10 Medical Breakthroughs of 2009

1. ReCell kit: New technique in burn treatment

More often than not, treatment for severe second-degree burns may add more to injury: Cutting a swath of skin from another area on the same patient in order to graft it over the burn. This procedure may add more pain for the burn victim and doubles the area of healing. And now a new discovery has revealed successful results to heal burns which are economical compared to skin grafts. The ReCell kit, slightly bigger than a designer sunglass case, accommodates a miniature lab for harvesting the skin basal cells. Using ReCell kit, marketed by Avita Medical, a United Kingdom-based regenerative-medicine company, and by a small skin biopsy, surgeons can create a suspension of the skin’s basal cells – the stem cells of the epidermis — and spray the solution directly onto the burn. The technology, developed by Australian surgeon Fiona Wood, relies on cells, such as skin progenitor cells and the colour-imparting melanocytes, that are most concentrated at the junction between the skin’s top two layers. With a small step-by-step kit dubbed ReCell, surgeons can harvest, process and apply these cells to treat a burn as large as 10.5 square feet. After removing a small swatch of skin near the burn site (the closer the biopsy, the better for precise matching of colour and texture), the surgeon places it in the kit’s tiny incubator along with an enzyme solution. The enzyme loosens the critical cells at the skin’s dermal-epidermal junction, and the surgeon harvests them by scraping them off the epidermal and dermal layers and suspending them in solution. The resulting mixture is then sprayed onto the wound, repopulating the burn site with basal cells from the biopsy site. This procedure has shown results comparable to those from skin grafts.

2. GP100: A new skin cancer vaccine

Vaccines, generally, work by training the body’s immune system to distinguish affected cells and attack them. The results of the clinical trials using the new vaccine — GP 100:209-217 or GP 100 — were presented at the American Society of Clinical Oncology meeting in Orlando, Florida by scientists from the National Cancer Institute. GP 100 could someday trigger a patient’s immune system to systematically kill off the skin cancer cells before they have a chance to spread further. GP 100 was shown to help shrink tumors and delay the progression of skin cancer. The theory is that once the body can distinguish cancer cells, it can kill them before cancer has an opportunity to recur in a patient. The vaccine GP 100 targets skin cancer (melanoma) and has shown improved response rates and progression-free survival for patients when combined with the immunotherapy drug, Interleukin-2. The vaccine helped to shrink tumors and delayed the worsening of the disease as well. It was found that approximately 22 per cent of patients given the vaccine and Interleukin-2 showed their tumors shrink by at least half, compared with 10 per cent of people getting Interleukin-2 alone. Additionally, vaccine users saw that cancer stabilized for three months versus half that time for the others. Additionally, patients treated with both the vaccine and interleukin-2 showed a distinct stabilization of their cancer for three months versus half that time for patients treated without the vaccine.

3. NT-3: Breakthrough in spinal cord reconstruction

It’s true. Researchers from the University of California have discovered a chemical that can rewire the spinal cord after injury. The University of California researchers have found a way to regenerate synapses (nerve junctions) that were damaged in rats. When Neurotrophin-3 (NT-3) was applied the nerves steered themselves to the correct destination synapse and supported the complete connection to the synapse. The researchers were able to prove that using electron microscopes that the nerves were moving to the correct locations and automatically healing. NT-3 has reached two important milestones of spinal cord damage research: a) To allow nerves to move through tissue towards a synapse that was originally used, and b) To complete the healing process so the connection worked as before. It is breathtaking to see how spinal cord injuries are worked on without the need of embryonic stem cells and NT-3 is not based on human tissue which means that there is not the threat of rejection.

4. New method for creating stem cells: Pluripotent

Dr. Andras Nagy of Mount Sinai Hospital, Toronto, Canada has in March, 2009 discovered a new method of creating stem cells from adult cells that could lead to possible cures for devastating diseases including spinal cord injury, macular degeneration, diabetes and Parkinson’s disease. Dr. Nagy discovered a new method to create pluripotent stem cells (cells that can develop into most other cell types) without causing harm to healthy genes. This method involves a novel wrapping procedure, in order, to deliver specific genes for reprogramming cells into stem cells. According to the scientific observers, this novel method for generating stem cells will not require embryos as starting points and adult tissues such as a patient’s own skin cells can be used to generate stem cells. These stem cells can form the basis for treatment of many disorders and conditions that are currently considered incurable. The study was published by Nature, accelerates stem cell technology and provides a road map for new clinical approaches to regenerative medicine. Previous approaches required the use of viruses to deliver the required genes, a method that carries the risk of damaging the DNA. Dr. Nagy’s method does not require viruses, and so overcomes a major hurdle for the future of safe, personalized stem cell therapies in humans.

5. Dabigatran: New drug for stroke patients

Dabigatran: A new drug has been found to prevent a higher number of strokes with less bleeding than existing medicines such as warfarin. The results of a study of 18,113 patients, with a mean age of 71 years and with atrial fibrillation and followed up for 2 years, a key risk factor for stroke confirmed this finding. This new drug waiting United States’ FDA approval has been considered to be more effective than the existing medications. It does not interact with many other medications; this negates the need to constantly test and adjust the dose.

Dabigatran was slightly more effective than warfarin at preventing stroke and embolism, especially the dosage of 150 mg given twice a day, and may reduce all-cause mortality.

