Alan List, M.D., leader of the Hematologic Malignancies Program at the H. Lee Moffitt Cancer Center & Research Institute, recently conducted a phase I/II trial of the experimental drug Revlimid showing promise as an innovative way to treat patients with myelodysplastic syndrome (MDS), a form of pre-leukemia. Given in pill form, Revlimid simultaneously blocks the growth of new blood vessels that nourish tumors (anti-angiogenesis) and stimulates the immune system to fight cancer cells. The study is reported in the Feb.10 issue of the New England Journal of Medicine.

Researchers have created a way to transform the dead bone of a transplanted skeletal graft into living tissue in an experiment involving mice. The advance, which uses gene therapy to stimulate the body into treating the foreign splint as living bone, is a promising development for the thousands of cancer and trauma patients each year who suffer with fragile and failing bone grafts. The findings were posted online Feb. 13 and will appear in the March 1 issue of Nature Medicine.

The procedure, designed by a team led by Edward M. Schwarz, Ph.D., associate professor of orthopedics and of microbiology and immunology at the University of Rochester Medical Center, is intended to eventually aid people with various cancers or injuries whose treatment involves the replacement of large sections of bone. Cancers such as osteosarcoma, one of the most common types of bone cancers, or tumors that occur adjacent to bones, often must be treated by removing the diseased section of bone and replacing it with the only alternative available – a donated section of comparable bone from a cadaver. The new splint of bone is then literally screwed into place, giving the patient most of the strength and support of the original bone. Bone, unlike any other tissue in the human body, can still perform one of its functions, structural support, even if all its cells are completely dead. A serious problem arises, however, when the bone wears over time.

The crystallized form of a molecular machine that can cut and paste genetic material is revealing possible new paths for treating diseases such as some forms of cancer and opportunistic infections that plague HIV patients.

Purdue University researchers froze one of these molecular machines, which are chemical complexes known as a Group I intron, at mid-point in its work cycle. When frozen, crystallized introns reveal their structure and the sites at which they bind with various molecules to cause biochemical reactions. Scientists can use this knowledge to manipulate the intron to splice out malfunctioning genes, said Barbara Golden, associate professor of biochemistry. Normal genes then can take over without actually changing the genetic code.

The results of the Purdue study are published in the January issue of the journal Nature Structural and Molecular Biology.