February 13, 2012, 9:47 PM EST

By Ryan Flinn

Feb. 14 (Bloomberg) -- Stem cells grown from patients’ own cardiac tissue can heal damage once thought to be permanent after a heart attack, according to a study that suggests the experimental approach may one day help stave off heart failure.

In a trial of 25 heart-attack patients, 17 who got the stem cell treatment showed a 50 percent reduction in cardiac scar tissue compared with no improvement for the eight who received standard care. The results, from the first of three sets of clinical trials generally needed for regulatory approval, were published today in the medical journal Lancet.

“The findings in this paper are encouraging,” Deepak Srivastava, director of the San Francisco-based Gladstone Institute of Cardiovascular Disease, said in an interview. “There’s a dire need for new therapies for people with heart failure, it’s still the No. 1 cause of death in men and women.”

The study, by researchers from Cedars-Sinai Heart Institute in Los Angeles and Johns Hopkins University in Baltimore, tested the approach in patients who recently suffered a heart attack, with the goal that repairing the damage might help stave off failure. While patients getting the stem cells showed no more improvement in heart function than those who didn’t get the experimental therapy, the theory is that new tissue regenerated by the stem cells can strengthen the heart, said Eduardo Marban, the study’s lead author.

“What our trial was designed to do is to reverse the injury once it’s happened,” said Marban, director of Cedars- Sinai Heart Institute. “The quantitative outcome that we had in this paper is to shift patients from a high-risk group to a low- risk group.”

Minimally Invasive

The stem cells were implanted within five weeks after patients suffering heart attacks. Doctors removed heart tissue, about the size of half a raisin, using a minimally invasive procedure that involved a thin needle threaded through the veins. After cultivating the stem cells from the tissue, doctors reinserted them using a second minimally invasive procedure. Patients got 12.5 million cells to 25 million cells.

A year after the procedure, six patients in the stem cell group had serious side effects, including a heart attack, chest pain, a coronary bypass, implantation of a defibrillator, and two other events unrelated to the heart. One of patient’s side effects were possibly linked to the treatment, the study found.

While the main goal of the trial was to examine the safety of the procedure, the decrease in scar tissue in those treated merits a larger study that focuses on broader clinical outcomes, researchers said in the paper.

Heart Regeneration

“If we can regenerate the whole heart, then the patient would be completely normal,” Marban said. “We haven’t fulfilled that yet, but we’ve gotten rid of half of the injury, and that’s a good start.”

While the study resulted in patients having an increase in muscle mass and a shrinkage of scar size, the amount of blood flowing out of the heart, or the ejection fraction, wasn’t different between the control group and stem-cell therapy group. The measurement is important because poor blood flow deprives the body of oxygen and nutrients it needs to function properly, Srivastava said.

“The patients don’t have a functional benefit in this study,” said Srivastava, who wasn’t not involved in the trial.

The technology is being developed by closely held Capricor Inc., which will further test it in 200 patients for the second of three trials typically required for regulatory approval. Marban is a founder of the Los Angeles-based company and chairman of its scientific advisory board. His wife, Lisa Marban, is also a founder and chief executive officer.

--Editors: Angela Zimm, Andrew Pollack

-0- Feb/13/2012 22:32 GMT

To contact the reporter on this story: Ryan Flinn in San Francisco at rflinn@bloomberg.net

To contact the editor responsible for this story: Reg Gale at rgale5@bloomberg.net

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Scarred Hearts Can Be Mended With Stem Cell Therapy, Study Shows

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13-02-2012 12:31 Sridhar Ramaswamy, MD, Tucker Gosnell Investigator and Associate Professor of Medicine, Massachusetts General Hospital Cancer Center, Harvard Medical School, Broad Institute of Harvard and MIT, and Harvard Stem Cell Institute, discusses ongoing research into drug tolerance and resistance, specifically the roll of dormant cancer cells. If a tumor goes into remission as a result of a cancer drug and then recurs it is likely that the tumor will still respond to the initial treatment. In the dormant state the cells are resistance, in the original they are sensitive. The exact mechanism behind this has yet to be discovered. In some cases giving a course, stopping, and then continuing later on can create an additive effect, an idea that Ramaswamy calls a drug holiday. A comparison is underway between drug and non-drug induced dormant cells in order to find the mechanism that causes resistance. The ultimate goal of the research is to be able to predict and stop drug resistance.

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Dr. Ramaswamy on Dormant Tumor Cells and Resistance - Video

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JERUSALEM--(BUSINESS WIRE)--

Gamida Cell announced today that the Gamida Cell-Teva Joint Venture (JV), equally held by Gamida Cell and Teva Pharmaceutical Industries, has enrolled the last of 100 patients in the international, multi-center, pivotal registration, Phase III clinical trial of StemEx, a cell therapy product in development as an alternative therapeutic treatment for adolescents and adults, with blood cancers such as leukemia and lymphoma, who cannot find a family related, matched bone marrow donor.

StemEx is a graft of an expanded population of stem/progenitor cells, derived from part of a single unit of umbilical cord blood and transplanted by IV administration along with the remaining, non-manipulated cells from the same unit.

Dr. Yael Margolin, president and chief executive officer of Gamida Cell, said, "The JV is planning to announce the safety and efficacy results of the Phase III StemEx trial in 2012 and to launch the product into the market in 2013. It is our hope that StemEx will provide the answer for the thousands of leukemia and lymphoma patients unable to find a matched, related bone marrow donor.”

Dr. Margolin continued, “StemEx may be the first allogeneic cell therapy to be brought to market. This is a source of pride for Gamida Cell, as it further confirms the company’s leadership as a pioneer in cell therapy. In addition to StemEx, Gamida Cell is developing a diverse pipeline of products for the treatment of cancer, hematological diseases such as sickle cell disease and thalassemia, as well as autoimmune and metabolic diseases and conditions helped by regenerative medicine.”

About Gamida Cell

Gamida Cell is a world leader in stem cell population expansion technologies and stem cell therapy products for transplantation and regenerative medicine. The company’s pipeline of stem cell therapy products are in development to treat a wide range of conditions including blood cancers such as leukemia and lymphoma, solid tumors, non-malignant hematological diseases such as hemoglobinopathies, acute radiation syndrome, autoimmune diseases and metabolic diseases as well as conditions that can be helped by regenerative medicine. Gamida Cell’s therapeutic candidates contain populations of adult stem cells, selected from non-controversial sources such as umbilical cord blood, which are expanded in culture. Gamida Cell was successful in translating these proprietary expansion technologies into robust and validated manufacturing processes under GMP. Gamida Cell’s current shareholders include: Elbit Imaging, Clal Biotechnology Industries, Israel Healthcare Venture, Teva Pharmaceutical Industries, Amgen, Denali Ventures and Auriga Ventures. For more information, please visit: http://www.gamida-cell.com.

Read more here:
The Gamida Cell-Teva Joint Venture Concludes Enrollment for the Phase III Study of StemEx®, a Cord Blood Stem Cell ...

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MARLBOROUGH, Mass.--(BUSINESS WIRE)--

Advanced Cell Technology, Inc. (“ACT”; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today the dosing of third patient in its Phase 1/2 trial for Stargardt’s macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The patient was treated on Monday (Feb. 6) by Steven Schwartz, M.D., Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA) and retina division chief at UCLA’s Jules Stein Eye Institute. The outpatient transplantation surgery was performed successfully and the patient is recovering uneventfully.

“With the treatment of this third Stargardt’s patient at Jules Stein Eye Institute, we have now completed the treatment of the first cohort of patients under our clinical protocol for phase I/II of our U.S. SMD trial,” said Gary Rabin, chairman and chief executive officer of ACT. “We will continue to regularly monitor the three SMD patients in this trial, and by early spring anticipate review of their progress and safety-related data by the Data and Safety Monitoring Board (DSMB). With approval of the DSMB, we would then advance to the next cohort of patients and administer a higher dosage of RPE cells. In the context of all three trials we have running, this patient is the fifth person worldwide to be treated with our hESC-derived RPE cells. To date, there have been no complications or side effects due to the RPE cells, and we remain cautiously optimistic that our ongoing clinical programs will demonstrate the safety and tolerability of ACT’s stem cell-derived RPE cells.”

Each of the three clinical trials being undertaken by the company in the U.S. and Europe will enroll 12 patients, with cohorts of three patients each in an ascending dosage format. These trials are prospective, open-label studies, designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with SMD or dry age-related macular degeneration (dry AMD) at 12 months, the study’s primary endpoint. Preliminary results relating to both early safety and biological function for the first two patients in the United States, one SMD patient and one dry AMD patient, were recently reported in The Lancet. On January 20, 2012, the first SMD patient to be enrolled in the Company’s U.K. clinical trial was treated at Moorfields Eye Hospital in London.

Further information about patient eligibility for the SMD study and the concurrent study on dry AMD is also available on www.clinicaltrials.gov; ClinicalTrials.gov Identifiers: NCT01345006 and NCT01344993.

About Stargardt's Disease

Stargardt’s disease or Stargardt’s Macular Dystrophy is a genetic disease that causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium, which is the site of damage that the company believes the hESC-derived RPE may be able to target for repair after administration.

About Advanced Cell Technology, Inc.

Advanced Cell Technology, Inc., is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.

