StemCell Therapy

Triad Business Journal

College hires leader for Biotechnology Center of Excellence Triad Business Journal Our Biotechnology Center of Excellence will be key to Alamance County's economic development and we are excited to have an administrator with the depth and breadth of experience as Yonnie Butler, said President Algie Gatewood. His range of ...

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College hires leader for Biotechnology Center of Excellence - Triad Business Journal

The Nasdaq Composite Index rose Wednesday, buoyed by broad advances at biotechnology and IT companies, after the Federal Reserve decided to raise interest rates for the second time in three meetings.

The technology-heavy index rose 0.7% to 5,900.05, its fifth advance in the last six sessions. The benchmark index settled just below all-time highs.

With the gain, the Nasdaq has returned nearly 10% for the year, outpacing the S&P 500 Index and Dow Jones Industrial Average.

A total of 2,009 companies listed on the Nasdaq reported gains, versus 888 that finished lower and 229 that went unchanged. A total of 166 companies reported new highs, versus 47 that set new lows.

Health stocks listed on the S&P 500 rose more than 1%, with pharmaceuticals, biotechnology and life sciences leading the rally. Information technology also advanced 0.6% as a sector.

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The Nasdaq Biotechnology Index rose 1.5% to 3,162.82.

The Federal Reserve raised its benchmark interest rate by a quarter point to 1% in a move that was widely anticipated by the markets. Policymakers continue to expect three rate increases this year, putting the central bank on course for two additional adjustments in 2017.

In terms of upcoming releases, the U.S. Labor Department will report on initial jobless claims on Thursday. Separately, the Commerce Department will report on housing starts and building permits. The Philadelphia Fed will also report on regional manufacturing conditions in the early morning.

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Nasdaq Composite Index Approaches Record High as Biotechnology, IT Advance - Economic Calendar

Sam Konduros(Photo: Provided)

Sam Konduros, a former executive director of Greenville Health Systems Research Development Corporation, was named on Monday as the new president and CEO of the S.C. Biotechnology Industry Organization.

SCBIO is a statewide, non-for-profit that represents and organizes innovators in medicine, medical devices and biomaterials.

Konduros is a currently a member of the SCBIO Board of Directors.

I greatlyappreciate the SCBIO boardspassion forservingand advancing South Carolinas rapidly growing life sciences community and industry sector, and for their vote of confidence in my leadership capabilities for the organization going forward, Konduros said in a statement.

Architecture and design firm adds new hire to Greenville office

Verizon looks to fill 100 positions at new telesales center in Greenville

Konduros, a business leader and biomedical and economic development consultant, is the founder of SK Strategies LLC, launched in 2004, and has led a number of state economic development efforts.

He was the founding president and CEO of theUpstate S.C. Alliance, and is a former chairman for the Greenville Chamber of Commerce and a former committee member of the S.C. Chamber of Commerce.

Konduros has a law degree from the University of South Carolina and an undergraduate degree from Clemson University. He is alsoa graduate of the Economic Development Institute at the University of Oklahoma.

Sams strengths in knowing the biotech sector and his deep experience in business and economic development were compelling, Erin Ford, chair of SCBIO, said in a statement. The board was won over by his vision for the growth of SCBIO.

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Sam Konduros named president and CEO of SC Biotechnology Industry Organization - Greenville News

More than 54 million adults in the United States -- or about one in four -- have arthritis, and almost 60 percent of these individuals are of working age (i.e., ages 18-64). What's worse is that more than 24 million adults with arthritis have activity limitations from their disease, with the percentage of these patients having grown from about 36 percent in 2002 to about 43 percent in 2014.

That's according to a CDC Morbidity and Mortality Weekly Report(www.cdc.gov) (MMWR) and an accompanying Vital Signs report(www.cdc.gov) released March 7. The reports were based on data from the CDC's 2013-2015 National Health Interview Survey (NHIS).

Physical limitations these adults experience may be as simple as having difficulty holding a cup, lifting a grocery bag or walking to their car -- but they still hinder their ability to pursue everyday activities.

"Arthritis symptoms keep millions of Americans from going about their daily routines," said CDC Acting Director Anne Schuchat, M.D., in a news release.(www.cdc.gov) "Doctors and loved ones can help people with arthritis by encouraging them to be as physically active as they can be. Physical activity is a proven strategy to ease pain and reduce symptoms among people with arthritis."

The CDC found that when patients with arthritis engage in physical activity, they can reduce their arthritis symptoms by as much as 40 percent. However, many adults with arthritis aren't physically active, with about one-third reporting they don't engage in physical activity during leisure time.

The reports also noted that adults with arthritis can reduce their symptoms by participating in disease management education programs. But these programs are being underutilized, with just one in 10 patients signing up. According to the CDC, the best way to improve sign-up rates is for health care professionals to recommend these programs, because adults with arthritis are significantly more likely to attend an education program when their physician suggests they do so.

As previous NHIS survey analyses have found, women, adults who were unable to work, those in fair or poor health, and those with obesity, heart disease or diabetes had higher age-adjusted prevalences of both arthritis and arthritis-attributable activity limitations.

Although the prevalence of arthritis among adults who were unemployed was similar to that among employed adults, unemployed respondents had a much higher prevalence of arthritis-attributable activity limitations. According to the CDC, this finding suggests that disease-attributable activity limitations may play a role in these people's unemployment.

Also as in past analyses, arthritis prevalence was similar among blacks and whites, but the prevalence of arthritis-attributable activity limitations was higher among blacks. Findings in Hispanics were mixed, with a much lower prevalence of arthritis but a proportionately higher prevalence of arthritis-attributable activity limitations.

"Our findings suggest that the burden of arthritis is increasing and requires more widespread use of existing, underused evidence-based interventions," the MMWR report concluded. "Physical activity is a proven strategy for managing arthritis, with known benefits for the management of many other chronic conditions."

The report went on to say that efforts to improve the health of adults with arthritis, including those with comorbid chronic conditions, should include wider dissemination and implementation of evidence-based interventions.

"These interventions meet the unique needs of adults with arthritis and have been found to reduce pain and improve function, mood and confidence to manage health and quality of life," the report said.

Because the CDC views arthritis as a large and growing clinical and public health problem, the agency is funding arthritis programs in 12 states this year to disseminate community-based arthritis-appropriate evidence-based physical activity and self-management education interventions.

"Its extremely important for primary care providers to encourage their patients with arthritis to be physically active," said CDC epidemiologist Kamil Barbour, Ph.D., in the news release. "It is just as important for them to motivate their patients to attend workshops to learn how to better manage their arthritis."

Related AAFP News Coverage New NIH Research Program Targets Health Disparity, Chronic Disease (9/7/2016)

More From AAFP American Family Physician: AFP By Topic: Arthritis and Joint Pain

Familydoctor.org: Rheumatoid Arthritis(familydoctor.org)

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Arthritis Limits Daily Activities of 24 Million U.S. Adults - AAFP News

If your dog could talk, being his best friend would be a lot easier. However, he cant tell you that his knees stiffen up after he plays too long or that his hips hurt when he goes up or down stairs. Hes counting on you to recognize signs of pain and decreased mobility and to have your veterinarian diagnose and treat the condition.

Its arthritis. And it hurts.

I have it, too. Most older people do. The Pfizer Animal Health and the Arthritis Foundation have joined together the first ever partnership between a human health disease foundation and an animal health disease to explore the insights, facts and treatment solutions for both human and canine arthritis sufferers alike.

Arthritis affects over 70 million humans and 11 million dogs. It is a chronic health problem for both people and their dogs, causing pain, loss of mobility and quality of life. We know humans suffer from stiff joints, soreness and inflammation due to arthritis. Upon observation, you can see how this affects our beloved canine friends. It is up to us to help them find relief through diet, exercise and medication.

Arthritis is a painful, degenerative joint disease that affects one in five adult dogs in the U.S.

It is even more common in older dogs (my very limited experience has been that larger dogs seem to be really susceptible, too). Unfortunately, many of the cases go undiagnosed because owners attribute the subtle changes in the dogs to old age or slowing down.

Any dog can develop arthritis, and knowing the signs and symptoms of pain will help you determine when your dog may need veterinary attention. Could your dog have arthritis? Ask yourself these simple questions: Does your dog limp or appear stiff during or after activity? Is your dog slow to rise from a resting position? Does your dog lag behind or tire easily during long walks? Is your dog reluctant to climb steps or jump up?

If your answers to these questions are yes, its possible that your dog is suffering from painful arthritis. The sooner your dog is properly diagnosed and treated, the sooner he can overcome the pain. If your dog has arthritis, pain relief is possible with Rimadyl, a medication for dogs that has been proven clinically effective for the relief of pain associated with arthritis.

I dont know exactly how long Rimadyl has been on the market, but I think it has been available by prescription for a relatively short time from your veterinarian. It is a non-steroidal, anti-inflammatory medication available in caplets and in easy-to-give chewable tablets that dogs love (all dogs, except mine). Signs of Rimadyl intolerance may include loss of appetite, vomiting and diarrhea, which could indicate side effects involving the digestive tract, liver or kidneys. This sounds horrible and even worse than the illness itself. Some of these reported side effects are really rare at less than 1 percent.