Dabigatran etexilate is an oral alternative to warfarin that directly inhibits thrombin. In this study, patients were randomized to receive dabigatran, 110 mg twice a day; dabigatran, 150 mg twice a day; or warfarin targeted to an INR (International Normalization Ratio) between 2.0 and 3.0. When compared with warfarin, the 150-mg dose of dabigatran was associated with a lower risk of stroke

6. New Year gives hope for a HIV vaccine

Two new antibodies that can offer promise as an HIV vaccine has been discovered by researchers at the International AIDS Vaccine Initiative and the Scripps Research Institute in La Jolla, CA and New York. These antibodies to be produced only by a minority of patients and are considered to be “broadly neutralising,” as they cripple many different strains of the deadly virus that causes AIDS. The scientists discovered two broadly neutralising antibodies to HIV that reveal a previously unknown site on the virus that could prove to be a good target for vaccine design. Researchers are working to develop immunogens, the active ingredient in vaccines in the hope of prompting the immune system to produce powerful antibodies that would protect from HIV infection.

The two newly discovered neutralising antibodies to HIV are the most potent to be identified while also maintaining strong breadth of neutralisation. They are the first to have been discovered in more than a decade and the first to have come from donors in developing countries, where the majority of new HIV infections occur. The identification of the antibodies and the site they target on HIV was made possible through a collaboration, orchestrated by IAVI, among researchers at IAVI, Scripps Research, the biotechnology companies Theraclone Sciences and Monogram Biosciences, and a group of clinical research centers in the United States, the United Kingdom, Australia, Thailand and Africa.

7. Stem cells for lung and heart disorders

Two recent studies published in the September issue of ‘Cell Transplanation’ have shown that transplanted human-derived umbilical cord blood (UCB) stem cells in an animal model had positive curative effects on specific lung and heart disorders in the animal model. The researchers have found that the stem cells have a protective effect against hypoxia-induced lung injury. The researchers expect that their findings can have major therapeutic potential for treating conditions such as hyperoxic neonatal lung disease, or bronchopulmonary dysplasia (BPD), in premature human infants that are presently considered untreatable.

8. New mechanism of insulin for betterment of diabetes

Researchers from the Hebrew University of Jerusalem, in collaboration with the Japanese and American universities have explored the role of LKB1, a gene that is involved in numerous cellular functions, and whose role in the functioning of the pancreas was never examined before. The researchers studied the implications of beta cell-specific loss of LKB1 gene, using a mouse model system and were successful to show that eliminating this gene from beta cells causes the production and secretion of more insulin than normal beta cells. The enhanced production of insulin leads to an improved response to increases in blood glucose levels. The findings are likely to bring smiles to those suffering from diabetes (excessive blood sugar) due to insufficient production of insulin.

LKB1 negatively regulates both insulin content and secretion thus enhancing insulin secretion.

9. Skin test for detecting Alzheimer’s

Blanchette Rockefeller Neurosciences Institute (BRNI) at West Virginia University, the United States researchers discovered a skin test that can detect the presence of Alzheimer’s disease. The test involves pricking of a finger to detect the presence of certain defective enzymes involved with memory function that are present in both brain and skin cells. The test results were noted to be 98 percent accurate in detecting Alzheimer’s, according to Daniel Alkon, MD, the institute’s scientific director. Till now, the Alzheimer’s disease can only be definitively diagnosed on autopsy. Physicians diagnose the disease in patients with a combination of cognitive tests to assess mental function, a neurological exam, and brain scans to rule out other problems like stroke or brain tumors. And it is difficult to distinguish between Alzheimer’s and other forms of dementia, especially at the early stages. BRNI researchers who developed the diagnostic have got the approval from US FDA (Food and Drug Administration) to test in humans an experimental drug that activates the enzymes–a mechanism that represents a new therapeutic approach to Alzheimer’s. In preliminary clinical trials, the skin test could accurately predict which patients had Alzheimer’s disease. BRNI, meanwhile, announced a partnership with Inverness Medical Innovations, a US medical diagnostics company, that will fund a larger clinical trial of thousands of patients that is necessary to commercialise the diagnostic.

10. Discovered: New genetic cause of cardiac failure

Unstable structural elements of the heart muscle lead to heart failure. In a lifetime, the heart pumps some 250 million litres of blood through the body and to do this, the muscle fibers of the heart have to be extremely durable. However, researchers headed by Dr. Wolfgang Rottbauer, vice-chair, Department of Medicine III at Heidelberg University Hospital in Germany have discovered a protein that is considered responsible for the stability of one of the smallest muscular unit, known as the sarcomere. They proved that mutations in this protein are the cause heart failure. Primary heart muscle disease with decreased cardiac pump function leading to enlargement of the heart chambers (dilated cardiomyopathy) is one of the most frequent causes of chronic heart failure. The heart disease weakens cardiac cells and the heart can no longer pump efficiently which leads to dilation of the cardiac chambers. Muscle activity takes place in the smallest unit of muscle fiber, the sarcomere. In the presence of an appropriate stimulus, actin and myosin filaments interact and contract the muscle. These movable elements are anchored in what are known as Z-disks. With every heartbeat, enormous forces act on the Z-disks. Sarcomere is one of the factors that are responsible for the stability of the heart muscles and abnormalities can cause the muscles to lose strength leading to weakening of the heart. Early treatment with medications to reduce stress on the heart in patients with such mutations might be beneficial. The results have been published in the November issue of ‘Nature Medicine’.

Sourced from the Net.

• Stanford medical research team builds tiny broken hearts – San Jose Mercury News
• Heart has cellular regeneration ability – Science News
• Roche Skin-Cancer Drug Shows Promise – Wall Street Journal
• Failing Hearts Healed With Stem Cells – WebMD
• Diabetics could soon ‘grow their own insulin’ – Daily Mail
• Stem Cell Therapy Shrinks Enlarged Hearts – BusinessWeek
• New Test May Predict If Cancer Will Spread – Daily Health Report
• Scientists identify possible human lung stem cell – CTV.ca
• Skin Cancer Breakthrough: Arthritis Drug Could Be New Weapon Against Melanoma – Medical News Today
• Pancreas cancer vaccine trialled – BBC News

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