Forward-Looking Statements

Statements in this news release regarding future financial and operating results, future growth in research and development programs, potential applications of our technology, opportunities for the company and any other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “will,” “believes,” “plans,” “anticipates,” “expects,” “estimates,” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements, including: limited operating history, need for future capital, risks inherent in the development and commercialization of potential products, protection of our intellectual property, and economic conditions generally. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in the company’s periodic reports, including the report on Form 10-K for the year ended December 31, 2010. Forward-looking statements are based on the beliefs, opinions, and expectations of the company’s management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. Forward-looking statements are based on the beliefs, opinions, and expectations of the company’s management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. There can be no assurance that the Company’s clinical trials will be successful.

Originally posted here:
ACT Announces Third Patient with Stargardt’s Disease Treated in U.S. Clinical Trial with RPE Cells Derived from ...

Recommendation and review posted by simmons

Stem cells grown from patients’ own cardiac tissue can heal damage once thought to be permanent after a heart attack, according to a study that suggests the experimental approach may one day help stave off heart failure.

In a trial of 25 heart-attack patients, 17 who got the stem cell treatment showed a 50 percent reduction in cardiac scar tissue compared with no improvement for the eight who received standard care. The results, from the first of three sets of clinical trials generally needed for regulatory approval, were published today in the medical journal Lancet.

“The findings in this paper are encouraging,” Deepak Srivastava, director of the San Francisco-based Gladstone Institute of Cardiovascular Disease, said in an interview. “There’s a dire need for new therapies for people with heart failure, it’s still the No. 1 cause of death in men and women.”

The study, by researchers from Cedars-Sinai Heart Institute in Los Angeles and Johns Hopkins University (43935MF) in Baltimore, tested the approach in patients who recently suffered a heart attack, with the goal that repairing the damage might help stave off failure. While patients getting the stem cells showed no more improvement in heart function than those who didn’t get the experimental therapy, the theory is that new tissue regenerated by the stem cells can strengthen the heart, said Eduardo Marban, the study’s lead author.

“What our trial was designed to do is to reverse the injury once it’s happened,” said Marban, director of Cedars- Sinai Heart Institute. “The quantitative outcome that we had in this paper is to shift patients from a high-risk group to a low- risk group.”

Minimally Invasive

The stem cells were implanted within five weeks after patients suffering heart attacks. Doctors removed heart tissue, about the size of half a raisin, using a minimally invasive procedure that involved a thin needle threaded through the veins. After cultivating the stem cells from the tissue, doctors reinserted them using a second minimally invasive procedure. Patients got 12.5 million cells to 25 million cells.

A year after the procedure, six patients in the stem cell group had serious side effects, including a heart attack, chest pain, a coronary bypass, implantation of a defibrillator, and two other events unrelated to the heart. One of patient’s side effects were possibly linked to the treatment, the study found.

While the main goal of the trial was to examine the safety of the procedure, the decrease in scar tissue in those treated merits a larger study that focuses on broader clinical outcomes, researchers said in the paper.

Heart Regeneration

“If we can regenerate the whole heart, then the patient would be completely normal,” Marban said. “We haven’t fulfilled that yet, but we’ve gotten rid of half of the injury, and that’s a good start.”

While the study resulted in patients having an increase in muscle mass and a shrinkage of scar size, the amount of blood flowing out of the heart, or the ejection fraction, wasn’t different between the control group and stem-cell therapy group. The measurement is important because poor blood flow deprives the body of oxygen and nutrients it needs to function properly, Srivastava said.

“The patients don’t have a functional benefit in this study,” said Srivastava, who wasn’t not involved in the trial.

The technology is being developed by closely held Capricor Inc., which will further test it in 200 patients for the second of three trials typically required for regulatory approval. Marban is a founder of the Los Angeles-based company and chairman of its scientific advisory board. His wife, Lisa Marban, is also a founder and chief executive officer.

To contact the reporter on this story: Ryan Flinn in San Francisco at rflinn@bloomberg.net

To contact the editor responsible for this story: Reg Gale at rgale5@bloomberg.net

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Scarred Hearts Can Be Mended With Novel Stem Cell Therapy, Study Finds

Recommendation and review posted by simmons

COLUMBIA, SC (WIS) - Cutting-edge arthritis treatment for our four-legged family members is now available in Columbia.

Banks Animal Hospital is the first in the area to offer in-house Stem Cell therapy. It uses your pets own body to heal itself.

Take 13-year-old Maggie, for example. The energetic pup has a limp that usually keeps her from jumping or going up stairs.

"Today when everybody's out there filming her little limp it's not as pronounced because she wants to please," said Maggie's owner, Beth Phibbs. "She's just a great dog."

But a great attitude wasn't enough to repair a bad case of cervical spine arthritis.

So Monday, Beth brought Maggie to Banks Animal Hospital for the Stem Cell therapy. Like many, Beth had never heard of Stem Cell work in animals. "Until Dr. Banks mentioned it to me I was like, beg your pardon?"

"There's no down side, no side effects because you're using your own cells," said Dr Ken Banks.

Banks and his staff first gather some of Maggie's blood and fat. Both are good places to find the repair cells they're after. Adult stem cells, not the controversial embryonic kind, are then separated and spun down.

"The repair system in Maggie's body has failed," said Jason Richardson of MediVet-America. "It's fallen asleep at the wheel, we're taking these repair cells, activating them so a chronic condition like osteo arthritis to Maggie will now be an acute illness."

This kind of treatment used to take days with material being shipped across the country, but now it can be done in hours.

"The ability to do it same day, convenience, the ability to do it in clinic saves a lot of money to the doctor which he can then pass on to the patient," said Richardson.

The treatment will still run you around $2,000, but Richardson says that's half of what the similar treatment use to cost.

When it's over, Maggie should be able to live out her life pain and drug free -- something Phibbs is looking forward to.

"I'm hoping in a couple of weeks she's gonna have a new lease on life," said Phibbs.

Copyright 2012 WIS. All rights reserved.

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Vet offers stem cell therapy for dogs

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WALKER, Mich. (WOOD) - Dogs who received the first in-clinic stem cell therapy in West Michigan returned to the vets who treated them Monday morning.

Boris and Natasha returned to Kelley's Animal Clinic for their 60-day checkup after receiving stem cell treatment in December 2011.

Dr. James Kelley and his staff of vets removed fat tissue from the dogs and activated it with an enzyme before injecting it into their back legs.

This adult animal stem cell technology is different from the controversial embryonic stem cell therapy.

Kelley said both dogs are doing amazingly well and that the procedure has done more than just help their arthritis.

"We're finding that not only the joints are affected, the rest of the animal is affected as well," said Kelley. "The skin is better. The attitude in these dogs is much improved."

Kelley and his staff have done 16 stem cell treatments since the first on Boris and Natasha, and he said all the dogs are showing signs of improvement after a short period of time.

Read more:
Dogs who got stem cell therapy are well

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MONDAY, Feb. 13 (HealthDay News) -- Stem cell therapy's promise for healing damaged tissues may have gotten a bit closer to reality. In a small, early study, heart damage was reversed in heart-attack patients treated with their own cardiac stem cells, researchers report.

The cells, called cardiosphere-derived stem cells, regrew damaged heart muscle and reversed scarring one year later, the authors say.

Up until now, heart specialists' best tool to help minimize damage following a heart attack has been to surgically clear blocked arteries.

"In our treatment, we dissolved scar and replaced it with living heart muscle. Such 'therapeutic regeneration' has long been the holy grail of cell therapy, but had never been accomplished before; we now seem to have done it," said study author Dr. Eduardo Marban, director of the Cedars-Sinai Heart Institute in Los Angeles.

However, outside experts cautioned that the findings are preliminary and the treatment is far from ready for widespread use among heart-attack survivors.

The study, published online Feb. 14 in The Lancet, involved 25 middle-aged patients (average age 53) who had suffered a heart attack. Seventeen underwent stem cell infusions while eight received standard post-heart attack care, including medication and exercise therapy.

The stem cells were obtained using a minimally invasive procedure, according to the researchers from Cedars-Sinai and the Johns Hopkins Hospital in Baltimore.

Patients received a local anesthetic and then a catheter was threaded through a neck vein down to the heart, where a tiny portion of muscle was taken. The sample provided all the researchers needed to generate a supply of new stem cells -- 12 million to 25 million -- that were then transplanted back into the heart-attack patient during a second minimally invasive procedure.

One year after the procedure, the infusion patients' cardiac scar sizes had shrunk by about half. Scar size was reduced from 24 percent to 12 percent of the heart, the team said. In contrast, the patients receiving standard care experienced no scar shrinkage.

Initial muscle damage and healed tissue were measured using MRI scans.

After six months, four patients in the stem-cell group experienced serious adverse events compared with only one patient in the control group. At one year, two more stem-cell patients had a serious complication. However, only one such event -- a heart attack -- might have been related to the treatment, according to the study.

In a news release, Marban said that "the effects are substantial and surprisingly larger in humans than they were in animal tests."

Other experts were cautiously optimistic. Cardiac expert Dr. Bernard Gersh, a professor of medicine at Mayo Clinic, is not affiliated with the research but is familiar with the findings.

"This study demonstrates that it is safe and feasible to administer these cardiac-derived stem cells and the results are interesting and encouraging," he said.

Another specialist said that while provocative and promising, the findings remain early, phase-one research. "It's a proof-of-concept study," said interventional cardiologist Dr. Thomas Povsic, an assistant professor of medicine at the Duke Clinical Research Institute, in Durham, N.C.

