What is autism? What makes autistic people different from others? What causes autism? There are hundreds of questions in people’s mind when it comes to this condition. What is it actually?

Autism is a mental condition that remains present from the early childhood days and lasts for a lifetime. It’s characterized by minor to major difficulties in social interaction and framing language abstract concepts. And the critical part is, it’s incurable. However, a new study shows that stem cell therapy may show great improvement in children with autism.

It’s the story of Gracie Gregory, one of the 25 autistic children who participated in the study at Duke University in Durham, North Carolina involving stem cells to tone down the issues associated with autism. The idea behind the research was to find out if a transfusion of the self-umbilical cord blood with the rare stem cells can cure autism or at least reduce the severity.

Gracie was diagnosed with autism at the age of 2. When Gracie’s mother first came to know about the research, she immediately enrolled her without knowing what to expect and what not to expect. Such was the difficulty; they were facing with their autistic child. There was a time when Gracie’s sister, Ryleigh was afraid of her because of her unmanageable behavior like hitting and kicking. But now that very Ryleigh thinks Gracie is “very sweet and kind.” And it only became possible after the stem cell therapy.

Gracie was on the mild to moderate scale of autism. During the trial, Gracie, then 5, started with the same symptoms like kicking, spitting, screaming and even hitting her therapists. It was almost impossible to make her sit at one place.

The results were beyond what Gracie’s parents were expecting! Her parents recollects how difficult their life was back then when managing her would take 75% of their daily life and now after the therapy she only consumes 10% of their daily time. When asked about rating her improvement on a scale of 1 to 10, her parents proudly rated 8/9.

She now attends regular school, plays with her sister, and enjoys life to the fullest, something that her parent would never have thought of.

According to her father, “We will say we don’t think it’s cured her. You still see some of the small idiosyncrasies that she does have. But again, I think it’s supercharged her learning curve. It’s pushed her to do things she normally wouldn’t do.” Her mother added, “She got better, and we’re just thankful for that — whether it be the stem cells or not. We’re just thankful for what changes have happened”.

Two thirds of the children, involved in the study showed great improvement. This is the results of the first phase of the study and a broader second level trail is on its way, which promises a long-term treatment for autistic children.

Skeptics are still saying that no matter what autism cannot be cured and there are quite a few unanswered questions that need to be addressed before going gaga over it. Even researchers at Duke acknowledge that the study is at a very early stage and still there is a long way to go. But for families like Gregorys, it’s a life-changing experience.

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Researchers were wondering from ages as to what could be the actual cause of Huntington’s disease which causes involuntary jerky body movements disabling the victim to exhibit physical and mental coordination. The awkward bodily momentum gradually worsens after the initial unsteady gait putting the victim in jeopardy. Parental inheritance being the focal reason, this disease is hard to prevent and has been affecting millions of people globally without a documented cure. However, the available treatments reduce the severity of the symptoms and aid the victim in lessening the care giving requirements.

To enhance the quality of life of the global populace affected with Huntington’s disease, researchers are looking forward to get core advancement in finding an explicit medical solution. However, the extensive research to find the root cause of Huntington’s disease has enabled the researchers to get a key which can be a potential advancement in finding a generic solution to lessen the severity of the ailment. It is established that a mutant of the Huntington (mHTT) protein is the fundamental cause of the disease though the researchers are still unclear as to how it disturbs the brain cells’ function. As per the established findings of late, mHTT vitiates the cellular transport process adding to the neurotoxicity eventually resulting in loss of neurons.

Caused by an extended CAG repeat in the Huntington (HTT) gene, this neurodegenerative disease with mHTT plays pivotal role in disrupting the transport system of a cell which normally requires mobilizing multiple elements from its nucleus to the other parts and vice-versa. It has been pragmatically realized that mHTT creates aggregates in the central operations area that is in the nucleus preventing it from passing through the nuclear pores.  This pile up in the mHTT following the stagnation in the nuclear passages kills the brain cells by shutting them down.

The new fact discovered can drive the researchers to find out new ways to fix the cellular traffic jams there by aiding the process of finding new treatment methodologies for the Huntington’s disease. Although it is a fact that this step towards an advanced research in finding the cause for the disease is in the initial stage, the researchers are excited with their discovery as these findings are the stepping stones in conquering the Huntington’s disease along with encouraging the search for new therapeutic modules.

Biotech companies and pharmacy companies are looking forward to dwell on this very concept to capitalize it and bring into existence various therapies for biological processes. Hope they get abundant clues to prove the fact that early birds get quick worms in the course of their research. Ultimately, it is the millions of people who are affected with the Huntington’s disease who get the best of the research.

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Over the years scientific development in neonatal medical care has been revolutionary. One of the best advances in this domain has been in the form of cord blood and umbilical cord tissue banking. Between the baby and the mother, the umbilical cord acts as the lifeline as it comprises of many blood vessels, through which the umbilical cord blood pulses. The umbilical cord tissue, on the other hand, enables the blood vessels to weather the stress and the presence of the cord protects and supports them from bending.

The umbilical cord tissue comes with a high concentration of mesenchymal stem cells or MSCs. A research article states that “MSCs derived from the umbilical cord tissue, termed UCX, were investigated for their immunomodulatory properties and compared to bone marrow-derived MSCs (BM-MSCs), the gold-standard in immunotherapy.”[1]

According to available proprietary technology, UCX is being sequestered from the umbilical cord tissue; the methodology has proven to be quite strong, reproducible and will work adequately for stem cell banking. [2] It has been seen to produce a greater number of cells along with approximately 100% success rate with limited bacterial impurities.

