NYAS Conference highlights latest advances in primary immunodeficiencies (SCID)

One-day conference scheduled at Rockefeller University on April 25

Thirty years ago, a film entitled "The Boy in the Plastic Bubble" starred John Travolta as a child stricken with Severe Combined Immune Deficiency (SCID) who lived in a plastic enclosure until his death. Until recently, a newborn diagnosed with SCID would not survive to his or her first birthday. Today, clinical diagnosis and treatment advances in PI have cured certain of the "Bubble Boy" diseases as well as other PI disorders.

To highlight the latest advances in the treatment of Primary Immunodeficiencies (PI), a group of life threatening disorders that afflict millions of people in the U.S. and around the world, the Jeffrey Modell Foundation (JMF) and the New York Academy of Sciences is cosponsoring a one-day conference, Primary Immunodeficiencies: Past, Present, Future, on April 25 at Rockefeller University from 8 a.m. to 5:30 p.m. Thirty of the world's leading immunology investigators will present their latest findings and more than 350 scientists and physicians are expected to attend.

Consisting of four plenary sessions, the symposium will provide an overview of PI and detail the immense progress that has been made within the last 20 years in this field. Advances include the high success rate for bone marrow transplantation for unrelated, matched donors, as well as newborn screening for SCID and treatments derived from gene therapy. In addition, researchers are optimistic that stem cell research may yield new treatments in the future. The conference coincides with the 20 year anniversary of the Jeffrey Modell Foundation, a not-for-profit organization dedicated to PI research, physician education and patient support.

"In the last twenty years, there has been an amazing number of scientific breakthroughs," said Fred Modell, co-founder of the Jeffrey Modell Foundation. "For example, it is now possible to have an over 90 percent success rate in bone marrow transplantation, which would have been unthinkable two decades ago. We've made tremendous strides in newborn screenings for SCID, and advances in gene therapy and stem cell research hold out promise that difficult cases may be treated, and in some cases, even cured."

Among the highlights of the conference are:

• Max Cooper, M.D., Howard Hughes Medical Institute, University of Alabama, Birmingham, AL.
  Topic: "Evolution of Immunology - Where is the Future," an overview on the progress made from the nearly hopeless state of diagnosis and treatment of PI 20 years ago versus the startling advances of today.
• Jennifer Puck, M.D., Division of Immunology & Rheumatology, University of California, San Francisco.
  Topic: "Translating Research Advances--Detecting Severe Combined Immune Deficiency (SCID) Early Through Newborn Screening," a report on a joint venture between NIH, Affymetrix Inc. and the Jeffrey Modell Foundation in developing genetic screening for SCID using microarray technology.
• Chaim Roifman, M.D., Division of Immunology & Allergy, Hospital for Sick Children, Toronto
  Topic: "Can We Finally Choose Mismatched Related vs Matched Unrelated Donors for Transplantation of SCID?" - an update on research published Feb. 1, 2006 in JAMA showing how matched unrelated donors provided better engraftment, immune reconstitution and survival for SCID patients undergoing bone marrow transplantation compared to mismatched related donors.
• Alessando Aiuiti, M.D., Ph.D., San Raffaele Telethon Institute for Gene Therapy, Milan.
  Topic: "Current Challenges and Future Perspectives for Gene Therapy," commentary on successful outcomes in gene therapy to cure PI in clinical trials.
• Irving Weissman, M.D., Ph.D., Director, Institute for Cancer and Cell Biology, Stanford University School of Medicine, Stanford, CA.
  Topic: "The Big Picture: Politics and Science"

One of the world's leading experts on stem cell research will deliver a provocative update on stem cell research, its potential for curing a wide range of diseases and the presence of politics and government in science.

For more information, please visit www.nyas.org. For information on the Jeffrey Modell Foundation, please visit http://www.info4pi.org.

Founded in 1817, the New York Academy of Science is an independent nonprofit organization of more than 24,000 members worldwide dedicated to serving science, technology, and society

Last reviewed: By John M. Grohol, Psy.D. on 21 Feb 2009
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