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“Look at me!” says 11-year-old Michaela, “I can walk now!” Michaela Sky Rodrigues is an exceptional girl with exceptional parents. Refusing to accept the bleak outlook doctors described for her, Michaela, who suffers from secondary dystonia, travelled with her family from her home in Australia to China for a series of pioneering umbilical cord blood stem cell treatments.

And the result? Michaela’s father emphasizes that stem cells are not a cure for dystonia; but they are an effective treatment: “small changes” he says, “lead to big changes.” Michaela can now walk, control her limbs and neck, use the toilet and eat solid foods. The list goes on and on, each a landmark for Mr and Mrs Rodrigues who are now looking forward to a bright future for their daughter: “Look what she’s accomplished,” Mr Rodrigues says, “in such a short time after stem cells… look how far she’s come.”

The clearest evidence of Michaela’s progress is the enormous smile on the face of both father and daughter, and the confidence with which Michaela addresses the video camera after her treatment. ‘Thank you,” she jokes, “and good night!”(1)

What is dystonia?

Dystonia is a painful and debilitating neurological movement disorder and although it affects around 70,000 people in the UK, many of us have never heard of it. Dystonia Awareness Week (5th – 13th May 2018) aims to raise awareness of the condition, enabling The Dystonia Society to support those affected.(2)

The cause of dystonia varies from patient to patient, though the condition is linked to the part of the brain that controls movement. The disorder may be inherited, or result from a related illness such as Parkinson’s disease, stroke, cerebral palsyor multiple sclerosis.

Dystonia can start at any age and might affect any part of the body. The symptoms vary and may be continuous or sporadic but often include uncontrolled muscle cramps and spasms; unusual twisting of parts of the body and uncontrolled blinking. (3)

Stem cell treatments for dystonia

Stem cell therapies for dystonia are currently being researched and trialled. One process involves taking a small skin sample from patients with dystonia, growing living fibroblasts from the skin, and then converting the fibroblasts into stem cells for making neurons. Although these cells, collected from the dystonic patient, cannot be used therapeutically, scientists hope that this process will increase understanding of the condition and future therapies.(4,5)

Umbilical cord blood stem cells and neurological disorders

In their 2016 paper, Application of Umbilical Cord Blood-Derived Stem Cells in Diseases of the Nervous System, a team of specialists surveyed the efficacy of cord blood stem cells in treating conditions including stroke, cerebral palsy, sceloris and glioblastoma. They concluded, “It is clear that UCB derived cells have profound neurogenic potential.”(6)

Since this time, we have witnessed the watershed study of cord blood stem cells for cerebral palsy at Duke University, which resulted in improved motor skills in some children: “We are encouraged by the results of this study,” said senior author Joanne Kurtzberg, M.D., director of Duke’s Pediatric Blood and Marrow Transplant Program, “which shows that appropriately dosed infusions of cord blood cells can help lessen symptoms in children with cerebral palsy.”(7)

With so many trials underway, and over 80 conditions already routinely treated with umbilical cord blood stem cells, including blood cancers and diseases of the immune system, scientists are cautiously optimistic about the future of regenerative medicine.

Sickle Cell Disease is a group of inherited conditions that affect the shape of red blood cells. Normal, healthy red blood cells are a circular, disc shape; whereas, red blood cells in sickle cell disease, have a crescent or “sickle” shape. The sickle shaped blood cells tend to stick together in clumps, leading to blockages in tiny blood vessels. This can result in pain crises, infection, chronic progressive organ damage, and eventually stroke. Also, sickle cells do not effectively transport oxygen around the body, so that patients with Sickle Cell Disease experience chronic anaemia, fatigue, and shortness of breath. 

Sickle Cell Anemia is one of the most common inherited blood disorders in the UAE, and the country is also reported to have one of the highest national frequencies of the disease in the world. Dr. Maryam Matar, founder of the UAE Genetic Disease Association said: 

"It’s incredibly important for expectant mothers who have a child with Sickle Cell Anemia to be informed about the ability to save their baby’s cord blood for potential transplant. A stem cell transplant has the ability to cure the disease, and all mothers should know this." 

Hamad’s family, from Abu Dhabi, wanted to improve the quality of life for their son and requested to transplant the cord blood from his younger brother Abdullah that was stored. The only cure for Sickle Cell Disease is through transplant of blood-forming stem cells such as those found in cord blood. Stem cell transplants are most effective in children and young adults with Sickle Cell Anaemia. When the stem cell donor is a compatible sibling, the outcomes are extremely positive: close to 90% sickle cell disease-free survival. Following successful stem cell transplants, sickle cell disease patients are symptom-free and their quality of life is greatly improved. 

