Archive for the ‘Stem Cells’ Category

Stem Cell Therapy for Autism… cont’d

Sunday, April 9th, 2017

As doctors try to understand and consider various emerging therapies for patients experiencing signs and symptoms of autism, the question of Stem Cell therapy has come to the fore. A Duke University professor barreled onto the scene, recently, with a pronouncement that sounds like a cure, even though it’s not.

Understanding the study
The project is Clinical Trial to Evaluate the Efficacy of Umbilical Cord Blood to Improve Outcomes for Children with Autism Spectrum Disorder. This was the first phase. The goals were to determine safety, and to evaluate the usefulness of a variety of tests to assess whether the treatment works.

Does giving a child’s own cells, which were collected from the umbilical cord at birth, back into their bloodstream, result in any adverse events? The report broadcast on CNN focused on a 7 year-old who seemed nearly OK, playing with her older, neurotypical sister. The treatment had taken place a couple of years earlier.

Dr. Joanne Kurtzberg, one of the researchers, proclaimed, “We saw improvements in 6 months…” She used the word curative twice in the same sentence, even though her point was ignorance of that outcome. She concluded the interview with, “of course we have to do a placebo controlled randomized trial to answer the question…” The Dad was more realistic, as he commented, “The autism is still there…”

The research involved 25 children, 2 – 6 years old, who had banked cord blood available. “Significant improvements in children’s behavior were observed,” in the majority of children, and “were greater in children with higher baseline nonverbal intelligence quotients.”

“Assessment of adverse events across the 12-month period indicated that the treatment was safe and well tolerated,” claims the abstract. In fact, agitation was a common complaint, requiring additional medications, as the infusion was administered. The authors admitted, “As an uncontrolled open-label study, it is not possible to determine whether the observed behavioral changes were due to the treatment or reflect the natural course of development during the preschool period.”

Discussion
When considering such an extreme treatment, the primary driving force should be the child’s degree of involvement with their developmental challenges. If your youngster is proceeding on an acceptable trajectory, 1) Is it worth the known, and unknown, risks? and, 2) What improvements are you actually seeking? In this study, as in other successful biomedical protocols, the less-affected patients showed the best improvement.

This investigation was done under rigorous conditions by highly trained university personnel, and utilized the patients’ own cord blood. Do stem cell centers around the world offer a similar degree of confidence as regards cleanliness, safety, follow-up, or the ability to handle emergencies? Are outcomes the same when using fat cells that have been turned into stem cells (explained in my previous blogs on this topic)?

The type of autism has got to be a factor, as well. Would a patient with a significant chromosomal variation or metabolic disease, for example, experience the same improvement?

On a positive note, it is encouraging to observe that the conventional research community implicitly concurs that successful treatment involves “modulating inflammatory processes in the brain addressing the reduction of body inflammation to improve ASD.”

Conclusion
We all wish to see a real breakthrough in autism treatment. It appears that stem cell therapy may represent a significant advance. But, that is all that it will represent. Children will still have yeast, and need follow-up labs, and ABA, and Speech therapy. Stem cell intervention seems to represent another, maybe better, certainly more costly, alternative protocol.

Thankfully, Phase II, a randomized, controlled study to assess efficacy, is now underway.

Medical Academy of Pediatric Special Needs 2016 Spring Conference

Sunday, May 1st, 2016

Practitioners, such as myself, find that it is necessary to attend the bi-annual Medical Academy of Pediatric Special Needs conference for two important reasons. First, to listen to experts from all over the world present their knowledge and latest research. Second, to network with, and learn from, other like-minded practitioners.

What I Liked Best
This year, I chose the ADHD path. The workups that were presented tended to be somewhat complex, and perhaps unattainable for many patients. The bottom line was to get a medical evaluation. The differential diagnosis ranges from thyroid to PANDAS. Mostly all agreed that stimulant and psychotropic meds should not be the first line in treatment. One professor spent some time questioning the diagnosis, itself, and how the modern world has contributed to the epidemic.

What I Liked Least
Traveling all the way to Costa Mesa, CA. Course work is 8 hours per day, so no time for Disneyland, etc.

