AUTISM AND STEM CELLS

A new study out of Harvard University, reported in Science, suggests that a transplant of stem cells into the brain may lead to future treatment for neural disorders, such as Autism and Parkinson's Disease, as well as spinal cord injuries and diseases, such as ALS. The researchers in the study focused on curbing obesity in mice, as it provides visual evidence as to whether the treatment was effective or not. Despite the focus on obesity, the study's results led the scientists to believe that this could potentially lead to further treatments for other diseases.

"[Researchers] investigated whether fetal neurons transplanted into a part of the mouse brain that does not normally produce new neurons of its own could repair an abnormal neural circuit. The recipients of the transplant treatment were genetically altered mice lacking the receptor for leptin, a hormone that regulates metabolism and body weight. In normal mice, leptin acts on neurons in a part of the brain called the hypothalamus, which regulates metabolism and other essential functions. But in the mutant mice, these neurons can't respond to leptin, and the mice become obese and diabetic. To see whether they could correct this defect, the researchers transplanted immature neurons taken from the hypothalami of fetal mice that had the normal leptin receptor gene into the same brain region of the obesity-prone mice.

Using electrodes to record the electrical activity of hypothalamic neurons, the researchers confirmed that the transplanted cells responded to leptin as expected and could communicate with the recipient mouse's own neurons. 'These newly incorporated neurons were in a sense acting as antennas for leptin and sending those signals into the brain.'"

AdvertisementThe implications of this study are staggering, namely that if researchers can pinpoint what neurons are specifically involved or responsible for the symptoms of a given neural disease, they may be able to introduce transplanted cells and guide their development, essentially repairing what has been damaged or what is malfunctioning. This runs counter to what scientists originally thought which was that one may simply infuse stem cells into an affected brain and the neural circuitry already present will know what to do, whereas in reality, stem cell therapies must be specifically selected and tailored for the affected area."We have used complex circuitry as a test case for whether precisely selected and controlled neuron transplants could rewire the brain. What we found is that these neurons not only turned into the right types of cells, but they sent signals to the recipient’s brain and received signals from the recipient’s brain. The next step for us is to ask parallel questions of other parts of the brain and spinal cord."

This study comes in light of the mulitude of other researchthat unequivocally determines that Autism is a neuralogical disease in origin and in manifestation. The hope for treatment and possible cure therefore lies in addressing the neural deficits specifically, such as with stem cell therapies, rather than special diets and avoidance of vaccines.

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Diabetes fight should be a priority

Print | Comments ()Posted by Marjorie Pritchard  November 18, 2011 02:00 PM

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John L. Brooks, 3d


Diabetes is now a global pandemic, afflicting 346 million people worldwide, and Massachusetts is squarely in the forefront of this unforgiving disease:
• About 407,000 adults are diagnosed with diabetes and another estimated 114,000 adults lhave type 2 diabetes, but do not know it.

• From 2005 through 2008, diabetes was the ninth leading cause of death in the Commonwealth.

• In 2009, nearly 5 percent of Massachusetts adults reported they had been diagnosed with pre-diabetes, which is blood glucose levels that are higher than normal but not high enough to be diagnosed as diabetes.

• The cost of diabetes in Massachusetts is $4.3 billion annually.

• Since 1999, there has been a 61 percent increase in the number of people diagnosed with diabetes in Massachusetts, a trend that will continue unless we all work together to change things.

Type 1 diabetes is predominantly initiated by family genetic factors. Type 2 diabetes, is accelerated by obesity, lack of exercise, lack of awareness, and a healthcare system that historically has focused on paying for downstream complications rather than prevention and proactive encouragement of healthy diets, exercise, medication adherence, and behavioral health issues.

As the United States faces the prospect of the incidence of diabetes rising from one in 10 citizens today, to one in three in the next 40 years, a new national commitment to address this disease head-on is essential.

At the Joslin Diabetes Center, we are catalyzing this call to action with the goal of a world free of type 1 and type 2 diabetes and their complications. We are reaching out across the Commonwealth to engage organizations, community centers, schools of public health and many others, to bring resources and energy to providing culturally tailored educational programs, tools, team-based resources and impactful care plans to inform and motivate families and their health teams to work collectively to reverse or slow this relentless diabetes tidal wave.

