"Guardian of the Genome" Therapies – A New Golden Age for Cancer R&D?
This is a guest post from Susan K Finston, President of Finston Consulting. Do you have a response to Susan’s post? Respond in the comments section below.
Last April, I wrote here about the paradox of cancer research funding where over $100 billion in R&D funding for genomic targeting of cancer tumors has yielded only modest gains for cancer patients. Despite the persistent lack of progress in curing most metastatic forms of cancer, risk averse, sclerotic, funding policies continue to throw good money after bad. As Nobel laureate Jim Watson points out:
“Targeted biological therapies don’t kill cancer cells, they are not curing cancer and it is unlikely that they can be made to do so in a practical or comprehensive way in the near future. It’s time for a change in strategy. We know the current approach is not working, because on the whole it has made no dent in cancer mortality.”
Now it looks like there is at least modest attention being given to an alternative approach to cancer R&D relating to the critical tumor suppressor protein, p53 (also cited by Watson).
Professor Sir David Lane first discovered the p53 protein in 1979, dubbing it “the guardian of the genome,” for the important role the protein plays in monitoring health of cells and preventing cancer. Mutation or deletion of p53 is highly correlated with growth of a majority of human tumors, and up to 90% of tumors for some cancer sub-categories like ovarian cancer. Overall, Lane asserts that “nearly every tumor has an affected or moderated p53 pathway,”
Finding ways to reactivate the key p53 protein has long been considered one of the ‘holy grails’ of cancer research. Now as reported by the New York Times, a number of the largest bio-pharma companies – including Roche, Merck, and Sanofi – are working actively on therapeutic approaches relating to reactivation of the p53 protein and effective against a range of cancer tumors.
Although not mentioned in the New York Times Article, a much smaller company, Cellceutix, may have the most promising p53 compound currently in human clinical trials at Harvard’s Dana Farber Cancer Center and Beth Israel Deaconess Medical Center.
Additional academic and bio-pharma start-ups also are pursuing p53 therapies at various stages of discovery and pre-clinical research.
These new therapies all focus on reactivation of the critical p53 tumor suppressor protein, responsible for controlling cell death and long recognized as the archetype of a molecular defect commonly associated with cancer tumors. If successful, this approach to develop “guardian of the genome” therapies would be a major departure from the genetic typing of tumors to a more holistic approach across cancer sub-types.
Renewed focus on p53 tumor suppressor therapies could truly be the start of a new golden age of cancer R&D that would bring more meaningful benefits to patients in the United States and globally – and coming not a moment too soon!
About the author:
President of Finston Consulting LLC since 2005, Susan works with innovative biotechnology and other clients ranging from start-up to Fortune-100, providing support for legal, transactional, policy and “doing business” issues. Susan has extensive background and special expertise relating to intellectual property and knowledge-economy issues in advanced developing countries including India and South Asia, Latin America and the Middle East North Africa (MENA) region. She also works with governments, and NGOs on capacity building and related educational programs through BayhDole25. Together with biotechnology pioneer Ananda Chakrabarty, she also is co-founder of Amrita Therapeutics Ltd., an emerging biopharmaceutical company based in India with cancer peptide drugs entering in vivo research. Previous experience includes 11 years in the U.S Foreign Service with overseas tours in London, Tel Aviv, and Manila and at the Department of State in Washington DC. For more information on latest presentations and publications please visit finstonconsulting.com.
I follow Cellceutix closely and I’m very encouraged with their trials to date. They are currently dosing for Cohort 7 at dosage levels equivalent in humans where there was tumor shrinkage and elimination in pre-clinical animal trials. I believe that there is much to be encouraged about with this company and their approaches to treating and possibly curing some types of cancers.
Yes, it is exciting to see Cellceutix and other companies moving p53 tumor suppressor therapies forward through preclinical and clinical testing. Another company, CDG Therapeutics, has completed Phase I Clinical Trials with a p53 targeting peptide therapy known as p28. Amrita Therapeutics Ltd., the company that I co-founded with Ananda Chakrabarty (he also co-founded CDG), is advancing a p53 peptide therapy known as AT-01 through preclinical testing. These could be real game-changers for cancer patients.