This Year’s Major Focus
Mitochondrial function continued to play a big role in the presentations. The advanced courses involved lots of methylation, detoxification, and energy production diagrams. The newest twist has been the addition of genetic testing to better determine the cause(s) of inadequately functioning biologic pathways. Single nucleotide polymorphisms (SNPs) and ‘epigenetics’ were the buzzwords – how individual genes interact with the environment and within the individual leading to dysfunction and downstream signs and symptoms.

Topics That Were Discussed in Passing
Microarray genetic testing, covering multiple genes, did not play a big part in this year’s talks. Discussions about childhood immunizations underlie a great deal of the members’ conversations; specifically the lack of solid scientific evidence for safety in high risk populations. Attendees are not against vaccinations, by the way. Lyme disease was discussed in general, and as that inflammatory process relates to other infectious-metabolic conditions.

Subjects Not Formally Presented
GcMAF and nagalase levels. Some patients have indicated that a useful, safe supply may become available, so that will help determine future use. As well, chlorine dioxide, hyperbaric oxygen treatment, helminths, medical marijuana, and stem cell therapy were not offered by this year’s presenters.

Conclusion
It is disappointing to return from such conferences without that ‘magic bullet’. Just standing around, listening to Sid Baker speak about how he got interested in autism, or asking him how the ‘ion cleaning’ footpath worked, is worth the price of admission, however. This science started with Dr. Baker, and he continues to be an inquisitive, gentle force for hope, 40 years later.

In the absence of a sufficient population of scientists who are willing and available to address this modern epidemic of childhood developmental problems, this meeting stands as a bastion against the current state of ignorance.

Understanding Autism Better

Sunday, May 17th, 2015

A growing number of children with tiny genetic differences, known as copy number variations, have accompanied the steady increase of patients who visit The Child Development Center.

Often, the conventional advice is that 1) such anomalies are probably not significant, and/or 2) no information is available about ‘that’ particular abnormality.
Do not believe it if your doctor has only told you that, “The chromosomes were normal.”
Take a moment to read the results yourself.

Chromosomes are structures inside our cells, mostly made of DNA. Genes, the basic building blocks of life, are located there. Suspected errors are sometimes only 1/1000 of an inch long! There seems to be a controversy about how different professionals understand the ‘not enough – too much – transposed’ pieces of DNA. How difficult is it then, for parents evaluate?

The basics about genetic results have been previously discussed here.
A specific description about copy number variation is described here.

Results are already available in children who have had a genetic testing, since they are included in the ‘Fragile X test’ (one of the known genetic causes of ASD).

Why perform a chromosomal test?
It should be required in all children who have a Spectrum diagnosis. It’s much more likely to be positive than an anesthesia-requiring MRI or EEG (especially in the absence of seizures).
The argument that testing is not necessary because, “The mom is not having any more children,” is specious. Such information can be quite important for the patient. If there are other people who have the same small chromosomal variations, they can add a great deal more knowledge about your child than merely Googling the cause and treatment of autism.
Additionally, as the future brings more and more information about the performance of those particular pieces of the chromosome – and the genes residing therein – there is added hope that it will lead to specific treatments, or regimens that might be avoided (such as, say, vaccinations) in affected individuals.

Which is the best chromosomal test?
For ninety-nine bucks, 23andme is not the best bet. A saliva sample yields information about ancestry, predisposition to certain traits (e.g., digestion, taste, metabolism, even HIV resistance). However, the SNPs (tiny genetic variations) that are tested are not indicative of ASD.

According to a formal consensus statement of genetic experts, chromosomal microarray (CMA) testing of the blood, “…offers a much higher diagnostic yield (15%–20%) for genetic testing of individuals with unexplained developmental delay, intellectual disability, ASD or multiple congenital anomalies…”
This is a >$1500 blood test (listed here) .

Lineagen advertises that their buccal smear test (a swab from inside the cheek) identifies the most number of changes that are related to autism diagnoses, and is superior to those offered by other labs.
The cost exceeds $5,000. Without insurance, that amount is often prohibitive. As data accumulates, so will the knowledge about the value and validity of this method.

