Category Archives: Treatment News

Cord Blood Stem Cell Treatment for Cerebral Palsy

Stem Cell treatment comes in many types.  Its been around now for several years.   When our daughter was born 11 years ago, we were just starting to hear about it.  In these last 11 years, clinics in places like China, Mexico, etc. have been offering stem cell treatments.  To our knowledge, no miracle cures have happened yet.   In fact, injecting anything into a child is dangerous business and some serious complications and even deaths have been reported.  China’s federal government has announced an official hold on stem cell treatments for cerebral palsy for fear of losing control over the many small clinics that have started to offer the treatment, however, my understanding is that the the network of Army hospitals operate independently and have continued to offer treatment.  Again, from what we have seen, there have been no miracle cures.

This brings us to the USA and two clinical trails being done on Cord Blood.  Here is a video that is quite interesting.

Cord Blood Stem Cell Treatment for Cerebral Palsy in Clinical Trial

The Medical College of Georgia and Dr. Joanne Kurtzberg at Duke University have begun FDA approved clinical trails using IV infusions of stem cells from a child’s own umbilical cord (autologous).

The Duke study is funded with a $10.2 million donation from the Robertson Foundation.

The mechanism that is at work is unclear, however Dr. Kurtzberg hypothesizes that cord blood cells can reduce inflammation in the brain, produce hormones to repair brain cells or possibly transform themselves into healthy brain cells to replace the damaged ones.

James Carroll, the head researcher for the clinical trials at MCG, has had success with treating oxygen related brain damage in animal tests. Rats with induced CP-like conditions were shown to have improved motor skills after multipotent adult progenitor stem cells were introduced.

In MCG’s trial, children 2 to 12 years old will be given IV infusions (half will receive cord blood stem cells, half placebo) and then examined three months later. At that point, the placeboed half of the patients will receive cord blood stem cells. Examinations will continue three and six months later.  The Duke study focuses on children 1 to 6 years old.

Outside of the trail, it is said Dr. Kurtzberg has already treated about 150 patients with positive results.   BRIGHT hopes to contact Dr. Kurtzberg or any of the parents who’s children have received these treatments.

Cerebral Palsy which affects 2-3 children per thousand in the US.  The Centers for Disease Control estimated in 2003 that the cost of caring for someone with CP over a lifetime is $921,000.

Link
 This is further justification for the use of music as a tool for treatment within the Snowdrop programme,both generally and using such tools as ‘The Listening Programme’ of which Snowdrop is a provider. With thanks to MNT
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Researchers have long debated whether or not language and music depend on common processes in the mind. Now, researchers at Georgetown University Medical Center have found evidence that the processing of music and language do indeed depend on some of the same brain systems.Their findings, which are currently available on-line and will be published later this year in the journal NeuroImage, are the first to suggest that two different aspects of both music and language depend on the same two memory systems in the brain. One brain system, based in the temporal lobes, helps humans memorize information in both language and music — for example, words and meanings in language and familiar melodies in music. The other system, based in the frontal lobes, helps us unconsciously learn and use the rules that underlie both language and music, such as the rules of syntax in sentences, and the rules of harmony in music.

“Up until now, researchers had found that the processing of rules relies on an overlapping set of frontal lobe structures in music and language. However, in addition to rules, both language and music crucially require the memorization of arbitrary information such as words and melodies,” says the study’s principal investigator, Michael Ullman, Ph.D., professor of neuroscience, psychology, neurology and linguistics.

“This study not only confirms that one set of brain structures underlies rules in both language and music, but also suggests, for the first time, that a different brain system underlies memorized information in both domains,” Ullman says. “So language and music both depend on two different brain systems, each for the same type of thing — rules in one case, and arbitrary information in the other.”

Robbin Miranda, Ph.D., currently a post-doctoral researcher in the Department of Neuroscience, carried out this research with Ullman for her graduate dissertation at Georgetown. They enrolled 64 adults. They used a technique called Event-Related Potentials, in which they measured the brain’s electrical activity using electrodes placed on the scalp.

The subjects listened to 180 snippets of melodies. Half of the melodies were segments from tunes that most participants would know, such as “Three Blind Mice” and “Twinkle, Twinkle Little Star.” The other half included novel tunes composed by Miranda. Three versions of each well-known and novel melody were created: melodies containing an in-key deviant note (which could only be detected if the melody was familiar, and therefore memorized); melodies that contained an out-of-key deviant note (which violated rules of harmony); and the original (control) melodies.

