The internet entrepreneur Sean Parker, founder of Napster and former president of Facebook is funding the first set of tests CRISPR gene-editing technology in human beings as a new cancer treatment , according to MIT Technology Review. This unique treatment is recently being reviewed and could become the first clinical trial involving CRISPR, a gene editing tool, according to the National Institutes of Health (NIH). The trial will be used to test the technology’s feasibility and safety in humans.
Parker, whose net worth is estimated at a hefty $2.4 billion, dived into the world of cancer research in April. The entrepreneur is planning to completely finance a $250 million grant to launch the Parker Institute for Cancer Immunotherapy, which will be a joint effort between scientists, technology experts and researchers to fund immunotherapy research initiatives and will be the biggest grant on immunotherapy research.
This move has been well appreciated by the medical and science communities as Parker’s decision will inspire other investors, particularly internet entrepreneurs, to make use of their financial standings to fund cancer research.
There is abundant motive and reasoning behind Parker’s decision as he is looking to control the cancer research market. Parker’s foundation is looking to control research patents and release treatment methods to the public, convincing scientists and researchers to make available their treatments for public use.
Clustered regularly interspaced short palindromic repeats (CRISPR) is a gene-manipulation immune therapy technology in which doctors extract a patient’s blood cells and genetically modify them using the technology to create special white blood cells called T-cells. The treatment, developed by University of Pennsylvania, utilizes modified blood cells, which are being used for treatment of all kinds of cancers including melanomas, myelomas and sarcomas.
The new cancer treatment uses gene-editing to modify T cells so that they more effectively target certain cancers other than leukemia. Previous trials have used a virus to insert a section of DNA that searches for cancerous mutations, before infusing the lab-made blood cells back into patients.
A proposal for a small safety study has been recently presented by Penn doctors Edward Stadtmauer, Jan Joseph Melenhorst , and Carl June, to the Recombinant DNA Advisory Committee (RAC), an NIH office in charge of reviewing gene therapy studies. Once it is cleared, up to 15 patients could receive the treatment, at three locations — University of Pennsylvania, the University of California and the MD Anderson Cancer Center. The study will revolve around complex safety issues, such as whether the altered genes using genetic engineering will be efficacious enough, and there is no guarantee it will go forward.
Parker, President of The Parker Foundation, said in a press statement, “We are at an inflection point in cancer research and now is the time to maximize immunotherapy’s unique potential to transform all cancers into manageable diseases, saving millions of lives. We believe that the creation of a new funding and research model can overcome many of the obstacles that currently prevent research breakthroughs. Working closely with our scientists and more than 30 industry partners, the Parker Institute is positioned to broadly disseminate discoveries and, most importantly, more rapidly deliver treatments to patients.”
“One of our key areas of focus is to develop novel approaches to modify T-cells to enhance their function,” said Jenifer Haslip, a spokesperson for the Parker Institute for Cancer Immunotherapy, headquartered in San Francisco.
“CRISPR technology provides an opportunity to profoundly manipulate cells,” according to a statement provided by Haslip. “We’re excited to be part of the first clinical effort in the United States to combine these two powerful therapeutic approaches to treat a devastating disease like cancer.”
Researchers are excited by the potential of using CRISPR as a genetic engineering technique in the fight against cancer, but scientists have also warned against the careless use of CRISPR to alter human genes. The technology could be used to create genetically perfected test-tube babies which is a subject of much controversy and could lead to harmful consequences down the line.
NIH Director, Dr. Francis Collins, reminded NIH’s commitment to encourage innovations in biomedical research in a manner that was both ethical and backed by scientific evidence and proper research. With organizations such as the RAC available to publicly discuss the scientific, safety, and ethical boundaries of such hi-tech clinical experiments, public safety is assured.
RAC approval in this case will be a major boost for human applications of CRISPR, but researchers still have a long way to go before the trial can begin, with the doctors involved needing similar responses from in-house ethics boards and the Food and Drug Administration. But even if the proposal does not go ahead, it seems to be a matter of when, not if CRISPR will be used in humans as other studies.