And Dr. Chip Lavie, medical director of Cardiac Rehabilitation and Prevention at the John Ochsner Heart and Vascular Institute, in New Orleans, also discussed the results. He said that while the study showed that the cardiac stem cells reduced scar tissue and increased the area of live heart tissue in heart attack patients with moderately damaged overall heart tissue, it did not demonstrate a reduction in heart size or any improvement in the heart's pumping ability.

"It did not improve the ejection fraction, which is a very important measurement used to define the overall heart's pumping ability," Lavie noted. "Certainly, much larger studies of various types of heart attack patients will be needed before this even comes close to being a viable potential therapy for the large number of heart attack initial survivors."

Povsic concurred that much larger studies are needed. "The next step is showing it really helps patients in some kind of meaningful way, by either preventing death, healing them or making them feel better."

It's unclear what the cost will be, Povsic added. "What society is going to be willing to pay for this is going to be based on how much good it ends up doing. If they truly regenerate a heart and prevent a heart transplant, that would save a lot money."

Marban, who invented the stem cell treatment, said the while it would not replace bypass surgery or angioplasty, "it might be useful in treating 'irreversible' injury that may persist after those procedures."

As a rough estimate, he said that if larger, phase 2 trials were successful, the treatment might be available to the general public by about 2016.

More information

The U.S. National Heart, Lung, and Blood Institute describes current heart attack treatment.

Visit link:
Stem Cell Treatment Might Reverse Heart Attack Damage

Recommendation and review posted by simmons

MARLBOROUGH, Mass.--(BUSINESS WIRE)--

Advanced Cell Technology, Inc. (“ACT”; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today the dosing of third patient in its Phase 1/2 trial for Stargardt’s macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The patient was treated on Monday (Feb. 6) by Steven Schwartz, M.D., Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA) and retina division chief at UCLA’s Jules Stein Eye Institute. The outpatient transplantation surgery was performed successfully and the patient is recovering uneventfully.

“With the treatment of this third Stargardt’s patient at Jules Stein Eye Institute, we have now completed the treatment of the first cohort of patients under our clinical protocol for phase I/II of our U.S. SMD trial,” said Gary Rabin, chairman and chief executive officer of ACT. “We will continue to regularly monitor the three SMD patients in this trial, and by early spring anticipate review of their progress and safety-related data by the Data and Safety Monitoring Board (DSMB). With approval of the DSMB, we would then advance to the next cohort of patients and administer a higher dosage of RPE cells. In the context of all three trials we have running, this patient is the fifth person worldwide to be treated with our hESC-derived RPE cells. To date, there have been no complications or side effects due to the RPE cells, and we remain cautiously optimistic that our ongoing clinical programs will demonstrate the safety and tolerability of ACT’s stem cell-derived RPE cells.”

Each of the three clinical trials being undertaken by the company in the U.S. and Europe will enroll 12 patients, with cohorts of three patients each in an ascending dosage format. These trials are prospective, open-label studies, designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with SMD or dry age-related macular degeneration (dry AMD) at 12 months, the study’s primary endpoint. Preliminary results relating to both early safety and biological function for the first two patients in the United States, one SMD patient and one dry AMD patient, were recently reported in The Lancet. On January 20, 2012, the first SMD patient to be enrolled in the Company’s U.K. clinical trial was treated at Moorfields Eye Hospital in London.

Further information about patient eligibility for the SMD study and the concurrent study on dry AMD is also available on www.clinicaltrials.gov; ClinicalTrials.gov Identifiers: NCT01345006 and NCT01344993.

About Stargardt's Disease

Stargardt’s disease or Stargardt’s Macular Dystrophy is a genetic disease that causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium, which is the site of damage that the company believes the hESC-derived RPE may be able to target for repair after administration.

About Advanced Cell Technology, Inc.

Advanced Cell Technology, Inc., is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.

Forward-Looking Statements

Statements in this news release regarding future financial and operating results, future growth in research and development programs, potential applications of our technology, opportunities for the company and any other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “will,” “believes,” “plans,” “anticipates,” “expects,” “estimates,” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements, including: limited operating history, need for future capital, risks inherent in the development and commercialization of potential products, protection of our intellectual property, and economic conditions generally. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in the company’s periodic reports, including the report on Form 10-K for the year ended December 31, 2010. Forward-looking statements are based on the beliefs, opinions, and expectations of the company’s management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. Forward-looking statements are based on the beliefs, opinions, and expectations of the company’s management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. There can be no assurance that the Company’s clinical trials will be successful.

See the rest here:
ACT Announces Third Patient with Stargardt’s Disease Treated in U.S. Clinical Trial with RPE Cells Derived from ...

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ALACHUA, Fla.--(BUSINESS WIRE)--

AxoGen, Inc. (OTCBB: AXGN.OB - News), an emerging regenerative medicine company focused on the commercialization of proprietary products and technologies for peripheral nerve reconstruction and regeneration, today announced Jill Schiaparelli will be joining as its Senior Vice President of Business Strategy and Marketing.

“Strong sales growth is key to our continued success,” states Karen Zaderej, Chief Executive Officer of AxoGen, Inc. “Jill’s extensive strategic marketing and leadership experience in the healthcare space will add significant value to the team and will be ideal as we execute our growth initiatives.”

Prior to joining the team, Ms. Schiaparelli was a principal of JS Strategic Partners, a consultancy she founded to provide business strategy, growth initiatives, and commercialization implementation to healthcare companies and service providers. Jill also served as Global Vice President of Commercial Strategy & Business Development for ApaTech – a world leader in orthobiologics acquired by Baxter Healthcare. Earlier in her career, she held several senior positions in strategic marketing, sales operations, and analytics for the Johnson & Johnson family of companies and spent nearly a decade in investment banking.

Ms. Schiaparelli received her MBA from the Stern School of Business at New York University and a Bachelor’s of Science in Business Administration from Boston University.

About AxoGen, Inc.

AxoGen (OTCBB: AXGN.OB - News) is a regenerative medicine company with a portfolio of proprietary products and technologies for peripheral nerve reconstruction and regeneration. Every day people suffer traumatic injuries or undergo surgical procedures that impact the function of their peripheral nerves. Peripheral nerves provide the pathways for both motor and sensory signals throughout the body and their damage can result in the loss of function and feeling. In order to improve surgical reconstruction and regeneration of peripheral nerves, AxoGen has developed and licensed patented and patent-pending technologies, which are used in its portfolio of products. This portfolio includes Avance® Nerve Graft, which AxoGen believes is the first and only commercially available allograft nerve for bridging nerve discontinuities (a gap created when the nerve is severed). AxoGen’s portfolio also includes AxoGuard® Nerve Connector, a coaptation aid allowing for close approximation of severed nerves, and AxoGuard® Nerve Protector that protects nerves during the body’s healing process after surgery. AxoGen is bringing the science of nerve repair to life with thousands of surgical implants of AxoGen products performed in hospitals and surgery centers across the United States, including military hospitals serving U.S. service men and women.

AxoGen (formerly known as LecTec Corporation) is the parent of its wholly owned operating subsidiary, AxoGen Corporation. AxoGen’s principal executive office and operations are located in Alachua, FL.

Cautionary Statements Concerning Forward-Looking Statements

This Press Release contains “forward-looking” statements as defined in the Private Securities Litigation Reform Act of 1995. These statements are based on management’s current expectations or predictions of future conditions, events or results based on various assumptions and management’s estimates of trends and economic factors in the markets in which we are active, as well as our business plans. Words such as “expects”, “anticipates”, “intends”, “plans”, “believes”, “seeks”, “estimates”, “projects”, “forecasts”, “may”, “should”, variations of such words and similar expressions are intended to identify such forward-looking statements. The forward-looking statements may include, without limitation, statements regarding product development, product potential or financial performance. The forward-looking statements are subject to risks and uncertainties, which may cause results to differ materially from those set forth in the statements. Forward-looking statements in this release should be evaluated together with the many uncertainties that affect AxoGen’s business and its market, particularly those discussed in the risk factors and cautionary statements in AxoGen’s filings with the Securities and Exchange Commission. Forward-looking statements are not guarantees of future performance, and actual results may differ materially from those projected. The forward-looking statements are representative only as of the date they are made, and AxoGen assumes no responsibility to update any forward-looking statements, whether as a result of new information, future events or otherwise.

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AxoGen Names Jill Schiaparelli Senior Vice President of Business Strategy & Marketing

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In Remaking Eden (Harper Perennial, 1998), geneticist Lee Silver envisioned a future in which humanity has split into two species: “Naturals,” the poor slobs who muddle along with the genes that nature gave them, and the “GenRich,” who can afford to boost their physical and mental talents via genetic engineering. Silver warns that over time, “the genetic distance between the Naturals and the GenRich has become greater and greater, and now there is little movement up from the Natural to GenRich class.”

We don’t have to wait until science catches up to science fiction for this unjust dystopia to be realized. It’s happening now, in the United States, as a result of policies that favor the rich at the expense of un-rich. Scholars are confirming with empirical studies what Occupy Wall Street protesters have been saying: our system is unfairly rigged in favor of the haves, who keep pulling away from have-nots.

Education can help the poor climb their way to a higher socioeconomic status. But according to Sabrina Tavernise of The New York Times, several studies have shown that “the achievement gap between rich and poor children is widening, a development that threatens to dilute education’s leveling effects.”

Race plays less of a role than it once did in this widening chasm. A study published last year by sociologist Sean Reardon found that the difference between standardized test scores of blacks and whites has narrowed since 1960, while the difference between low-income and wealthy students has surged 40 percent. “We have moved from a society in the 1950s and 1960s,” Reardon told The Times, “in which race was more consequential than family income, to one today in which family income appears to be more determinative of educational success than race.”