For listeners familiar with a melody, an in-key deviant note violated the listener’s memory of the melody — the song sounded musically “correct” and didn’t violate any rules of music, but it was different than what the listener had previously memorized. In contrast, in-key “deviant” notes in novel melodies did not violate memory (or rules) because the listeners did not know the tune.

Out-of-key deviant notes constituted violations of musical rules in both well-known and novel melodies. Additionally, out-of-key deviant notes violated memory in well-known melodies.

Miranda and Ullman examined the brain waves of the participants who listened to melodies in the different conditions, and found that violations of rules and memory in music corresponded to the two patterns of brain waves seen in previous studies of rule and memory violations in language. That is, in-key violations of familiar (but not novel) melodies led to a brain-wave pattern similar to one called an “N400” that has previously been found with violations of words (such as, “I’ll have my coffee with milk and concrete”). Out-of-key violations of both familiar and novel melodies led to a brain-wave pattern over frontal lobe electrodes similar to patterns previously found for violations of rules in both language and music. Finally, out-of-key violations of familiar melodies also led to an N400-like pattern of brain activity, as expected because these are violations of memory as well as rules.

“This tells us that these two aspects of music, that is rules and memorized melodies, depend on two different brain systems — brain systems that also underlie rules and memorized information in language,” Ullman says. “The findings open up exciting new ways of thinking about and investigating the relationship between language and music, two fundamental human capacities.”

For more information: http://networkedblogs.com/yv0WS

Self-Motivation Key to Neural Regeneration

Some extremely important work is being done by Grégoire Courtine, a neuroscientist at the Swiss Federal Institute of Technology Lausanne.

His group has achieved the very important milestone of being able to rehabilitate a rat to walk again after causing hind leg paralysis by near complete severing of the spine.

This type of injury is common in Spinal Cord Injury and results in very weak signals being transmitted with not enough bandwidth to create any meaningful motor movement.

Next Dr. Courtine inject drugs that mimic the normal chemical signals from the brain into the damaged spinal cord.   They also insert two electrodes and stimulate across the injury site.

Animals left lying idle during recovery showed improved nerve growth, but did not regain function.

Animals put on a treadmill with assisted leg movements showed some improvement, but nothing that would be considered a breakthrough.

However, when a third element was added, a goal of food, then the rats not only responded, but they learned to walk again.  Some animals even regained the ability to run and climb.

This is an extraordinary result that has not been seen before.   The success appears to have been achieved by three factors.

  • Improved chemical environment for neural signaling using drugs and electric stim.
  • The use of assisted motor training that used a robotic harness to remove balance and weight issues and allowed the animal to focus on forward movement.
  • The final and most important factor seems to be the introduction of a goal and positive achievement of that goal.  In this case, running after some food and finally after 30 minute work-outs achieving that goal multiple times.

Examples the concept of “success” based motor learning is seen over and over.  One example would be the reliance on the “good arm” of stroke patients and consequently the loss of motor learning in the “bad arm”.

The work of Dr. Courtine could prove to be a breakthrough for Spinal Cord Injury patients and a confirms the thesis of BRIGHT that “success” based motor learning is likely a key therapeutic approach for children with Cerebral Palsy as well.

Nanoparticles help deliver drug to targeted brain cells for Acute Cerebral Palsy Treatment

There are two phases to brain injury.  The first is the phase that lasts for the first few days.  During this time, there is what is called a biochemical cascade which actually causes more healthy brain cells to die.  The second phase lasts for the rest of our lives.   So treatment also follows the two phases.  If you can treat early enough (hours after the injury) then there is a chance to prevent the further damage caused by the cascade.  Brain Cooling is one approach.  Another is anti-inflammatory drugs.  If you can’t treat in the first few hours, then treatment is focused around how to deal with the dead brain cells.  I personally believe that plasticity is the best approach to deal with dead brain cells (i.e. retraining other parts of the brain to take over for the dead parts.)

Below is a new article announcing some exciting results of a new way to deliver drugs safely to the part of the brain that needs treatment.  This has been one of the issues with brain cooling, drug treatment, etc.  You can just treat the entire brain.  You need to target the correct cells.  These nano-particles are helping to do this.

