Cluster-busters against Cancer and a Potential New Gene Therapy for LCA

TumorGen and PhenoVista Biosciences partnered to explore metastatic cancer cell clusters to validate technology targeting these clusters and prevent cancer metastasis, which causes 90% of cancer-related deaths. 

Using PhenoVista’s analytical imaging technology, the two companies, who partnered last year, reported TumorGen’s micro-fluidic technology platform was able to detect multiple metastatic clusters from several lung cancer patients. The platform showed “tremendous sensitivity and specificity,” which proved to be more effective than previous efforts to collect and characterize the clusters. 

The clusters contained cancerous and non-cancerous cells, James Evans, CEO of PhenoVista, noted in the announcement. The companies announced the advancements could lead to improved therapeutic options for these patients.

The study, which the National Cancer Institute supported, notes that these metastasized clusters can be captured. This will allow for multiple analyses that could identify weaknesses and potential therapeutic approaches, which TumorGen dubbed “cluster busters.” This therapeutic could then be able to target the cells and prevent metastasis.

Jeffrey K. Allen, founder and president of TumorGen, noted in the company’s announcement that there is a “tremendous unmet medical need” for anti-metastatic therapies that can prevent tumors from forming across the body. He said the company’s platform is able to identify where these metastatic cancer cell clusters are vulnerable, enabling the development of those needed therapeutics.

He told BioSpace the company is energized to pursue therapeutic targets that can lead to a whole new class of anti-metastatic drugs that will save lives.  

“Our next steps will be to expand our efforts beyond lung cancer to other solid tumor types, all of which have been shown to release circulating metastatic cancer cell clusters,” he said. “We have also begun discussions with a biopharmaceutical company about beginning to use our technology to characterize MCCCs, confirm biomarkers of interest, and evaluate therapeutic targets within their pipeline.”

When it comes to looking for a partner, Allen said TumorGen hopes to pair up with a company that “is looking to differentiate themselves in the oncology space by focusing on stopping metastatic disease before it spreads.”

Allen said that the majority of cancer therapies remain focused on the primary tumor, while the different mechanisms controlling metastasis remain unaddressed. Since MCCCs are often heterogeneous and may contain immune cells that protect them from immune surveillance, Allen said they hope to find a partner interested in investigating targeting more than just the cancer cells within the cluster.

Gene Therapy Developed for Rare Blindness Disease

Researchers from the National Institutes of Health have developed a potential new gene therapy approach to Leber congenital amaurosis (LCA), a disease that causes blindness in children. 

A research team from the National Eye Institute, a division of the NIH, revealed that mutations cause a type of LCA in the NPHP5 gene, which leads to defects in the primary cilium found in cells throughout the body, Technology Networks reported. A deficiency in NPHP5 can cause blindness in children. However, the team explained that deficiency could lead to kidney disease in severe cases. 

Defects in 25 different genes can cause LCA. The FDA has approved Spark Therapeutics gene therapy Luxturna for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy, a form of LCA. That leaves other forms of this disease without treatment. 

Using stem cells collected from two patients with NPHP5 deficiency, the NIH team generated retinal organoids, which possess functional features of the natural retina. A deficiency of NPHP5 was found in the retinal organoids, along with lower levels of the protein CEP-290, which works alongside NPHP5. 

Using an adeno-associated viral vector that contained a functional version of NPHP5, the NIH researchers were able to target these extracted stem cells and demonstrate a “significant restoration of opsin protein concentrated in the proper location in outer segments.” The NIH research warrants further study and suggests a functional protein could prevent blindness in these children.

Further research is warranted, particularly in more severe forms of the disease.