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Neural stem cells, mesenchymal stem cells, induced pluripotent stem cells, oh my!
These may be intimidating terms, but with Dr. Atul Malhotra’s help, they don’t have to be! A neonatologist at Monash Newborn, Monash Children’s Hospital and research academic at Monash University, Dr. Malhotra joined Hope for HIE in a live Q&A to break down the complex topics of cell therapies and early detection measures for cerebral palsy.
Here are our key takeaways:
To better comprehend the role of stem cells and cell therapies in treating and understanding HIE, we need to understand what stem cells are. Dr. Malhotra says stem cells [cells that come from the human body] have two main properties: the ability to self-renew [create more cells when one is dying] and the ability to differentiate [turn into different cells of various organs or tissues].
Stem cells, though, are not exclusive to cell therapies, which involve the transplantation, injection, and/or administration of human cells into a human body or tissues that are damaged. Other biological cells are being studied for these purposes, and while they may not have the ability to self-renew or differentiate, they do have the potential ability to repair, regenerate, or protect a damaged organ.
Right now, therapeutic hypothermia – the process of lowering the body temperature to 91 degrees Fahrenheit for around 72 hours to help the body focus on healing the brain – is the standard of care for those who meet its strict criteria, including the timing of implementation [within six hours] and how sick a baby is when born with HIE [moderate to severe presentation]. It is not a cure, but it can lower death and disability.
Cell therapies, like the studies being done with cord blood, may be beneficial in conjunction with cooling or for babies outside of that treatment window.
Doing this, of course, takes time, funding, and the right infrastructure: while Dr. Malhotra recognizes the progress made and that “we’re getting there,” there’s a lot more work to be done.
Speaking of work being done, Dr. Malhotra and his group at Monash University in Australia are not the only ones studying the effects of various stem cells and other biological cells on repairing or protecting damaged organs and tissues, like the brain. Research teams from all over the world – like the US, the Netherlands, Denmark, South Korea, China, and Japan – are all communicating and collaborating on their findings.
Through their work, they hypothesize that there are three types of cells that may be considered for treating HIE: the first is cord blood cells, which are derived from the umbilical cord and placenta; the second are cells derived from the bone marrow; the third is called induced pluripotent stem cells. These cells do not have stem-like properties to start, but they can be induced to self-renew and differentiate.
When it comes to the cell types being studied by these teams, there is a difference between those that are showing the most promise [showing the best benefits in the lab] and those that are most advanced in the pipeline of clinical research [closest to clinical use].
Cord blood, rich in the source of stem cells and other cells used in cell therapies, is the most advanced in the clinical trial pipeline. Active studies are further along that show the effectiveness of cord blood in treating preterm injuries and neonatal stroke because these occurrences are higher in the population than in those with HIE.
Like other research with HIE, cell therapy research begins with smaller non-human, lab-based trials, including various animal models. Typically, these trials start with an established “mouse model” and work up to bigger animals before trialing in healthy adults. It is then that trials make their way to HIE babies. Safety is the top goal and is prioritized before the ability to fully test and evaluate the effectiveness of cell therapy treatments.
Not to worry, though, for Dr. Malhotra reminds us that progress, while perhaps slow, is still being made. For instance, it took the practice of therapeutic hypothermia nearly 15 years before it was put into clinical use.
So, while there are currently no big clinical trials for cell therapies and HIE, smaller trials are working on modifying the protocol to assess the most beneficial timing and dose of cell administration. To become aware of these studies, Dr. Malhotra first recommends starting a line of communication with your child’s healthcare professional or medical-care team. In the meantime, there is information on the Clinical Trial Registry page that you may find helpful, the link for which is below.
Any clinical trial your family is interested in registering for should undoubtedly be FDA-regulated, meaning they are designed and conducted according to federal law and sound, evidence-based clinical practices. They should also have an ethics board or committee that responsibly manages the trial and assess its risk-to-benefit ratio. Last but not least, make sure that you ask what type of cell is being administered for the treatment since – as you may recall – stem cells are not always the ones used in cell therapies.
In addition to studying cell therapies and their role in treating various types of perinatal brain injuries, Dr. Malhotra is passionate about researching early detection measures of cerebral palsy. With the most recent standardized assessments in place, he says providers can officially diagnose cerebral palsy as early as 3-months of age. This is impressive, especially considering that these diagnoses used to happen closer to 18 months or 2 years of age.
An early diagnosis is critical because it sooner opens the door for early interventions to take place. While 3 months is too young to determine the baby’s learning or behavior outcomes, a doctor can assess his or her physical outcomes and offer referrals for physical or occupational therapy, etc. The earlier these therapies begin, the better: when babies are young, their brains are more plastic [moldable] and can easier adapt to the resources they have.
There are resources like Save the Cord Foundation, the Stem Cell Network, and the Umbilical and Placental Cell News that can guide you to more information about the current state of research and cell therapies. Dr. Malhotra also says that his own institution’s research is available via the Cell Therapy and Regenerative Medicine page, which is also linked below.
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