Please dont let your dog suffer with the pain of arthritis.

For the animals, thank you for caring.

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It's arthritis and it hurts - Buffalo Bulletin

fox6now.com

Canine cannabis: Dogs with anxiety, arthritis and more benefiting from products derived from hemp plant fox6now.com MILWAUKEE Pet owners across the country are going crazy over a hemp product they claim helps improve the health of their dogs and cats. Veterinarians are now jumping on board and recommending it to clients instead of animal pharmaceuticals.

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Canine cannabis: Dogs with anxiety, arthritis and more benefiting from products derived from hemp plant - fox6now.com

Scientists have long known that there is a link between schizophrenia and rheumatoid arthritis. People who suffer from schizophrenia tend not to develop rheumatoid arthritis, and people with rheumatoid arthritis are at low risk for schizophrenia.

Though this relationship has been clear for more than 50 years, nobody really knew why the link existed, according to University of Pittsburgh professor of psychiatry and human geneticsVishwajitNimgaonkar.

We thought that there might be genetic factors that might explain this phenomenon, he said.

Nimgaonkarshunch has been backed up bynew research identifying 29 genetic markersin the human genome that are associated with both diseases.

One form of the marker increases the risk for schizophrenia and another form of the marker increases the risk for rheumatoid arthritis, he said.

A genetic marker is an individual gene or a sequence of DNA.

Nimgaonkarsaid all 29 of the markers they identified were within eight genes already thought to be possible culprits for both schizophrenia and rheumatoid arthritis.

Nimgaonkarsaid he hypothesizes that there is a complex relationship between the proteins produced by these genes and the likelihood someone might develop either disease. Genes produce proteins that carry out the genetic instructions to generate particular attributes such as hair color, height or susceptibility to specific diseases.

Probably there are thousands of genes, each of small effect, that may be together causing the risk for this illness and interacting with each other to produce the risk, said Harvard Medical School psychiatry professorMatcheriKeshivan.

KeshivansaidNimgaonkarswork is consistent with the theory that schizophrenia might have some auto-immune component, similar to rheumatoid arthritis, wherein the immune system attacks healthy joint cells.

Rheumatoid arthritis is an autoimmune disease in which the bodys immune system attacks healthy joint cells, creating inflammation, leading to pain and swelling. About 1.5 million Americans suffer from rheumatoid arthritis, which can cause permanent joint damage and disfiguring if not treated early, according to theArthritis Foundation.

Keshivansaid that in patients with schizophrenia, genes might overproduce proteins that are meant to clean up neurons in the brain, a process known as synaptic pruning, which can lead to healthy neurons being damaged.

Keshivansaid hes hopeful that better understanding of the genetic causes of schizophrenia can lead to new treatments and added that the disease is likely caused by genetic factors.

But at the same time, we do not have a very good idea as to which particular genes might be causing this disease, Keshivan said.

According to theSchizophrenia and Related Disorders Alliance of America, orSARDAA, about 3.5 million Americans and 1.1 percent of the worlds population are affected by the disease, which is characterized by delusions, hallucinations and disorganized speech and behavior.

Keshivansaid other symptoms of the disease include a lack of motivation and impaired cognition, which he called negative symptoms. He said, while treatments currently available are reasonably effective at treating the delusions and hallucinations, treating the negative symptoms is much more difficult. He said hes hopeful that understanding more about the genetic causes of the disease can lead to more holistic therapies.

SARDAAreports that about 75 percent of patients reach full or partial recovery, while a quarter do not see improvements with existing therapies, which include medication and psychotherapy.

While there are many questions left unanswered,Nimgaonkarsaid the findings represent a good starting point for further research. Because the analysis depended completely on existing datasets, he said laboratory experiments will help confirm and clarify his teams results.

What we are trying to do is to reach out to our colleagues inrheumatology, and what wed like to do is test these theories using actual tissues from patients: brain tissue, synovial (joint) tissues, or blood cells, he said.

The findings are published in the journalnpjSchizophrenia.

Photo Credit: Timothy K. Hamilton

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Pitt Study Sheds Light On Genetic Link Between Schizophrenia And Arthritis - 90.5 WESA

March 14, 2017

An international research team, led by scientists at University of California San Diego School of Medicine, has created the first cellular model of anorexia nervosa (AN), reprogramming induced pluripotent stem cells (iPSCs) derived from adolescent females with the eating disorder.

Writing in the March 14th issue of Translational Psychiatry, the scientists said the resulting AN neuronsthe disease in a dishrevealed a novel gene that appears to contribute to AN pathophysiology, buttressing the idea that AN has a strong genetic factor. The proof-of-concept approach, they said, provides a new tool to investigate the elusive and largely unknown molecular and cellular mechanisms underlying the disease.

"Anorexia is a very complicated, multifactorial neurodevelopmental disorder," said Alysson Muotri, PhD, professor in the UC San Diego School of Medicine departments of Pediatrics and Cellular and Molecular Medicine, director of the UC San Diego Stem Cell Program and a member of the Sanford Consortium for Regenerative Medicine. "It has proved to be a very difficult disease to study, let alone treat. We don't actually have good experimental models for eating disorders. In fact, there are no treatments to reverse AN symptoms."

Primarily affecting young female adolescents between ages 15 and 19, AN is characterized by distorted body image and self-imposed food restriction to the point of emaciation or death. It has the highest mortality rate among psychiatric conditions. For females between 15 and 24 years old who suffer from AN, the mortality rate associated with the illness is 12 times higher than the death rate of all other causes of death.

Though often viewed as a non-biological disorder, new research suggests 50 to 75 percent of risk for AN may be heritable; with predisposition driven primarily by genetics and not, as sometimes presumed, by vanity, poor parenting or factors related to specific groups of individuals.

But little is actually known about the molecular, cellular or genetic elements or genesis of AN. In their study, Muotri and colleagues at UC San Diego and in Brazil, Australia and Thailand, took skin cells from four females with AN and four healthy controls, generated iPSCs (stem cells with the ability to become many types of cells) from these cells and induce these iPSCs to become neurons.

(Previously, Muotri and colleagues had created stem cell-derived neuronal models of autism and Williams syndrome, a rare genetic neurological condition.)

Then they performed unbiased comprehensive whole transcriptome and pathway analyses to determine not just which genes were being expressed or activated in AN neurons, but which genes or transcripts (bits of RNA used in cellular messaging) might be associated with causing or advancing the disease process.

No predicted differences in neurotransmitter levels were observed, the researchers said, but they did note disruption in the Tachykinin receptor 1 (TACR1) gene. Tachykinins are neuropeptides or proteins expressed throughout the nervous and immune systems, where they participate in many cellular and physiological processes and have been linked to multiple diseases, including chronic inflammation, cancer, infection and affective and addictive disorders.

The scientists posit that disruption of the tachykinin system may contribute to AN before other phenotypes or observed characteristics become obvious, but said further studies employing larger patient cohorts are necessary.

"But more to the point, this work helps make that possible," said Muotri. "It's a novel technological advance in the field of eating disorders, which impacts millions of people. These findings transform our ability to study how genetic variations alter brain molecular pathways and cellular networks to change risk of ANand perhaps our ability to create new therapies."

Explore further: Stem cell-derived 'mini-brains' reveal potential drug treatment for rare disorder

More information: P D Negraes et al, Modeling anorexia nervosa: transcriptional insights from human iPSC-derived neurons, Translational Psychiatry (2017). DOI: 10.1038/tp.2017.37

Using "mini-brains" built with induced pluripotent stem cells derived from patients with a rare, but devastating, neurological disorder, researchers at University of California, San Diego School of Medicine say they have ...

Researchers at University of California, San Diego School of Medicine and Rady Children's Hospital-San Diego have created the first stem cell-derived in vitro cellular model of a rare, but devastating, neurodegenerative condition ...

With the help of mouse models, induced pluripotent stem cells (iPSCs) and the "tooth fairy," researchers at the University of California, San Diego School of Medicine have implicated a new gene in idiopathic or non-syndromic ...

In a study spanning molecular genetics, stem cells and the sciences of both brain and behavior, researchers at University of California San Diego, with colleagues at the Salk Institute of Biological Studies and elsewhere, ...

An international team of scientists led by researchers at the University of California, San Diego School of Medicine have used induced pluripotent stem cells (iPSCs) derived from patients with amyotrophic lateral sclerosis ...

Researchers at Karolinska Institutet collaborating in the large-scale Karolinska Schizophrenia Project are taking an integrative approach to unravel the disease mechanisms of schizophrenia. In the very first results now presented ...

To combat the effects of a poor diet, probiotics may be just the thing. However, surprising new research from UNSW suggests probiotics are much less effective when taken alongside a balanced diet, and could even impair certain ...

In a small-scale study of women with previously diagnosed mood disorders, Johns Hopkins researchers report that lower levels of the hormone allopregnanolone in the second trimester of pregnancy were associated with an increased ...

An international research team, led by scientists at University of California San Diego School of Medicine, has created the first cellular model of anorexia nervosa (AN), reprogramming induced pluripotent stem cells (iPSCs) ...