At the beginning of 2017 Hamad’s parents had made the decision to use the cord blood stem cells from his brother for a transplant to try and save his life. The transplant was carried out in the United States at Cincinnati Children’s Hospital Medical Centerand  the sample was shipped after in-depth testing in compliance with strict AABB and FDA criteria. 

The transplant took place on 23rd June 2017, and almost a year later, Hamad is now completely cured and living a happy, healthy life, disease-free. Mai Ibrahim, Chief Operating Officer said, "It is such a great honour to be contributing to saving lives here in the United Arab Emirates, and we want to support those tackling the disease in the country, where it is so common. 

 "We were worried but our local peadiatrician was positive. He asked us to observe him for another six months. We saw no changes in his behaviour." - Apurba Dey Singha, Apratim’s father.

 Apratim saw a specialist in Delhi who diagnosed him as having Autism Spectrum Disorder (ASD). Autism refers to a range of conditions characterized by challenges with social skills, repetitive behaviors, speech and nonverbal communication, as well as by unique strengths and differences. Each person with autism presents differently, but has enough of the traits to be recognized as being on the spectrum.

 Apratim’s family researched interventions to improve his language skills and tried several approaches. They learned that in 2014, the same year Apratim was born, Duke University launched the first in a series of clinical trials treating autism with cord blood stem cells. 

Results from the first Duke trial of cord blood therapy for autism were published open access in April 2017. In that pilot study, participants showed significant improvements in measures of social skills, expressive vocabulary, severity of autism behavior patterns, and eye-tracking response to stimuli. The children’s scores were measured by both parents and clinicians using established behavioral scales developed for autism. The observed improvements appeared within 6 months of cord blood therapy.

 In a press release from Duke University, lead investigator Dr Joanne Kurtzberg expressed “cautious optimism” but urged parents not to jump to conclusions:

 "Parents of children with autism should not interpret these results as conclusively showing effectiveness of this treatment. There is much work still to be done in much larger, randomized clinical studies before we can draw any firm conclusions about effectiveness." - Joanne Kurtzberg, MD.

 Apratim’s cord blood had been stored since birth .His parents asked to participate in a clinical trial at Duke, and after a number of tests he was accepted. The therapy took place in the United States in July 2017.

In the 9 months since the cord blood therapy, his father says he has seen significant changes in Apratim. He is communicating more, his cognitive skills have improved, he’s started school, and he likes playing with his friends.

 “We were happy that we made a wise decision of preserving our baby's umbilical cord stem cells at birth, which were used to treat my son for autism. Today, we are seeing great progress in terms of development milestones in my son." - Apurba Dey Singha.

According to the public relation of Royan Stem Cell Technology Company, Dr.Ashrafi said:” The injection of cord blood stem cells derived from hematopoietic or mesenchymal sources to the children suffering from spastic cerebral palsy at the age of 4 to 14 years old was started as a research project since last summer.”

He also said that there were 108 children enrolled in this project who were categorized into 3 groups. The first group received the stem cells derived from hematopoietic sources, the second group received mesenchymal stem cells and the third group was considered as the witness. So far about half of the patients have been observed and entered in this project.

He later added that this project was first approved at Tehran Medical University and was launched under the support of  Hormozgan Medical University, State Welfare organization of Iran, Blood Transfusion Organization and Royan Stem Cell Technology Company.

He pointed to this fact that putting this project into effect in full success, patients would not need to go to other countries such as China, India, Ukraine or even Germany to receive treatments which by itself impose lots of expenses to them coming along with some more problems. Launching such successful project can be considered as a means of developing health tourism industry in the country as well in which many neighboring countries such as Iraq and Azerbaijan would be the best potentials showing great interest in receiving such medical services in Iran.

Dr.Ashrafi said: ”This project was initially funded by Hormaozgan Medical University and we hope we can receive more financial aids from other sources to put this project into the effect thoroughly and to its full end.”

He declared that so far we have concluded that the best treatment source for treating such malignant are cord blood stem cells which comes to the success rate up to 20 to 60 percent with no negative side effects.   