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A team of scientists from Johns Hopkins and elsewhere have developed nano-devices that successfully cross the brain-blood barrier and deliver a drug that tames brain-damaging inflammation in rabbits with cerebral palsy.

 A report on the experiments, conducted at Wayne State University in collaboration with the Perinatology Research Branch of the National Institute of Child Health and Human Development, before the lead and senior investigators moved to Johns Hopkins, is published in the April 18 issue of Science Translational Medicine.

For the study, researchers used tiny, manmade molecules laced with N-acetyl-L-cysteine (NAC), an anti-inflammatory drug used as antidote in acetaminophen poisoning. The researchers precision-targeted brain cells gone awry to halt brain injury. In doing so they improved the animals’ neurologic function and motor skills.

The new approach holds therapeutic potential for a wide variety of neurologic disorders in humans that stem from neuro-inflammation, including Alzheimer’s disease, stroke, autism and multiple sclerosis, the investigators say.

The scientists caution that the findings are a long way from human application, but that the simplicity and versatility of the drug-delivery system make it an ideal candidate for translation into clinical use.

“In crossing the blood-brain barrier and targeting the cells responsible for inflammation and brain injury, we believe we may have opened the door to new therapies for a wide-variety of neurologic disorders that stem from an inflammatory response gone haywire,” says lead investigator Sujatha Kannan, M.D., now a pediatric critical-care specialist at Johns Hopkins Children’s Center.

Cerebral palsy (CP), estimated to occur in three out of 1,000 newborns, is a lifelong, often devastating disorder caused by infection or reduced oxygen to the brain before, during or immediately after birth. Current therapies focus on assuaging symptoms and improving quality of life, but can neither reduce nor reverse neurologic damage and loss of motor function.

Neuro-inflammatory damage occurs when two types of brain cells called microglia and astrocytes — normally deployed to protect the brain during infection and inflammation — actually damage it by going into overdrive and destroying healthy brain cells along with damaged ones.

Directly treating cells in the brain has long proven difficult because of the biological and physiological systems that have evolved to protect the brain from blood-borne infections. The quest to deliver the drug to the brain also involved developing a technique to get past the brain-blood barrier, spare healthy brain cells and deliver the anti-inflammatory drug exclusively inside the rogue cells.

To do all this, the scientists used a globular, tree-like synthetic molecule, known as a dendrimer. Its size — 2,000 times smaller than a red blood cell — renders it fit for travel across the blood-brain barrier. Moreover, the dendrimer’s tree-like structure allowed scientists to attach to it molecules of an anti-inflammatory NAC. The researchers tagged the drug-laced dendrimers with fluorescent tracers to monitor their journey to the brain and injected them into rabbits with cerebral palsy six hours after birth. Another group of newborn rabbits received an injection of NAC only.

Not only did the drug-loaded dendrimers make their way inside the brain but, once there, were rapidly swallowed by the overactive astrocytes and microglia.

“These rampant inflammatory cells, in effect, gobbled up their own poison,” Kannan says.

“The dendrimers not only successfully crossed the blood-brain barrier but, perhaps more importantly, zeroed in on the very cells responsible for neuro-inflammation, releasing the therapeutic drug directly into them,” says senior investigator Rangaramanujam Kannan, Ph.D., of the Center for Nanomedicine at the Johns Hopkins Wilmer Eye Institute.

Animals treated with dendrimer-borne NAC showed marked improvement in motor control and coordination within five days after birth, nearly reaching the motor skill of healthy rabbits. By comparison, rabbits treated with dendrimer-free NAC showed minimal, if any, improvement, even at doses 10 times higher than the dendrimer-borne version. Animals treated with the dendrimer-delivered drug also showed better muscle tone and less stiffness in the hind leg muscles, both hallmarks of CP.

Brain tissue analysis revealed that rabbits treated with dendrimer-borne NAC had notably fewer “bad” microglia — the inflammatory cells responsible for brain damage — as well as markedly lower levels of other inflammation markers. They also had better preserved myelin, the protein that sheaths nerves and is stripped or damaged in CP and other neurologic disorders. And even though CP is marked by neuron death in certain brain centers, animals who received dendrimer-borne NAC had higher number of neurons in the brain regions responsible for coordination and motor control, compared with untreated animals and those treated with NAC only.

