Falcon Genomics continues to work toward its goal of seeing its research-use-only Cancer Biochip System adopted in a clinical setting.
As part of that effort, the company recently demonstrated in two papers the ability of the platform to test the effectiveness of different short-interfering RNAs targeting genes in breast cancer cells to reveal therapeutic targets. In addition, Falcon is developing a higher-throughput, next-generation version of the system, according to a company executive.
Rula Abbud-Antaki, Falcon Genomics' president and chief scientific officer, told BioArray News this week that the company now feels that its CBCS platform is "ready for clinical use." As evidence of its clinical utility, she cited several validation studies, including one published last week in PLoS One, and another in June in Hormones and Cancer.
As described in the more recent PLoS One paper, Abbud-Antaki and colleagues looked at the effectiveness of 40 different siRNA targeting genes in breast cancer cells that were cultured on the CBCS three-dimensional platform, and were able to observe tumor growth on the chip in real time, as well as identify siRNAs that blocked such growth, she said.
Another outcome of the study was the discovery that specific siRNAs, such as ß-actin siRNA, acted as "potent" suppressors of breast cancer growth in certain patients, potentially revealing new therapeutic targets, she said. Abbud-Antaki credited the CBCS 3D technology with the results.
"We believe that [ß-actin] has been overlooked by previous investigators because they grew cells on flat surfaces, whereas our assay has higher translatability to the in vivo milieu," Abbud-Antaki said. "The scientific basis for this finding also makes a lot of sense because several cancer therapies actually target the actin cytoskeleton," she added.
Abbud-Antaki co-founded Falcon Genomics with James Antaki and Victor Keyloun in 2004. The Pittsburgh-based firm's CBCS platform uses a three-dimensional biochip to enable the high-throughput, automated, and quantitative identification and validation of inhibitors of cells.
According to US Patent No. 8,110,375, which the firm received in February of this year, each chip consists of a slide coated with a soft agar matrix that supports anchorage-independent, three-dimensional cell growth. Spots containing active agents are placed in or on the matrix, and cells are plated on the matrix and allowed to grow in the presence of the active agents.
To date, the chip has been available for research use, but Abbud-Antaki said that the recent validation studies could enable the firm's entry into the clinical market.
"In fact, during our validation studies, we found some results from patients with very aggressive tumors and possible options for treatments that we wish we could have reported back to the attending physician," said Abbud-Antaki. "All we really need now is to get the word out, and that is why we are so excited about these recent publications."
In Abbud-Antaki's opinion, the CBCS could provide "two very unique benefits" to clinicians. If drug therapies exist for a certain patient's disease, the CBCS could be used to select the most effective treatment, she said. And if the patient's cancer is not presently treatable, she said the CBCS could provide them with an assessment of the aggressiveness of their cancer, "as opposed to the 'let's wait and see approach' that is the current alternative."
To get the chip into the clinic, Falcon is seeking "all types" of funding to expand its validation studies to a larger number of breast cancer patients, and looking for collaborators.
"Ideally, we hope to find a pharma partner that can help accelerate the translation to clinical practice," she said. Abbud-Antaki added that Falcon Genomics has had "very preliminary discussions" with potential partners, but declined to elaborate.
Abbud-Antaki believes that the CBCS will not require US Food and Drug Administration clearance, but that it does need to "demonstrate sufficient efficacy to convince clinicians to adopt the technology."
She said that the opinion of Falcon Genomics regulatory experts is that as long as the assay is run in a lab that is compliant with the US Clinical Laboratory Improvement Amendment — and the company publishes the more validation studies in peer-reviewed journals — that "will be sufficient" to see the platform into the clinic.
While Falcon works toward that major goal, it is also developing a new version of the CBCS that will allow the firm to test "each and every abnormally expressed tumor gene," Abdul-Antaki said.
In the two most recent studies, the first-generation CBCS was used to look at fewer than 100 siRNAs, but according to the '375 patent, each slide could technically hold up to 150,000 siRNAs. Not only will the new version of the CBCS offer higher-multiplex assays, it will also be "highly miniaturized," Abbud-Antaki said. In the '375 patent, the company describes matrices containing spots that are between 65 and 120 micrometers in diameter.
Falcon Genomics is currently applying to the National Institutes of Health for Phase II Small Business Innovative Research funding. Last December, the firm completed the milestones from a $270,000 Phase I SBIR grant it received two years ago, Abbud-Antaki said (see BAN 10/26/2010).
In the meantime, Falcon Genomics will continue to analyze patient tumor samples on the CBCS, and is working on two additional papers with results from several types of breast cancers, Abbud-Antaki said.