How to read the results of chromosomal microarray testing?
Unless the microarray contains the text, “XX Normal Female,” or “XY Normal Male, no abnormalities reported,” continue reading and ask the lab or a trusted professional to interpret.

How to use the results of chromosomal microarray testing?
Go to the database SFARI gene,
Click on the Copy Number Variant button,
Click on the gene(s) with variation, and follow the table to the letter that best matches your child’s findings.

Conclusions:
Parents often exclaim, “I don’t care what the exact diagnosis is, just make my child improve!” However, the more precise the analysis, the more likely it is that treatment will better target each individual’s physiology.

Knowing this information about your child is valuable – not only in the future, but adds to understanding and treatment for the present situation, as well.

Addendum:

(New York Times 5.14.15)
U.S. Introduces New DNA Standard for Ensuring Accuracy of Genetic Tests

(Journal American Medical Association (6.15)
Copy Number Variations and Cognitive Phenotypes in Unselected Populations

Chromosome 7 flaws alter chemical tags throughout genome

 

Where Have All Those Autism Treatments Gone?

Sunday, March 8th, 2015

Secretin, OSR, Namenda (memantine), Bumex (bumetanide), Actos, Spironolactone, and more, have all been prescribed to improve the signs and symptoms that ASD individuals experience and display. Some are only used in rare circumstances, others are no longer even available. Now, it’s GcMAF.

The Situation:
For several years, that macrophage-boosting blood product has been used to improve immune functioning in various conditions, including ASD. There have been reports of successful amelioration of negative behaviors and facilitating communication.

At The Child Development Center, there have been 25 patients who received the product, either by subcutaneous or sublingual route. As reported here, ten of the children improved enough for the parent to re-order a ~$1000US solution. Improvements were reported in cognition, communication, and even toilet training.

Without any explanation regarding why the company no longer produces, GcMAF.eu now redirects the user to GcMAF.se (France to Switzerland). The site has the following disclaimer:”… it is not possible to purchase GcMAF here… A dozen companies have claimed to make GcMAF. Most of them failed…”

The website offers alternatives that are less than reassuring. “Immunobiotech.eu, … the most professional company in this field.” “An Israeli company was offering GcMAF at $1,000 a shot, but many of the people using it did not believe it worked. It was probably inactive.” Or, they plainly disparaged the product. “Saisei Mirai – a Japanese company… Somewhat risky to inject in our opinion, and their research papers don’t seem to show good results”.

The Outcome:
Presently, when one door closes on the medical treatment of autism, there are few remaining ones to explore. Conventional prescriptions gain a stronger foothold, no matter how limited they perform, or how serious are their side effects.

Professionals feel compelled to assist in ameliorating aggressive behaviors with strong CNS medications, such as Abilify, Risperidone, or even Zoloft, Prozac, and Klonopin. No medication promises to assist the acquisition of speech and language.

Alternative treatments, which may carry reduced evidence of usefulness or safety take a stronger hold, as well. Parents who are seeking improvements are neither foolish nor ignorant – they are desperate. Families are left to evaluate anecdotal reports and Internet stories.

The Conclusion:

A great deal can be learned by the experience of those treatments
that have become less popular, or even extinct.

With an ever-increasing incidence of ASD, more therapies are bound to be invented and evaluated. Parents are not going to give up their fight simply because they are admonished about being ‘unscientific’ or ‘over-‘ emotional.

As more professionals experience affected patients, newer trials are bound to take place. When pharmaceutical companies see an improved return on their investments, they are more likely to join the effort. Increased prevalence boosts enrollment in scientific research.

There is a common theme of gut improvement and addressing immune function. Mostly, what the storage locker of trial treatments contains, are some important keys to understanding, treatment and prevention.

Stem Cell Therapy for Autism – Final Word

Friday, May 4th, 2012

This has been a pretty exhaustive series of posts exploring and explaining the most recent developments in stem cell therapy as they pertain to ASD. These blogs are meant for parents who are considering the procedure (wiki isn’t helping entirely, right?), but many readers may choose this conclusion as their only ‘read’, which is fine. However, the extensive material in the preceding articles can assist parents who want to be better informed.