The findings suggest that the treatment not only reduces inflammation in the cells, but may also prevent cell damage and cell death, the researchers said. The Kannans, who are married, say they plan to follow some treated animals into adulthood to ensure the improvements are not temporary.

A separate study, led by Rangaramanujam Kannan, has already demonstrated the therapeutic benefits of this approach in reversing retinal damage in rats with macular degeneration, the vision-robbing eye disorder that affects millions of older adults.

Source : Johns Hopkins Medical Institutions

http://www.biologynews.net/archives/2012/04/23/nanodevices_that_cross_bloodbrain_barrier_open_door_to_treatment_of_cerebral_palsy.html

Cure not Care!

“Cure not Care”, “Cure Warriors”, “Cure Tribe”

These are just a few of the phrases coined by the Spinal Cord Injury community to express their dedication to the idea that a cure is very possible.

Charles Carson, was founder of the Spinal Cord Society – the first cure advocacy group dating to the late 1970s, and trademarked the slogan “cure not care”.

Sam Maddox writes these words: “Chuck, who died a year ago, believed that science and medicine could fix SCI with a focused effort. He used to suggest cure research would happen more quickly if scientists were given epidural nerve blocks to induce paralysis – to sharpen the sense of urgency. He also used to wonder whether progress was impeded by the rehab and caring industries, whose bottom line would suffer if people started dumping their wheelchairs. Carson used to host an annual SCS convention. Scientists meet the consumers. Even in the most incomprehensible neuro-babble, there is hope. As Carson often said, “A lot more people have died from no hope than from false hope.”

“The biggest difference now as opposed to 30 years ago: the warriors are much better informed. And they all seem to know each other, thanks to a networked world. There is more hope now, or at least there are more clinical trials. Better biology and more shoulders to the wheel. But today there’s still just as much impatience, passion, and anger as there was in Chuck’s day.”

Sam’s and Chuck’s words are so true.   BRIGHT’s members are most defiantly “Cure Warriors”.     Check the Treatments and Research Category frequently to see what is on the horizon as a possible cure to help recovery after Brain Injury.

Care not Cure

Care not Cure

60 Minutes Investigation – Stem Cell Fraud

I would like to make a comment on this 60 minutes video. First off, I am a sucker for the underdog. Although I am not defending this man, I have to say the reporting and investigation is a bit dramatic and one sided.   Chris Arnold from the SCI forum comments “Could it be that the primary aim of this production was a good dollop of shock/horror that will guarantee good viewing numbers? “

Perhaps the real story that should have been investigated is why is it that so little progress has been made toward a Cure for Brain Injury?   Where is the money that is being invested into research actually going?   Is it being spent  efficiently?   Is there accountability?  But that would not make for very dramatic reporting…

Regarding the risk of Stem Cell treatment and other non-standard treatments.  Yes, they can have huge risks.   However, here is the issue. All of medicine is filled with risk. No doctor or no amount of clinical trials can tell you if a procedure is effective or risk free for you. The issue is that when it comes to humans, the sample size is ONE. There is only one you and when you take any drug, undergo any procedure, etc. How your body reacts is going to be unique. Yes, aspirin is safe for a large part of the population; however it kills thousands of people each year! Thousands!

The July 1998 issue of The American Journal of Medicine explains it as follows: “Conservative calculations estimate that approximately 107,000 patients are hospitalized annually for nonsteroidal anti-inflammatory drug (NSAID)-related gastrointestinal (GI) complications and at least 16,500 NSAID-related deaths occur each year among arthritis patients alone.”

With regard to Stem Cells, I think the ideal approach is to allow people to make their own informed choices. To assume that all people who chose to perform a risky procedure are “tricked” or “stupid” and therefore need to be told what procedures can and can’t be used by government is utterly wrong.

Doctors routinely prescribe off-label medications. Some estimates say it accounts for 50 percent of all drug use in the USA. Getting back to the 60 minutes interrogation question “if stems cells had been clinically tested”. Off-label prescriptions are a similar offense. The FDA tests all therapies on a specific disease, and the pharmacies sell the approved drugs for that condition only. But doctors can prescribe them regardless, and this is called off-label and no one dares to call the doctor a con-man.

