In patients with low and intermediate risk myelodysplastic syndrome and deletion 5q (del(5q)) treated with lenalidomide, monitoring of cytogenetic response is mandatory, since patients without cytogenetic response have a significantly increased risk of progression. Therefore, we have reviewed cytogenetic data of 302 patients. Patients were analyzed by karyotyping and fluorescence in situ hybridization. In 85 patients, del(5q) was only detected by karyotyping.

It has recently been shown that patients with myelodysplastic syndrome (MDS) may find that their time to stem cell engraftment post transplant is delayed beyond that seen by Acute Myeloid Leukemiaterm transplant patients (a more serious disease.) The thought behind these results is that the bone marrow's stroma (matrix cells) was not providing sufficient signal to the blood progenitor cells before the transplant took place, whereas the AML patients may have had progenitor cells with more serious mutations in their dna which would not have responded properly even if the stroma's signals were being provided. Therefore in many AML patients, transplanted stem cells are able to engraft quickly because the matrix environment is healthy and able to support the division and growth of new progenitors.

HOUSTON - A new scoring system for a form of leukemiaterm known as myelodysplastic syndrome (MDS) identifies patients who appear to have low-risk disease but actually have poor prospects of survival, researchers at The University of Texas M. D. Anderson Cancer Center report online at the journal Leukemia.

"We know an undefined group of MDS patients who are classified as low-risk by our present prognostic models will at some point have a sudden worsening of their disease. Right now, we don't know who these people are, but if we can identify them, we can start those with a poor prognosis on early treatment," says lead author Guillermo Garcia-Manero, M.D., associate professor in M. D. Anderson's Department of Leukemia.

Pharmion Corporation (NASDAQ:PHRM) today announced interim data from a Phase 2 clinical trial evaluating three alternative five-day dosing schedules for Vidaza(R) (azacitidine for injection) that demonstrated safety and response profiles which are consistent with those achieved with the FDA-approved seven-day regimen, as reflected in rates of hematologic improvement (HI) and red blood cell (RBC) transfusion independence. These responses were observed in a broad range of MDS patients, including FAB low-risk patients. These data were presented at the 49th Annual Meeting and Exposition of the American Society of Hematology (ASH) in Atlanta.

"The data from this study suggest that a five-day schedule is as effective as the currently approved seven-day schedule at achieving RBC transfusion independence and hematologic improvement in MDS patients," said Dr. Andrew R. Allen, executive vice president and chief medical officer of Pharmion. "A dosing schedule that circumvents the need for weekend dosing would provide a meaningful benefit in terms of convenience to these patients and clinicians."

• Exjade offers new alternative to burdensome standard therapy in children and adults who require blood transfusions for chronic anemias
  
• Approval makes iron chelation more accessible to patients suffering from diseases such as thalassemia, sickle cell and myelodysplastic syndromes

Basel, November 2, 2005 – Novartis announced today the first approval worldwide for Exjade^® (deferasirox) – the first and only once-daily oral iron chelator – by the US Food and Drug Administration. Exjade has been approved for the treatment of chronic iron overload due to blood transfusions in adults and children age two and older.

The European Myeloma Platform (EMP) says updated data for previously treated patients on REVLIMID (lenalidomide) plus the steroid dexamethasone shows median survival of 35 months, nearly three years. The data are pooled from two large randomized controlled Phase III trials that enrolled more than 700 patients from nearly 100 clinical sites worldwide. These updated results were presented at the 11th International Myeloma Workshop in Kos, Greece.

"Here in Kos we are seeing what is truly a paradigm shift in treatment for multiple myeloma patients," said Greetje Goossens, EMP member and leader of the Belgium myeloma support group. "The experts here are saying that nearly three years of median over all survival for REVLIMID patients is the longest they have seen in myeloma to-date from large clinical trials."

Disorders of blood cells may begin in the biological environment where the cells develop, not just with the cells themselves, according to a study from researchers at the Massachusetts General Hospital (MGH) and the Peter MacCallum Cancer Center (Peter Mac) in Melbourne, Australia. In the June 15 issue of Cell, the investigators describe finding that genetic alterations in the bone marrow of mice can cause a type of myeloproliferative syndrome, an overproduction of certain blood cells that also occurs in human patients.

