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Drug Helps Patients at High Risk for Leukemia
Gentle Therapy Gives Some Elderly More Options and Hope

January 2005 - A drug gentle enough for patients in their 70s or 80s is showing promise for the treatment of myelodysplastic syndrome (MDS), a pre-leukemic disorder that can progress to acute myelogenous leukemia (AML), according to study results presented in December at the annual meeting of the American Society of Hematology.

The drug R115777, or tipifarnib (Zarnestra®), produced results ranging from complete responses to improvements in blood counts in about one-third of 82 patients, says the studys lead investigator, Razelle Kurzrock, M.D., director of the Phase I Program in the Division of Cancer Medicine at M. D. Anderson. Study participants were treated at seven hospitals in the United States, Canada and Europe.

Therapy gives new treatment option to MDS patients

The treatment outcome, which includes well-tolerated side effects, gives the mostly elderly patients who develop MDS a much-needed alternative, Kurzrock says. It is one more drug that can be tried to help improve blood counts and prevent leukemia development in these patients, she says.

When the study began, there was no approved therapy to treat MDS, but recently, the U.S. Food and Drug Administration (FDA) approved use of azacytidine (VidazaTM), a chemotherapy drug administered by injection.

Tipifarnib helps about as many patients as azacytidine, but for diseases like MDS you need more than one drug because the syndrome is made up of many subtypes and therefore only a limited number of patients will respond to any one drug, Kurzrock says. Therefore, if one drug doesnt help, then the other might; or they could potentially be used together.

Drug can help patients with mutated and normal genes

Tipifarnib belongs to a group of drugs known as farnesyl transferase inhibitors, which block enzymes needed for the activation of cancer-promoting proteins.

The drug was initially believed to act primarily on the ras gene, which regulates the growth of cells. Normal cells need a good ras gene in order to grow and make new DNA.

While the ras gene is mutated in about 25% of MDS patients, recent studies, including this one, demonstrate that patients whose ras gene is normal can benefit, Kurzrock says. It has become apparent that tipifarnib regulates other important cancer genes, although we dont know which ones they are.

http://www.cancerwise.org/January_2005/display.cfm?id=B9D1F0F1-9D19-46EC-B1FAA3F752FACB34&method=displayFull&color=red

Cord Blood from Two Donors Okay for Leukemia

NEW YORK (Reuters Health) - Feb 4, 2005 - Transplanting umbilical cord blood from two separate donors appears to be a safe and effective treatment for leukemia, new research suggests. The findings suggest that umbilical cord blood transplants could be made available to thousands more adolescent and adult patients, Dr. Juliet N. Barker of the University of Minnesota in Minneapolis and colleagues write in the medical journal Blood.

Umbilical cord blood transplants have advantages over bone marrow transplantation--including rapid access and little risk of graft-versus-host disease (GVHD), a serious condition that occurs when blood cells present in the transplant actually attack the recipient. However, umbilical cord blood donations to adults have been sharply limited by the small size of a single unit, Barker and her team note.

The researchers conducted the current study to determine if this limitation could be overcome by giving adults two units of partially matched umbilical cord blood from separate donors. Twenty-three patients received the transplants for severe forms of leukemia.

While two of the patients died of infection shortly after transplantation, the umbilical cord blood transplant successfully took root or "engrafted" in all of the other patients. Engraftment is the main goal for any type of transplant.

At one year, 72 percent of the patients who had received the transplant during remission were still alive. Moreover, the rate of GVHD was no greater than that seen with single-unit umbilical cord blood transplants.

The authors conclude: "Transplantation of two partially...matched umbilical cord blood units is safe, and may overcome (the barriers that limit) the use of umbilical cord blood in many adults and adolescents."

SOURCE: Blood, January 25, 2005.

Nelly's Sister Passes From Leukemia

By Remmie Fresh
3/24/2005 - Sadly, Nelly has lost his sister Jackie Donohue to a years-old bout with leukemia. Donohue, 31, died Thursday morning after the cancer came out of remission.

"We are deeply saddened to announce the passing of Jackie Donahue, 31, who lost her 3-year battle with leukemia this morning," the family told AllHipHop.com in a statement. "We want to thank everyone for their love and support of Jackie. In particular, we want to thank those who participated in the Jes Us 4 Jackie bone marrow drives."

In March of 2001, Donahue was diagnosed with Acute Myelogenous Leukemia and had beaten the disease until she suffered a relapse in January 2003.

Consequently, she and Nelly established the non-profit Jes-US-4-Jackie as a way to raise awareness about leukemia and to find her a possible bone marrow donor.

"We are very proud of her efforts to educate and raise awareness about the disease and the need for African-Americans to join the National Donor Registry. She will always be remembered for her loving spirit, energy, courage and unshakeable faith."

Nelly cancelled his recent concert appearances after learning about his sisters passing.

Funeral details have yet to be announced.

Donations can be made to:

The Jacqueline Donahue Trust
C/O US BANK SLMOL2PB
721 Locust Street
St. Louis, MO. 63101

New Research Shows Promise for Treating Bone Marrow Failure in MDS Patients

A study published in the August 6, 2002 edition of Annals of Internal Medicine, volume 137: 156-163, revealed that the immunosuppressive drug antithymocyte globulin (ATG) was effective in treating bone marrow failures associated with MDS. Dr. Jeffrey J. Molldrem and his colleagues at The University of Texas M.D. Anderson Cancer Center and the National Institutes of Health conducted the study. All 61 patients enrolled in the study received ATG (ATGAMñ Pharmacia Corporation) a medicine typically used in organ transplant patients. Within eight months of initial treatment, the need for red blood cell transfusions was eliminated in 21 of the 61 patients (34%) enrolled in the study. For 16 of the 21 responding patients, positive results were seen within three eight months of treatment.

On a theory that MDS has an autoimmune component, Dr. Molldrem and his colleagues elected to treat MDS as an autoimmune disease and administered ATG based on its success in treating patients with aplastic anemia, a bone marrow disorder. From 1994 to 1998, researchers enrolled MDS patients who were dependent on blood transfusions and studied their response to the drug, as well as long-term disease outcomes. Among the patients who responded to ATG, 80 percent continued making enough blood cells to be transfusion-free after five years. More importantly, none of these responding patients progressed to leukemia, a common outcome of MDS. Response was associated with a statistically significant longer survival and an almost significant decreased time to disease progression. Further research is being conducted at M.D. Anderson using other immune suppressing drugs and combinations of drugs, including ATG, to improve the response to treatment.

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