The simplest explanation for the divide is that the rich can afford to send their children to better schools, hire private tutors for them and give them other advantages. In 1972, affluent parents spent five times as much on their children, on average, as low-income parents; by 2007, that difference had almost doubled, to nine to one. “The pattern of privileged families today is intensive cultivation,” sociologist Frank Furstenberg told The Times.

The federal tax code is also stacked against the poor. The code caps taxes on long-term capital gains and dividends at 15 percent, which is why Mitt Romney is taxed at a lower rate than a grade-school teacher. Far from being progressive, with percentages rising with income, the tax code is regressive in this key area. Those who work for a living pay more in taxes, percentage-wise, than those who live off investments.

Political scientist Andrew Hacker documents the depths of our inequality in “We’re More Unequal Than You Think,” in The New York Review of Books this month. He estimates that since 1985 “the lower 60 percent of households have lost $4 trillion, most of which has ascended to the top 5 percent.” U.S. economic policies, Hacker says, now serve as a “giant vacuum cleaner” sucking money from low-income people and showering it upon the rich.

Economists quantify the inequality of a society on a scale called the Gini index. If everyone has the same income, the Gini index is zero; if one person makes all the moola, the Gini index is one. The U.S. Gini index has risen from .359 in 1972 to .440 in 2010, an increase of more than 20 percent, Hacker reports. In contrast, the Gini index of socialist Sweden is .230.

Hacker notes that “in a not-so-distant past, families of modest means made enough to put something aside for their children’s college fees. That cushion is gone, which is why millions of undergraduates are now forced to take much larger loans. Adding interest and penalties, many will face decades paying off six-figure debts.” (I’m facing this financial challenge myself; my son is entering college next fall and my daughter a year later.)

The U.S. exemplifies the Matthew effect, a sociological term that alludes to a passage in the Gospel of Matthew: “For to all those who have, more will be given, and they will have an abundance; but from those who have nothing, even what they have will be taken away.”

Our current presidential race features several Christian candidates—Rick Santorum, Newt Gingrich and Romney—who seem to view the Matthew effect as the Eleventh Commandment. These men trumpet their religiosity and rectitude, and yet they advocate economic policies that benefit the rich and hurt the poor, violating the most basic rules of moral decency. Naturals must join together with rich people with a conscience to create a more economically just society.

Image courtesy Wikimedia Commons, http://www.flickr.com/tracy_olson.

Excerpt from:
Education Isn't Helping Americans Overcome Deepening Inequality

Recommendation and review posted by Bethany Smith

Public release date: 13-Feb-2012
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Contact: Cathia Falvey
cfalvey@liebertpub.com
914-740-2100
Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY -- Children are naturally drawn toward gaming and other types of technology, creating an ideal opportunity to design interactive media tools to encourage physical activity and promote healthy eating habits, according to an article in a special issue of the journal Childhood Obesity celebrating the second anniversary of First Lady Michelle Obama's Let's Move! initiative. The issue includes a special Foreword by Mrs. Obama and is available free online at http://www.liebertpub.com/chi.

"Let's Get Technical! Gaming and Technology for Weight Control and Health Promotion in Children," an article by Tom Baranowski, PhD and Leslie Frankel, PhD, USDA/ARS Children's Nutrition Research Center, Baylor College of Medicine, Houston, Texas, describes the ongoing research effort to identify and develop the most effective approaches for using gaming and interactive media to deliver health promotion messages to children of all ages.

This special Let's Move! issue has a wide range of contributions from leaders in the fight against childhood obesity including Secretary of Agriculture Tom Vilsack, NFL quarterback Drew Brees, Stephen Daniels, MD, PhD, Sandra Hassink, MD, Margo Wootan, DSc, and Editor-in-Chief David Katz, MD, MPH.

The issue covers a broad range of topics including creating environments that support routine physical activity and a healthy lifestyle, after-school obesity prevention programs, nutrition standards for school meals, faith-based advocacy efforts to end childhood obesity, gaming and technology for weight control, parent training programs for 2-4 year old Latino children, the role of sleep in childhood obesity, a roundtable discussion about what we don't know about childhood obesity, industry efforts to help children make healthy food choices, and success stories from the Let's Move! initiative.

"We know that 'screen time' is a contributor to childhood obesity. But we also know it's not going away. Thought leaders like Dr. Baranowski are showing how to convert parts of the problem into parts of the solution," says David L. Katz, MD, MPH, Editor-in-Chief of Childhood Obesity and Director of Yale University's Prevention Research Center. "We are honored to feature such pragmatic expertise on the pages of the Journal."

###

Childhood Obesity is partly funded by a grant from the W.K. Kellogg Foundation to ensure that the Journal is accessible as widely as possible, and to provide a framework that addresses the social and environmental conditions that influence opportunities for children to have access to healthy, affordable food and safe places to play and be physically active.

Childhood Obesity is a bimonthly journal, published in print and online, and the journal of record for all aspects of communication on the broad spectrum of issues and strategies related to weight management and obesity prevention in children and adolescents. The Journal includes peer-reviewed articles documenting cutting-edge research and clinical studies, opinion pieces and roundtable discussions, profiles of successful programs and interventions, and updates on task force recommendations, global initiatives, and policy platforms. It reports on news and developments in science and medicine, features programs and initiatives developed in the public and private sector, and a Literature Watch. Tables of content and a free sample issue may be viewed online T http://www.liebertpub.com/chi.

Mary Ann Liebert, Inc. is a privately held, fully integrated media company known for establishing authoritative medical and biomedical peer-reviewed journals, including Games for Health Journal, Telemedicine and e-Health, Population Health Management, Diabetes Technology & Therapeutics, and Metabolic Syndrome and Related Disorders. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 70 journals, newsmagazines, and books is available on our website at http://www.liebertpub.com.

Mary Ann Liebert, Inc.
140 Huguenot St., New Rochelle, NY 10801-5215
Phone: 914-740-2100
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AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert! system.

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Games and interactive media are powerful tools for health promotion and childhood obesity prevention

Recommendation and review posted by Bethany Smith

Bone marrow drive at Guam Premier Outlets: Bone marrow drive at Guam Premier Outlets Written by Pacific Daily News
MYTHS AND FACTS

Myths and facts about bone marrow donation:
? Myth: All bone marrow donations involve surgery.
? Fact: The majority of donations do not involve surgery. Today, the patient's doctor most often requests a peripheral blood stem cell donation, which is non-surgical. The second way of donating is marrow donation, which is a surgical procedure. In each case, donors typically go home the same day they donate.

?Myth: Donating is painful and involves a long recovery.
?Fact: There can be uncomfortable but short-lived side effects of peripheral blood stem cell donation. Due to taking a drug called filgrastim for five days leading up to donation, peripheral blood stem cell donors may have headaches, joint or muscle aches, or fatigue. Donors are typically back to their normal routine in one to two days. Those donating marrow receive general or regional anesthesia, so they feel no pain during donation. Marrow donors can expect to feel some soreness in their lower back for one to two weeks afterward. Most marrow donors are back to their normal activities in two to seven days.

?Myth: Donating is dangerous and weakens the donor.
?Fact: Though no medical procedure is without risk, there are rarely any long-term side effects. Be The Match carefully prescreens all donors to ensure they are healthy and the procedure is safe for them. We also provide support and information every step of the way.

Because only 5 percent or less of a donor's marrow is needed to save the patient's life, the donor's immune system stays strong and the cells replace themselves within four to six weeks.

?For more myths and facts, and more information about bone marrow donation, visit http://www.bethematch.org. Be The Match Registry is operated by the National Marrow Donor Program.

More In News

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UPDATE: Sign up for the National Bone Marrow Registry now

Recommendation and review posted by Bethany Smith

BOCA RATON—

A physician from Indianapolis met the woman who saved his life on Sunday morning, providing an emotional kick-off for the second annual Walk for Life, sponsored by the Gift of Life Bone Marrow Foundation.

"It's almost like a total out of body experience," said Scott Savader, 53, moments after he embraced former Sunrise resident Jill Rubin, who provided the stem cells that were transplanted into Savader's body nearly two years ago.

As the two met for the first time, about 300 people cheered before heading off on a 5K walk at Florida Atlantic University. The effort is part of a campaign to raise awareness and raise $100,000 for lab tests necessary to match donors and recipients.

Savader said receiving the transplant was "like being plucked from a fire or a sinking car. There is a bond there now that transcends just knowing somebody. If not for her generosity, I would have died."

Each year, 10,000 people in the U.S. are diagnosed with a disease treatable with a bone marrow transplant. Yet only about half find the donor who could save them, according to Jay Feinberg, the Delray Beach resident who started the foundation after he was diagnosed with an aggressive form of leukemia.

He received a transplant in 1995 and has since dedicated his life to making matches for others.

Savader, a radiologist, was diagnosed with myelofibrosis in 2008.

Rubin, 45, a physical therapist, said she registered as a bone marrow donor 10 years ago while attending a fair in Plantation. She and her family have since moved to Deland.

"This is very emotional for me," said Rubin as she and Savader posed for pictures.

After spending a little time with Savader and his family Sunday, Rubin said she felt even better about her gift to him.

She also learned that Savader grew up and went to high school in Cooper City. "Small world," she said.