“Previously all myeloproliferative syndromes have been considered to be intrinsic to the blood cells themselves,” says Louise Purton, PhD, of the MGH Center for Regenerative Medicine, formerly of Peter Mac, who led the study. “This discovery may help us find better therapies for these disorders, which can be quite difficult to treat, and also for some leukemias.”

New updated results from a pivotal Phase II trial evaluating Revlimid in patients with an incurable blood cancer known as myelodysplastic syndromes (MDS) were presented today by Dr. Alan List, from the H. Lee Moffitt Cancer Center & Research Institute, at the 9th International Symposium on MDS currently taking place in Florence, Italy. Breakthrough data presented at the meeting showed that Revlimid, or lenalidomide, can provide long-term survival benefit and prevent disease progression in MDS patients with chromosome 5q deletion.

"These landmark data demonstrate that Revlimid, in many cases, can help patients with MDS live transfusion free for several years. More importantly, we found that Revlimid can provide a significant long-term survival advantage, with 87 percent of cytogenic responders having a ten-year survival estimate," said Dr. List, Professor of Oncology and Medicine and Chief, Division of Malignant Hematology at Moffitt, and lead investigator of the study. "It is very rewarding to see patients treated with Revlimid, living longer, living three or four years transfusion free and having a better quality of life overall."

Vidaza, Revlimid, or straight to stem cell transplant? 

A good review of current techniques used for the treatement of myelodysplastic syndromes

 [via Expanded Academic ASAP Document]:

    The FDA has approved orphan drug status for Seattle Genetic’s two drugs SGN-33 and SGN-35. Orphan drug status allows Seattle Genetics to obtain marketing exclusivity of the drug upon approval. More importantly, it also allows Seattle Genetics an opportunity to obtain grant funding from the U.S. government to support clinical trials and defray costs. The Orphan Drug Act encourages companies to develop treatment for diseases that affect fewer than 200,000 people a year.

    SGN-33 has shown anti-tumor effects in phase I studies. SGN-33 (lintuzumab) is a humanized monoclonal antibodyterm that targets the CD33 antigen. SGN-33 is expected to enter clinical phase II by the end of 2007 testing mainly older patients with AML and MDS.

    Announced February 13th on Rigel's site, the second of several phase I studies to evaluate the safety and initial efficacy of the candidate R763 in different types of cancers is starting. The trials will tests the inhibitor in solid tumors, hematological malignancies, and in combination with standard therapies.

    Merck Serono licensed development and commercialization rights to R763 and Rigel's Aurora kinase program in October 2005. Aurora kinases are a family of kinases (proteins that put phosphate groups on other proteins) that regulate mitosis (cellular division). It is thought that these kinases are deregulated in cancer, and culture cell studies have shown Aurora kinases have anti-tumor effects.

When a patient undergoes chemotherapyterm, all dividing cell types are affected. Cancers are rapidly dividing cells and this is the property that chemotherapy looks to exploit. Unfortunately, since this is a non-targeted approach, several other dividing cells types in the body are also affected. White blood cells are one such affected group of cells along with Red blood cells and platelets (which are all derived from the Bone Marrow, which is a rapidly dividing cell type). This depression of WBC numbers is known as the Myelosuppressive effect. This usually ends up being the limiting factor on how much dosage a patient can withstand. If too many WBCs are destroyed, then it will lead to a weakened immune system and that will lead to secondary infections and sepsis. So to counter this, patients undergoing chemotherapy to treat breast cancer are given a supplement of a certain kind of growth factors called Cytokines that stimulate the growth of subtypes of white blood cells.

Please read the About Us section if you have been diagnosed with Treatment related MDS. Leave your questions in the forums and I will be happy to help you with the latest research.

    Women with breast cancer who receive compounds that stimulate white blood cell production to help their bodies better tolerate chemotherapyterm are at an increased risk of developing a type of leukemiaterm or a condition called myelodysplastic syndrome, according to a new study in the February 7 Journal of the National Cancer Institute.