A dietary supplement kit, created to counter mood-altering brain changes linked to depression, virtually eliminated the "baby blues" among women in a new study at Toronto's Centre for Addiction and Mental Health (CAMH).

A study has shown thatcontrary to popular beliefolder people make riskier decisions than younger adults. Older people's generally more positive emotions make them more optimistic when gauging risks. In addition, older ...

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Researchers create model of anorexia nervosa using stem cells - Medical Xpress

IANS | New Delhi March 14, 2017 Last Updated at 22:14 IST

Stem cell treatment, which is effective in several health conditions, including spinal problems, can also help cure extreme blood platelet problems such as thrombocytopenia, say doctors.

The medical procedure has advantage over available treatments for blood platelet problems such as corticosteroids, blood or platelet transfusions and immunoglobulins medicines.

In a case study published in International Archives of Medicne, a 25-year-old man was treated for thrombocytopenia through stem cell therapy at a city based hospital. His laboratory examinations showed that his platelet count was 0.70 m3.

The patient underwent stem cell therapy wherein he was injected with 1 mL stem cells daily through an intravenous route.

"The patient was monitored regularly for the occurrence of any reactions during the whole therapy. Platelet count increased to 1.01m3 following the treatment and there were remarkable improvements in other symptoms," said Geeta Shroff, Stem Cell Specialist, Director, Nutech Mediworld.

Shroff has also conducted successful research on patients with spinal problems, anterior cruciate ligament tear and curing them through stem cell treatment.

According to Shroff, thrombocytopenia is defined as the reduction in blood platelet count below the normal platelet count distribution (1.5m3). It is the second most common hematological disorder after anaemia and equally affects both men and women.

The decrease in the platelet number increases the bleeding and blood loss; and when coupled with other clotting disorders can lead to serious morbidity or death.

"The proliferation and differentiating ability of stem cells has made this therapy an attractive therapeutic option. Stem cell therapy are being explored as regenerative medicine for treating various diseases due to their potential to multiply, proliferate and differentiate into any cell type.

At the injured site, stem cell produce different trophic factors and reduce the cell loss, promote host regeneration, hence, restore the function," said Shroff.

--IANS

rup/lok/dg

(This story has not been edited by Business Standard staff and is auto-generated from a syndicated feed.)

Stem cell treatment, which is effective in several health conditions, including spinal problems, can also help cure extreme blood platelet problems such as thrombocytopenia, say doctors.

The medical procedure has advantage over available treatments for blood platelet problems such as corticosteroids, blood or platelet transfusions and immunoglobulins medicines.

In a case study published in International Archives of Medicne, a 25-year-old man was treated for thrombocytopenia through stem cell therapy at a city based hospital. His laboratory examinations showed that his platelet count was 0.70 m3.

The patient underwent stem cell therapy wherein he was injected with 1 mL stem cells daily through an intravenous route.

"The patient was monitored regularly for the occurrence of any reactions during the whole therapy. Platelet count increased to 1.01m3 following the treatment and there were remarkable improvements in other symptoms," said Geeta Shroff, Stem Cell Specialist, Director, Nutech Mediworld.

Shroff has also conducted successful research on patients with spinal problems, anterior cruciate ligament tear and curing them through stem cell treatment.

According to Shroff, thrombocytopenia is defined as the reduction in blood platelet count below the normal platelet count distribution (1.5m3). It is the second most common hematological disorder after anaemia and equally affects both men and women.

The decrease in the platelet number increases the bleeding and blood loss; and when coupled with other clotting disorders can lead to serious morbidity or death.

"The proliferation and differentiating ability of stem cells has made this therapy an attractive therapeutic option. Stem cell therapy are being explored as regenerative medicine for treating various diseases due to their potential to multiply, proliferate and differentiate into any cell type.

At the injured site, stem cell produce different trophic factors and reduce the cell loss, promote host regeneration, hence, restore the function," said Shroff.

--IANS

rup/lok/dg

(This story has not been edited by Business Standard staff and is auto-generated from a syndicated feed.)

IANS

http://bsmedia.business-standard.com/_media/bs/wap/images/bs_logo_amp.png 177 22

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'Stem cell treatment can cure blood platelet problems' - Business Standard

Monday, March 13, 2017 3 a.m. CDT

BRUSSELS (Reuters) - Belgian biotech group Tigenix said on Monday its medical trial with a novel treatment for patients at risk of heart failure after a coronary attack was successful.

The group said patients treated in its PhaseI/II trial of donor-derived expanded cardiac stem cells (AlloCSC) showed no side-effects and all of them continued to live after 30 days, six months and a year.

Tigenix added that in one subgroup of trial patients associated with a poor long-term outlook, there was a larger reduction in the size of infarction, tissue death due to inadequate blood supply.

"This is the first trial in which it has been demonstrated that allogeneic cardiac stem cells can be transplanted safely through the coronary tree," one of the doctors in the trial said.

The group said it would now analyze the data from the trial and decide on how to proceed with its research.

(Reporting by Robert-Jan Bartunek; editing by Philip Blenkinsop)

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Belgium's Tigenix says heart attack stem cell trial successful - KFGO

DECATUR A 24-year-old Decatur man is being held in jail on $50,000 bond after he was arrested for allegedly kicking a woman in the face repeatedly, sending her to the hospital for treatment of multiple facial fractures and possible blindness in one eye.

Casio Devine, allegedly was at a residence in the 800 block of East Rogers Avenue between 3 and 4 a.m. Sunday, when he became upset about social media posts his female acquaintance had made.

Casio punched her several times in the face with a closed fist, the victim told police.

After she fell to the floor, Casio then kicked her approximately eight times in the head, said a probable cause affidavit by Decatur patrol officer Joseph Kish.

A young child told police she saw the suspect punch and kick the victim several times. An adult relative of the victim said Casio told her he wished he would have killed the victim.

An emergency room physician at Decatur Memorial Hospital told police the victim may lose the use of her right eye. He said he was going to transfer her to another hospital for specialized treatment.

Kish observed that the victim's right eye was bruised and swollen shut. The swelling was about the size of a golf ball. Blood was coming out from her swollen eye. The victim also suffered bruises and abrasions on her back.

Devine was arrested about 11 a.m. Sunday and booked into the jail on a preliminary charge of aggravated domestic battery. He is due in court for his arraignment by Monday.

Devine has three prior criminal convictions, including a 2010 conviction for manufacture/delivery of cocaine, for which he was sentenced to six years in the Illinois Department of Corrections.

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Man faces battery charge; attack may cause partial blindness - Herald & Review

The finding raises the possibility that human retinas can be induced to regenerate, naturally repairing damage caused by degenerative retinal diseases and injury, researchers say.

Scientists have identified a chemical signal in the zebrafish brain that helps it regenerate retina, a finding that may help cure blindness in humans.

The discovery raises the possibility that human retinas can be induced to regenerate, naturally repairing damage caused by degenerative retinal diseases and injury, including age-related macular degeneration and retinitis pigmentosa, researchers said.

The prevailing belief has been that the regeneration process in fish retinas is triggered by secreted growth factors, but our results indicate that the neurotransmitter GABA might initiate the process instead, said James Patton, professor at Vanderbilt University in the US.

All the regeneration models assume that a retina must be seriously damaged before regeneration takes place, but our studies indicate that GABA can induce this process even in undamaged retinas, said Patton.

The discovery raises the possibility that human retinas can be induced to regenerate. (Shutterstock)

It turns out that the structure of the retinas of fish and mammals are basically the same.

Although the retina is very thin less than 0.5 millimetres thick it contains three layers of nerve cells: photoreceptors that detect the light, horizontal cells that integrate the signals from the photoreceptors and ganglion cells that receive the visual information and route it to the brain.

In addition, the retina contains a special type of adult stem cell, called Muller glia, that span all three layers and provide mechanical support and electrical insulation. In fish retinas, they also play a key role in regeneration.

When regeneration is triggered, the Muller glia dedifferentiate, begin proliferating, and then differentiate into replacements for the damaged nerve cells. Muller glia are also present in mammalian retinas, but do not regenerate.

Graduate student Mahesh Rao got the idea that GABA normally a fast-acting neurotransmitter best known for its role of calming nervous activity by inhibiting nerve transmission in the brain might be the trigger for retinal regeneration. He was inspired by the results of a study in the mouse hippocampus which found that GABA was controlling stem cell activity.

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Working with Patton and assistant professor Dominic Didiano, Rao designed a series of experiments with zebrafish which determined that high concentrations of GABA in the retina keep the Miller glia quiescent and that they begin dedifferentiating and proliferating when GABA concentrations drop.

They tested their hypothesis in two ways: By blinding zebrafish and injecting them with drugs that stimulate GABA production and by injecting normal zebrafish with an enzyme that lowers the GABA levels in their eyes.

The research was published in the journal Stem Cell Reports.

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Fish eyes may hold key to cure blindness in humans, finds study - Hindustan Times

Many intriguing articles have been written about investing in biotechnology. Biotechnology investment has been referenced by many knowledgeable and respectable authors as controversial, out of favor, and even sexy (this article by Stephen Simpson, CFA, is must read). Surviving current trends in biotechnology stock price manipulation can be both stressful and disappointing. This leaves us all to wonder is it even worth it to try speculative biotechnology as an investment option?