Temperatures in the 40s and a chilly wind did little to dampen enthusiasm for the walk. Participants were inspired by Savader and Rubin and other success stories.

Among the latter were 6-year-old Matthew Welling, on hand with his parents Michael and Susie Welling of Port Chester, N.Y., and Boca Raton resident Jill Goldsmith, who donated the bone marrow that reversed the boy's osteoporosis in 2007.

"It was an amazing, life-changing experience," said Goldsmith, 50, as she watched Matthew dance happily around a field at the university.

"What I had to do to save a life was so easy," said Goldsmith. "And to see him now, well, I feel proud and honored and so blessed."

During last year's walk, more than 1,000 new donors were added to the registry and resulted in 14 matches for patients throughout the U.S. They joined a total registry of nearly 200,000, said Feinberg.

Volunteering to become a potential donor begins with an oral swab that is then tested for tissue type. Most of the foundation's money goes toward paying for those lab tests, which cost about $55 each, said Feinberg.

For information, go to mwclary@tribune.com">http://www.giftoflife.org.

mwclary@tribune.com or at 954-356-4465

Read more:
Bone marrow recipient meets donor who gave him gift of life

Recommendation and review posted by Bethany Smith

BOCA RATON—

A physician from Indianapolis met the woman who saved his life on Sunday morning, providing an emotional kick-off for the second annual Walk for Life, sponsored by the Gift of Life Bone Marrow Foundation.

"It's almost like a total out of body experience," said Scott Savader, 53, moments after he embraced former Sunrise resident Jill Rubin, who provided the stem cells that were transplanted into Savader's body nearly two years ago.

As the two met for the first time, about 300 people cheered before heading off on a 5K walk at Florida Atlantic University. The effort is part of a campaign to raise awareness and raise $100,000 for lab tests necessary to match donors and recipients.

Savader said receiving the transplant was "like being plucked from a fire or a sinking car. There is a bond there now that transcends just knowing somebody. If not for her generosity, I would have died."

Each year, 10,000 people in the U.S. are diagnosed with a disease treatable with a bone marrow transplant. Yet only about half find the donor who could save them, according to Jay Feinberg, the Delray Beach resident who started the foundation after he was diagnosed with an aggressive form of leukemia.

He received a transplant in 1995 and has since dedicated his life to making matches for others.

Savader, a radiologist, was diagnosed with myelofibrosis in 2008.

Rubin, 45, a physical therapist, said she registered as a bone marrow donor 10 years ago while attending a fair in Plantation. She and her family have since moved to Deland.

"This is very emotional for me," said Rubin as she and Savader posed for pictures.

After spending a little time with Savader and his family Sunday, Rubin said she felt even better about her gift to him.

She also learned that Savader grew up and went to high school in Cooper City. "Small world," she said.

Temperatures in the 40s and a chilly wind did little to dampen enthusiasm for the walk. Participants were inspired by Savader and Rubin and other success stories.

Among the latter were 6-year-old Matthew Welling, on hand with his parents Michael and Susie Welling of Port Chester, N.Y., and Boca Raton resident Jill Goldsmith, who donated the bone marrow that reversed the boy's osteoporosis in 2007.

"It was an amazing, life-changing experience," said Goldsmith, 50, as she watched Matthew dance happily around a field at the university.

"What I had to do to save a life was so easy," said Goldsmith. "And to see him now, well, I feel proud and honored and so blessed."

During last year's walk, more than 1,000 new donors were added to the registry and resulted in 14 matches for patients throughout the U.S. They joined a total registry of nearly 200,000, said Feinberg.

Volunteering to become a potential donor begins with an oral swab that is then tested for tissue type. Most of the foundation's money goes toward paying for those lab tests, which cost about $55 each, said Feinberg.

For information, go to mwclary@tribune.com">http://www.giftoflife.org.

mwclary@tribune.com or at 954-356-4465

Original post:
Bone marrow recipient meets donor who gave him the gift of life

Recommendation and review posted by Bethany Smith

LONDON: British scientists are claiming a breakthrough after creating brain tissue from human skin.

The researchers have for the first time generated a crucial type of brain cells in the laboratory by reprogramming skin cells.

They say it could speed up the hunt for new treatments for conditions such as Alzheimer's disease, epilepsy and stroke.

Advertisement: Story continues below

Until now it has only been possible to generate tissue from the cerebral cortex, the area of the brain where most serious neurological diseases occur, by using controversial embryonic stem cells, obtained by the destruction of an embryo. This has meant the supply of brain tissue available for research has been limited due to ethical concerns and limited availability.

Scientists at the University of Cambridge say that they have overcome this problem, showing for the first time that it is possible to re-program adult human skin cells so that they develop into neurons found in the cerebral cortex.

Initially, brain cells grown in this way could be used to help researchers gain a better understanding of how the brain develops and what goes wrong when it is affected by disease. They could also be used for screening new drug treatments.

Eventually, they hope the cells could be used to provide healthy tissue that can be implanted into patients to treat neurodegenerative diseases and brain damage.

Dr Rick Livesey, who led the research at the university's Gurdon Institute, said: ''The cerebral cortex makes up 75 per cent of the human brain. It is where all the important processes that make us human take place. It is, however, also the major place where disease can occur.

''We have been able to take reprogrammed skin cells so they develop into brain stem cells and then essentially replay brain development in the laboratory.

''We can study brain development and what goes wrong when it is affected by disease in a way we haven't been able to before. We see it as a major breakthrough in what will now be possible,'' said Dr Livesey, whose findings are published in the journal Nature Neuroscience.

The cerebral cortex is the part of the brain that is responsible for most of the high-level thought processes such as memory, language and consciousness.

Telegraph, London

See more here:
Scientists grow brain cells from human skin

Recommendation and review posted by Bethany Smith

Eventually they hope the cells could also be used to provide healthy tissue that can be implanted into patients to treat neurodegenerative diseases and brain damage.

Dr Rick Livesey, who led the research at the University of Cambridge's Gurdon [corr] Institute, said: "The cerebral cortex makes up 75% of the human brain, is where all the important processes that make us human take place. It is, however, also the major place where disease can occur.

"We have been able to take reprogrammed skin cells so they develop into brain stem cells and then essentially replay brain development in the laboratory.

"We can study brain development and what goes wrong when it is affected by disease in a way we haven't been able to before. We see it as a major breakthrough in what will now be possible."

The cerebral cortex is the part of the brain that is responsible for most of the major high-level thought processes such as memory, language and consciousness.

While human brain cells have been created from stem cells before, this has relied upon embryonic stem cells. Attempts to make them from skin cells have produced neurons that are not found in the cerebral cortex.

Dr Livesey and his colleagues were able to create the two major types of neuron that form the cerebral cortex from reprogrammed skin cells and show that they were identical to those created from the more controversial embryonic stem cells.

Dr Livesey, whose findings are published in the journal Nature Neuroscience, said this may eventually lead to new treatments for patients where damaged tissue could be replaced by brain cells grown in the laboratory from a sample of their skin.

He said: "You don't need to rebuild damage to recover function as the brain is quite good at recovering itself – it does this after stroke for example. However, it may be possible to give it some extra real estate that it can use to do this.

"We can make large numbers of cerebral cortex neurons by taking a sample of skin from anybody, so in principal it should be possible to put these back into the patients."

Dr Simon Ridley, head of research at Alzheimer's Research UK, which funded the study alongside the Wellcome Trust, added: "Turning stem cells into networks of fully functional nerve cells in the lab holds great promise for unravelling complex brain diseases such as Alzheimer's."

Excerpt from:
Human brain cells created from skin

Recommendation and review posted by Bethany Smith

London, Feb 12 (ANI): British scientists have for the first time generated crucial types of human brain cells in the laboratory by reprogramming skin cells, which they say could speed up the hunt for new treatments for conditions such as Alzheimer's disease, epilepsy and stroke.

Until now it has only been possible to generate tissue from the cerebral cortex, the area of the brain where most major neurological diseases occur, by using controversial embryonic stem cells, obtained by the destruction of an embryo.

This has meant the supply of brain tissue available for research has been limited due to the ethical concerns around embryonic stem cells and shortages in their availability.

However, scientists at the University of Cambridge now insist they have overcome this problem after showing for the first time that it is possible to re-programme adult human skin cells so that they develop into neurons found in the cerebral cortex, the Telegraph reported.

Initially brain cells grown in this way could be used to help researchers gain a better understanding of how the brain develops, what goes wrong when it is affected by disease and it could also be used for screening new drug treatments.

Eventually they hope the cells could also be used to provide healthy tissue that can be implanted into patients to treat neurodegenerative diseases and brain damage.

The cerebral cortex is the part of the brain that is responsible for most of the major high-level thought processes such as memory, language and consciousness.

"The cerebral cortex makes up 75 percent of the human brain, is where all the important processes that make us human take place. It is, however, also the major place where disease can occur," said Dr Rick Livesey, who led the research at the University of Cambridge's Gurdon [corr] Institute.

"We have been able to take reprogrammed skin cells so they develop into brain stem cells and then essentially replay brain development in the laboratory.

"We can study brain development and what goes wrong when it is affected by disease in a way we haven't been able to before. We see it as a major breakthrough in what will now be possible," he added.

Dr Livesey and his colleagues were able to create the two major types of neuron that form the cerebral cortex from reprogrammed skin cells and show that they were identical to those created from the more controversial embryonic stem cells.

He said this may eventually lead to new treatments for patients where damaged tissue could be replaced by brain cells grown in the laboratory from a sample of their skin.