On November 19 our community of doctors, patients, families, friends and advocates will gather for Joslin’s annual High Hopes Gala and World Diabetes Day Celebration, our most important night of the year. This event supports innovation and research aimed at creating a world free of diabetes and its complications.
We must build partnerships and cost-effective, risk-sharing collaborations with other healthcare providers, payers, and federal, state and city agencies to share and leverage our expertise in novel and measurable ways. This is what is needed to help patients, families, primary care doctors and other healthcare providers and payers to tackle this disease and its dreaded complications. We must make this a priority. Our lives -- and the lives of our loved ones -- depend on it.
John L. Brooks 3d is president and CEO of the Joslin Diabetes Center.

DIABETES AWARENESS MONTH BEGINS

Submitted by Lorenzo.Piemonte on Tue, 11/08/2011 - 09:26

In many parts of the world, the beginning of November marks the start of a month-long series of activities aimed at raising diabetes awareness and calling for urgent action to tackle the diabetes epidemic. Here's a reminder of why we all have to 'Act on Diabetes. Now.':

• New figures recently launched by the International Diabetes Federation indicate that the number of people living with diabetes has risen to 366 million.
• Diabetes is responsible for 4.6 million deaths a year - 1 every 7 seconds.
• Healthcare spending on diabetes has reached USD 465 billion.
• Diabetes is among the top 10 causes of disability, resulting in devastating complications such as blindness and lower limb amputations.
• All nations – rich and poor – are suffering the impact of the diabetes epidemic
• Diabetes is undermining global development.
• Diabetes hits the poorest hardest

World’s first stem cell bandage in human clinical trials

Press release issued 14 November 2011

The company behind a pioneering stem cell bandage, believed to be the world’s first adult and autologous (patient’s own) stem cell treatment designed to heal torn meniscal cartilage, can now take the technology to human clinical trials thanks to an investment from one of the UK’s most successful entrepreneurs.Mr Hugh Osmond, a partner of Sun Capital — who helped build Pizza Express into the UK’s largest sit-down restaurant chain and founded one of the country’s largest pub companies, the Punch Group — has completed a £0.65 million funding round for Azellon Cell Therapeutics Ltd, the University of Bristol spin-out company behind the therapy that has to date raised £2.25 million.
The company, which has received approval from the Medicines and Healthcare products Regulatory Agency (MHRA) for the world's first clinical trial using its Cell Bandage product, is funded by existing investors IP Group plc, the developer of intellectual property based businesses — and Oxford Technology Management as well as new investors including lead funder, Mr Osmond.
Azellon’s Cell Bandage has been designed as an alternative to the current treatment of surgical removal of the meniscus (meniscectomy), a procedure that more than 1.7 million people around the world per year are estimated to undergo. This common orthopaedic procedure often results in the early onset of osteoarthritis, leading to further joint surgery including total knee replacement.
The Cell Bandage, which in vitro (tissue culture) has shown great promise for the healing of meniscal tears, is grown from the patient’s own stem cells and will be transplanted in the patient’s knee joint within two weeks of extracting the stem cells from bone marrow.
The MHRA approved Phase I/IIa trial will treat ten meniscal tear patients with a cell bandage product, seeded with the patient's own stem cells. The trial will be undertaken at Southmead Hospital in Bristol and is scheduled to begin in May 2012 with interim data available within 18 months.
Azellon is co-founded by Professor Anthony Hollander at the University of Bristol, who came to national prominence as part of the academic team that saved the life of Claudia Castillio, after developing the first tissue-engineered trachea (windpipe) using the patient’s own stem cells. This fully functioning airway was transplanted into the patient and saved her life.
Professor Anthony Hollander, Chief Scientific Officer of Azellon Cell Therapeutics Ltd and Head of the School of Cellular and Molecular Medicine at the University of Bristol, said: “With permission for a trial from MHRA and completion of this funding round, we are now ready to get going on our safety trial; it’s an important moment for Azellon and for stem cell research.”
Alan Aubrey, CEO of IP Group plc, said: “Azellon’s stem cell bandage is targeted at a very large and growing market with a clear medical need and we are pleased to support the company as it moves into its Phase I/IIa trial.”
Hugh Osmond, who has a medical degree from Oxford University, said: “As a keen sportsman who has had multiple knee operations myself, I believe that this procedure has the potential to be a major breakthrough in treating knee and eventually other joint injuries. For many of the 1.7 million people a year who have operations to repair torn knee cartilage, it could be the difference between an active old age or spending their pension years in a wheel chair. I am very excited.”