Again, I am not defending this particular stem cell clinic. I am not defending stem cells. What I am defending is the right of an individual to make an informed decision on their own, with-out interference from institutions that would have you believe that they are infallible, but in fact they are far from that. (As the thousands of deaths that occur annually from FDA approved drugs attest).

Another thought goes to the pace of progress in the current USA driven research model. Yes, you will argue that the pace of progress must be slow in order to ensure optimal safety and effectiveness. Sure, I understand all that. But tell that to someone whose life is slipping away from them. Tell that to a family who is watching their child suffer each day.

My thought is that there must be room for a fast track. If you want to take the slow and safe route because you feel better about that approach, that is fine. However, there should be fast track options for those who do not have the luxury to wait. In many cases, this is exactly what doctors do with off-label prescriptions. It is also what clinics operating outside the USA and offering potentially dangerous, but potentially groundbreaking treatments are offering. There needs to be a place for this fast track approach and by definition, if it’s not approved by the APA and the FDA, it should not be called a con.

People are smarter then professionals would like to admit. We need to challenge the status quo. We need to demand results based funding. We need some doctors and researchers to live each day like it was soon to be their last in order to accelerate the pace of discovery.

60 Minutes – Stem Cell Fraud

Geron Corp. says stem cell thearpy not profitable. Cancels research.

Economics, not science, thwarts embryonic stem cell therapy

Geron Corp. pulls the plug on its ambitious effort to reverse spinal damage and will focus instead on financially less risky cancer drugs.

November 21, 2011|By Eryn Brown, Los Angeles Times

For patients paralyzed by spinal cord injuries, Geron Corp.’s stem cell research was the shining hope.

The biotech firm showered scientists with millions of dollars to develop a treatment to reverse spinal damage. The therapy was the first treatment derived from embryonic stem cells to be cleared by the Food and Drug Administration for testing in humans.

But last week, Geron abruptly pulled the plug on its pioneering trial and the rest of its stem cell business, including early work on treatments for heart ailments, diabetes and other diseases. Pursuing futuristic cures through regenerative medicine was financially riskier than focusing on the company’s two cancer drugs, which were further along in development, company executives said.

People who had pinned their hopes on stem cells reacted with dismay.

“It was like someone ripped my heart out,” said Sabrina Cohen, who was paralyzed in a car accident in 1992 and runs a research foundation in Miami.

At first blush, Geron’s decision called into question the plausibility of curing diseases with embryonic stem cells. They have the ability to grow into any kind of cell in the body, and laboratories around the world have spent hundreds of millions of dollars trying to turn them into replacement parts such as insulin-secreting islet cells for people with diabetes.

Industry watchers say the science of stem cell medicine remains sound. It’s just the economics that are shaky.

Geron, based in Menlo Park, Calif., was always fighting an uphill battle, seeking funding for a venture fraught with medical and regulatory obstacles at a time when skittish investors wanted certainty. Faced with dwindling resources, Geron had a safer option and ran with it.

“Companies are looking to double down on their most promising programs,” said Dr. George Q. Daley, director of the stem cell transplantation program at Children’s Hospital Boston.

Geron made history in January 2009, when the FDA allowed it to test the safety of a stem-cell-based product known as GRNOPC1. Scientists hoped the therapy would help rebuild fragile neurons in the spinal cord that were damaged in car crashes, falls and other accidents, restoring sensation and mobility to paralyzed patients. The first of five patients to participate in the Phase 1 safety trial got an injection in October 2010.

Everything seemed to be proceeding smoothly. In May, the California Institute for Regenerative Medicine granted Geron a $25-million loan to support the trial. In October, Geron reported that four patients who had received doses of GRNOPC1 tolerated the treatment well. (Stanford announced Friday that its doctors had treated a fifth patient on Wednesday, even though the trial had been suspended.)

But Geron’s funds were running low. Rather than try to raise money in a tight market, the company decided to devote the roughly $150 million it had left to push its two cancer drugs through ongoing clinical trials.

Geron announced last Monday that it would eliminate 66 jobs — almost 40% of its workforce — and seek “partners” to take over its stem cell business. It repaid the $6.4 million it had thus far borrowed from the California institute, plus interest.

