Promising Results from Trial of Gene Therapy for 'bubble Boy' Disease

Hematopoietic stem cell transplantation (HSCT), once considered an effective yet risky alternative to drug therapy for blood cancer, has become more

Hematopoietic stem cell transplantation (HSCT), once considered an effective yet risky alternative to drug therapy for blood cancer, has become more accessible and successful in a wide range of patients as a result of major advances in transplant strategies and technologies. Several studies representing these advances were presented today during the 55th American Society of Hematology Annual Meeting and Exposition in New Orleans.

HSCT is effectively used today as a form of "replacement" therapy for patients with hard-to-treat blood cancers, providing healthy cells from either the patient (autologous transplantation) or from a donor (allogeneic transplantation) to better equip patients to fight the disease on their own. Historically, clinicians evaluating a patient for transplant have sought to identify donor cells that are perfectly matched to the patient's cell type, which is considered to be the optimal approach to help ensure successful outcomes and to minimize risk of graft-versus-host disease (GVHD), a serious and potentially life-threatening complication that occurs when the donated immune cells attack the patient's cells as foreign tissue.

Advanced Heart Failure Patients Benefit From Long-lasting Gene Therapy
The long-term benefits of a single dose of their gene therapy AAV1/SERCA2a in advanced heart failure patients was reported by researchers.

Today, researchers are challenging traditional assumptions about transplant eligibility and donor matching in an effort to expand the universe of patients who can benefit from this treatment while improving long-term success rates. New findings presented today indicate that with proper considerations, haploidentical (half-matched) transplants or transplants using cord blood cells can be viable, effective alternatives when a fully matched donor is not available. Researchers also contend that transplant outcomes can be further improved by identifying patients who are at high risk for certain complications, such as cognitive decline, or by employing post-transplant treatments to reduce their risk of relapse.

"The exciting research results being presented today underscore how transplants are becoming an increasingly successful treatment option for more patients with blood cancer than ever before," said Jeffrey Miller, MD, moderator of the press conference and Deputy Director of the Masonic Cancer Center and the Clinical and Translational Sciences Institute at the University of Minnesota in Minneapolis. "Whereas transplant would not have been an option for many patients without a sibling or other perfectly matched donor even several years ago, exciting new strategies have afforded them the opportunity to gain significant benefit from this procedure today. As we are now able to focus our efforts on improving the overall patient experience and reducing the risk of relapse, the leading cause of death after transplant, we have greatly improved long-term survival outcomes for patients who before might not have had another treatment option."

This press conference will take place on Saturday, December 7, 2013, at 10:00 a.m. CST.

Immunosuppression may Not be Required in Persistent Gene Therapy in Muscle
Previous research has indicated that successful gene therapy is based on the effective delivery and maintained expression of healthy copies of a gene into tissues of patients.

Encouraging Outcomes in Older Patients (Pts) Following Nonmyeloablative (NMA) Haploidentical Blood or Marrow Transplantation (haploBMT) With High-Dose Posttransplantation Cyclophosphamide (PT/Cy) [158]

To more fully demonstrate the utility of haploidentical bone marrow or peripheral blood stem cell transplants (haploBMT) as an alternative to fully matched transplants, this study evaluated nonmyeloablative, related haploBMT (with post-transplantation cyclophosphamide) among patients aged 50 – 75 with poor-risk hematologic malignancies. Results of 273 such transplants performed at Johns Hopkins were compared to determine the impact of older age on outcomes. The two-year probability of progression-free survival was very similar among patients in their 50s, 60s, and 70s (39, 36, and 39%, respectively), as was the two-year probability of overall survival (51, 56, and 44%). Among these age groups, there were also no statistically significant differences in the risks of non-relapse death or severe graft-versus-host disease.