Students sign up to be life-savers

Published on Tuesday 8 November 2011 07:08

STUDENTS at Wigan and Leigh College responded superbly to a charity’s call to become lifesavers by signing up to the blood stem cell donor register.

London-based organisation Anthony Nolan held the recruitment drive at Leigh Sixth Form College, on Sale Way, to try and help increase the currently low numbers of men aged between 18 and 30 years old who are on the register, a campaign backed by Leigh MP Andy Burnham.

Around 30 students at the college signed up for the donation register, with other students who were not able to become donors as they were aged under 18 pre-registered for when they are old enough or given literature about stem cell donation.

More than 80 per cent of blood stem cell donations are from men, and men aged 18-30 are considered to be the ideal donors most likely to be asked to donate, yet just 12 percent of donors on Anthony Nolan’s register are from this age group.

To improve these figures, the charity has launched an ambitious recruitment drive to get 10,000 men in the target age group signed up, and Mr Burnham, who visited the recruitment drive at Leigh Sixth Form, is urging any fit and healthy young men in Wigan and Leigh to consider becoming a donor.

A college spokesman said: “Both the trust and the college were really pleased with the turnout, and with how many young people signed up. It was a very positive response. We weren’t quite sure how our student community would respond, as we all know how few donors there are but that doesn’t always translate into people taking the step to signing up, but there was lots of interest and they obviously wanted to make a difference.”

To join the register, all applicants have to do is fill out a short medical questionnaire and provide a sample of saliva.

A stem cell donation and transplant could save the life of someone with a blood cancer such as leukaemia, but currently Anthony Nolan is only able to find tissue type matches for half the patients who come to them. To become a donor, register online at www.anthonynolan.org

New “braking system” makes stem cell therapy safer

04/11/2011 02:51:00

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Houston, TX - Cancer treatment with stem cell transplantation and specially modified immune system components called T-cells can enhance the chance of recovery from diseases such as leukemia or lymphoma.

Now these treatments can be made safer by using a new braking system that almost immediately controls the therapy if things go wrong, said researchers from the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children’s Hospital and The Methodist Hospital in a report in the current issue of the New England Journal of Medicine.  

Dr. Malcolm Brenner (www.bcm.edu/genetherapy/faculty-brenner), director of the   Center for Cell and Gene Therapy (www.bcm.edu/genetherapy/), and his colleagues have been developing special anti-tumor cells called T-cells that harness the body’s immune system to fight cancer.  Although they and other investigators have successfully developed ways of targeting these cells to attack cancer, the T-cells can grow in number in the patient and survive many years, which make any side effectssevere, long-lived and often uncontrollable. These treatments would be much safer if researchers could turn the cells off if patients suffered a bad reaction. However, no effective technology existed to rapidly accomplish this outcome.

The Cell and Gene Therapy researchers tested the new safety switch in patients who developed graft versus host disease after they received a stem cell transplant as part of their cancer therapy. During stem cell transplantation, a patient’s own stem cells are eradicated with drugs or radiation.  They then receive an infusion of blood-forming stem cells along with T-cells from a related donor, which help to kill any remaining cancer and to fight infections. However, sometimes the transplanted T-cells attack the patient’s own tissues. The result is graft versus host disease, which is disabling and often fatal. Turning off the T-cells in the transplant would stop the disease. The new treatment accomplishes just that – killing more than 90 percent of the cells within 30 minutes after a single treatment.

To make the safety switch, the researchers introduced into the T-cells a gene called iCasp9, which produces programmed cell death. When the patient receives a specific drug, it joins together two single molecules of iCasp9 (a process called dimerization) and makes it active – rapidly triggering the death of the cell.

“The patients come in with a severe itchy rash all over their bodies,” said Brenner. “We give them the drug and we can literally watch the rash melt away.”

In the study, the investigators gave the genetically modified T-cells to five patients who had received a blood stem cell transplant for relapsed leukemia (a blood cancer). The transplanted T-cells divided in the patients’ blood over time and helped their immunity recover.

Four patients developed the potentially fatal graft versus host disease and received the special activating drug. Within 30 minutes, 90 percent of the T-cells were dead and their graft versus hostdisease began to disappear. The T-cells continued to decrease in number over the next 24 hours and their graft versus host disease went away completely. The small number of T-cells that remained were able to expand and were able to fight infections, but did not cause further graft versus hostdisease

This kind of rapid cellular braking medicine would extend to other T-cell therapies, he said, such as that recently reported in the New England Journal of Medicine [N Engl J Med. 2011 Aug 10. [Epub ahead of print]  (http://www.nejm.org/doi/full/10.1056/NEJMoa1103849), and may ultimately be applicable to other forms of stem cell therapy in general, hastening their safe introduction to the clinic.

