Friday, 11 April 2014

Update 39 - Is HSCT the answer for CIDP?

Being well aware of the limited success of recovery from CIDP, I have been trying to find out about the muted alternatives such as HSCT (haematopoietic stem cell transplantation) can be seen as AHSCT (Autologous!).

Now there seems to be a lot written about HSCT being a miracle cure and there appears to be a large amount of smoke and mirrors going on, however these claims seem to be promising at best and dubious at worst.  Yes there is proof that trials have successes against all diseases, but there have also been issues.  In the U.S. limited trials appear to being performed, into MS & CIDP etc.  There also seem to be a significant cost to these treatments and significant risks (that are missed by certain clinics/practices).

I have currently found a number of articles/posts that I believe sum up the current situation:

Report from the US June 2013:

Only 70-80% of patients with chronic inflammatory demyelinating polyneuropathy (CIDP) respond satisfactorily to the established first-line immunomodulatory treatments. Autologous haematopoietic stem cell transplantation (AHSCT) has been performed as a last treatment resort in a few therapy-refractory cases with CIDP. We describe the results of AHSCT in 11 consecutive Swedish patients with therapy-refractory CIDP with a median follow-up time of 28 months.
Case data were gathered retrospectively for AHSCT treatments in 11 patients with CIDP refractory to the first-line immunomodulatory treatments, intravenous high-dose immunoglobulin, corticosteroids and plasma exchange and to one or more second-line treatments used in 10 of the 11 patients.
The median Inflammatory Neuropathy Cause and Treatment (INCAT) score within 1 month prior to AHSCT was 6 and the Rankin score 4. Total INCAT and Rankin scores improved significantly within 2-6 months after AHSCT and continued to do so at last follow-up. The motor action potential amplitudes (CMAP) improved already within 4 months (median) after AHSCT. Three of the 11 patients relapsed during the follow-up period, requiring retransplantation with AHSCT in one. Eight of the 11 patients maintained drug-free remission upon last follow-up. AHSCT was safe but on the short term associated with a risk of cytomegalovirus (CMV) and Epstein-Barr virus reactivation, CMV disease, haemorrhagic cystitis and pancreatitis.
Our results though hampered by the limited number of patients and the lack of a control group suggest AHSCT to be efficacious in therapy-refractory CIDP, with a manageable complication profile. Confirmation of these results is necessary through randomised controlled trials.

Information from DIADS web site into MS

Hope not hype
Despite the best efforts of researchers, stem cells are often portrayed as a miracle cure, and many MS patients who learn about stem-cell therapy from breathless newspaper articles or television features face let-downs. Even the clinicians had to learn some hard lessons in early studies. "In our HSCT clinical trial, we started with patients who had fairly advanced disease, wheelchair-bound or even bed-ridden," says Richard Nash, a specialist in immunotherapy at the Colorado Blood Cancer Institute in Denver, Colorado "and we found that for many of these patients, even after transplantation they are going to continue to get worse."
The outcomes of subsequent trials have been markedly improved by the recognition that patients with advanced MS might have crossed a threshold of nervous-system degeneration beyond which anything short of neuronal regrowth or replacement is likely to fail. This can be a difficult message for individuals with severe MS to hear. "The only time that patients get mad at me is when we don't offer the transplant," says Burt. "It's hard to get patients to understand that this isn't going to help them."
In some cases, patients have pursued treatment at so-called 'stem-cell clinics' around the world, where they pay tens of thousands of dollars to be injected with cells of dubious provenance in an environment with minimal regulatory oversight. These clinics claim to treat any number of conditions with stem cells but offer little in the way of peer-reviewed efficacy data. At least one published report described a patient who developed tumours after a clinic transplanted fetal stem cells, and in May 2011 Germany shut down the XCell Centre, where a young patient died from complications following autologous MSC transplantation. More recently, a case report from a team of neurologists in Arizona described how a teenage MS patient suffered a severe and debilitating inflammatory response during a course of stem-cell treatments at a clinic in Costa Rica. The physicians were unable to determine the extent to which this strong immune response was attributable to the transplant, but they cite this example as justification for restricting experimental stem-cell treatments to clinical trial settings with proper oversight and safeguards. "We are really fighting those clinics," says Martino, who has collaborated with colleagues in a survey of clinical stem-cell research in MS. "We prepared this leaflet that anybody can easily download from various MS society websites, where we explained exactly what stage we're at with the different types of stem cells."
Most stem-cell researchers see cause for optimism but point out that good science and good medicine require considerable amounts of both time and effort. "The stem cell you use and how you use it will depend on the disease you're treating as well as the stage, and it's just beginning," says Burt. "When I first started I thought I'd have all the answers in five years. But it doesn't work that way — it takes time."

YouTube Web Link to a CIDP story

To me - all this says progress is being made, but there are still issues and not enough is known/proven.  From an outside perspective it is easy to look at all the good news stories and headlines and go rushing to a clinic and signing up for $40,000??? to go through this, to recover from CIDP.  But the headline from the MS story of Hope not Hype sums it up.

Maybe it is because I have got a reasonably normal life back, so I am not desperate to be cured?  Others will think the risks and the costs are worth it - and good luck to them.

If there is more relevant stuff out there, I will include it and I am still digging!