In Youngest Babies, This Type of Blood Cancer Is Most Challenging

Pediatric oncologists have identified specific genes, dubbed partner genes, that fuse with another gene to drive an often-fatal form of leukemia in infants. By more accurately defining specific partner genes, researchers expect to better predict which infants may benefit from particular treatments.

Oncologists also aim to use this latest knowledge to develop new and more effective therapies for this difficult-to-treat type of blood cancer, called acute lymphoblastic leukemia (ALL). Their goal is to target treatments to specific genes and other associated factors that become abnormal because of the gene fusions.

Blaine W. Robinson, Ph.D., a research scientist at The Children’s Hospital of Philadelphia, will present research findings in infant ALL at the annual meeting of the American Society of Hematology on Dec. 8. His group collaborated with the Children’s Oncology Group (COG), a cooperative, multicenter research organization, on this research, sponsored by the Leukemia & Lymphoma Society.

ALL is the commonest of all the pediatric cancers. While the survival rate for children older than one year of age with ALL has increased over time with advances in chemotherapy, the outlook for infants (patients less than one year old) with the disease generally has been grim. Infants with ALL have a poor prognosis and a much higher mortality rate compared to other children, and curative treatments for them are far behind the therapy for childhood ALL.

For the majority of these high-risk infants, the problem is within the structure of a specific chromosome. In an abnormality called the MLL translocation, the MLL gene on chromosome 11 breaks and joins with any one of many different “partner” genes from other chromosomes. The rearranged genetic region, called a translocation, leads to the production of a fusion gene and an abnormal protein and, ultimately, to leukemia.

The current study covered 221 infants with ALL in a COG clinical trial. Researchers detected MLL translocations in the ALL cells of 74 percent of the patients. The two most common partner genes that fused with the MLL gene were AF4 on chromosome 4 and ENL on chromosome 19. Both of these translocations were associated with a very poor prognosis; event-free survival (EFS) rates were 34 percent with AF4 and 29 percent with ENL, compared to the overall EFS rate of 46 percent among all infants in the study–still far inferior to survival rates that are seen in children above age one.

The EFS rates with these two partner genes were even lower when the infants were less than 90 days old at diagnosis. Conversely, the survival rates were better when these partner genes fused to MLL in the leukemia cells of older infants. Though age was already known to be a classic prognostic factor in infant ALL, the differences in survival in younger versus older babies when these specific partner genes are involved had not been so clear.

In contrast, outcomes were better for infants with ALL when the third most common partner gene, AF9, fused to MLL, or when the MLL gene was unaffected. In these patients, the respective EFS rates were 68 and 66 percent. The researchers also analyzed white blood cell counts (WBC)–another classic prognostic factor in leukemia. They found that when MLL was fused to AF4, the infants were far more likely to have higher WBC, while the WBC was lower when MLL fused to AF9.

More refined knowledge of how the different partner genes of MLL in infant ALL are connected to the underlying molecular biology of the disease may guide the researchers to more appropriate treatment decisions. “Our ability to classify ALL based on specific partner genes of MLL may provide a new way to categorize which infants might benefit from specific types of treatment,” said senior author Carolyn A. Felix, M.D., a pediatric oncologist and expert in infant leukemia at Children’s Hospital, and a professor of Pediatrics at the University of Pennsylvania School of Medicine. “We also hope these findings will contribute to the development of new, molecularly targeted therapies for infants with this grim form of cancer that we seek to conquer.”

Gregory Reaman, M.D., chair of the Children’s Oncology Group, added, “As infants with ALL represent the group of children with the highest risk of treatment failure, despite successive attempts to intensify conventional therapy, these clues to potentially tailoring molecularly targeted treatment approaches are very exciting.”

Major grant support for this research is from a Specialized Center of Research grant from the Leukemia & Lymphoma Society. Felix is the principal investigator of this multicenter grant, focused on developing innovative treatments for infant leukemia. Other major support came from a National Cancer Institute grant to the Children’s Oncology Group.

Specific MLL Partner Genes in Infant Acute Lymphoblastic Leukemia (ALL) Associated with Outcome Are Linked to Age and White Blood Cell Count (WBC) at Diagnosis: A Report on the Children’s Oncology Group (COG) P9407 Trial. Abstract 907, to be presented at the 51st Annual Meeting of the American Society of Hematology, Dec. 8, 2009.

Source: The Children’s Hospital of Philadelphia


Chemistry World recently highlighted a new report published by the Pharmaceutical Research and Manufacturers of America (PhRMA) that identifies 97 new drugs and vaccines in development for HIV/AIDS and related conditions.

The report found that the 97 products in development include 23 vaccines and 54 antivirals. These drugs are either in human clinical trials or awaiting approval by the U.S. Food and Drug Administration (FDA).

“We are greatly encouraged by these critically important medicines and vaccines in development to treat and prevent HIV infection,” said PhRMA President and CEO Billy Tauzin. “Pharmaceutical researchers are continuing their efforts to develop new therapies and vaccines to improve and lengthen the lives of HIV-infected patients.”

“As a result of HIV/AIDS medicines, a disease that was once a virtual death sentence can now be controlled and treated as if it were a chronic disease,” stated Tauzin. “And the new medicines our scientists are working on right now bring hope for even more promising results in the future.”

December 1 marks the 21st anniversary of “World AIDS Day” – a global awareness campaign that originated at the 1988 World Summit of Ministers of Health on Programmes for AIDS Prevention.

