[sammy65Participant]

Oh, and regarding Scottish medicine approval system, I have checked information available on the net. It appears that before a medicine can be prescribed in Scotland it has to be accepted by Scottish Medicine Consortium SMC, however the licencing of medication falls under the UK licencing body for the UK, the Medicines and Healthcare products Regulatory Agency (MHRA). SMC cannot approve a medication unless it has been licenced in the UK. I think SMC is equivalent of NICE in Scotland.

[sammy65Participant]

I was diagnosed one year later than you, in January 2022 followed by autologous transplant in June 2022. My disease was caught early, but I had one small lesion on T4. That lesion has healed after treatment to form a fatty tissue in the hole. The appearance of my bone marrow has changed from variegated to smooth. So, the medicines do work, but at what side effects and for how long. I would imagine your bones also healed, but because during the healing the bone does not regrow unfortunately, you must take extra care. I am still in remission, but it’s called very good partial remission. It reminds of school grades. It means I have a constant ‘safe’ amount of paraprotein left (if it goes up, I relapse), I take lenalidomide maintenance which isn’t easy. To be honest, I am disappointed how fatigued I am, and nothing can be done about it, I have found out that doctors can’t really treat this type of fatigue. I returned to work full time after one year, nearly two and a half years ago. In my measures, I have been lucky to have returned to work, but no one would bat an eye lid if had not. What concerns me is the lack of innovative potentially low side effects treatments in the UK, such as the CAR-T therapy, which also provides a deep remission to many. I am aware that bispecific have been approved on NHS, but these have huge side effects probably incompatible with working. The recent Trojan horse treatment, aka belantamab mafodotin is the latest addition over here after it has been retested and dusted off the shelf. It can cause a severe vision damage, so it is working, but at a very high cost of harsh side effects. It is a very unkind medication selection. At the end of the day the UK is lagging most of the world in Car-T treatment, it is a sad fact, no trojan horse will make it look better. Three specifics maybe on the horizon, as they are being tested in a trial by Dr Poppat, London based professor. There are three Car-T trials in the UK- all of which are randomised to standard type of medication, so 50-50 chance that a patient will get Car-T. One is with added chemo in both groups; one trial is for newly diagnosed only. I hope that ice will thaw on Car-T approval, I also hope that it will get approved in Scotland, it might get approved first in Scotland, however Car-T is not yet licenced in the UK for treatment of myeloma. These are sad times.

[sammy65Participant]

Hi, I was diagnosed in 2022 and have been very hopeful for the Car-T cell therapy to come. However, its approval in the UK could not be completed because the manufacturer did not have confidence in NICE approval process and withdrew from the deal. This new era of cell therapy treatments for myeloma has been used in the USA and non-brexit Europe for some time now. Write to your local MP as I have done to keep knocking on the thick gate. I have written to NICE and they were not helpful, just a factual ‘no’.

[sammy65Participant]

Hi. Depends what test the GP booked. Light chains quantitative test is not a single myeloma diagnostic test. When you have increased or low light chains, doctor may order a special test to check what those light chains are doing. There is another part of the chains called heavy chains which will show if they were secreted by cancerous bone marrow. The GP may check your urine for Jones Bence protein and order electrophoresis test. That test will find out if monoclonal heavy chain/paraprotein is present. If positive for paraprotein you will be referred to haematology under cancer pathway for further investigation. There is a 2% chance that people have myeloma without secreting light or heavy chains affected by cancer, however the GP will monitor you to find out. I was initially not as suspect for myeloma, not anaemic, bones were fine, no pain, normal kidneys and calcium, but was fatigued; and paraprotein test showed suspect for myeloma.

[sammy65Participant]

Hi Anne,

I understand that the provider has withdrawn their proposal due to manufacturing issues of Car-T in UK. As you have said, the reasons are more complex than the one major reason. I would like to know what happens next to making Car-T available as treatment protocol in the UK. I have not looked at Car-T in US in detail as I am aware that it has been available in US for some time. The EU case is more interesting as it is more recent and its treatment ‘lines’ are similar to UK. Car-T has become available to EU patients recently, in February 2024 for people who who have received at least 1 prior therapy.

It was approved on the basis of a clinical trial which compared a medical specialist’s choice of either pomalidomide, bortezomib and dexamethasone (PVd) or daratumumab, pomalidomide and dexamethasone (DPd) until disease progression. It was found that people who received Car-T had a stronger and deeper remission compared to people in the standard treatment group.

Here is the link for Cartitude 4 trial: https://www.nejm.org/doi/full/10.1056/NEJMoa2303379

Regarding the side effects, Car-T has fewer side effects on immunosuppression than standard medications. Its main side effects are during administration of the treatment.

I would like to know if Myeloma UK is undertaking some actions on the government level to make Car-T available in UK. There is clearly a gap for a treatment with less immunosuppressive side effects that is already on the market. For people who are on lenalidomide maintenance like myself, neutropenia side effect can lead to breaks in the treatment and premature death. Most standard medications have this side effect, so Car-t can in principle give people a form of respite from those traditional side effects while keeping myeloma supressed.

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