Others who took part in this work include Dr. Antonio Di Stasi,  Dr. Siok-Keen Tey, Dr. Gianpietro Dotti, Yuriko Fujita,  Alana Kennedy-Nasser,  Caridad Martinez, Dr. Karin Straath, Enli Liu, April G. Durett, Bambi Grilley, Dr. Hao Liu,  Dr. Conrad R. Cruz,, Dr. Barbara Savoldo, Dr. Adrian P. Gee, Dr. Robert A. Krance, Dr. Helen E. Heslop, Dr. David M. Spencer, and Dr. Cliona M. Rooney, all of BCM and Dr. John Schindler of The University of Texas Southwestern Medical School.

Funding for this work came from the National Institutes of Health, the National Heart, Lung and Blood Institute and the National Cancer Institute. The dimerizing drug was supplied by Bellicum Pharmaceuticals.

Learn more at www.methodisthealth.com. Follow Methodist on Twitter athttp://twitter.com/MethodistHosp and Facebook at http://www.facebook.com/methodisthospital.

PERSONAL STEM CELL BANKS

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Drs. Xiao-Dong Chen and Qian Wang of The University of Texas Health Science Center San Antonio are members of the research team that discovered a young microenvironment could stimulate old stem cells to expand more rapidly. Credit: UT Health Science Center San Antonio

Old stem cells can be rejuvenated by being placed in a young microenvironment, research from The University of Texas Health Science Center San Antonio shows. This raises the possibility that patients' own stem cells may one day be rescued and banked to treat their age-related diseases.

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Stem cells are immature cells that have the potential to convert into bone, muscle,blood vessels, nerve fibers, and other body cells and tissues. It's no wonder medical science seeks to utilize these versatile cells to restore tissues deteriorated by age, disease or injury.

Older stem cells are not as robust as young ones, however — a challenge to clinicians who seek to use patients' own stem cells to treat age-related diseases.

"The number and quality of those cells decline with age, that is very clear," said Xiao-Dong Chen, M.D., Ph.D., a stem cell researcher at the UT Health ScienceCenter. "And, using the patient's own cells can impact results."

Dr. Chen's team recently made a discovery in mice that, if translated to humans, could solve this predicament.

Old cells expand when grown on a young scaffold of tissue

Dr. Chen suspected that giving stem cells a youthful environment for growth would cause them to regenerate faster. His team extracted mesenchymal stem cells from the bone marrow of 3-month-old mice and 18-month-old mice. The group also obtained extracellular matrix (ECM) from mice of both ages. ECM is a scaffold of connective tissue, such as collagen, which constitutes a majority of the body's structure.

The lab team seeded half of the older stem cells on ECM from the 3-month-old mice and half on ECM from the 18-month-old mice. Likewise, half of the young stem cells were seeded on the young ECM and half were seeded on the old ECM.

Young and old cells showed a 16.1-fold and 17.1-fold expansion, respectively, when grown on ECM from young mice, compared to a 4.1-fold and 3.8-fold expansion when grown on ECM from old mice.

Finding confirmed in rodent implants

Next, under the skin of mice, Dr. Chen's group implanted artificial scaffolds seeded with stem cells of both ages that had been grown on young or old ECM. These were left to grow for eight weeks. The researchers targeted bone formation. When the implants were removed, the team found that old cells that had been grown on a young ECM produced just as much bone as young cells, while old cells grown on an old ECM produced no bone. The results were published in the FASEB Journal earlier this year.

"If this research transfers successfully to clinical application in humans, we could establish personal stem cell banks," Dr. Chen said. "We would collect a small number of older stem cells from patients, put those into our young microenvironment to rescue them — increasing their number and quality — then deliver them back into the patient."

This stem cell rescue and infusion could be done as often as disease treatment requires it, he said. The next step is to repeat the study in human stem cells and ECM.

Dr. Chen, an associate professor of comprehensive dentistry in the Health Science Center Dental School, discussed the finding at the Strategies for Engineered Negligible Senescence conference (SENS,http://www.sens.org/conferences/sens5) held at Queens' College in Cambridge, U.K.

Provided by University of Texas Health Science Center at San Antonio