EryDex is a slow-release system, he says, and research has found that it ''prevents the typical steroid side effects." These can include thinning of bones, high blood sugar, cataracts, and high blood pressure.
Research has shown the treatment can delay symptoms and the course of the disease. In one study, 18 patients given monthly infusions for 6 months had a better score on a commonly used scale to assess motor skills and many other aspects of daily living. The company is conducting its final study before seeking FDA approval. Results of that study are expected by the second half of 2019.
The treatment has been granted orphan drug status by the FDA. That means the drug is designed to treat rare diseases or is unlikely to ever be profitable to a drug manufacturer. Besides ataxia telangiectasia, the company is looking at other diseases, including cancer, their system could treat.
Rubius Therapeutics in Cambridge, MA, is also studying superblood treatment for rare diseases, cancers, and immune system disorders. It has raised more than 0 million in financing for research.
Advantages of Superblood, How It Compares With Other Therapies
The less-toxic superblood approach also allows the drugs to target tumors more directly, experts say. The drugs also stay in the body longer, so superblood may be more effective in the long term.
Experts say the new technology builds on another treatment advance known as CAR T-cell therapy. CAR T-cell therapy draws on the power of the body's T cells, known as the workhorses of the immune system, due to their ability to find and kill diseased cells.
In the CAR T process, blood is drawn from a patient, T cells are separated and genetically engineered to boost their tumor-fighting ability, then returned to the patient through an IV.
Compared to CAR T, superblood has more advantages, experts say. The superblood treatment is easier to use, can be made more quickly, and is long lasting.
The prestigious honorary medical society, founded in 1885, serves as one of the nation's leading professional organizations for physician scientists. Each year, nominees for membership undergo rigorous review and a competitive selection process.
An expert in the field, Li is recognized by AAP for his work in cancer immunology and immunotherapy. He and his research team have made pivotal contributions to understanding the immunological properties of heat shock proteins in cancer immunotherapy and immune tolerance. He was specifically cited for establishing the roles of GP96/GRP94 in immunity and cancer. He also uncovered the roles of TGFbeta-GARP axis on platelets in immuno-tolerance as well as discovered CNPY2 as a key initiator of the unfolded protein response.
Li serves as chair of the Department of Microbiology and Immunology at the Medical University of South Carolina and is co-leader of the Cancer Immunology Research Program at Hollings Cancer Center, South Carolina's only NCI-designated Cancer Center. A board-certified medical oncologist specializing in bone marrow and blood stem cell transplantation, Li is the South Carolina SmartState Center of Economic Excellence Sally Abney Rose Chair in Stem Cell Biology & Therapy.
"I am excited and honored to be a member of the Association for American Physicians and to have the opportunity to learn from the best and brightest physician scientists in the world," he said.
Nominations which are only accepted from AAP members, are determined by the nominee's advancement of medical knowledge through translational science. Inductees were recognized at the annual joint meeting for physician scientists April 20 through 22 in Chicago.
About Hollings Cancer Center
The Hollings Cancer Center at the Medical University of South Carolina is a National Cancer Institute-designated cancer center and the largest academic-based cancer research program in South Carolina. The cancer center is comprised of more than 120 faculty cancer scientists with a research funding portfolio of million and a dedication to reducing the cancer burden in South Carolina. Hollings offers state-of-the-art diagnostic capabilities, therapies and surgical techniques within multidisciplinary clinics that include surgeons, medical oncologists, radiation therapists, radiologists, pathologists, psychologists and other specialists equipped for the full range of cancer care, including more than 200 clinical trials. For more information, please visit /www.hollingscancercenter.org.