"You don't need to rebuild damage to recover function as the brain is quite good at recovering itself - it does this after stroke for example. However, it may be possible to give it some extra real estate that it can use to do this," Dr Livesey said.

"We can make large numbers of cerebral cortex neurons by taking a sample of skin from anybody, so in principal it should be possible to put these back into the patients," he added.

Dr Simon Ridley, head of research at Alzheimer's Research UK, which funded the study alongside the Wellcome Trust, said: "Turning stem cells into networks of fully functional nerve cells in the lab holds great promise for unravelling complex brain diseases such as Alzheimer's."

The findings were published in the journal Nature Neuroscience. (ANI)

View post:
Brain cells created from human skin

Recommendation and review posted by Bethany Smith

JERUSALEM--(BUSINESS WIRE)--

Gamida Cell announced today that the Gamida Cell-Teva Joint Venture (JV), equally held by Gamida Cell and Teva Pharmaceutical Industries, has enrolled the last of 100 patients in the international, multi-center, pivotal registration, Phase III clinical trial of StemEx, a cell therapy product in development as an alternative therapeutic treatment for adolescents and adults, with blood cancers such as leukemia and lymphoma, who cannot find a family related, matched bone marrow donor.

StemEx is a graft of an expanded population of stem/progenitor cells, derived from part of a single unit of umbilical cord blood and transplanted by IV administration along with the remaining, non-manipulated cells from the same unit.

Dr. Yael Margolin, president and chief executive officer of Gamida Cell, said, "The JV is planning to announce the safety and efficacy results of the Phase III StemEx trial in 2012 and to launch the product into the market in 2013. It is our hope that StemEx will provide the answer for the thousands of leukemia and lymphoma patients unable to find a matched, related bone marrow donor.”

Dr. Margolin continued, “StemEx may be the first allogeneic cell therapy to be brought to market. This is a source of pride for Gamida Cell, as it further confirms the company’s leadership as a pioneer in cell therapy. In addition to StemEx, Gamida Cell is developing a diverse pipeline of products for the treatment of cancer, hematological diseases such as sickle cell disease and thalassemia, as well as autoimmune and metabolic diseases and conditions helped by regenerative medicine.”

About Gamida Cell

Gamida Cell is a world leader in stem cell population expansion technologies and stem cell therapy products for transplantation and regenerative medicine. The company’s pipeline of stem cell therapy products are in development to treat a wide range of conditions including blood cancers such as leukemia and lymphoma, solid tumors, non-malignant hematological diseases such as hemoglobinopathies, acute radiation syndrome, autoimmune diseases and metabolic diseases as well as conditions that can be helped by regenerative medicine. Gamida Cell’s therapeutic candidates contain populations of adult stem cells, selected from non-controversial sources such as umbilical cord blood, which are expanded in culture. Gamida Cell was successful in translating these proprietary expansion technologies into robust and validated manufacturing processes under GMP. Gamida Cell’s current shareholders include: Elbit Imaging, Clal Biotechnology Industries, Israel Healthcare Venture, Teva Pharmaceutical Industries, Amgen, Denali Ventures and Auriga Ventures. For more information, please visit: http://www.gamida-cell.com.

Go here to see the original:
The Gamida Cell-Teva Joint Venture Concludes Enrollment for the Phase III Study of StemEx®, a Cord Blood Stem Cell ...

Recommendation and review posted by Bethany Smith

A team of researchers from Iowa State University (ISU) and the Fred Hutchinson Cancer Research Center utilized the Life Sciences Collaborative Access Team 21-ID-F x-ray beamline at the APS and beamline 5.0.2 at the Advanced Light Source at the Lawrence Berkeley National Laboratory to take a close look at a group of fused proteins that can control genes. Their work has revealed the crystallographic structure of a protein encoded by an important group of harmful plant pathogens that has evolved to manipulate host gene expression in a specific yet highly adaptable manner.

Xanthomonas bacteria inject TAL effector proteins into the cells of plants that they infect. The proteins wind themselves into the major groove of specific sections of the plants' DNA. This switches-on genes that benefit the invading bacteria. Conversely, the same binding might activate genes that stimulate the plants' defense systems against the pathogens. The DNA binding step, which involves a molecular recognition process, requires tandem repeat units of the protein building blocks, amino acids, usually 34 amino acids long and ending with a half repeat. The repeating patterns have allowed researchers to predict the likely targets for these binding regions and so engineer novel sequences that can also bind to specific strands of DNA.

This research has stimulated renewed interest in the possibility of designing artificial "TAL nuclease" (TALEN) proteins that could target DNA and thereby modify specific genes for a wide variety of purposes in crop plants or possibly to even treat human genetic diseases. Adam Bogdanove, of Iowa State University, and colleagues have been building on these discoveries for several years. In 2011, Bogdanove and a team at the University of Minnesota, led by Dan Voytas, formerly of ISU, and a second ISU group led by Bing Yang demonstrated in the laboratory that such a notion was potentially viable as they could create TALENs to manipulate genes and gene functions.

Now, writing in the journal Science in January 2012, Barry Stoddard and colleagues at the Fred Hutchinson Cancer Research Center in Seattle, including post-doc Amanda Mak and faculty member Phil Bradley, have collaborated with Bogdanove and Andres Cernadas to use multiple expression clones and data relating to target site sequences for co-crystallization. The team used a TAL effector, PthXo1, produced by the rice pathogen Xanthomonas oryzae and used x-ray diffraction data collected at the two DOE light sources to solve its three-dimensional (3-D) structure bound to a 36-base pair DNA duplex containing a sequence found in the rice genome itself.

To solve the structure, Bradley developed a high-throughput computational structure prediction method for TAL effectors using the Rosetta software package to generate a highly accurate molecular model for molecular replacement phasing. The resulting structure was then validated against the peaks obtained for selenomethionyl derivative, in which anomalous peaks would match up with the original data only if the model were correct.

Having obtained the 3-D structure of a TAL effector and shown how it physically interacts with double-stranded DNA, the researchers can now figure out the details of the recognition and binding process involved and so get a clearer picture of how they work in nature and how they might be manipulated in the laboratory.

They conclude in their latest Science paper that the work "reveals the hitherto enigmatic structural nature of a simple solution that an important group of pathogens has evolved to manipulate host gene expression in a specific yet highly adaptable manner." Bogdanove describes the structure the team has obtained as "really quite beautiful." "So far there is nothing else in nature quite like it," he said.

More information: Amanda Nga-Sze Mak, Philip Bradley, Raul A. Cernadas, Adam J. Bogdanove, and Barry L. Stoddard, “The Crystal Structure of TAL Effector PthXo1 Bound to Its DNA Target,” Science 335(6069), 716 (10 February, 2012). http://www.science … 716.abstract

Provided by Argonne National Laboratory (news : web)

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Manipulating genes with hidden TALENs

Recommendation and review posted by Bethany Smith

CAMBRIDGE, Mass.--(BUSINESS WIRE)--

Foundation Medicine, Inc., a molecular information company that brings comprehensive cancer gene analysis to routine clinical care, and Dana-Farber Cancer Institute today announced the Nature Medicine publication of results from their collaborative next-generation sequencing (NGS) study to assay cancer-relevant genes in 24 non-small cell lung cancer (NSCLC) and 40 colorectal cancer (CRC) cases. In this study, 59% of the samples were found to have genomic alterations directly associated with a clinically-available targeted therapeutic or a relevant clinical trial of a targeted therapy. Two novel gene fusions, KIF5B-RET in NSCLC and C2orf44-ALK in CRC, were discovered among the potentially druggable alterations identified in the study. Both of these findings may expand therapeutic options for a subset of cancer patients. This publication demonstrates that using targeted NGS to profile patient tumors for molecular alterations associated with therapeutic responses may have an important clinical impact in cancer treatment.

“In this collaboration, we detected clinically-relevant genomic alterations in more than half of the samples profiled, and, because Foundation Medicine’s NGS assay detects all classes of alterations with clinical-grade sensitivity, this research was able to identify both expected as well as completely novel alterations,” said Maureen Cronin, Ph.D., senior vice president, research & product development of Foundation Medicine and co-author of the study. “The discovery of novel rearrangements and fusions, such as KIF5B-RET and C2orf44-ALK, supports an important role for NGS in the clinical understanding and treatment of cancer.”

“In a common indication like NSCLC, identifying even a small subpopulation of individuals with gene fusions who may be responsive to a targeted therapy has the potential for major therapeutic impact,” said Phil Stephens, Ph.D., executive director, cancer genomics of Foundation Medicine and co-author of the study. “This joint research with Dana-Farber translates genomic research to the clinic and we expect that it may quickly have a positive impact for patients.”

Clinically-relevant alterations, which are defined here as being associated with an available clinical treatment option or ongoing clinical trial investigating a new targeted therapy, were identified in 72% of NSCLC tumor samples and 52.5% of CRC tumor samples.

The novel, recurrent KIF5B-RET fusion was identified by the NGS assay in one patient with NSCLC. In subsequent screening, 11 additional RET fusions were identified in 561 lung adenocarcinoma samples from a cohort of never or limited former smokers with NSCLC. In common with known oncogenic alterations in EGFR and EML4-ALK, the KIF5B-RET gene fusion was found more than twice as often in NSCLC samples from individuals of Asian descent (0.8% (1/212) of the Caucasian samples and 2% (9/405) of the Asian patient samples). Additionally, none of the fusion-positive tumors contained alterations in any of the other known oncogenes that drive lung cancer (EGFR, ERBB2, BRAF or KRAS or rearrangements of EML4-ALK or ROS1).Tumors with this fusion were specifically sensitive to targeted drugs that inhibit RET, suggesting that prospective clinical trials of RET-targeted therapeutics may benefit individuals with NSCLC with KIF5B-RET rearrangements.