StrongBio believes it is worth it, even if it results in losses that are hard to endure. In the end, contributions to healthcare from growing sources of capital are extremely important for improved patient care (termed supply-side capital). As these contributions have grown, however, so too has waste. With proper selection, timing, and diversification (three pillars of biotechnology investment), the common retail investor can eventually be financially rewarded as philanthropic goals of the population are met.

Simply put, biotechnology companies focus on drug development aiming to treat an unmet or under-met disease or medical condition. Companies that have succeeded have net sales in the tens of billions and total market values in the hundreds of billions of dollars. Speculative biotechnology companies, in contrast, differ from proven top biotechnology companies in that they often have no approved products or revenues.

Gary Pisano of Harvard Business School has done extensive modeling of biotechnology (and other technology) returns. Reports between 2006 and 2012 indicate that average biotechnology returns have been historically unimpressive; with 25-year returns of "market baskets" of biotech stocks yielding only about 10% per year. This means that much of the legendary "opportunity" in biotechnology stocks revolves around successful portfolio management of technological trend shifts and timing positions accordingly.

Much of the challenges lie in the fact that science experts tend to focus into niches instead of pursue interdisciplinary science. Scientists tend to lack fundamental economics or business expertise and vice-versa, with business leaders lacking science background.

So what if a speculative biotechnology company has shown positive data in a curative treatment for cancer? Many things can still go wrong for an investor. One should always have a plan for setbacks and delays. Sometimes clinical setbacks can occur requiring a company to delay a trial until regulatory requirements are met. Other delays are more business-oriented, with slated clinical trials held up due to lack of funding such as in poor economic times.

Even legal setbacks occur and can cost both time and money. And then we have the gatekeeper: FDA, and regulatory setbacks that can occur. The fact is most biotechnology projects fail. According to Pisano, the average biotechnology company is likely to fail 90% of the time, with companies that make it all the way to Phase 3 experiencing approximately 50% chance of success.

Multidisciplinary investment management increases the likelihood of a success, meaning, that many common retail investors are going to have to try to wear multiple hats when performing qualitative analyses. That's what StrongBio calls work. It's a lot of work. But knowing what to look for in each discipline can be of great service to the retail investor.

And if all of those pitfalls are not enough, going back to our legitimate cancer data success scenario, market manipulation and fake news from negative press can still make investors feel like their winners are losing investments for quite some time. Take for instance the 2016 situation with Celator Pharmaceuticals, which was driven down to $0.79 cents per share and rose 1600% when whatever market forces that were holding it down, along with negative press, finally gave up the fight to an obvious winner (having been bought out by Jazz Pharmaceuticals (NASDAQ:JAZZ).

The oppressive forces on the stock persisted right up until FDA review. Other company shareholders, like those of Northwest Biotherapeutics (NASDAQ:NWBO), allege that negative press and stock manipulation are linked. Immunomedics's (NASDAQ:IMMU) stock see-sawed back and forth several times between $2 and $5 (and even drew a halt from the SEC), market cap between 180 million and 500 million respectfully, in 2016 with alternating negative legal press by no less than 20 law firms and positive research press and stock price volatility. Extreme patience is required while waiting for "fair value."

The University of Chicago oncologist Mark Ratain postulated that a company with a market cap of less than $300 million is unlikely to succeed. Commonly known in biotechnology investment circles, the Feuerstein-Ratain rule, was a solid predictor in the past. This year companies are defying the 300 million rule. StrongBio believes the rule used to hold water because there was a predictable method to the involvement of big pharma in purchasing speculative biotechnology cancer stocks.

So either big pharma is no longer able to identify useful technologies and many are slipping through the cracks, which is unlikely, or something in the markets is changing. It is also possible that it was getting predictable to pick biotechnology successes based upon the highly successful metric by the well-respected Feuerstein and Ratain, so market makers have changed it up a little. Past open market buyout periods of obtaining shares of speculative stocks drove prices up as a whole (or held them flat for long periods of time) to approximately 300 million market caps. Accumulation such as this no longer seems to be in effect.

Whatever the mechanism of value assignment by the market, it is clear there is a new market pattern emerging in biotechnology, with lower than normal market caps. StrongBio believes there may be several contributing reasons for this. One, investment levels are predicted to be the lowest that they have been since 1947. This is also true in the investment banking sector, a big source of biotechnology funding.

Simply put we have not had a great investment economy, and risky biotechnology may be regarded as an irresponsible investment during tough financial times. Because new patterns on speculative biotechnology company stocks show suppression over periods of months and years, it is possible there just is not as much retail and/or institutional support as in years past.

Two, is the SEC unconscious at the wheel or did someone outsource that job to Asia? This query addresses concern over foreign-based or hostile entities' ability to starve funding for cash hungry technology companies when they need to sell stock. In recent years, an increase in foreign companies cheaply acquiring U.S. biotechnologies developed at tax-payer-supported universities and other technologies funded by state and local governments has plagued markets.

New stock exchanges like IEX have even been set up in an attempt to thwart different kinds of financial manipulation utilizing delays in trade execution (read this book or a synopsis about it in Flash Boys; it's fascinating). However, the market is currently responding to new executive political leadership in a corrective way. One can always hope that a nation of laws will have proper enforcement.

Three, short sellers have influenced stock prices (being respectful of regulations) for a long time, but not to the extent that manipulation is occurring now. Foreign countries like those in Western Europe, Australia and Canada have entirely outlawed the practice of shorting on their own stock exchanges. This indicates these countries have identified that stocks were oversold and manipulated and regulations and laws limiting short sales were not able to control it.

It would appear pretty obvious to those following speculative biotechnology that the same is occurring in the U.S. For instance, one exploring and mining company presented evidence to the SEC of a naked short position in the hundreds of billions of shares. Regardless of mechanism there may be a way to estimate increases in short-selling using well established metrics in biotechnology.

StrongBio cites the 2016 failures (and likely more in 2017) of the long-established Feuerstein-Ratain rule as evidence NOT that the metric is somehow flawed, but rather that market conditions have changed. The 300 million "rule" is now as dated as 4-inch tile countertops for kitchens and bathrooms, and has likely been rendered obsolete by rampant stock price manipulation. But that does not mean that one should abandon biotechnology investments.

Eventually, a fair value is decided between a suitor and company management if something in the pipeline passes FDA and can be sold. Since Celator (NASDAQ:CPXX) rendered the Feuerstein-Ratain rule obsolete at a market cap of less than $100 million, and Immunomedics obtained FDA breakthrough therapy status in triple negative breast cancer at about $160 million, we know the static threshold value of 300 million is no longer even close to critical mass for the metric.

Out of fairness, the metric can be influenced by other factors such as a changing FDA landscape as well, but that wouldn't explain the difference in market cap as the FDA does not participate in stock pricing or market making per se. It follows that if market regulation returns to prior levels, the metric threshold would increase back to 300 million.

How much lower can the Feuerstein-Ratain critical threshold go? That may depend in part how many shares can be shorted in a given company. StrongBio cites here mention of a gnarly cancer drug company from a recent article by an author of the metric that has approvable Phase 3 data. According to a report by H.C. Wainright, this 40 million market cap company, CytRx (NASDAQ:CYTR), is likely to get some kind of approval pathway for aldoxorubicin on its statistically significant Phase 3 sarcoma data, where it outperformed all 5 other drugs in the study.

One might approximate that if short selling is the cause for lower market cap FDA approvals in cancer, estimates based upon how much additional share supply exists now versus when the rule was working can be made. 300 million, the past metric threshold, divided by 40 million, the current potential low, gives a WHOPPING 7.5-fold more potential share supply (provided to the market by short selling). This assumes that the relationship between stock price and shorting of shares has some kind of linear and direct relationship, such as common economic supply and demand curves.

This implies the Feuerstein-Ratain metric tool is a dynamic sliding-threshold subject to changing market factors. One might argue that a linear relationship would be less representative for a "real world" sigmoidal supply and demand model, such as that proposed by Alfred Marshall. These curves break from a linear path to form a smooth parabolic curve with sigmoid limits because of factors such as wear and tear of production equipment, transportation limits, and other practical factors in supply-side analysis. In an electronic market system of a thinly traded biotechnology stock, it is unlikely these factors are relevant.

This emerging scenario creates an even greater margin for profit if one can properly select stocks that are at a record low market cap threshold for FDA approval in cancer, sometimes called short squeeze. At some point, speculative biotechnology companies get a fair value assigned if management is honest enough to serve their fiduciary duty to shareholders. It is at the point of buyout that fair value was reached for CPXX. Fair value soon will be reached for IMMU based upon significant partnership (Seattle Genetics, SGEN) and possibly even CYTR.

StrongBio cautions the reader that CytRx has been accused of hiring stock promotion media in the past, and its pillar of honor might have been "pierced." Nonetheless it appears that after a substantial period of risk everything that was promoted is likely to be true. In addition, remember that biotechnology investment in a "market basket" of companies typically returns about 10%. StrongBio does not recommend deviating from this basket approach, but rather by changing weighting late into development and after dilutions, obtain higher than 10% returns when possible.