Abstract

Multiple myeloma (MM) is a malignant plasma cell (PC) disorder, characterized by a complex interactive network of tumour cells and the bone marrow (BM) stromal microenvironment, contributing to MM cell survival, proliferation and chemoresistance. Mesenchymal stem cells (MSCs) represent the predominant stem cell population of the bone marrow stroma, capable of differentiating into multiple cell lineages, including fibroblasts, adipocytes, chondrocytes and osteoblasts. MSCs can migrate towards primary tumours and metastatic sites, implying that these cells might modulate tumour growth and metastasis. However, this issue remains controversial and is not well understood. Interestingly, several recent studies have shown functional abnormalities of MM patient-derived MSCs indicating that MSCs are not just by-standers in the BM microenvironment but rather active players in the pathophysiology of this disease. It appears that the complex interaction of MSCs and MM cells is critical for MM development and disease outcome. This review will focus on the current understanding of the biological role of MSCs in MM as well as the potential utility of MSC-based therapies in this malignancy.

Read more: www.nature.com/articles/s41375-018-0061-9

My distinguished colleagues, before I became a member of this incredible team, I had never thought that words “work” and “pleasure” are compatible. But, working with you, I have become more and more convinced that it’s possible. Work is an integral part of our life and also a good source of happiness. That’s why I wish that your job bring you personal growth and career advancement, moral satisfaction, warm trustful relationships and wealth in the coming year. As the New Year approaches, give a moment of thought to the achievements and the failures of this year so that we know the right path for us.

We are looking forward to a new year that brings new challenges and opportunities. Happy and Prosperous New year!

Wish you all  very fruitful days in the coming year! Happy Holiday!

MORTEZA ZARRABI

MANAGING DIRECTOR

For Natasha Spencer, mother of Krabbe disease sufferer Kenan, and passionate campaigner for newborn screening, this is a bitter-sweet success. Spencer’s Facebook post yesterday brimmed with heartfelt emotion. “Today,” she wrote, “the next generation of expectant, Illinois parents can take for granted the test Kenan never received. Today, we will find you, needle-in-a-haystack child, and identify you before the disease does.”
Kenan’s prenatal and newborn screening tests
When Natasha Spencer went for her 21-week ultrasound scan her doctor reassured her that all her baby’s tests were clear, adding that her unborn son had “a beautiful brain.” Natasha already had a daughter, Tamsen and she and her husband, Dann were looking forward to meeting their son.
 
3 months later, Kenan was born. He weighed 8 pounds, 11 ounces and had a full head of hair. Natasha introduced him to her Facebook friends with a photo of him swaddled and sleeping. “We are in love!” she wrote.
Later, a nurse pricked Kenan’s heel and used special filter paper to collect blood that would be screened for dozens of rare diseases. When Natasha heard that “All results are normal” she wasn’t surprised. Kenan gave every appearance of being a healthy baby boy.
 
Kenan’s first few months
For the next 6 months, the Spencer family continued their lives as normally as any family with a newborn baby. Kenan grew alongside his big sister as his parents proudly noted their developmental landmarks: “Kenan’s laugh … around 5 months!” Natasha wrote on Facebook. “What a joy it is to hear!”
 
Then, just a few weeks later everything began to change. Instead of progressing as Tamsen had progressed, Kenan seemed to begin to regress. At first he became fussy and difficult to please, then his head began to tilt at an awkward angle. In just two weeks he was unable to use his hands. The change was so rapid and frightening that rather than waiting for an MRI scan scheduled for the next week, Natasha rushed Kenan to the emergency department of her nearest hospital.
It took two weeks to confirm a diagnosis of an aggressive form of Krabbe disease.
 
What is Krabbe disease?
Krabbe disease is an accute lysosomal disorders. Babies with Krabbe (pronounced crab-AY) are born with a “beautiful brain” that later deteriorates. Their lysosomes don’t have enough of an enzyme that helps sustain the protective coating around nerve fibers. Without that protection, nerves throughout the body can’t transmit their signals properly.
The babies’ bodies go limp; their jaws go slack. They lose their sight, their hearing and their sense of taste. As the swallow reflex wanes, they choke on their own saliva. The mere act of holding them upright can restrict an airway. Children with the most aggressive form of Krabbe disease typically die before age 2.
New parents are used to chronicling the firsts of childhood: the first time a baby smiles, sits up, crawls. But for parents of infants with Krabbe, this tradition is turned inside out. Instead, they are mindful of the lasts: the last time a baby smiles, laughs, turns his head to make eye contact.
The use of cord blood to treat Krabbe disease
Studies have shown that a transplantation of umbilical cord blood can significantly improve the neurological health of infants with Krabbe disease, ‘favourably altering the natural history of the disease’. Infant screening for Krabbe disease would provide the opportunity to treat babies with umbilical cord blood transfusion before the disease became established.
 

