An international clinical study demonstrated astounding recovery of patients with multiple sclerosis after a stem cell transplant. Scientists claim these transplants are dramatically better than standard drugs at halting the spread of multiple sclerosis and improving its symptoms.
Multiple sclerosis (MS) is a lifelong condition which can affect the brain and/or spinal cord. In most cases, it causes problems with vision, arm or leg movement, sensation or balance and reduces life expectancy. A global study in 2013 estimated the number of people with MS at 2.3 million.
This study compared hematopoietic stem cell transplantation (HSCT) with drug treatment. HSCT involves: 1) collecting blood-cell-producing stem cells from bone marrow or blood, 2) wiping out a patient’s immune system using cancer (chemotherapy) drugs, 3) restoring the immune system with a stem cell transplant.
Prof Richard Burt, the lead investigator from Northwestern University Chicago, said to BBC: “The data is stunningly in favor of transplant compared to current best available drugs – the neurological community has been skeptical about this treatment, but these results will change that.”
The process is suitable for patients with relapsing-remitting MS – where attacks or relapses are followed by periods of remission. Scientists relied on findings from an earlier study on patients with relapsing-remitting MS. The process itself can be exhausting and few weeks in an isolation room whilst immune system rebuilds is obligatory. 110 patients took part in the trial, in hospitals in Chicago in the US, Sheffield in the UK, Uppsala in Sweden and Sao Paolo in Brazil.
The provisional results were released at the annual meeting of the European Society for Bone and Marrow Transplantation in Lisbon. They are yet to be published in a peer-reviewed journal.
Prof Basil Sharrack, the neurologist at Royal Hallamshire Hospital, told BBC: “This is interim analysis, but with that caveat, this is the best result I have seen in any trial for multiple sclerosis.”
After one year, only one relapse occurred among the stem cell group compared with 39 in the drug group. After three years, the transplants had failed in three out of 52 patients (6%), compared with 30 of 50 (60%) in the control group. Altogether, transplant group experienced a reduction in disability and the drug group symptoms progressed.
Prof John Snowden, director of blood and bone marrow transplantation at Sheffield’s Royal Hallamshire Hospital, told BBC: “We are thrilled with the results – they are a game changer for patients with drug-resistant and disabling multiple sclerosis.”
The stem cell therapy might get patients off lifelong treatments and provide them with results that have never been achieved before. The transplant costs around £30,000 at this time, about the same as the annual cost of some MS drugs. Still, scientists have many obstacles to overcome in treating MS. Hopefully, they will find a way to lower possibility of infections and toxic side effects as well as find proper treatments for other types of MS.
Director of research at the UK-based MS Society Dr. Susan Kohlhaas said: “The MIST results are important and show this area needs further research. While HSCT appears to be effective for some people with MS, it remains a high-risk treatment that won’t be right for everyone. We now need to know how HSCT compares to existing, less aggressive, MS treatment options.”
The BBC filmed one study patient, Louise Willetts undergoing her transplant in October 2015 and her recovery.
Louise is now symptom-free. “The worst time was not being able to get out of bed because I had no stability in my body – I struggled to walk and even spent time in a wheelchair. MS also affected my cognition – it was like a brain fog and I misread words and struggled to keep up with conversations,” she said for BBC.
Learn more about the trial in the video below:
By Andreja Gregoric, MSc