Personalized Cancer Vaccine for Multiple Myeloma

Overseen By Amishi Vora

Age: 18+

Sex: Any

Trial Phase: Phase < 1

Sponsor: M.D. Anderson Cancer Center

Must not be taking: Corticosteroids, Chemotherapy, Radiotherapy, others

Disqualifiers: Myeloma events, Plasma cell leukemia, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 16

Duration: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to explore the side effects of a personalized vaccine designed to help the body's immune system fight cancer cells in patients with smoldering multiple myeloma, a type of blood cancer. The trial has two parts: in one, participants receive a custom-made vaccine; in the other, they receive the vaccine along with lenalidomide. It suits individuals diagnosed with smoldering multiple myeloma who have a higher risk of disease progression. Participants must provide blood and bone marrow samples to create the personalized vaccine. As an Early Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the chance to be among the first to receive this innovative therapy.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it does not allow treatment with corticosteroids (except for low doses) or prior chemotherapy for smoldering multiple myeloma. Bisphosphonates are allowed.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that personalized cancer vaccines are generally safe. In one study, the vaccine did not cause serious side effects, and many participants lived longer. Another study found the vaccine safe and practical for patients with smoldering multiple myeloma, a condition where unusual cells grow in the bone marrow.

When combined with lenalidomide, a drug commonly used for multiple myeloma, the vaccine was also well-tolerated. Studies have reported long-lasting remissions without major side effects. Lenalidomide is already approved for treating multiple myeloma, which adds confidence in its safety.

Overall, evidence suggests that both the personalized vaccine alone and with lenalidomide are generally safe for patients. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about these treatments because they offer a personalized approach to fighting multiple myeloma. Unlike standard treatments like chemotherapy or stem cell transplants, the personalized cancer vaccine is tailored specifically to each patient's unique cancer profile. This means the immune system is trained to target and destroy the cancer cells more effectively. In Stage II, the addition of lenalidomide enhances the vaccine's impact by boosting the immune response further. This personalized strategy not only promises to be more effective but also aims to minimize side effects compared to traditional therapies.

What evidence suggests that this trial's treatments could be effective for multiple myeloma?

Research has shown that personalized cancer vaccines can help the immune system fight cancer. In this trial, participants will receive a personalized vaccine, with some receiving it alone and others in combination with lenalidomide. Some studies indicated that patients who received these vaccines did not experience serious side effects, and many lived for several years. When combined with lenalidomide, previous studies did not show a significant increase in complete recovery rates, but it did help stabilize the disease in several patients. Although these findings are preliminary, they suggest that personalized vaccines could be promising for treating multiple myeloma, especially when used with other treatments. More research is needed to confirm these effects. ^{1 2 5 6 7}

Who Is on the Research Team?

Krina Patel

Principal Investigator

M.D. Anderson Cancer Center

Are You a Good Fit for This Trial?

This trial is for adults with smoldering multiple myeloma at intermediate or high risk of progression. Participants must have certain levels of plasma cells, kidney function, and blood counts, and be able to consent. Excluded are those with active myeloma, prior SMM treatment, other cancers, infections like TB or hepatitis B/C, pregnant women, and people with HIV.

Inclusion Criteria

My condition is confirmed as smoldering multiple myeloma without any signs of further complications.

I am at intermediate or high risk of my condition worsening to multiple myeloma according to specific health criteria.

I have a diagnosis of smoldering multiple myeloma at an intermediate or high risk.

Exclusion Criteria

I have been diagnosed with plasma cell leukemia.

I have not had systemic treatment for SMM, including the prohibited ones.

Pregnant or lactating females

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Patients receive personalized vaccine subcutaneously on days 1 and 15 of cycles 1-2 and on day 1 of cycles 3-6. Treatment repeats every 28 days for up to 6 cycles.

24 weeks

12 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

4 visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Vaccine Therapy

Trial Overview The study is testing a personalized vaccine made from the patient's own blood and bone marrow to see if it can stimulate the immune system to destroy cancer cells in patients with smoldering multiple myeloma. It also involves radiography of biopsy specimens and Lenalidomide therapy.

How Is the Trial Designed?

Group I: Stage II (personalized vaccine, lenalidomide) Experimental Treatment 3 Interventions

Patients undergo collection of blood and bone marrow for making the vaccine. Patients then receive personalized vaccine SC on days 1 and 15 of cycles 1-2 and on day 1 of cycles 3-6. Patients also receive lenalidomide PO on days 1-21. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity.

Group II: Stage I (personalized vaccine) Experimental Treatment 2 Interventions

Patients undergo collection of blood and bone marrow for making the vaccine. Patients then receive personalized vaccine SC on days 1 and 15 of cycles 1-2 and on day 1 of cycles 3-6. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity.

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials

3,107

Recruited

1,813,000+

Published Research Related to This Trial

Chimeric antigen receptor T cells targeting the BCMA protein show promise as a potent treatment for multiple myeloma, based on preliminary results from an ongoing phase I clinical trial in China.

The therapy was well tolerated by patients and led to complete and durable responses in those with relapsed or refractory multiple myeloma.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sending CAR T Cells After Multiple Myeloma. [2018]

In a study of 44 patients with multiple myeloma, the two-dose SARS-CoV-2 mRNA vaccination showed low rates of adverse reactions, similar to those seen in general vaccine trials.

A high percentage of patients (93% on therapy and 94% not on therapy) developed detectable antibodies after the second dose, indicating strong immunogenicity and providing reassurance for vaccine-hesitant patients with multiple myeloma.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and antibody response to two-dose SARS-CoV-2 messenger RNA vaccination in patients with multiple myeloma. Greenberg, RS., Ruddy, JA., Boyarsky, BJ., et al.[2023]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11331005/

Advances and challenges in anti-cancer vaccines for multiple ... Myeloma vaccination has so far shown limited clinical efficacy. However, further studies are essential to optimize various aspects, including antigen and ...

2. mountsinai.org mountsinai.org/about/newsroom/2025/personalized-cancer-vaccine-proves-promising-in-a-phase-1-trial-at-mount-sinai

Personalized Cancer Vaccine Proves Promising in a ... Early results show that PGV001 did not cause serious side effects, and at five-year follow-up, of the 13 patients treated, six patients survived ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT03631043

Personalized Vaccine in Treating Patients With Smoldering ... This early phase I trial studies the side effects of personalized vaccine in treating patients with smoldering multiple myeloma. Vaccines made from a person's ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S0305737222000470

The current clinical landscape of personalized cancer ... Reduction of primary tumor burden and lung metastasis. Vaccine induced neoantigen specific T-cell responses. 70% survival for vaccinated mice compared to 20% ...

5. nature.com nature.com/articles/s41541-024-00979-w

DNA vaccines against GPRC5D synergize with PD-1 ... The mouse vaccine alone was effective in preventing myeloma growth but required PD-1 antibodies to inhibit established MM tumors. We next ...

6. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/10.1111/ajco.70006

Personalized Cancer Vaccines in the Clinical Trial Pipeline After a median follow-up of 36 months, the recurrence-free survival rate in the vaccinated cohort was 87.5% (95% CI: 72.7%–100%) [14].

7. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/4737/533688/Personalized-Neoantigen-Peptide-Vaccine-with-or

Personalized Neoantigen Peptide Vaccine with or without ... Conclusions: This study demonstrates the feasibility and safety of a personalized NeoAg peptide vaccine in patients with SMM.