According to the public relation of Royan Stem Cell Technology Company, here on Tuesday, 13th, February, 2018, Professor Jason Acker, the Head of Society of Cryobiology and the manager of Biobanking Project of Alberta University in Canada, had a visit to Royan Cord Blood Bank and Stem Cell Technology Company.
This visit came along with a scientific meeting with the experts of various departments and laboratories through which Professor Acker got familiar with all the procedures and tasks performed and handled by the staff of different departments since collection to the storage of cord blood stem cells samples. 
In the interview made, Professor Jason Acker stated:” I have seen all the stages and steps taken throughout different parts and laboratories of high quality and in total compliance with international standards and criteria". He also added that he is ready to make a full support to Royan Cord Blood and Stem Cell Technology Company in realm of Cord Blood and Stem Cells and that would be good opportunities to set up scientific collaboration so that both sides can take advantage of mutual benefits and opportunities rising ahead. 

The majority of twins are fraternal, occurring when the mother releases two separate eggs that are both fertilized. The birth rate of identical twins, which occurs when a single fertilized egg spontaneously splits into two eggs, has not changed and is only 1 in 285 pregnancies. In other words, it used to be that half of twins were identical, but nowadays only 1 in 3 sets of twins are identical.

Genetically, fraternal twins are the same as any other siblings, they just happen to be born at the same time. Both fraternal twins and siblings have a 25% of having matching antigen types. That is a 1 in 4 chance of being a perfect match for a cord blood transplant or any other stem cell therapy that requires HLA matching.

The fact that twins share the same womb means that they tend to be smaller babies. Since the 1960s, it has been established that the average birth weight of full-term babies in the United States is about 8 pounds (3.6 kg) and babies smaller than  5.5 pounds (2.5 kg) are classified as low birth weight. However, within the past decade research has changed medical ideas about what is normal for a twin. Singleton babies are expected to spend a full 39-40 weeks in gestation, but studies show that in order to avoid growth restrictions it is best for twins to be born at 37 weeks. The average birth weight of twins born at 37 weeks or later is 5.5 pounds.

Nonetheless, the fact that twins are smaller babies does mean that they have smaller placentas and less umbilical cord blood. Parents of twins are often concerned10,11 about cord blood banking: On the one hand they worry that their babies may experience the learning delays that are often associated with early birth and low birth weight, and which can now be treated with cord blood therapy13-15. On the other hand, they worry that the collected cord blood will not be enough to be useful for therapy.

The bank Cryo-Cell has conducted the only study of cord blood collections from multiple births in the setting of family cord blood banking. Since 3 out of every 100 births are twins, for their study they pulled data from their last 300 multiple births and their last 10,000 singleton births. Not surprisingly, the median volume of cord blood collected was 10% smaller in multiple births. The good news was that the cord blood units from multiple births had nearly 4 times more Total Nucleated Cells (TNC) cells and nearly 8 times more CD34+ cells than the minimums required for storage. The reassuring conclusion of the Cryo-Cell study is that cord blood units collected from multiple births have sufficient numbers of stem cells to treat children for cerebral palsy and other conditions identified in infancy, under current dosing guidelines

Regardless of what each nightfall steals from our life, may dawn give us another day to weave new hopes and dreams. I wish you Happy New Year from the bottom of my heart. Leave the sorrows, pain, and sadness behind and let’s welcome the brand New Year with a smile.

 Beta Thalassemia Major is an inherited blood disorder that occurs when the genes governing the production of haemoglobin (the protein in red blood cells that binds to oxygen and carries it throughout the body) are flawed. The most common treatment is to receive regular blood transfusions to keep the body stocked with healthy red blood cells, but frequent blood transfusions can lead to other health problems.
 The parents of Zayed and Mohammed had taken the decision to store their younger son’s cord blood with CryoSave Arabia in Dubai Healthcare City. Thankfully, detailed HLA typing showed the siblings were a close enough match for Mohammed to donate his stem cells to Zayed. CryoSave Arabia was responsible for the shipment of the cord blood stem cells after in-depth testing in compliance with strict AABB and FDA criteria. CryoSave Arabia is the only facility in the UAE accredited by AABB and this marks the first release of privately stored cord blood stem cells from the UAE to the US.  
The transplant was carried out in late 2014 in the United States at the Johns Hopkins University School of Medicine, Baltimore, Maryland. Zayed underwent a combined transplant using stem cells from both cord blood and bone marrow to optimize the chances of success. Since the transplant, the family are back home in Abu Dhabi where Zayed has made a full recovery and no longer suffers signs of Thalassemia, nor has he required regular blood transfusions.  
  ‘’Our son is in much better condition after the transplant, it has been remarkable. Stem cell storage is so valuable even though it is something that you hope you will never need, like insurance. In our case we did need it and we are incredibly glad we made the decision to store the cord blood in the first place, otherwise our family’s future would be very different’’ said Zayed’s father. 
Kenneth R. Cooke, MD, Director of the Pediatric Blood and Marrow Transplantation Program at Johns Hopkins University School of Medicine said: ‘’ I would always recommend families to store their child’s cord blood whether they know of any possible health issues or not, as it can be used in the future for theirs and possibly other family members’ benefit.” 
Mai Ibrahim, CryoSave Arabia’s Lab Director and Head of Quality: “It is such a great honour to be working in a field that saves lives and the Dubai facility reflects our desire to be the best. We are trusted with families’ valuable stem cells so it is absolutely essential that we ensure each sample is processed, stored and released successfully. There is no room for error.” 
 