The primary pillar of biotechnology stock investing, selection, is obviously a critical factor, standing tall in front of the pillar of timing. How can we avoid picking a loser? The second pillar of timing comes down to choosing a company with favorable Phase 3 data as they meet with FDA in a type B meeting (where FDA reviews the data outcomes of a clinical study) and an abnormally low stock price compared to the annual market its product will serve.

These two pillars stand in synergism, as one doesn't have a favorable investment with only one solid pillar supporting a portfolio candidate under current market conditions. It is important to invest lightly at first so that lower prices do not cause harm to a portfolio. If the stock runs up just be happy you had a little.

The pitfall of regulatory hurdles is always a major concern, but there is circumstantial evidence that the FDA will be easing some burdens. Cancer drug shortages (such as existed for doxorubicin in December 2016) can be thwarted by increasing the number of suppliers and approving safer more effective derivatives. The FDA may favor competition to lower prices of potentially egregious monopolies. Cancer treatment in the hospital environment is currently trending towards increasing physician options and information as well as for patient-physician interaction as well.

For instance, some drugs may be hard on the liver and not be good for alcoholics, whereas other drugs may be rough on the heart, and be contra-indicated for heart attack sufferers. So demonstration of comparable efficacy may be acceptable if safety is improved for subsets of patients. Whether the desire for increased options will spread from cancer to other indications remains to be seen.

However it is no secret that Trump intends to "slash restraints" artificially put on drug makers by the FDA. If these trends come to fruition, StrongBio expects the chance of success of biotechnology companies to increase, but the markets for some drugs may sink into smaller niches of sales with greater total options available.

So there are certainly the same past investment risks that the FDA will not view data as favorable or that companies will have a hard time proving they can meet production standards for NDA approval, including lot to lot consistency. Oftentimes a speculative biotechnology company can partner this production with a number of firms.

But the reward to risk ratio can at least be dynamically tuned for investment success. With proper selection, timing, and diversification, StrongBio estimates that new regulatory policies and market conditions will make biotechnology investment potentially more common and successful as the outdated thresholds of the past are readjusted to guide investors.

Disclosure: I am/we are long IMMU CYTR.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Editor's Note: This article covers one or more stocks trading at less than $1 per share and/or with less than a $100 million market cap. Please be aware of the risks associated with these stocks.

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Why Should Investors Consider An Allocation In Speculative Biotechnology: A Sector Analysis - Seeking Alpha

On March 9, 2017, the National Academies of Sciences, Engineering, and Medicine (National Academies) published a report entitled Preparing for Future Products of Biotechnology, prepared by the Committee on Future Biotechnology Products and Opportunities to Enhance Capabilities of the Biotechnology Regulatory System (Committee). The Committee was asked to describe the possible future products of biotechnology that will arise over the next five to ten years, as well as provide some insights that can help shape the capabilities within the U.S. Environmental Protection Agency (EPA), the U.S. Food and Drug Administration (FDA), and the U.S. Department of Agriculture (USDA) as they move forward. According to the Committee, agencies may be overwhelmed by the number and diversity of new biotechnology products. The Committee states that the agencies should increase their scientific capabilities, tools, and expertise in key areas of expected growth. The report reflects the Committees deliberations regarding the future products of biotechnology that are likely to appear on the horizon, the challenges that the regulatory agencies might face, and the opportunities for enhancing the regulatory system to prepare for what might be coming. The Committee reached consensus on conclusions and recommendations that are based on extensive information gathering, Committee discussions, and input from a wide variety of communities interested in biotechnology. A copy of the slides used during a National Academies webinar on the report can be found on the National Academies website.

On July 2, 2015, the White House Office of Science and Technology Policy, the Office of Management and Budget, the U.S. Trade Representative, and the Council on Environmental Quality issued a memorandum, Modernizing the Regulatory System for Biotechnology Products, directing EPA, FDA, and USDA to update the Coordinated Framework for the Regulation of Biotechnology (Coordinated Framework). The Obama Administration asked the agencies to accomplish three tasks:

Clarify the current roles and responsibilities of the EPA, FDA, and USDA in the regulatory process;

Develop a long-term strategy to ensure that the federal regulatory system is equipped to assess efficiently the risks, if any, of the future products of biotechnology; and

Commission an expert analysis of the future landscape of biotechnology products.

As reported previously, on January 4, 2017, the White House announced the release of the 2017 Update to the Coordinated Framework for the Regulation of Biotechnology. The 2017 Update provides a comprehensive summary of the roles and responsibilities of EPA, FDA, and USDA with respect to regulating biotechnology products. Together with the National Strategy for Modernizing the Regulatory System for Biotechnology Products, published in September 2016, the 2017 Update offers a complete picture of a robust and flexible regulatory structure that provides appropriate oversight for all products of modern biotechnology. Within that regulatory structure, the federal agencies maintain high standards that, based on the best available science, protect health and the environment, while also establishing transparent, coordinated, predictable and efficient regulatory practices. More information is available in the White House blog item, Increasing the Transparency, Coordination, and Predictability of the Biotechnology Regulatory System.

The July 2, 2015, memorandum called for the commission of an external, independent analysis of the future landscape of biotechnology products. EPA, FDA, and USDA commissioned the National Academies to prepare an analysis to identify potential new risks and frameworks for risk assessment and areas in which the risks or lack of risks relating to the products of biotechnology are well understood. This analysis is presented in the report prepared by the Committee that was released on March 9, 2017.

The Committee was tasked to:

Describe the major advances and the potential new types of biotechnology products likely to emerge over the next five to ten years;

Describe the existing risk-analysis system for biotechnology products including, but perhaps not limited to, risk analyses developed and used by EPA, USDA, and FDA, and describe each agencys authorities as they pertain to the products of biotechnology;

Determine whether potential future products could pose different types of risks relative to existing products and organisms. Where appropriate, identify areas in which the risks or lack of risks relating to the products of biotechnology are well understood; and

Indicate what scientific capabilities, tools, and expertise may be useful to the regulatory agencies to support oversight of potential future products of biotechnology.

Human drugs and medical devices were not included in the purview of the study.

To address its statement of task, the Committee gathered information from a number of sources, and heard from over 70 speakers over the course of three in-person meetings and eight webinars. The Committee received responses to a request for information from a dozen federal agencies, and solicited statements and written comments from members for the public. According to the report, the Committee defined biotechnology products as products developed through genetic engineering or genome engineering (including products where the engineered DNA molecule is itself the product, as in an engineered molecule used as a DNA information-storage medium) or the targeted or in vitro manipulation of genetic information of organisms, including plants, animals, and microbes. The term also covers some products produced by such plants, animals, microbes, and cell-free systems or products derived from all of the above.

The Committee grouped future products into three major classes:

Open-release products: The open-release products that the Committee saw on the horizon include plants, animals, microbes, and synthetic organisms that have been engineered for deliberate release in an open environment. According to the report, the ability to sustain existence in the environment with little or no human intervention is a key change between existing products of biotechnology and some of the future ones anticipated in this class. The report states that the Committee thought that future open-release products would be developed for familiar uses, such as agricultural crops, but would also likely be developed for uses such as cleaning up contaminated sites with engineered microbes, replacing animal-derived meat with meat cultured from animal cells, and controlling invasive species through gene drives;

Contained products: The Committee concluded that future biotechnology products that are produced in contained environments are more likely to be microbial based or synthetically based rather than based on an animal or plant host. According to the report, organisms of many genera are used in fermenters to produce commodity chemicals, fuels, specialty chemicals or intermediates, enzymes, polymers, food additives, and flavors. When considering the laboratory as a contained environment, the report states that many examples of transgenic animals from vendors are widely used today for research and development. Because performing biotechnology in contained environments allows higher control over the choice of host organism, systems with advanced molecular toolboxes are already in high use; and

Platforms: Biotechnology platforms are tools that are used in the creation of other biotechnology products, according to the report, including products that are traditionally characterized as wet lab, such as DNA/RNA, enzymes, vectors, cloning kits, cells, library prep kits, and sequencing prep kits, and products that are dry lab, such as vector drawing software, computer-aided design software, primer calculation software, and informatics tools. The report states that these two categories continue to meld as newer approaches are published or commercialized.

The report notes that there are a variety of technical, economic, and social trends that drive and will continue to drive the types of biotechnology products developed in the next decade. Technical and economic trends in the biological sciences and biological engineering are accelerating the rate at which new product ideas are formulated and the number of actors who are involved in product development. The report states that with regard to social trends, it was evident to the Committee that there are many competing interests, risks, and benefits regarding future biotechnology products. According to the report, it was clear that the U.S. and international regulatory systems will need to achieve a balance among these competing aspects when considering how to manage the development and use of new biotechnology products.

The Committee found that the Coordinated Framework appears to have considerable flexibility in statutory authority to cover a wide range of biotechnology products. The jurisdictions of EPA, FDA, and USDA are defined in ways that may leave gaps or redundancies in regulatory oversight, however. According to the report, even when jurisdiction exists, the available legal authorities may not be ideally tailored to new and emerging biotechnology products. Other agencies will likely have responsibilities to regulate some future biotechnology products, and their roles are not well specified in the Coordinated Framework.

