Dear ASGCT Members and Colleagues:
Well it is hard to believe Y2K was 10 years ago! A lot has happened during the last decade but it is ending on a very positive note for many in our Society. Gene transfer and cell therapy have grown in scope and capabilities, and our Society has seen members who have capitalized on these advances. This year we saw the publication of three important clinical studies showing benefit in patients with genetic disorders. In January the New England Journal of Medicine reported work by Aiuti et al. demonstrating long-term clinical benefit in patients treated for adenosine deaminase deficiency. In November, Lancet published a manuscript by Maguire et al. providing additional encouraging responses for patients treated with Leber's Congenital Amaurosis. In that same month, Science published results by Cartier et al. demonstrating evidence that lentiviral gene transfer may slow disease progression in adrenal leukodystrophy.
While these results are encouraging, and very exciting for the patients who have benefited from these treatments, we still have much to do. Long-term benefit and safety must be the focus of ongoing work. Moreover, expanding the indications to cancer, infectious disease, and other disorders is still challenging.
As we look to the New Year, the Society will continue to focus on enhancing the careers of our members. This year we will offer the Clinical Training Course just prior to our Annual Meeting. This in-depth conference deals with the challenges of bringing novel gene and cell therapies to the clinic. The Annual Meeting is shaping up to offer an outstanding program. As usual, the Education Program will bring participants up to speed on basic and clinical aspects of gene and cell therapy. Our George Stamatoyannopoulos Lecture will be given by Hal Broxmeyer, Ph.D. whose pioneering work in hematopoiesis led to the development and clinical implementation of cord blood transplantation. He has also been active in evaluating the use of integrating vectors in stem cells and his work represents the synergy possible when cell and gene transfer technologies are combined. The Presidential Symposium will feature talks by Jean Bennett, MD, PhD who will speak about the experience in treating congenital blindness. The other speaker will be David Williams, MD who will discuss his work in hematopoietic stem cell biology and gene transfer. Many of you know David as the Editor-in-Chief of Molecular Therapy and he completed his 5-year term on December 31 of 2009. The Society owes a remarkable debt of gratitude to Dave for his vision and dedication, making Molecular Therapy the leading journal in our field.
So what will the New Year bring? First up, Malcolm Brenner begins his 5-year term as Editor-in-Chief of Molecular Therapy. Barrie Carter and I will head to the NIH in February and speak with NIH directors to ensure they are aware of the advances in our field and the value of cell and gene therapy. Xandra Breakefield is leading a Task Force looking at how to best represent gene and cell therapy within the Society. Jude Samulski is working on improving our website. Barrie Carter and John Englehardt have presented revisions to the Society's committees and abstract categories that better represent the cell therapists within our membership. Moreover, the ASGCT staff and our dedicated members will be reviewing abstracts and preparing for the Annual Meeting.
So I hope everyone had a wonderful Holiday and is ready for a great year ahead. I hope you can join us in Washington D.C. - and get working on those abstracts!
Ken Cornetta, MD
ASGCT President