Stem and reboot

Banking on cord blood Banking on cord blood: Inside the Cordlife lab in Kolkata.

Efforts to treat autism using the child’s own cord blood

Rajiv was two when his parents noticed he was different. “If he got attracted to something he would do that for hours together. If one tried to get him out of it, he would get angry, cry and shout without a pause. Unless he was given the same thing, he would not go silent,” said Rajiv’s father, Rakesh Goel, a Kolkata businessman. “My wife and I used to scold him. But he never improved.” Goel took him to a doctor.

And then came the blow: doctors told the family that Rajiv was an autistic child. His paediatrician suggested they consult a neurosurgeon, who told them that the child’s social interaction was limited because he was autistic. The only option was psychological and behavioural therapy as neurological methods may not help.

India has more than one crore autistic children. Experts predict that in 20 years, the number could be five times more. These children grow up to become dependent adults.

In autism or Autism Spectrum Disorder, the ill-developed or maldeveloped inter-neuronal connections impair the brain’s ability to process information from the environment. There is no cure yet for ASD; often behavioral therapy is prescribed. Stem cell therapy is a new frontier that experts are exploring as a treatment modality for ASD. Cordlife Sciences India, which banks umbilical cord blood, has engaged US-based child neurologist Michael Chez for a human trial-based research project on autism in five locations in the country, including Kolkata. According to Chez, the clinical trial he has done on stem cell therapy for autism has shown positive results. He will guide a team of doctors in India to carry out clinical trial on autistic patients who have banked their umbilical cord with either the company or elsewhere. “This is a double blind cross over study similar to my trial in the United States,” said Chez.

Said Meghnath Roy Chowdhury, managing director of Cordlife India, “We are excited to have Chez in India with us to work with physicians here to study the treatment of childhood autism using cord blood stem cells. Consistent with our mission, this initiative demonstrates our commitment to play our part in contributing towards medical options available to families who have stored their children’s cord blood.”

Said Dr Proshanto Chowdhury, technical director of Cordlife India: “It’s not necessary that they bank their umbilical cord blood with us. If they have banked with other organisations, public or private, we will allow the kids afflicted with autism to get these benefits.”

The company has secured regulatory approval from the Indian Council of Medical Research (ICMR) for the trial, which will begin shortly. But those who have not stored their umbilical cord blood will not qualify for the trial. According to doctors, only autologous umbilical cord blood (one’s own cord blood) is safe and ethical for use. This disqualifies Rajiv as his umbilical cord blood was not banked.

Chez is optimistic but does not guarantee cure. “The mechanism of umbilical cord blood stem cells may moderate or change certain things which add to the clinical symptoms of autism,” said Chez. “We believe this mechanism may undo some of the damage or dysfunction in the brain. But none can claim currently that stem cell therapy will definitely cure autism. But that is what the trial is all about. We are very optimistic.”

The history of medical science owes some of its fantastic human survival stories to stem cell therapy. Some 27 years ago, in one of the first umbilical cord stem cell transplants, a boy was cured not only of his blood disorder for which the treatment was administered but also his critical heart disease.

“We have numerous such success stories which in fact gave us strength to go for human trials,” said Proshanto Chowdhury. “The patient would get almost a new brain as the cells and blood vessels would be new and if they are suffering due to damaged brain cells, the chances are high that they would be cured through this process.”

Chez claimed that recent human trials around the world have suggested that IQ levels of the patients improve manifold and they respond quickly to various social messages in a few days of transplantation. Said he: “All results have shown quick recovery but we could not claim they have been cured medically. That can only be said once the study is finished and research is completed following human trial.”

Not all human stem cell trials have been free from adverse events. Fourteen of the eighteen autistic participants of Chez’s trial last year showed adverse reaction after transplantation with one showing serious reaction. All survived. The reactions are usually dark urine, gastrointestinal symptoms and fever with vomiting, in extreme cases. Chez believed that Indian hospitals have enough facilities to handle the post-transplant adverse reactions. According to Cordlife India, there would be multi-sector research with the findings of the human trials. The research would determine if such application will be part of treatment for autism in India.

Rajiv is now seven years old and undergoing physiotherapy. His doctors know he may not be completely cured. Neither do they claim physical exercise will improve his condition. But they know it is for the better when they see him interact with children his age and sometimes respond positively.

* The umbilical cord blood stem cells are suspended in 100ml saline and injected through a peripheral vein. Some doctors prefer not to use saline suspension. It is infused through the intrathecal route but the intravenous route is equally efficacious and is less risky and painful
* Infusion of cord blood stem cells leads to neural regeneration, new vascularisation
* Symptoms of autism will likely go away with the formation of new nerve cells

* Autism is a neurodevelopmental disorder, characterised by difficulties in social communication and interaction and restricted and repetitive patterns of behaviour.
* Causes include genes and environmental factors. The symptoms are visible by age two.
* Boys are at a higher risk than girls, thanks to genetics.
* There are screening tests that can be done between 18 months and age two.
* Though autism cannot be cured, certain therapies can help like behavioural management therapy, cognitive behaviour therapy and speech-language therapy.

Interview/ Dr Michael Chez
No claim of cure

Dr Michael Chez

How many children are autistic around the world?

Incidence varies but in western Europe and the United States it is increasing every two years. Latest [figures] in the US shows 1:68 overall, 1:186 girls, 1:42 boys. These are similar or at least 1:100 children by age 6 globally.

Some Asian and African cultures may under-report but in India there are now at least 1:200 children or higher. Singapore around 1:217, and China and the Philippines between 1:100 to 1:200. In India, 5-10 million children is the conservative estimate.

How does cord blood stem cell cure autism?

This is not what anyone claims currently—that stem cell therapy will definitely cure autism. What we hope is that autism has many causes and some subsets of patients may respond more than others. Epilepsy is similar with many causes and genetic risks.

We prefer to think that mechanisms of umbilical cord blood stem cells may moderate or alter certain things that may add to the clinical symptoms of autism. We think these mechanisms may undo some of the damage or dysfunction.

Current research is trying to assess if there is a way out and, in addition, which type of children can respond to umbilical cord blood. In future what we learn to safely do with stem cells may be specific to some genetic or other autism subtypes and be even more effective.

What treatment is currently available for autism? Can surgery improve the child’s condition?

There are no definite neurosurgical methods to help, unless it is a rare case of hydrocephalus or Chiari [malformation] that has early autism delays but responds to improved brain pressure. However, this is an extremely small subset of patients. But in reality [there are] no definite neurosurgery methods to help at this time. In addition, we mainly treat co-morbidities with psychiatric medications, epilepsy drugs, behavioural or sleep medications. There are no specific language treatments or socialisation therapies except the mainstay of applied behavioural therapy like ABA [Applied Behaviour Analysis] and speech, play and supportive therapies.

How much research has been done to determine the effectiveness of cord blood stem cell transplantation for autism?

Doctors say, in 80 diseases so far which respond to stem cell therapy, the success rate is not above 8 per cent.
Research is just starting and is hopeful.

My study will be among the first reported this year. We have some numbers of case reports in a published Chinese study showing positive outcomes, but none claims cures. Language and IQ may improve, social or toy interests also; however, no one yet reports curing autism to date. Safety, especially with autologous umbilical cord blood, seems to be definite.

Does this promise a trouble-free future for the autistic child?

This again will vary from case to case as not all children have the same degree of impairment or IQ levels. No one can promise anyone a trouble-free life with or without autism.

However, autism patients are not being promised independent adult life with any current therapy. All we can hope is that any improvement from cord blood may lead us to find better ways to treat the condition.

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