My goal was to review the current state-of-the-art regarding Stem Cell Therapy, in as neutral a manner as possible. The medical community is fairly dogmatic against high cost, potentially high risk procedures which have little scientific confirmation, and practitioners of such therapies are often labeled as charlatans who prey upon desperate families. My personal bias is, likewise, to be very suspicious of such miracle cures. However, as I have written previously, traditional medicine has so f*#%ed up the diagnosis, etiology and treatment of the condition we call autism that it is no wonder that parents seek assistance elsewhere.

The ‘pros’ of Stem Cell Centers can be found at websites such as this. The cons are exposed in detail at quackwatch. And, don’t forget the schlep to India. So far, this is my  dollars-worth of advice, “What would you do if it were your child, Dr. Udell?” – from a practicing pediatrician’s point of view:

One of the most troubling contentions that I have encountered when interviewing Stem Cell proponents is, “There are practically no risks to the patient because the type of cells that are now used (whatever type that they are using) are safe.” First, nothing that a doctor does is perfectly safe. I even have to counsel parents about the potential die-off from probiotic treatment that can result in regressive behaviors, lack of sleep, rashes, bowel problems and such. Second, there are other increased-risk modalities that often accompany stem cell treatment including; lumbar puncture, intravenous line placement, steroid usage, antibiotics or anesthesia. Third, “Studies have shown safety…” Since when did the Autism community believe that statement? And what about long-term complications? No one can answer that honestly, because no one knows.

This story is a clear example of the issue. In the past month, several children in my medical practice have suffered some especially disruptive behaviors. One particular beautiful child just got her caretaker (of 2 years) to quit because she broke the poor lady’s arm. Her mom said, “You know, I spoke to a woman who has lots of bucks, who had a child – although it was different sex, different age, different diagnosis – who said that there was an improvement with the therapy. Even if I got a small improvement in ‘Suzie’s’ (self-injurious) behavior, I would be glad to spend the $20K.” Now, I had to speak to this mother for 20 minutes to convince her to give a short course of steroids, and it was a struggle. What is the attraction?

Parents can be driven to the point of trying, literally, anything to create a break in the action, even if only is only temporary. Very few people experience or can even comprehend the complicated feelings of parents with severely affected children. That’s why this mom wasn’t considering transplant risks. “Just give me a light in the middle of the tunnel,” is a fair plea. What patients don’t hear about is when a treatment has failed or turned out to be a waste of time and money – there are no blogs about that. There is even one patient in our practice who developed ASD after a stem cell transplant (required for a different medical condition),

What should be happening? There should be more research centers, if not performing the procedure, at least collecting reliable data from the patients who have undergone the treatment. The patients need to be assessed in some formal way about their level of ASD involvement and improvement(s), if any. Those parents so set on going forward with the procedure should be enrolled in a proper setting with all the accepted standards. For those professionals performing the protocol, it’s the only way that they are going to be believed, anyway.

You can’t ‘buy’ your way out of ASD. I takes lots of love, time, work, perseverance and luck and involves a combination of treatments, especially targeting the social domain. Some improvements may take time to evolve into useful behaviors. Steps forward by an autistic patient tend to be incremental and require reinforcement and practice so that neuro-typical behaviors can become embedded into the personality.

No, I can’t recommend the procedure at this time. I can, however, understand how parents arrive at the decision to move forward with the treatment, but not without exhausting all other reasonable and better documented therapies. If you have the time and money and your child continues to be severely affected, at least be well informed and maintain reasonable expectations.

Addendum:

 January, 2014

I recently interviewed a parent who related the following story: “I was so thrilled after the treatment. The child was more calm than ever. I really had hope, for the first time in years.” 
“Would you do it again,” I asked?
“Well, no,” Mom said. “After about a month or so, all those gains in behavior were lost. That was too much money for such a short period of improvement.”

July 2014
Duke University Trial:
http://sfari.org/news-and-opinion/news/2014/experts-balk-at-large-trial-of-stem-cells-for-autism

“There’s no question Kurtzberg is the right person to do this,” says Emanuel DiCicco-Bloom, professor of neuroscience, cell biology and pediatrics at the Rutgers Robert Wood Johnson Medical School in New Jersey. Still, he says, “I think it’s early times.