Georgina Russell from Preston, near Manchester in the UK, was four months pregnant when she received the terrible news that her older brother, Ashley, had a rare, slow-growing form of the brain tumor, glioblastoma.
 Glioblastoma is associated with a wide range of commonly occurring symptoms, such as headaches and nausea. For this reason it can take a long time for sufferers to recognize the seriousness of their condition. Ashley had been suffering with vague symptoms for months before changes to his co-ordination and vision prompted him to seek medical advice. Having confirmed glioblastoma with MRI images, Ashley’s doctors estimated that he had just five years to live.
 Ashley is 34 years old and worked for the Royal Navy until his diagnosis in August 2017. He and his wife have a daughter, Alexis, who has recently turned two. "She’s so like her Dad,” says Georgina, “Ashley’s always been a family man. At least now he has his family around him and he’s able to spend time with his daughter. That’s the best thing for him really." 
Determined to help her brother in any way possible, Georgina spent the days following his diagnosis researching his condition and emerging treatments. Then, late one night she made the discovery that could save Ashley’s life. "I knew that the placenta was nutritional and could help the recovery of the body," says Georgina "and I thought, what a shame for that to go in the bin. Then my cousin sent me a link to a story about breast milk being healing and used to fight infection. This was interesting, but it wasn’t enough. I started typing things in and eventually came across a site that mentioned umbilical cord blood. I thought, oh my goodness! Doctors are using stem cells from the cord to treat different cancers!"
 "I was reading this and thinking, I've got this cure inside me right now!" 
 
Georgina was shocked to learn that cord blood stem cells have been used in over 40,000 transplants to treat over 80 diseases of the blood and immune system, and yet no one she knew had heard of cord blood storage. "It should become a part of our education." She says, "It’s so important. In schools and childbirth clinics people need to know that their child or a member of the family might need these cells. This is Ashley’s life. A life that’s ending far too early." 
The next day, Georgina compared umbilical cord blood banks in the UK and spoke to Nikki at Biovault Family. Nikki talked her through the unique properties of umbilical cord blood and the process of collection and storage. “I realized, this could be it," Georgina says, "with Ashley being my brother there would be a good chance of a match." 
Feeling reassured that the family had a plan, Georgina tried to carry on as normal. Then, six weeks early and in the middle of a theatrical performance of Sister Act, Georgina’s waters broke. "I was at the Winter Gardens in Blackpool" Georgina says, "and I felt a gush. I stood up and ran to the toilets. Luckily a retired midwife had spotted me and saw that I was bleeding. My partner rushed us in a taxi to the hospital where they did a scan and discovered a dark line between the womb and placenta. I had clots and had been bleeding for a long time. After two days in hospital I was taken for a C-section." 
Despite everything she had been through, Georgina was determined that the umbilical cord be collected safely. "I was in trouble, my baby was in trouble but I just kept saying: Do not throw away that cord! Biovault was amazing. It was a bank holiday but they all pulled together. Nikki rang me and told me not to worry; they would get a kit out to me. As soon as that was sorted out I could relax.” 
Her daughter Charley was delivered safely and has had a powerful effect on Georgina who feels closer than ever to her family. "We are five siblings," she says, “and there are so many photos of the five of us together as children. To remove Ashley is like removing a piece from the picture; a piece of the jigsaw puzzle would be missing. Since I’ve had Charley I can begin to imagine how our mum is feeling right now. It’s beyond anything I’ve ever felt, it’s heart wrenching." 
"Our hope is that because his tumor is slow growing, and medicine is moving fast, the medicine will progress quicker than the tumor." 
With Charley’s cord blood and tissue safely stored away, Ashley’s family and friends have pulled out all the stops to organize fundraising events to pay for his treatment. "Ashely is overwhelmed by all of the support." Georgina says, "He can't believe that so many people care so much. But that’s human nature. If someone can help, they will"