The second novel finding in the study was a potentially clinically-relevant gene fusion between C2orf44 and ALK identified in one CRC patient. Additional assays suggest this fusion gene yields 90-fold overexpression of anaplastic lymphoma kinase (ALK), the target of crizotinib, a U.S. FDA approved therapy for NSCLC. Given the structure of the rearrangement that generated the C2orf44-ALK fusion, it is unlikely that current clinical detection methods would have detected this alteration. This research thus suggests that a previously unrecognized subset of individuals with CRC may harbor genetic alterations that may make them responsive to ALK-inhibitor treatment.

The assay used for the testing described in this Nature Medicine paper is analytically validated to have a false discovery rate of less than 1% with at least 99% sensitivity for base substitutions occurring with at least 10% frequency.

The paper, “Identification of new ALK and RET gene fusions from colorectal and lung cancer biopsies” by Lipson, D. et al. is now available online.

About Foundation Medicine’s Comprehensive Cancer Genomic Test

Foundation Medicine’s comprehensive cancer genomic test uses next-generation sequencing to analyze routine clinical specimens (i.e., small amounts of formalin fixed, paraffin embedded tumor tissue) for all classes of genomic alterations (point mutations, copy number alterations, insertions/deletions, and select rearrangements) in approximately 200 cancer-related genes. The test is optimized for clinical-grade analysis of tumor tissues, overcoming multiple complexities (such as purity, ploidy and clonality) inherent to tumor genomes. Results are designed to serve as a helpful decision-support tool for physicians to evaluate cancer treatment approaches tailored to each patient’s molecular subtype. Each patient report is reviewed and annotated by a molecular oncologist and consists of scientific and medical literature relevant to that patient’s genomic alterations and includes information on targeted therapies and clinical trials supported by scientific and medical research.

About Foundation Medicine

Foundation Medicine is dedicated to improving cancer care through the development of comprehensive cancer diagnostics that will help physicians inform treatment decisions based on an individual patient’s molecular cancer subtype. Foundation Medicine’s first laboratory developed test, based on a next-generation sequencing platform, is designed to accommodate a broad landscape of cancer genome information and a growing repertoire of targeted treatments and clinical research opportunities. Foundation Medicine’s test will assist physicians to make prompt and informed determinations about the best cancer treatments and clinical trial options for each patient, taking into account each patient’s unique cancer-associated alterations alongside publicly available scientific and medical information. The company’s founding advisors are world leaders in genome technology, cancer biology and medical oncology; they, alongside clinicians, biotech and molecular diagnostics industry leaders, are working to harness emerging technologies to develop unparalleled tests that will identify and interpret an ever-growing set of actionable genomic alterations, truly enabling personalized cancer medicine. For more information, please visit the company’s website at http://www.foundationmedicine.com.

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Foundation Medicine and Dana-Farber Cancer Institute Identify Novel Genomic Alterations in Lung and Colorectal Cancer

Recommendation and review posted by Bethany Smith

DENVER, Feb. 13, 2012 /PRNewswire/ -- According to a new study the preference for fatty foods has a genetic basis, and those with certain forms of the CD36 gene may like high-fat foods more than those who have other forms of this gene. The results may help explain why some people struggle when placed on a low-fat diet and may one day assist people in selecting diets that are the best fit for them to follow.  Atkins™, the original and leading low-carb weight-loss plan based on an extensive scientific body of research, is a better alternative for those who require a low-carb, higher-fat diet in order to lose and maintain weight.

"Fat is universally palatable to humans," says lead author Kathleen Keller, assistant professor of nutritional sciences, Penn State. "Yet we have demonstrated for the first time that people who have particular forms of the CD36 gene tend to like higher-fat foods more and may be at greater risk for obesity compared to those who do not have this form of the gene. Our study is one of the first to show this relationship in humans."

According to Colette Heimowitz, M.Sc., vice president of nutrition and education for Atkins Nutritionals, Inc., "This study illustrates why some diets simply do not work for certain people who would fare better on a higher-fat, low-carbohydrate plan. We already know that Atkins offers a scientifically proven alternative that delivers better weight loss and health marker improvements among those who have carb intolerance, including those with metabolic syndrome, pre-diabetes or insulin resistance.  Now we have a test that can help individuals identify their individual tendency and therefore prevent issues of sugar metabolism disorders by following a higher-fat, lower-carbohydrate program before obesity occurs."

Given that the Atkins Diet™ reduces carbohydrates during weight loss, the dieter is directed to increase their healthy carb intake until they find their personal carb balance – the level where their body can effectively metabolize carbs and burn fat for fuel while maintaining long-term weight loss. Atkins is unique in that no other weight-loss and maintenance program does this.  The Atkins Diet is backed by more than 80 published, peer-reviewed studies conducted over the past several decades.

The study "Common Variants in the CD36 Gene are Associated with Oral Fat Perception, Fat Preferences, and Obesity in African Americans," was led by a team of scientists from Penn State, Columbia University and Rutgers University who examined 317 African-American males and females because individuals in this ethnic group are highly vulnerable to obesity and thus are at greatest risk for obesity-related diseases.

"Our results may help explain why some people have more difficulty adhering to a low-fat diet than others and why these same people often have better compliance when they adopt higher-fat, low-carbohydrate diets.  The Atkins Diet is one example," says Keller.

About Atkins Nutritionals, Inc.

Atkins Nutritionals, Inc. is a leader in the $2.4 billion weight control nutrition category, and offers a powerful lifetime approach to weight loss and management. The Atkins Diet focuses on a healthy diet with reduced levels of refined carbohydrates and added sugars and encourages the consumption of protein, fiber, fruits, vegetables and good fats. Backed by research and consumer success stories, this approach allows the body to burn more fat and work more efficiently while helping individuals feel less hungry, more satisfied and more energetic.

Atkins Nutritionals, Inc., manufactures and sells a variety of nutrition bars and shakes designed around the nutritional principles of the Atkins Diet™.  Atkins' four product lines: Advantage®, Day Break™, Endulge™ and Cuisine™ appeal to a broad audience of both men and women who want to achieve their weight management goals and enjoy a healthier lifestyle. Atkins products are available online at atkins.com and in more than 30,000 locations throughout the U.S. and internationally. For more information, visit atkins.com.

 

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Atkins Diet Plan Ideal for Those with Fat Preference Gene

Recommendation and review posted by Bethany Smith

BRIDGEPORT -- Every person is unique. Thus, it follows that no two people will respond the same exact way to the same illness, the same medication or even the same food.

At least, that's the theory behind much of what's taught at the University of Bridgeport's College of Naturopathic Medicine, where students are trained in providing a more personalized kind of medical care. "Rather than (advocating) the practice of `Everyone with this disease gets this drug,' we focus on treating the individual," said Dr. David M. Brady, UB vice provost for health sciences.

The college will soon be able to expand its training and provide more naturopathic health services to the greater Bridgeport community with the development of its new Center for Excellence in Generative Medicine, which is slated to open in late March or early April. The center will be located in a grand, white former Victorian across the street from UB's Health Sciences Center, where many naturopathic students are currently trained.

The Center for Excellence will build on the work of its new director, Dr. Peter D'Adamo, supervising physician of the personalized medicine shift at the Health Sciences Center. D'Adamo is perhaps best known for writing the best-selling 1996 book "Eat Right 4 Your Type," which advocates the theory that your blood type is a genetic marker that dictates which types of foods are best for you.

As a doctor, he's designed a software program, called SWAMI, that uses various pieces of genetic information about a patient to determine which foods are most beneficial for him or her. "How does one person thrive on a low-fat diet and one person thrive on a low-carb diet?" D'Adamo said. "The truth is, there are probably as many different kinds of diets as there are people."

The SWAMI software is used as part of the naturopathic college's curriculum. He's also working on a form of software that uses genetic information and other data to determine the best medical therapies for an individual patient.

D'Adamo said, too often, physicians overlook things like genetic markers and nutrition when determining the best way to treat a patient. Among its many missions, the Center of Excellence will sponsor research in the fields of nutrigenomics and epigenetics, which examine how human genes interact and are affected by the environment, lifestyle and diet.

Brady and D'Adamo said the new center is in keeping with the university's commitment to both education and to serving the community. The university's health programs -- which include not just the naturopathic college, but also schools of acupuncture, chiropractic medicine, dental hygiene, human nutrition and a physician assistant school -- conduct about 20,000 patient visits a year and provide several million dollars worth of medical care. The care is provided at a low cost or no cost.

"The nice thing about this is we're offering a high level of health care to the medically underserved," Adamo said.

Many of UB's naturopathic students are looking forward to the opening of the new center, including Kristin Tomko, a fourth-year naturopathic medicine student from Jericho, Vt. "I think it's a great idea," she said.

acuda@ctpost.com; 203-330-6290; twitter.com/AmandaCuda; http://blog.ctnews.com/whatthehealth/

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University of Bridgeport to open 'Center of Excellence'

Recommendation and review posted by Bethany Smith

By Julian Gavaghan

Last updated at 11:00 AM on 13th February 2012

Twin brothers have told of their ‘devastation’ after both dramatically lost their sight to a rare condition that affects just a few hundred people.