The report states that the Committee found that the complexity of the existing biotechnology regulatory system, which could appear fragmented, results in a system that is difficult for product developers -- including individuals, nontraditional organizations, and small enterprises, as well as consumers, product users, and interested members of the public to navigate. The complexity can cause uncertainty and a lack of predictability for developers of future biotechnology products and creates the potential for loss of public confidence in oversight of future biotechnology products.

According to the report, the increased rate of new product ideas means that the types and number of biotechnology products in the next five to ten years may be significantly larger than the current rate of product introduction. The report cautions EPA, FDA, USDA, and other relevant agencies to prepare for this potential increase, including finding effective means of evaluation that maintains public safety, protects the environment, and satisfies the statutory requirements appropriate for each agency. The increased number of actors involved in product development means that the regulatory agencies will need to be prepared to provide information regarding the regulatory process to groups that may have little familiarity with the Coordinated Framework.

According to the report, advances in biotechnology are leading to products that involve the transformation of less familiar host organisms, have multiple engineered pathways, are comprised of DNA from multiple organisms, or are made from entirely synthetic DNA. Such products may have few or no comparators to existing nonbiotechnology products, which function as the baseline of comparison in current regulatory risk assessments of biotechnology products.

For future biotechnology products in all degrees of complexity and novelty, the Committee considered the risk assessment endpoints related to human health or environmental outcomes, such as illness, injury, death, or loss of ecosystems function. The Committee concluded that these endpoints are not new, but the intermediate steps along the path to those endpoints may be more complex, more ambiguous, and less well characterized than those for existing biotechnology products. According to the report, the scope, scale, complexity, and tempo of biotechnology products likely to enter the regulatory system in the next five to ten years have the potential to critically stress EPA, FDA, and USDA, both in terms of capacity and expertise.

At a high level, the Committee found that there are existing frameworks, tools, and processes for risk analyses and public engagement that can be used to address the issues likely to arise in future biotechnology products in a way that balances competing issues and concerns. Given the profusion of biotechnology products that are on the horizon, however, there is a risk that the capacity of the regulatory agencies may not be able to provide efficiently the quantity and quality of risk assessments that will be needed. The report states that an important approach for dealing with the increase in the products will be the increased use of stratified approaches to regulation, where new and potentially more complex risk analysis methods will need to be developed for some products, while established risk analysis methods can be applied or modified to address products that are familiar or that require less complex risk analysis. To help articulate what capabilities, tools, and expertise might be useful to meet these objectives, the Committee created a conceptual map for decision-making aimed to assess and manage product risk, streamline regulation requirements, and increase transparency.

The Committee identified the following broad themes regarding future opportunities for enhancement of the U.S. biotechnology regulatory system:

The bioeconomy is growing rapidly and the U.S. regulatory system needs to provide a balanced approach for consideration of the many competing interests in the face of this expansion;

The profusion of biotechnology products over the next five to ten years has the potential to overwhelm the U.S. regulatory system, which may be exacerbated by a disconnect between research in regulatory science and expected uses of future biotechnology products;

Regulators will face difficult challenges as they grapple with a broad array of new types of biotechnology products -- for example, cosmetics, toys, pets, and office supplies -- that go beyond contained industrial uses and traditional environmental release (for example, Bacillus thuringiensis (Bt) or herbicide-resistant crops);

The safe use of new biotechnology products requires rigorous, predictable, and transparent risk-analysis processes whose comprehensiveness, depth, and throughput mirror the scope, scale, complexity, and tempo of future biotechnology applications; and

In addition to the conclusions and recommendations from this report, EPA, FDA, USDA, and other agencies involved in regulation of future biotechnology products would benefit from adopting recommendations made by previous National Academies committees related to future products of biotechnology that are consistent with the findings and recommendations in this report.

On the basis of its conclusions, the Committee developed a number of detailed recommendations regarding actions that can be taken to enhance the capabilities of the biotechnology regulatory system to be prepared for anticipated future products of biotechnology.

EPA, FDA, USDA, and other agencies involved in regulation of future biotechnology products should increase scientific capabilities, tools, expertise, and horizon scanning in key areas of expected growth of biotechnology, including natural, regulatory, and social sciences;

EPA, FDA, and USDA should increase their use of pilot projects to advance understanding and use of ecological risk assessments and benefit analyses for future biotechnology products that are unfamiliar and complex and to prototype new approaches for iterative risk analyses that incorporate external peer review and public participation; and

The National Science Foundation, the Department of Defense, the Department of Energy, the National Institute of Standards and Technology, and other agencies that fund biotechnology research with the potential to lead to new biotechnology products should increase their investments in regulatory science and link research and education activities to regulatory-science activities.

The report is well written and contains a significant amount of new and valuable information on the types of new biotechnology products being innovated and coming into commerce, trends of note regarding future products, and regulatory gaps and redundancies that need to be addressed. This background information is clearly presented and supports well the conclusions that are essential to understand, and the recommendations that are in urgent need of response.

That the federal agencies tasked with regulating biotechnology products need increased funding and organizational retooling to address the challenges eloquently and convincingly described in the report are truths beyond dispute. In this political climate, and under this Administration, meeting these needs will be challenging. Shareholders of all sorts in the biotechnology area -- businesses, innovators, environmental and public health activists -- are urged to weigh in and express support for the allocation of resources needed to fulfill the reports recommendations. Future generations of biotechnology products are on the line and at risk if these recommendations fall on deaf ears.

2017 Bergeson & Campbell, P.C.

Continued here:
National Academies Report Finds Future Biotechnology Products May Overwhelm Agencies - The National Law Review

March 14, 2017 7:54am NASDAQ:IBB

From Zacks: 2016 was a tough year for biotech stocks with the sector facing a lot of criticism for rising drug prices. Although shares did rally post-election in November on hopes that drug pricing would not be a key focus area under a Donald Trump presidency, the rally turned out to be short-lived following the Presidents views regarding drug pricing.

Trump made it clear that he does not like what happened to drug prices and he will bring them down.

Drug pricing was not the only issue that impacted the sector last year 2016 was also disappointing from an R&D perspective with a fewer number drugs managing to gain FDA approval. There were some high-profile pipeline failures as well. Other factors that weighed on the sector include mixed results, slower-than-expected new product launches and increasing competition.

The impact of these issues resulted in the NASDAQ Biotechnology Index declining 19.1% in 2016. However, the sector is showing signs of recovery this year with the index gaining 12.4%year-to-date (YTD). (Read: Top-Ranked ETFs That Crushed S&P 500 in the Bull Market)

Drug Pricing Will Remain a Headline Risk

With drug pricing being a populist issue, it looks like the spotlight will remain on rising drug prices in 2017 as well. According to the Jan 2017 Kaiser Health Tracking poll, affordability of prescription drugs remains at the top of the publics priority list for the President and Congress focus should be on ensuring the affordability of high-cost drugs to people who need them and taking steps to lower prescription drug prices.

President Trumps recent tweet that he is working on a new system where there will be competition in the Drug Industry and pricing for the American people will come way down will keep the biotech industry on edge. While it is clear that the government intends to address the drug pricing issue, there is no clarity on what steps will be taken.

Biosimilars Pose a New Threat

Another challenge being faced by the sector is the recent entry of biosimilar competition in the U.S. While a relatively new area, the market for biosimilars is huge and highly lucrative with several blockbuster biologics including Humira and Lantus slated to lose patent protection by 2020. Biosimilars are expected to reduce healthcare costs and provide a large number of patients with access to much needed biologic treatments. Biosimilars are also gaining acceptance across formularies. (Read: Trumps Defense Spending Plans Make These ETFs Buys Again)

Deals to Pick Pace?

Licensing agreements and deals including those with opt-in arrangements should continue being signed with immuno-oncology remaining a favorite area. Moreover, major biotech and pharma companies should gain from Trumps proposed tax plan and proposal to repatriate corporate profits held offshore at a one-time tax rate.

Given the possibility of repatriation of funds, chances are that M&A activity will pick up as the year progresses big companies with deep pockets often look to replenish and boost their pipelines as well as portfolios by acquiring companies with innovative pipelines and technology. Meanwhile, small bolt-on acquisitions will continue. (Read: Play These Stocks & ETFs If Fed Acts in March)

Companies with innovative technologies and pipelines are highly sought after. Niche disease areas like nonalcoholic steatohepatitis (NASH), immuno-oncology and multiple sclerosis are in demand. Treatments for orphan diseases are also much sought after with quite a few deals being signed in these areas.

New Products Should Gain Traction

Highly-awaited new products that gained approval over the last couple of years should contribute significantly to revenues. The FDA approved 22 new drugs in 2016 including Exondys 51 (Duchenne muscular dystrophy), Epclusa (hepatitis C virus), Ocaliva (rare, chronic liver disease), Zinbryta (multiple sclerosis), and Venclexta (chronic lymphocytic leukemia in patients with a specific chromosomal abnormality) among others. The agency also expanded the label of cancer drugs like Kyprolis and Imbruvica.