China stops all stem cell tourism

China’s instant ban on unapproved stem cell treatment will affect some US patients, and a handful of specialist medical tourism agencies who have been accessing China for treatment that is not available in the US or most European countries.

In recent years, China has attracted foreign citizens to experimental hospitals and doctors willing to treat seriously ill patients with unproven stem-cell therapies.

The Ministry of Health
announced that the country has stopped all unproven and experimental stem-cell treatment programmes and refuses to accept any new applications for approval of stem-cell treatments. The ban will initially last until the end of June 2012, but the chances of the ban being lifted quickly after then seem very slim.

China’s experimental stem-cell treatments have been used on patients with severe neurological diseases, other chronic illnesses and injuries. While some patients have said that these unproven stem cell therapy programs in China have done them good, critics argue that the benefit is purely in the mind.

The government seeks to bring under control its growing but loosely regulated industry. It realizes that an official ban will not work unless it can be enforced. A ban on organ transplants is still blatantly ignored by some hospitals. The ministry is implementing a year long campaign to halt unauthorized stem cell therapy trials.

Regulations on stem cell treatments were relatively relaxed in China compared to other nations and the country is often seen as a last hope for people suffering from serious medical problems ranging from cancer to spinal cord injuries. But there are many horror stories where unapproved untested stem cell treatment has led to death, disease and infection.

Stem cell therapies introduce new cells into damaged tissue in order to treat disease or injury. Patients, both local and from overseas, can pay tens of thousands of dollars in a last ditch attempt to restore back functions or sight. The lucrative nature of the treatment for hospitals and agencies, as many patients will pay anything asked, means that an official ban will reduce it, but may just drive it underground.

China has no specific policy for clinical trials or applications of stem cell technology. Such trials are subject to the same general regulations governing all medical practices, such as the importance of volunteers’ rights and health. Stem cell treatment and trials already approved by the State Food and Drug Administration will continue. Those that are not approved must be stopped immediately-even in mid trial.

The Ministry of Health and State Food and Drug Administration are working together to seek to control the expanding stem cell industry. Stem cells have the ability to become any type of cell in the human body, and researchers around the world are studying them to see whether they can cure disease. The potential of Chinese stem-cell research is high and the long-term aim of the government is to get stringent regulation working and seen to be working, so that it can sell research results, treatment and drugs to overseas countries.

A growing number of hospitals and clinics in large cities in China have been offering stem cell therapies for treatment of diseases ranging from cancer and Alzheimer’s to spinal cord injuries, treatments that are backed by little or no scientific evidence and which are considered at best experimental. Some of these involve large general hospitals where patients pay thousands — or even tens of thousands — of dollars for treatments that are advertised online, which attract both Chinese patients and those from overseas. According to patients, doctors and relatives of patients, patients have come away with little or no improvement and a number have died. The ministry has made it clear that health providers can no longer charge money for experimental stem cell applications under the new order.

Whether the government can enforce the ban is questionable, as many hospitals making money from stem cell treatment are affiliated with government organisations such as the army, the PLA, and the domestic police forces, each more politically powerful than either of the enforcing government departments. They were unable to enforce a 2009 order that hospitals and clinics offering advanced and experimental medical technology had to obtain approval or face closure.

Days before the Chinese ban, the United States’ Food and Drug Administration (FDA) issued a warning about unproven stem cell claims, where patients are vulnerable to unscrupulous providers of stem cell treatments that are illegal and potentially harmful. The FDA warned consumers that any stem cell treatment they are considering must be approved by FDA or studied under a clinical investigation that has been submitted to and allowed to proceed by FDA. The FDA has recently participated in activity to fight illegal activity involving stem cells. In December 2011, three men were arrested and charged with 15 counts of criminal activity related to manufacturing, selling and using stem cells without FDA sanction or approval. One of these was a consultant at a university in South Carolina who used university facilities to manufacture stem cell products, then sold them to a man posing as a doctor who travelled to Mexico to perform unapproved stem cell procedures on people suffering from cancer, multiple sclerosis and other autoimmune diseases. The three defendants received more than $1.5 million from patients seeking treatment for incurable diseases.

Medical tourists may turn to clinics and hospitals in Mexico, India, Turkey, Russia and elsewhere for stem cell therapies that have not undergone clinical trials, are not recognised as standard treatment, and where governments have a more relaxed view of a practice that brings in income from overseas patients.