April 8, 2021
As we have reported, it is really exciting that the first anti-BCMA CAR T cell-therapy for myeloma, Abecma, was approved by the U.S. Food & Drug Administration. This very active new immune therapy is now available for patients with relapsed or refractory multiple myeloma who have received four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody.
Abecma will be available only at designated centers in the U.S.
According to news reports, bluebird bio Inc. and Bristol Myers Squibb have set the wholesale list price for Abecma at $419,500. However, the out-of-pocket expenses for patients will most likely be very manageable through a variety of payers. At the same time, the Institute for Clinical and Economic Review (ICER) released a report with a much lower recommended pricing, based on an arbitrary $150,000-per-Quality-Year-of-Life assignment, which remains a controversial methodology.
It remains to be seen how the rollout of this exciting and important new therapy option proceeds. What is very clear is that for patients, the potential for deep and potentially lasting treatment-free remissions is extremely attractive.
Additional data from the second anti-BCMA CAR T-cell therapy were presented at the Virtual 47th Annual Meeting of the EBMT (European Society for Blood and Marrow Transplantation). This was part of what is called a rolling submission for a biologics license application to support the approval of the investigational BCMA-directed CAR T-cell therapy called “cilta-cel” (ciltacabtagene autoleucel) for the treatment of relapsed/refractory myeloma. This CAR T-cell therapy has produced an overall response rate of 96.9% with 67% stringent CRs, with 33 patients achieving MRD negativity (57.9%). With a toxicity profile similar to Abecma, it is wonderful to have a second CAR T-cell therapy moving forward in the approval process. No dates have yet been released for potential CARTITUDE-1 trial data review and approval.
As of April 8, 2021, 171 million COVID-19 vaccine doses have been administered in the U.S., according to the CDC COVID Data Tracker. Approximately 76% of those over 65 have received at least one dose of vaccine and over 56% are fully vaccinated. This is really great news and moves us ever closer to a high level of immunity in community settings. A recent article in the New York Times, “What Can You Do Once You’re Vaccinated?,” provides excellent guidance about how to assess your risk as community activities re-open.
Although all vaccines continue to be safe, there is one caution about the AstraZeneca vaccine regarding blood clots. Although rare, very unusual blood clotting has been noted in the brain sinuses as well as the blood vessels linking to the spleen in the belly area (not typical areas for blood clots). There were 62 cases, mostly in women under 60 and within 2 weeks of vaccination. Although this is not enough to halt vaccinations, it is an important alert for health-care workers.
For myeloma patients there has been a natural concern that COVID-19 vaccination could actually trigger the myeloma (or even MGUS or SMM). However, there have been no such reports of triggering myeloma or any underlying disease, although some temporary increases in immunoglobulin and/or light chain levels have been noted. Careful tracking post-vaccination is recommended to make sure there is no major impact. IMF staff members, including our InfoLine team, and myeloma support group leaders are on alert to be aware of any such emerging issues should they occur and keep everyone informed.
As the vaccines roll out, new COVID-19 variants continue to emerge. The B-117 variant, which originated in the U.K., is now widespread in the U.S. and is clearly more infectious and dangerous. This new virus strain, now the most common strain in the U.S., is linked to increasing numbers of COVID-19 cases in five states: New York, Michigan, Florida, Pennsylvania and New Jersey. These cases account for 43% of all new cases in the U.S. now (196,400 cases out of 453,360 cases).
Lately, new patterns of spread involving younger people and group sports activities are occurring. In a very sad case in Minnesota, a myeloma patient became infected after returning to work as a youth hockey referee. This highlights the need for myeloma patients to continue to be cautious, even if vaccinated. Antibody response levels may be low in patients receiving ongoing myeloma therapy, and emerging regional COVID-19 variants may be especially dangerous. Please continue to wear masks to avoid community exposure.
In addition, what are being called double variants are occurring in California, New York and probably elsewhere. These appear to be particularly dangerous.
It is SO important to limit and control what is currently the beginning of a fourth surge. Local “super spreader” events can occur with the re-opening of indoor events such as sports practices and stadium events, where viruses have the ability to grow and morph into new variants that threaten everyone in the community.
Some good news
Additional vaccines are being developed for broader use, especially outside the U.S. One new vaccine is called NDV-HXP-S. This can be grown easily in chicken eggs, just like the flu vaccine, making it much cheaper and globally accessible. Clinical trials are currently ongoing in Brazil, Mexico, Thailand and Vietnam. The need to equitably distribute vaccines around the world and control COVID-19 infections cannot be overemphasized.
RESEARCH DURING THE PANDEMIC
Myeloma research remains very active during the pandemic. Next week I will survey some amazing new myeloma research which can help move us closer to a cure. This week comes thought-provoking news from out there in the world of physics. It could answer a central question: “Is outer space empty or is it filled with dark matter that is an alternate reality or universe with lots going on (which is important for us to know)?”
This new research results suggest that a muon—which is similar to an electron (part of all atoms in our bodies) but 207 times heavier with a magnetic spin—can possibly provide an answer. This is especially exciting for everyone because the results were blinded to avoid any unintended bias. When the coding envelopes were opened, the researchers were stunned to see the positive result.
It seems the muon is reacting to something in dark matter which can be truly revolutionary: the first window into a whole unseen world around us! (I recommend Frank Close’s book, Antimatter, for a short look at this fascinating topic.) To give a practical example, PET scanning, which many myeloma patients have had, results from a reaction between matter (the bone marrow) and antimatter (unseen). This allows us to visualize the presence of myeloma.
Who knows where the new research on muons will lead?
ADORABLE PINK DOLPHINS RETURN
The beautiful pink dolphins in Hong Kong harbor are returning. Due to the pandemic, there is much lighter cross-channel ferry activity and likely less pollution in the Pearl River that feeds into the harbor, resulting in 30% or more dolphins. The dolphins are pink because they have more blood vessels close to the skin, which helps lower their temperatures while swimming in warmer waters.
It is always great to look for a silver lining (in this case pink!), which can help us retain a positive attitude and resilience in the face of many challenges. Let’s stay focused on the notion that we will get through this pandemic together. And with increasing vaccination, a higher community immunity will start to save us all.
Dr. Brian G.M. Durie serves as Chairman of the International Myeloma Foundation and serves on its Scientific Advisory Board. Additionally, he is Chairman of the IMF's International Myeloma Working Group, a consortium of nearly 200 myeloma experts from around the world. Dr. Durie also leads the IMF’s Black Swan Research Initiative®.