Meanwhile, so far in 2017, the FDA has approved 5 new drugs including Trulance (treatment of chronic idiopathic constipation in adults) and Parsabiv (treatment of secondary hyperparathyroidism in adult patients with chronic kidney disease undergoing dialysis).

ETFs in Focus

Highlighted below are some biotech ETFs ETFs present a low-cost and convenient way to get a diversified exposure to the sector.

iShares Nasdaq Biotechnology (IBB)

IBB, launched in Feb 2001 by BlackRock Investments LLC, tracks the Nasdaq Biotechnology Index. The fund mainly covers biotech stocks (81.6%) with pharma accounting for 10.8%, life sciences tools & services for 7.3%, Health care technology for 0.1%, Health care equipment for 0.11% and Health care supplies for 0.07%. The top 3 holdings include Amgen Inc. (9.23%), Celgene Corporation (7.72%) and Biogen Inc. (7.42%). The total assets of the fund as of Mar 7, 2017 were $8.38 billion representing 162 holdings. The funds expense ratio is 0.47% while dividend yield is 0.16%. The trading volume is roughly 1,491,728 shares per day.

SPDR S&P Biotech ETF (XBI)

XBI, launched in Jan 2006 by State Street Global Advisors, tracks the S&P Biotechnology Select Industry Index. The fund covers health care stocks only. The top 3 holdings include ARIAD Pharmaceuticals, Inc. (3.98%), Clovis Oncology, Inc. (3.76%), and ACADIA Pharmaceuticals Inc. (2.74%). The total assets of the fund as of Mar 8, 2017 were $2.9 billion representing 88 holdings. The funds expense ratio is 0.35% while dividend yield is 0.21%. The trading volume is roughly 4,427,722 shares per day.

First Trust NYSE Arca Biotech ETF (FBT)

FBT, launched in Jun 2006 by First Trust Advisors, tracks the NYSE Arca Biotechnology Index. The top 3 holdings include Kite Pharma, Inc. (4.77%), Alnylam Pharmaceuticals, Inc. (3.98%) and Nektar Therapeutics (3.77%). The total assets of the fund as of Mar 7, 2017 were $902 million representing 30 holdings. The funds expense ratio is 0.55% while dividend yield is nil. The trading volume is roughly 44,417 shares per day.

VanEck Vectors Biotech ETF (BBH)

BBH, launched in Dec 2011 by Van Eck, tracks the Market Vectors US Listed Biotech 25 Index. The fund covers health care stocks. The top 3 holdings include Amgen Inc. (12.13%), Celgene Corporation (10.73%) and Gilead Sciences Inc. (10.72%). The total assets of the fund as of Mar 8, 2017 were $702.5 million representing 26 holdings. The funds expense ratio is 0.35% while dividend yield is 0.21%. The trading volume is roughly 50,198 shares per day.

PowerShares Dynamic Biotech & Genome ETF (PBE)

PBE, launched in Jun 2005 by Invesco PowerShares, tracks the Dynamic Biotech & Genome Intellidex Index. The top 3 holdings include Incyte Corporation (5.38%), Vertex Pharmaceuticals Inc. (5.19%), and Regeneron Pharmaceuticals, Inc. (5.1%). The total assets of the fund as of Mar 8, 2017 were $245.6 million representing 31 holdings. The funds expense ratio is 0.50% while dividend yield is 0.34%. The trading volume is roughly 27,517 shares per day.

Conclusion

Although it may take a while for the dust around the drug pricing issue to settle down, pipeline success in innovative and important therapeutic areas, cost-cutting, share buybacks, new products, increased pipeline visibility and appropriate utilization of cash should help restore investor confidence in biotech stocks.

The iShares NASDAQ Biotechnology Index ETF (NASDAQ:IBB) fell $0.66 (-0.22%) in premarket trading Tuesday. Year-to-date, IBB has gained 13.28%, versus a 6.39% rise in the benchmark S&P 500 index during the same period.

IBB currently has an ETF Daily News SMART Grade of A (Strong Buy), and is ranked #2 of 36 ETFs in the Health & Biotech ETFs category.

This article is brought to you courtesy of Zacks Research.

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Have Biotech ETFs Finally Bottomed? - ETF Daily News (blog)

Millions of people around the world are living with arthritis. In the United States alone, the Centers for Disease Control puts that figure at nearly 23 million, or one in every four adults. But as common as it is, arthritis is actually just one word used to describe the joint inflammation associated with over 200 musculoskeletal conditions that affect everyone differently. This means people who seek out information about their condition online are often met with a barrage of confusing literature or conflicting pieces of advice that dont address their individual symptoms.

So British charity Arthritis Research UK and IBM Watson are trying to solve that problem with the introduction of a web and mobile-based virtual personal assistant for people in the UK, where about 10 million people are living with the condition. Created with the Watson Conversation API which allows for quick, simple building of chatbots or virtual agents across mobile devices, messaging platforms or physical robots the digital assistant draws information from the Arthritis Research UK library and specific input from the user to offer personalized, immediate advice on symptom management, treatment options and any other questions they may have.

We know that there are millions of people in the UK living with arthritis whose lives are severely limited as they struggle with unanswered questions. We want to ensure that everyone has access to information and support, whenever and wherever they need it, Arthritis Research UKs CEO Liam OToole said in a statement. Were really excited to be working with IBM Watson on this innovative new service that will enable us to have conversations with anyone seeking help, that we simply wouldnt be able to answer so quickly otherwise. Were confident that this new virtual assistant willhelp more people push back the ways arthritis limits their lives.

The tool is currently being tested by 300 people with arthritis in the UK, and it was developed over five months using Arthritis Research UKs eight decades of research and expertise as well as advice from health care professionals and IBM Watson cognitive computing experts. Additionally, more than 350 people with arthritis helped to develop, test and improve the tool, which will learn over time to develop more personalized feedback to users.

The eventual goal -- that users will simply speak to an AI-powered chatbot about their specific condition and receive highly personalized advice in return -- wont be available right away. The initial UK deployment will start out slowly, with users typing their inquiries into the site and the virtual assistant providing general information about arthritis and exercise that users can save online or print out. As use increases, the knowledge base will grow, enabling Arthritis Research UK to answer more questions such as those around diet and treatment options.

Considering the joint pain and limited dexterity in their hands many people with arthritis experience, its expected they will want an option that relieves them of the need to type. There is a plan for that. IBM Watson computing will learn from each interaction to refine the information retrieval tool for each user, and eventually, the tool will leverage the Watson cognitive voice input and output features (as well as location services) to understand questions spoken to the virtual assistant.

Arthritis Research UK developed the Watson-powered digital personal assistant themselves, providing a terrific example of how IBM's open, cloud-based Watson development platform is making cognitive computing broadly accessible to organizations and individuals worldwide, Cameron Brooks, IBM European Director for Watson in the Public Sector, said in a statement. Further, Arthritis Research UKs use of Watson APIs is a model for organizations thinking about how they might integrate cognitive computing into their services in order to positively impact the lives of people living with a serious health condition.

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UK arthritis resource charity taps IBM Watson to build virtual assistant - MobiHealthNews

Cancer is currently one of the top killers worldwide, and the number of cancer cases is only expected to rise. Although there are a number of therapies available, most of them are toxic and cause serious side effects. New research examines the impact of the natural vitamin C on cancer cell growth.

Cancer is the second leading cause of death and disease worldwide, accounting for almost 9 million deaths in 2015, according to the World Health Organization (WHO).

The global number of new cases of cancer are expected to grow by around 70 percent in the next 20 years.

In the United States, the National Cancer Institute (NCI) estimate that almost 40 percent of U.S. men and women will have developed cancer at one point during their lives.

There are various treatment options available for cancer, but they are not always effective; most of them are toxic, and they tend to have a variety of side effects.

In some more aggressive cases, the cancer does not respond to treatment, and it is believed that cancer stem-like cells are the reason why the cancer comes back and metastasizes.

New research, published in the journal Oncotarget, examines the effectiveness of three natural substances, three experimental drugs, and one clinical drug in stopping the growth of these cancer stem cells (CSCs.)

The study was conducted by researchers from the University of Salford in Manchester in the United Kingdom, and was led by Dr. Gloria Bonuccelli.

In total, the researchers measured the impact of seven substances: the clinical drug stiripentol, three experimental drugs (actinonin, FK866, and 2-DG), and three natural substances (caffeic acid phenyl ester (CAPE), silibinin, and ascorbic acid (vitamin C).)

The research focused on the bioenergetic processes of CSCs, which enable the cells to live and multiply. The study aimed to disrupt the CSCs' metabolism and ultimately prevent their growth.

Of all the substances tested, the team found that actinonin and FK866 were the most effective. However, the natural products were also found to prevent the formation of CSCs, and vitamin C was 10 times more effective than the experimental drug 2-DG.

Additionally, the study revealed that ascorbic acid works by inhibiting glycolysis - the process by which glucose is broken down within the cell's mitochondria and turned into energy for the cell's proliferation.