Stem Cell Therapy for Autism 3

Monday, April 30th, 2012

The condition currently designated as ASD is epidemic. Reports about the alarming numbers abound, but it doesn’t seem to stick. “Is there really an increase in actual cases?” is the most common question that I get asked about my medical practice, even by professionals. From my perspective, media reports and journal articles can’t stop “exposing” the recent increase in measles – 3 times as many cases as last year. The rise in such a preventable infectious disease is regrettable and dangerous. However, it isn’t necessarily occurring in non-vaccinated children, it represents a couple hundred cases – not tens of thousands of patients – and no child has died (or suffered reported permanent damage). That contradiction leaves some families with an autistic child feeling lost and neglected by the conventional medical community and so they seek ‘miracle’ cures.

Stem cells have been successfully transplanted into corneas since 2003. In 2005, the FDA approved, and in 2006, “Neurosurgeons and physicians at Doernbecher Children’s Hospital, Oregon Health & Science University… performed the first transplant of purified human fetal neural stem cells into the brain of a study participant…” who suffered from a rare, inherited brain condition. The material came from dead human fetuses, in the hope that the new tissue would replace a missing enzyme in affected patients. In that study, performed on 6 patients, a billion stem cells were directly transplanted into the patients’ brains and steroids were administered for a year. Although the safety of the project was demonstrated, the ability of the new tissue to reverse that condition has not been documented. Research was discontinued and the company that makes the product hopes to perform the procedure on less-sick patients in the future.

Compare that carefully controlled study to the procedures that are offered at the various Stem Cell Transplant Centers that reside outside the US. Such protocols use cells of various origin (fat, blood) and various injection sites (lumbar puncture, veins) with various additional medications (antibiotics, steroids) on various patients (ages, sex, level of ASD).

Stem cell therapy is experimental and only in its earliest stages of proficiency. Specifically for ASD, which is an enigmatic, multifactorial, multi-system condition with a widely variable presentation and outcome, there is no research to document improvement and only anecdotal evidence that it is either helpful or safe. A conclusion – a scientific one – CANNOT be reached with this level of information. So, the family that chooses this endeavor is working on faith, the (limited) experience of others, and luck when it comes to “the best course of action.”

In addition to betting on “whether” the treatment might “work” (as always, don’t expect miracle cures, just expect miracles), the concerned parent must consider:

1. The cost of Stem Cells and implantation (>$20,000), as influenced by:

a. Which cells lines will be harvested and grown?

b. How the cells are delivered to the patient (artery, vein, spinal fluid)?

c. Other medications, laboratory testing, procedures, or anesthesia if necessary?

d. Complications, and there are never no complications.

d. Insurance – not covered!

e. Travel, including hotel stays.

2. Alternatives

a. Conventional – this should be the first therapy once an autism diagnosis is established or suspected. It includes a neurologic evaluation, a thorough medical evaluation, appropriate laboratory testing and follow-up. This should be followed by Occupational, Speech and Language, Physical and Behavioral Therapy as it applies to your child’s level of function.

b. Biomedical – this will assist many patients, especially the younger ones, toward recovery or significant improvement, especially when accompanied by conventional therapies.

c. HBOT – this therapy may be chosen by many parents as an earlier adjunct to the other biomedical techniques or when they see their child continuing to fail to meet social, academic and developmental milestones and demonstrate significant behavioral concerns.

d. Other – including RDI, Listening therapies, NAETSensory therapies, Neurofeedback, Hippotherapy, etc.

3) Opportunity costs. $20,000 = 3 months of 25 hours/week proven or combination therapies; 4 weeks of summer camp for 6 years; more than 1 year of assistance services (cleaning, caring, tutoring) in your home; 1 used, low pressure HBOT chamber on eBay; 75 high pressure, high oxygen HBOT ‘dives’ in a safe, reliable, clean Center; 4 MNRI™ conferences, many years of neurofeedback, lots of NAET, or ten years of care at The Child Development Center of America (that’s me), including labs, supplements and medications. That dollar figure also equals the low estimation of 1 year of extra costs for each child with Spectrum Disorder.

4) Risks, which will be covered at the conclusion of this series.

