Compassionate-use experiments have revealed an extraordinary result. It looks like stem cell treatments may provide an effective way to ease muscular dystrophy. While stem cells can’t cure patients’ underlying genetic condition, their many proven therapeutic properties might help ease the symptoms. So what’s the story?
What is ‘compassionate use’ in drug testing?
‘Compassionate use’ means making a new, unapproved drug available to a seriously ill patient when there are no other treatments. The drugs are usually being tested but haven’t yet been approved, and they’re often called ‘investigational drugs’.
What is muscular dystrophy?
Muscular dystrophy is an umbrella name covering a number of muscle diseases caused by genetic mutations, where the muscles steadily become weaker. Because more than 30 separate genes are involved the origin of these diseases, it’s complex, and that’s why there’s very little available in the way of drugs to treat the condition.
There are many types of muscular dystrophy and each kind affects a different group of muscles. The symptoms vary from mild to severe and can be treated or even reversed, but not cured. In some cases the disease can be life threatening or fatal, ending in either respiratory failure or cardiomyopathy, a disease of the heart muscle.
An exciting compassionate-use study involving stem cells
One study has revealed ‘promising results’ from treating muscular dystrophies with mesenchymal stem cells from a substance called Wharton’s jelly. Taken from the umbilical cord, this is also called WJ-MSCs. The Polish study achieved ‘significant improvement’ across a number of muscles for most patients, and there weren’t any dangerous side effects.
While using WJ-MSCs in neurological patients remains controversial, the research proves stem cell therapy testing is a reasonable experiment under the circumstances. And this brings hope to people suffering from Muscular Dystrophy.
Current treatments for muscular dystrophy
Current treatments for the disease include supportive care and drugs. Steroids have proved incredibly useful but can have side effects that are hard to handle, things like severe weight gain, delayed puberty, behaviour problems and bone fractures.
The results of the stem cell research
The study took place in real-life settings and involved 22 people with different types of muscular dystrophy.
- The ratio of men to women was 50-50
- The median age was 33
- People were given between one and five intravenous and/or intrathecal (spinal column) injections per course of treatment
- They were given 1-2 two courses every two months
- The team assessed people’s muscle strength using CQ Dynamometer computerized force meters
The patients experienced significant improvement in several body muscles, including limbs, hips, elbows and shoulders. In the most successful case the patient could move without a crutch, call a halt to their rehabilitation, and take on a full-time job.
The results are impressive, but doctors say it’s still too early to confirm MSCs as a treatment for muscular dystrophies. There are several unknowns to be resolved first:
- How long the effects last
- Whether the treatment will need to be repeated regularly
- The exact treatment protocol over the long term
- The potential synergy with existing drugs and rehab
The team concludes that their results are ‘cautiously encouraging’, especially since there aren’t any other effective treatments. In the words of Anthony Atala, M.D and Editor-in-Chief of STEM CELLS Translational Medicine, “We look forward to the continuation of this research to further document clinical efficacy.”
Umbilical cord stem cells hold out hope in so many ways
The unusual power of umbilical stem cells is being tested around the world in all sorts of ways. These cells keep on delivering hope to people who suffer from a wide variety of conditions. When you save your cells during the birth of your child, and we store them for the future, you’re investing in potential cures for your child and other family members for decades to come. That’s what we call magic. Have you considered umbilical stem cell storage?
Ref: The use of umbilical cord-derived mesenchymal stem cells in patients with muscular dystrophies: results from compassionate use in real-life settings by Beata Świątkowska-Flis, Izabela Zdolińska-Malinowska, Dominika Sługocka and Dariusz Boruczkowski, 27 July 2021, Stem Cells Translational Medicine.
BSc (Hons) Microbiology
Biovault Family CEO, Kate Sneddon, joined Biovault in July 2009 and became Chief Executive Officer in 2016. As health industry professional her experience includes working as a microbiologist and leader at GSK for over 10 years. Her expertise in cord blood banking has been recognised in her awards, features in Parliamentary Review and Parents Guide to Cord Blood, as well as contributions to research with UCL and others.