Dr. Michael P. Lisanti, professor of translational medicine at the University of Salford, comments on the findings:

"We have been looking at how to target cancer stem cells with a range of natural substances including silibinin (milk thistle) and CAPE, a honey-bee derivative, but by far the most exciting are the results with vitamin C. Vitamin C is cheap, natural, nontoxic and readily available so to have it as a potential weapon in the fight against cancer would be a significant step."

"This is further evidence that vitamin C and other nontoxic compounds may have a role to play in the fight against cancer," says the study's lead author.

"Our results indicate it is a promising agent for clinical trials, and as an add-on to more conventional therapies, to prevent tumor recurrence, further disease progression, and metastasis," Bonuccelli adds.

Vitamin C has been shown to be a potent, nontoxic, anticancer agent by Nobel Prize winner Linus Pauling. However, to the authors' knowledge, this is the first study providing evidence that ascorbic acid can specifically target and neutralize CSCs.

Learn how 300 oranges' worth of vitamin C can impair cancer cells.

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Vitamin C can target and kill cancer stem cells, study shows ... - Medical News Today

Xconomy National

Its a struggle that comic-book fans know well. Ordinary people, bestowed with super powers from a spider bite or gamma rays, struggle to harness their own abilities.

T cells, key soldiers of the immune system, are wrestling with new-found super powers, too. Certain T cells hunt down invasive viruses, bacteria, and the bodys own bad seedsthe early signs of cancer. But cancer has ways of evading the immune system, so researchers are engineering T cells to give them extra cancer-fighting talents. These so-called CAR-T treatments, administered experimentally in clinical trials, have helped dozens of patients with otherwise untreatable blood cancers.

Now the fields first-ever regulatory approval is looking like a strong possibility, making this a watershed year for CAR-T therapy. But the supercharged T cells remain an enigma. Experts are wrestling with the super-hero problem that threatens to limit their usefulness: How can the cells be engineered to kill cancer without running amok?

Its more important than anything else to keep learning how CAR-T cells work and how they potentially carry risk, says Amir Fathi, an assistant professor of medicine at Harvard Medical School. As director of the leukemia program at Massachusetts General Hospital in Boston, Fathi treats patients with experimental CAR-T therapies.

(CAR-T stands for chimeric antigen receptor T cells, which describes the modification that picks up the signals of tumor cellssensitive T cell spyware, if you will.)

With Kite Pharma (NASDAQ: KITE) of Santa Monica, CA, nearly ready to ask the FDA to review its CAR-T axicabtagene ciloleucel as a treatment for adults with non-Hodgkin lymphoma (NHL), the fields first-ever approval could be months away. International drug giant Novartis (NYSE: NOV) might not be far behind, with a CAR-T for pediatric patients with acute lymphoblastic leukemia (ALL).

For now, the Kite and Novartis products are meant for narrow groups of patients who have failed to improve with other therapies, including chemotherapy and bone-marrow transplant. Their doctors are thrilled to have the first generation CAR-Ts. The patients whom these trials are targeting, theyre typically highly refractory, they have no options left, says Krishna Komanduri, director of the Sylvester Cancer Center Adult Stem Cell Transplant Program at the University of Miami.

But is a last-ditch lifeline for patients with severe cases of two blood cancers the ceiling for CAR-T? Doctors, patients, and investors who have poured billions of dollars into the field have much higher hopes that the living cells will stand alone as long-term treatments, even cures, not just improvements upon current standards or temporary bridges to get patients healthy enough for bone-marrow transplantswhich are fraught with risks, too.

The risk of dying from transplants has come down under 20 percent in recent years, which is still higher than the overall death risk from CAR-T. But transplants are proven to cure cancer; CAR-T therapy has a long way to go. I think we need to see no evidence of disease [in patients] for several years, ideally three to five, before even comparing it to transplant, says Vinay Prasad, a blood cancer specialist at Oregon Health & Science University in Portland, and a critic of what he sees as a rush to useand pay forexpensive new medicines and procedures.

Ambitions also include eventual treatment of more common solid tumorsbreast, colon, and lung, for example. But the obstacles in realizing those ambitionsunderstanding CAR-Ts potentially deadly side effects, training a wide range of medical staff, and convincing healthcare insurers that the treatments are worth paying forwill not be easy to surmount. As Komanduri notes, bone marrow transplants from donors have been known for decades to cure lymphoma, but inadequate Medicare coverage has held the field back. The endgame should not be proving that a technology works in the clinical trial setting and securing FDA approval, says Komanduri, but ensuring we have a framework that maximizes access to curative treatments in a sustainable way.

SIDE EFFECTS, DEATHS, AND RELAPSES

As noted in Xconomys searchable CAR-T clinical results database, many programs have had stunning early results. More than 80 percent of leukemia patients, for example, have shown no signs of cancer a few weeks after treatment. Relapse rates climb after a few months in most studies. Lymphoma patients have had lower rates of remission, but in the Kite Pharma program that could be up for approval this year, the rates have stayed stable for six monthsa big deal in the CAR-T world.

Significant side effects are likely to limit CAR-T therapy to major treatment centers that can handle their unpredictability and sudden severity. Doctors working on CAR-T studies say theyre getting a handle on one common side effect, called cytokine release syndrome, with steroids and other drugs. But CAR-T treatments are also causing Next Page

Alex Lash is Xconomy's National Biotech Editor. He is based in San Francisco.

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Possible Cures. Mystery Deaths. Daunting Costs. Can CAR-T Be Tamed? - Xconomy

BRUSSELS Belgian biotech group Tigenix said on Monday its medical trial with a novel treatment for patients at risk of heart failure after a coronary attack was successful.

The group said patients treated in its PhaseI/II trial of donor-derived expanded cardiac stem cells (AlloCSC) showed no side-effects and all of them continued to live after 30 days, six months and a year.

Tigenix added that in one subgroup of trial patients associated with a poor long-term outlook, there was a larger reduction in the size of infarction, tissue death due to inadequate blood supply.

"This is the first trial in which it has been demonstrated that allogeneic cardiac stem cells can be transplanted safely through the coronary tree," one of the doctors in the trial said.

The group said it would now analyze the data from the trial and decide on how to proceed with its research.

(Reporting by Robert-Jan Bartunek; editing by Philip Blenkinsop)

NEW YORK The Trump administration told states on Monday that it was opening the door for them to make changes to their health insurance programs by using a section of the 2010 Affordable Care Act that gives them the flexibility to do so.

Local transmission of the Zika virus in Florida may have occurred as early as June 15 of last year and likely infected people who lived not only in Miami-Dade County, but in two nearby counties, U.S. health officials said on Monday.

(Reuters Health) - - U.S. patients are more likely to experience gaps in coordination among healthcare providers than their counterparts in other high-income nations, a new study suggests.

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Belgium's Tigenix says heart attack stem cell trial successful - Reuters

LIMA He has been a familiar voice for radio listeners in Delphos and on radio stations for Lima-based Childers Media Group, and on Monday, Bob Ulm brought his unique perspective to the Lima Rotary Club, a perspective grown not only out of more than four decades in local radio, but also developed while being blind nearly his entire life.

With his 4-year-old black Labrador Retriever guide dog Pippa by his side, Ulm, 61, related to those in attendance at the Veterans Memorial Civic and Convention Center what life has been like for him since losing his eyes at a year old from retinal blastoma. For him, life has not been any less fulfilling despite not having his vision.

I think I have found ways to experience all the joys that people in life could have, he said. Rather than seeing things, I just hear them, or feel them, if you will.

Ulm credited his parents and his experiences as a student at Delphos St. Johns with helping him ensure that being blind did not become an excuse for not reaching his potential.

I was extremely blessed, and many people are not, with parents who demanded a great deal of me, he said. As I said in my presentation, they loved me when I needed it, spanked me when I needed it, treated me like any other kid and taught me the importance of not feeling sorry for myself because its entirely nonproductive.

During his presentation, Ulm related how technology has helped him become an effective news director for Childers Media Group, able to use computers to work with email and assemble news reports all without looking at a screen or using a mouse. Ulm also said technology will have to be embraced by those with disabilities such as blindness if they want to contribute to society.

Of the estimated 4 million people in the U.S. who are either totally blind, like me, or partially blind, the latest estimates show that between 60 and 70 percent of those folks are not able to find work, he said. Many of them who want to find work are unable to find employment in the job market today, and that simply means that those people are not able to become contributing members of society, to support their families and themselves, as Ive been fortunate to do, and thats really unfortunate.

Childers Media Group news director Bob Ulm, with his 4-year-old guide dog, Pippa, gave a presentation to the Lima Rotary Club on Monday addressing his years in radio and how his blindness has not decreased his quality of life.

http://limaohio.com/wp-content/uploads/2017/03/web1_LimaRotary.jpgChilders Media Group news director Bob Ulm, with his 4-year-old guide dog, Pippa, gave a presentation to the Lima Rotary Club on Monday addressing his years in radio and how his blindness has not decreased his quality of life. Craig Kelly | The Lima News

Blindness doesnt stop Ulm

Reach Craig Kelly at 567-242-0390 or on Twitter @Lima_CKelly.

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Blindness not slowing Ulm down - Lima Ohio