Source: Pharmaceutical Research and Manufacturers of America


Multiple sclerosis patient finds hope through stem cell treatment

  • Author: Health Informer
  • Filed under: Health News
  • Date: Dec 6,2009

Electromyogram (EMG) findings show improvement in conduction speed and latency -RNL Bio’s stem cell therapy demonstrates effective outcomes

RNL Bio Co., Ltd, a leading biopharmaceutical company specialized in adult stem cell therapeutics announced today that a 46-year-old female, Kang Sook Park’s Multiple Sclerosis improved tremendously after receiving stem cell treatment. Park was suffering from MS (Multiple Sclerosis), an autoimmune disease that affects the central nervous system (brain and spinal cord).

Multiple Sclerosis is a disease in which the nerves of the central nervous system degenerate. MS can cause problems with muscle control and strength, vision, balance, feeling, and thinking. MS is caused by damage to the myelin sheath, the protective covering that surrounds nerve cells. When the myelin sheath is damaged, nerve impulses slow down or stop responding. From the many symptoms of MS, numbness or abnormal sensation in any area is predominant.

For the past 20 years, Park suffered from MS. For stem cell injection, she was admitted into Choyang Hospital of Regenerative Medicine in Yanji, China for three weeks. She received stem cells intravenously as well as intrathecally. The injections were given in five intervals — a total of 1.2 billion cells.

Prior to stem cell treatment, Park suffered from Optic Neuritis — a common symptom of MS. Some of the symptoms of Optic Neuritis include double vision, vision loss, and uncontrollable rapid eye movements. She was not able to recognize any writing due to her vision loss. After six months of receiving stem cell therapy, Park experienced phenomenal changes. Her vision improved drastically — she was able to read. Furthermore, it also became possible for her to move her legs using her own muscular strength.

“My legs felt like hollow logs. They were so numb and stiff. However, after receiving stem cell treatment, I can actually feel my legs. I can also tell the difference in cold and warm temperatures. Stem cell therapy brought incredible changes to my physical condition,” stated Kang Sook Park.

Park also added, “Receiving stem cell turned my life around. I feel like I am reborn. The fact that I am in this condition brings me new joy. I love the fact that I no longer need anyone’s help to get from place to place. I would like to share my experience to others who may have this rare disease. I want to let others know that they can find hope through stem cell treatment, just the way I did.”

Dr. Chang Won Kim reported Park’s EMG findings before and after the stem cell treatment. “Patients who have an uncommon disease such as MS rarely show improvement or are completely cured. EMG results showed that Park’s movement increased both in upper and lower limbs. Reduction in latency suggests that demyelination was also improved. It is extremely difficult for MS patients to use their own strength to even make small movements in the arms or legs. These changes in Park were possible through stem cell therapy.”

President and CEO of RNL Bio, Dr. Jeong Chan Ra expressed, “The effective outcomes from stem cell treatment for rare diseases like Multiple Sclerosis can be used in the future. I will do my best to help improve the quality of life for people who are suffering from diseases like MS.”

Source: RNL Bio Co., Ltd


WHO launches new tobacco control effort in Africa

  • Author: Health Informer
  • Filed under: Health News
  • Date: Dec 5,2009

The World Health Organization (WHO) is increasing its attention to tobacco control in Africa with the overall goal of preventing tobacco use from becoming as prevalent there as it is in other parts of the world.

The focus of the programme will be on strengthening countries’ ability to implement the WHO Framework Convention on Tobacco Control (WHO FCTC), the international health treaty that guides national efforts to counter the tobacco epidemic, and the establishment of a regional centre of excellence to support the development of countries’ capacity to resist the spread of tobacco use.

“Tobacco use is the most preventable cause of illness and death,” said WHO Assistant Director-General for Noncommunicable Diseases and Mental Health Dr Ala Alwan. “It kills more than 5 million people per year. Unchecked, it will kill more than 8 million people per year by 2030, with more than 80% of those deaths occurring in developing countries. Although tobacco use is less prevalent in Africa than in other regions of the world, that will change unless we act.”

Tobacco use is a risk factor for the major noncommunicable diseases – heart attacks, strokes, cancers, diabetes and asthma and other chronic diseases – which together account for 60% of all deaths. In the 46 countries of WHO’s Africa region (AFRO), noncommunicable diseases are expected to account for 46% of deaths by 2030, up from 25% in 2004.

“Tobacco use in Africa is more than a health problem,” Dr Alwan said. “It’s a development problem, too. Tobacco breeds poverty, killing people in their most productive years. It consumes family and health-care budgets. Also, money spent on tobacco products is money not spent on such essentials as education, food and medicine.”

“WHO’s new and innovative tobacco control work in Africa will help to bring solutions within reach,” Dr Alwan added.

“We must act now to implement the WHO FCTC in order to prevent a tobacco epidemic in Africa,” said Dr Paul Samson Lusamba Dikassa, Director of Programme Management at the WHO Regional Office for Africa (AFRO). “WHO in the African region has an important role to play in working with countries to control tobacco.”

“There is a pressing need to formulate effective strategies against tobacco use,” said Dr Douglas Bettcher, Director of WHO’s Tobacco Free Initiative. “Working with governments and partner organizations, we can help in preventing tobacco from gaining the upper hand.”

The work will be financed in part by a grant of US $10 million from the Bill & Melinda Gates Foundation. The grant is the largest that WHO has received for tobacco control in Africa and is an important means to address the noncommunicable diseases gap in the international development agenda.

Source: World Health Organization (WHO)


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