In the US, a cord blood bone marrow transplant to treat sickle cell disease costs approximately $250,000 to $500,000. Since stem cell transplantation for ASD is not being studied at any US institution, parents seek care in Mexico and other Central America countries, Europe, China, South Korea and the Ukraine (more to follow, I’m sure). Depending on the Center, costs vary, starting at over $15,000 (various sites, parent’s information) for treatment alone (not including travel). Patients are required to remain for between 1-2 weeks for follow-up.

That concludes some basic background information that ought to be considered in order to make an informed decision about this complicated topic. The next post will sum up all of this and offer some advice.

Stem Cell Therapy for Autism – Part 1

Stem Cell Therapy for Autism – Part 2

Stem Cell Therapy for Autism 2

Monday, April 23rd, 2012

Previously, I discussed the potential that stem cells can exhibit to differentiate into useful tissue. Families interested in curing, reversing or overcoming symptoms of their child’s autism with this technology ought to be knowledgeable about whether the process works for other medical conditions. Unarguably, even by those who perform the procedure for autism, the evidence is in it’s earliest stages.

Current Stem Cell treatment that is medically utilized (United States):

Stem cells are useful when bone marrow requires rejuvenation after chemotherapy or radiation to wipe out cancer cells.

Pretty close to acceptance:

Heart attacks with cardiac damage

Ongoing investigations include:

Bladder repair / replacement

Retina repair

Cornea replacement

Strokes

Spinal cord repair. FDA approval for a study. A recent citation entitled, Human Umbilical Cord Blood Stem Cells Infusion in Spinal Cord Injury: Engraftment and Beneficial Influence on Behavior actually refers to rodent recipients.

Central Nervous System Conditions such as Multiple sclerosis or Parkinson’s Disease

Evidence (not necessarily experience) lacking:

Autism Spectrum

Cerebral palsy

Brain injury

Other Central Nervous System Conditions (e.g., Alzheimer’s)

The information will advance at a rapid rate, so my advice is to discuss the information as it pertains to your child, with a knowledgeable, independent and trusted practitioner.

1. Only consider human trials. We’re talking about a loved one here. A recent Cancer Research article was entitled, Human Neural Stem Cell Transplantation Ameliorates Radiation-Induced Cognitive Dysfunction. Although it looks very promising, the procedure was performed on rats. I can’t help thinking how easily someone with a vested interest in influencing a particular outcome might use that headline in order to justify or provide evidence of stem cells’ efficacy.

2. Surf wisely. Do your own research. Since much of that involves the ‘net, you are observing edited presentations of patients who are only like your child in their families’ desire to help heal their child. Are the images you watch of male or female children? What are their ages, and other co-morbid medical conditions? Did they present after vaccination (as always, an impossibility according to the AAP), like your kids, or were the children atypical since birth? Did they have GERD, or frequent antibiotic usage, or abnormal stooling since they came home from the hospital? What other treatments, such as antibiotics or steroids, were given with the transplant? Anecdotal evidence is very difficult to evaluate from youtube.com or parental testimonials. Editing is the whipped cream that can convince hopeful parents to take the plunge (and often, music is the cherry-on-top).

3. Learn when Stem Cell Treatment didn’t work. The first questions I ask when presented with any autism protocol are, “Who didn’t get better?”, and of course, “Who got worse?” These procedures are being performed in foreign countries. Even if, as is the usual contention, the criteria for certification or cleanliness are as stringent as they are in the US, the reporting agencies may not be. Centers should provide well documented outcomes and transparent (and reproducible) statistics. Follow up studies are scant. Since I know that many parents have grave concerns over administering oral melatonin to assist sleep, I can’t imagine how those families deal with this level of investigation.

I am always interested to learn from patients who have had the procedure performed on their children, some multiple times, and some who are looking to do it again. In the limited population that I have examined, the children appear to respond the same as with most biomedical treatments in that the more affected the child, the more treatment(s) is required. I have not yet noted more improvement than in those children who were treated with less complicated biomedical interventions.

Next up: Choosing a Stem Cell Center

Stem Cell Therapy for Autism – Part 1

Stem Cell Therapy for Autism – Conclusions

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Brian D. Udell MD
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