Michael and Dan Smith, 20, are still determined to finish university and get good jobs after being left almost totally blind by Leber's Optic Neuropathy.

Michael, who is in football training for this year's Paralympic Games, lost his sight in a matter of weeks while in his first year at Bart's and the London School of Medicine.

Rare condition: Dan (left) and Michael Smith suffer from Leber's optic Neuropathy

Less than a year later, his brother Dan suffered the same fate while in his second year studying aeronautical engineering at the University of Bristol.

The identical pair, who can only make out shadowy shapes, have been forced to adapt their lives, learning Braille and re-learning how to cook and choose clothes.

 

They also plan to embark on a 350-mile tandem bike ride from London to Amsterdam in April.

Michael first noted changes to his vision in November 2009, forcing him into the ‘crushing’ realisation he could not continue his degree in medicine.

WHAT EXACTLY IS LEBER'S OPTIC NEUROPATHY?

Leber's Optic Neuropathy is a hereditary condition passed on by the mother’s genes.

The genetic defect, which can lead to optic nerve damage – or neuropathy, is carried by roughly one in every 9,000 people.

But genes, which can cause eye cells to die off quickly, can pass though several generations without triggering the condition.

Around 50 per cent of men and 80 per cent of women with it will suffer no loss of vision. No reason is known for the difference between the sexes.

In the Smith brothers’ case, they have been unable to find any family history of sudden onset blindness going back four generations.

The main symptom is the sudden loss of vision, which is caused by a death of cells in the optic nerve responsible for relaying visual information from the eyes to the brain.

Once cells start dying, affected eyes will begin seeing colours that appear more washed out. Within a period of eight weeks, it can lead to near or total blindness.

In many cases, only one eye is affected and patients may not be aware of the loss of colour vision until the doctor asks them to cover the healthy eye.

Most of the time, however, both eyes are affected. Sometimes vision improves, although this is very unusual.

‘My symptoms came on suddenly - one day I was in a lecture and I could not make out the projector in front of me, it was really hazy,’ he said.

‘I lost 70 per cent to 80 per cent of vision in my left eye in a very short space of time.

'I continued at medical school for a couple of weeks but it was taking me half an hour to read a page of A4 and I was breaking down in tears.

‘I did not know what was happening to me. I could not recognise people's faces and I quickly lost my independence.’

A genetic test eventually revealed Michael was suffering from the rare genetic condition, which also caused rapid sight loss in his right eye.

‘It was the end of life as I knew it,’ he said. ‘I could not carry on at medical school and that was incredibly distressing. In fact, it was crushing.

‘I can never describe how devastating it was to give up on medicine.

‘Not only that but I couldn't go for a run on my own without it being dangerous, I could not prepare food, I did not know what I was wearing.’

Dan also ‘could not comprehend what was happening’ when he heard Michael's news.

‘Michael has been the closest person to me all my life and this struck him in his first week at university,’ he said. ‘It was incredibly hard to see this happening to my brother.

‘He called me to say he thought something was wrong - he wasn't able to recognise people but didn't know why. That conversation still haunts me.

‘Doctors initially thought he had a brain tumour but tests revealed that he had this rare genetic disorder.

‘The whole family was devastated as we tried to comprehend what had happened.

‘It was the cruellest of timing because it was my brother's first term at medical school and he had his whole life ahead of him.’

But further terrible news was still to come. Because the pair are identical twins, Dan was told he had a 60 per cent to 70 per cent chance of also going blind.

Optic neuropathy: Cracks show nerve damage in the eye, caused by cells dying

‘Knowing I could lose my sight was psychologically very, very tough,’ he said.

‘Leber's Optic Neuropathy is known as 'The Sword of Damocles', based on the Greek parable, because your life goes from being great, in a period of heightened happiness, to hitting rock bottom in an instance.

‘I had a pretty tough time knowing that this dark cloud was hanging over me during my first two years at university.

‘It was effectively like sitting on a time bomb that could have gone off at any moment.’

In Easter 2010, Dan began to lose sight in his left eye and within three weeks there was also ‘full scale deterioration’ in his right eye.

Michael said: ‘Once I was diagnosed, the one thing I wanted in life was for this not to happen to my brother too.’

Although Leber's Optic Neuropathy is a hereditary condition, the brothers have been unable to find any family history going back four generations.

The twins, from Barnet, north London, can now make out shadows but use white canes to help them move around.

It is not known if they will lose their remaining sight but their condition is extremely unlikely to improve.

Nevertheless, they are determined to live life to the full. Michael is now studying geography at King's College London and wants to be a disability lawyer when he graduates.

Dan is still studying aeronautical engineering and aims to go into investment banking.

Michael said: ‘The last two years have been the most difficult but also the most exciting of my life.

‘We have had to learn new skills such as Braille and a new computer language that coverts text to speech. In lectures, we have note-takers.

‘Everything is through touch now, I select clothes through touch and texture and cooking is through touch and smell.

‘I thought I would never smile again, but we wanted our lives back.’

Damage: The normally smooth contour of the retina has large black areas where photoreceptors have been lost due to the macular degeneration

Michael plays for the England blind football team and is hoping to get picked for this year's Paralympic Games.

‘It's incredibly competitive but I'm training all the time,’ he said.

The Arsenal fans will undertake the London-Amsterdam tandem bike ride on April 6 to raise awareness of their condition.

They hope to raise over ?3,000 for Blind in Business, a charity which helps blind and partially sighted people into work.

Dan Mitchell, training and fundraising manager at Blind in Business, said: ‘Having the Smith brothers embark on such a challenging journey to raise money for this small charity shows they always want to work towards bigger challenges.

‘They have both been challenged academically and have pushed themselves as visually impaired people, working towards careers in engineering and law.’

Leber's Optic Neuropathy mostly strikes young men and is caused by complex genetic defects.

Vision loss results from the death of cells in the optic nerve responsible for relaying visual information from the eyes to the brain.

Although central vision gradually improves in a small number of cases, for most people vision loss is permanent.

Michael and Dan can be sponsored via http://www.justgiving.com/sevenmenfivebikes.

 

 

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Twin brothers' devastation after both rapidly lost their sight due to rare genetic condition

Recommendation and review posted by Bethany Smith

John Arispizabal

"The Creative Destruction of Medicine" by Eric Topol M.D.

In “The Creative Destruction of Medicine,’’ Eric Topol, a cardiologist and geneticist at Scripps Health and Scripps Research Institute in San Diego, argues that we are on the brink of a revolutionary transformation in which recent technological and scientific advances will enable the personalization of medicine in ways that would have seemed like science fiction only a short while ago.

The convergence of six major technological advances - cellphones, personal computers, the Internet, digital devices, genetic sequencing, and social networks - are, in Topol’s view, making the “creative destruction of medicine’’ inevitable.

Topol borrows the term from economist Joseph Schumpeter, who coined it to describe the way major innovations tend to prove disruptive, rendering existing systems and technologies obsolete, as a necessary step on the path to new and better ways of doing things.

The new technologies will, he argues, bring about radical changes in the ways scientific knowledge is processed and shared more quickly and broadly; patient data collected, with real-time monitoring and diagnosis; and treatment becomes increasingly individualized. He predicts much of this will be driven by patients and that doctors and others who might be inclined to resist these changes will be under pressure either to embrace, or at the very least, to step aside and not interfere with them. And these looming developments will improve not just patient care but the efficiency of the entire health care system.

One area Topol identifies as ripe for change involves how prescription medicines are used in the country, practices that run more than $300 billion a year. He cites studies showing that popular medications such as Lipitor and Crestor have been proven to benefit only 1 to 2 percent of patients taking them who do not already have heart disease. When it becomes possible to predict in advance which patients will benefit from these medicines, a large part of the $26 billion spent annually on them could be saved, to say nothing of preventing their sometimes serious adverse effects.

Plavix, a drug given to prevent blood clots (on which $9 billion was spent in 2010), is another example. It is now known that at least 30 percent of people are unable to convert it into its active form due to genetic mutations. Not only does giving it to someone who cannot metabolize it place that person at risk for developing a dangerous blood clot, it, too, wastes resources.

Genetic variability explains why some people respond well to certain medications while others do not or suffer adverse effects from them. While the genetic mutations that determine responsiveness to Plavix can be tested for (and are, in certain medical centers), many others that affect other medications remain unknown. It is likely that as more are discovered, genetic sequencing will become a routine part of how specific treatments are chosen for patients.

This runs against the current model of using data derived from population studies of groups of patients to inform treatment decisions. Currently, there is considerable pressure from insurers on physicians to adhere to guidelines based upon data from population studies, and those who do not are penalized under the banner of “pay for performance.’’ Yet biology is more complex than standardization allows for, and simply drawing straight lines through uneven scatters of data points doesn’t change this fact.

A small warning: This book is packed with information and so some spots may be slow going for the less quantitative among us. But Topol does an excellent job of explaining all, and his enthusiasm for the possibilities of what the future holds is infectious. It can only be hoped, as the convergence he so convincingly predicts materializes, that the barriers erected by the gatekeepers of yesterday’s paradigms will be easily dismantled so as not to impede the benefits it promises.

Try BostonGlobe.com today and get two weeks FREE. Dennis Rosen, a pediatric pulmonologist, can be reached at dennis.rosen@childrens.harvard.edu.

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‘The Creative Destruction of Medicine’ by Eric Topol - The Boston Globe

Recommendation and review posted by Bethany Smith