In the present scenario, stem cells found in the cord blood are being used in the treatment of a number of blood-related diseases. However, there is a major hindrance as the volume of blood collected from the umbilical cord is low and can pose a drawback in terms of transplants. Studies and researches are currently underway to understand possible methods to increase the efficiency of cord blood transplants. Some investigations have delved into “ex vivo expansion of the hematopoietic stem cells before transplant, intensifying the homing of hematopoietic stem cells to the patient’s bone marrow, direct injections into the patient’s femoral bone, and transplants with multiple cord blood units” as stated in Banking Stem Cells From Umbilical Cord Tissue For Future Regenerative Medicine Applications. [3] A more direct technique is to make use of another stem cell population – MSCs found in the umbilical cord tissue – for co-transplantation with a cord blood unit, could well be the solution.

With technology to extract MSCs from cord tissue, the first step is to mince the tissue. This is further supported by an enzymatic breakdown process to “melt” away the cord tissue that in turn releases the native MSCs. An alternative way to preserve MSCs is to process the tissue and extract the cells thus securing them in a “treatment ready” form.

The clinical tests conducted on these cells have revealed affinity towards treating auto-immune diseases (diabetes, Crohn’s disease, graft-vs-host-disease), neurodegenerative disorders (Parkinson’s disease, Alzheimer’s) and cardiovascular diseases (acute myocardial infractions, ischemia).[4]

Deciding to store the umbilical cord tissue will give you the opportunity to take advantage of the therapeutic potential of stem cells in regenerative medicine. Umbilical cord tissue stored in cord blood banks represents an additional source of stem cells that may have both immediate and future applications. However, the choice to store your child’s umbilical cord tissue is only available to you once, therefore, think carefully while making your decision.

Sources used:

• http://www.bloodjournal.org/content/111/1/430.short?sso-checked=true
• http://www.ncbi.nlm.nih.gov/pubmed/23755729
• http://www.ncbi.nlm.nih.gov/pubmed/23998751
• https://stemcellres.biomedcentral.com/articles/10.1186/scrt394
• http://www.assembly.wales/NAfW%20Documents/hhs74_-_anthony_nolan.pdf%20-%2024012011/hhs74_-_anthony_nolan-English.pdf
• http://www.hindawi.com/journals/sci/2015/583984/#B30
• http://www.ncbi.nlm.nih.gov/pubmed/23895058

[1] What Makes Umbilical Cord Tissue-Derived Mesenchymal Stromal Cells Superior Immunomodulators When Compared to Bone Marrow Derived Mesenchymal Stromal Cells? – R. N. Bárcia,1 J. M. Santos,1 M. Filipe,1 M. Teixeira,1 J. P. Martins,1 J. Almeida,1 A. Água-Doce,2 S. C. P. Almeida,2 A. Varela,2 S. Pohl,3 K. E. J. Dittmar,3 S. Calado,4 S. I. Simões,4 M. M. Gaspar,4 M. E. M. Cruz,4 W. Lindenmaier,3 L. Graça,2 H. Cruz,1 and P. E. Cruz1

[2] R. I. Ganchas Soares, M. C. Baptista Coelho, J. M. Silva Santos, et al., Isolation Method of Precursor Cells from Human Umbilical Cord, edited by INPI, Medinfar, ECBio, Lisbon, Portugal, 2008.

[3] Banking Stem Cells From Umbilical Cord Tissue For Future Regenerative Medicine Applications – Rouzbeh R. Taghizadeh, PhD, & Kyle J. Cetrulo

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Miracles happen. How many times have we heard this? But have you actually ever seen a miracle happen. This is the story of one man’s fight against blindness which eventually gave hope to millions of people.

It was on a family road trip to New Hampshire when the 54-year-old Doug Oliver was first made aware of how advanced his macular degeneration had become when he failed to see two pedestrians in a crosswalk and almost ran them down. The incident resulted in Doug receiving a $500 fine from a police officer who had witnessed the near accident and was also made to give up his driver’s license.

Doug had been previously diagnosed almost 20 years ago, with macular degeneration. However, back then his doctors had predicted that his vision would last until the age of 60. But following the accident, when his eyesight was reviewed, it revealed that he was almost blind. The tests showed he had 20/2000 vision in the left eye and 20/400 in the right. This new found disability severely affected his career as an IT specialist and social policy consultant. He had no choice but to depend on his monthly disability check to make ends meet.

His doctor advised Doug that the only way to find a cure for his ailment was to participate in clinical trials. After vociferously searching for such trials, Doug was finally able to reach out to a surgeon, Dr. Jeffrey Weiss who was conducting an experiment on treating macular degeneration through stem cell banking. After flying out to Florida, Doug met with the doctors who were participating in the study. They extracted a bone marrow sample from his hip bone and with the help of an FDA-approved centrifuge, isolated the stem cells which were then injected into his eyes. Within 48 hours his vision improved. The vision in the left eye was 20/40 and in the right eye was 20/30.

Following the success of the treatment, Doug said, “I could see the boats and the little wake lines that you see from 30,000 feet. I acted like a 6-year-old and loved it”.

In order to garner government funding for such procedures, Senate Health Committee Chairman Lamar Alexander invited Doug to Washington in June 2016. Doug was asked to speak to Mr. Alexander’s colleagues regarding his success story, in the hope that it would convince them to support the passing of the 21st Century Cures Act which legislates the funding of high risk, high reward clinical trials. He believes that the passing of this Act can revolutionize the healthcare industry.

So as the story goes… miracles do happen, or at least in Doug Oliver’s case, a modern ‘medical miracle’ did happen.

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