private sector.”

The group’s capabilities have recently been bolstered by the creation of Global SPARK, a network linking 13 SPARK programs on five continents. “The network will help us exchange information, as well as facilitating interaction between accelerated projects and international advisors. We hold regular meetings to track progress and discuss projects,” Ferreira said.

SPARK-Supernova’s initial focus is on the discovery and development of drugs for clinical use. “But the idea is to expand our field of action as soon as we’re able to bring in more consultants,” he added.

Mutual benefits

One of the representatives of the pharmaceutical industry in attendance at the event was GSK’s Kevin Madauss, who presented Trust in Science, a research funding initiative designed to build long-term partnerships with leading scientific research centers in Latin American and African countries, among others.

“In Brazil we’re partnering with FAPESP and the National Scientific & Technological Development Council, CNPq, through this program,” Madauss said. “In FAPESP’s case, the focus is advancing through CENTD in the initial stages of the drug discovery process, especially validation of therapeutic targets.”

The numbers presented by Madauss illustrate why partnering with academic research centers is worthwhile for the pharmaceutical industry. On average, he said, the industry takes about 12 years to develop a new drug and invests some US$1.3 billion in the process.

“Most of the cost is incurred in the final stages, which include clinical trials. The discovery of new targets and new molecules, which runs from the first to the fourth year, costs much less. If, with the help of academic researchers, we can eliminate unpromising candidates early on and reduce failures in the more advanced stages, we’ll save a huge amount of time and money,” Madauss said.

Fernando Perez, CEO of Recepta Biopharma, said partnerships with universities were essential to his firm’s business model.

“In Brazil, the most highly qualified professionals are at universities and won’t want to leave a tenured position to face risks in industry,” Perez said. “We’ve opted to place people paid by Recepta in university labs to be sure all the processes are properly performed and recorded.”

Perez stressed the need for Brazilian industry and academia to create a common language and promote a cultural change at research institutions so that scientists become more open to partnering with business organizations.

Also attending the event were Camille R. Silva (Pharmagenix), Eyleen O’Rourke (University of Virginia, USA), Eric Gross (Stanford University School of Medicine), Artur T. Cordeiro (National Bioscience Laboratory, LNBio, Brazil), Carsten Wrenger (ICB-USP) and Jackson C. Bittencourt (ICB-USP).