For the first time, researchers at New York University have successfully transplanted a genetically engineered kidney from a pig into a human patient. This is a remarkable milestone as animal organs have traditionally been rejected by humans. The development could play a critical role in the treatment of the 100,000-plus patients awaiting organ donation in the United States. We also explore a gene-editing breakthrough in children with a rare immune disorder, the U.S. Food and Drug Administration’s proposal to expand hearing aid access to millions of Americans, thoughts for clinical trial executives on reducing the cost of decentralized clinical trials, and artificial intelligence in breast cancer screening.
Each week we highlight five things you need to know about in the life sciences industry. Here’s the latest.
Gene-edited pig kidney successfully implanted in human
Xenotransplantation of animal tissues into humans has the potential to improve or save the lives of countless patients on organ transplant lists. In the United States alone, over 100,000 people are waiting for an organ (90,000 need a kidney), and through September, only 31,000 transplants have been possible. But findings from a new research surgery have the potential to alleviate this situation. Surgeons were able to successfully transplant a genetically modified kidney from a pig into a human patient; the organ began functioning immediately and was not rejected during the study. To be clear, the transplant took place on a patient with no brain function, on life support and was only monitored for 54 hours. However, because of incompatibility of the human immune system with non-human tissues, transplants have historically been rejected very quickly. In this instance the pig kidney had been genetically modified to eliminate the enzyme that triggers rejection.
Children born with deadly immune disorder remain healthy 11 years later
Eleven years ago, researchers at UCLA began using gene therapy to treat an immune disorder in children known as ADA-SCID, or adenosine deaminase-deficient severe combined immunodeficiency. The researchers removed blood-forming stem cells from each child’s bone marrow, then used a specially modified virus, originally isolated from mice, to guide healthy copies of the ADA gene into the DNA of the stem cells. They then transplanted the cells back into the children’s bone marrow. At the time, researchers did not know the success the treatment would have. Eleven years later and in a promising success, nine of the 10 children treated are experiencing restored immune function.
FDA issues proposal to improve access to hearing aid technology
According to a release from the FDA, approximately 15% of American adults (37.5 million) age 18 and over report having some trouble hearing. Even so, only about one-fifth of this population uses a hearing aid. Following the Biden administration’s July executive order on Promoting Competition in the American Economy, the FDA issued a proposal intended to improve access to hearing aids and lower their cost through the creation of an over-the-counter hearing aid category.
Decentralized clinical trials offer cost savings
Clinical trial executives often evaluate trial design with patient centricity at the forefront. Costs receive less scrutiny than items affecting the patient experience, resulting in persistent opportunities related to cost reduction in the decentralized clinical trial model. This article explores shifts in thinking that may help executives pinpoint those cost-reduction opportunities while maintaining a patient-centric experience.
AI is helping to make breast cancer history
October is Breast Cancer Awareness Month, and while significant advances in screening, detection and treatment have happened over the last several decades, about 700,000 women globally still die every year from the disease. Early detection and access to health care have greatly improved outcomes, but the pandemic has introduced a new set of challenges for preventative medical treatment: an overall decrease in frequency of non-emergency care; a growing backlog of necessary treatments and procedures; and depleted health systems. In the United Kingdom alone, the National Health Service said that it would need 2,000 more radiologists just to clear the backlog of scans and keep up with growing demand. To augment the capacity of health care providers and support better patient outcomes, AI is being developed and deployed in a multitude of ways, several of which are specifically targeted at breast cancer.
The NHS has secured funding to study how AI can be used to screen for breast cancer, and startups are also leveraging AI to increase the rate and accuracy of traditional screenings, as well as to develop new low-cost and less-technology-reliant methods to bring access to rural and low-income populations. Additionally, a cancer hospital in France has developed AI that can identify four out of five cases at high risk of relapse, an important step in reducing a cancer that persists in one in 10 patients. While there is still work to do, innovations such as these are a promising development in the fight against cancer.
