[Ann Dildy]

BFCR4350A for multiple myeloma

by Ann Dildy on Wed May 02, 2018 7:16 am

Does have any information about BFCR4350A as a possible treatment for multiple myeloma?

What is known about it's possible efficacy and safety?

As a bit of background, I was diagnosed with multiple myeloma in 2013. Since then, I have been through two stem cell transplants and seven different protocols. In early March my LDH took a climb to about 900. A non specific indicator, but one that concerned my doctor. Bone marrow biopsy showed 75% cancerous plasma. We opted for a week in the hospital doing a VRD-PACE. Seven different drugs infused over four days. The result was that it knocked it down to 25%. I was not able to go on a protocol (Darzalex, dex, and 4 mg Pomalyst) while recovering.

It has been six weeks. I had an appointment at the Mayo Clinic last Friday and my bone marrow malignancy is back up to 81% in six weeks. My bone marrow biopsy shows no 13 or 17 chromo­somes. I am at a very tricky place, so I am considering taking part in a Phase 1 trial of BFCR4350A. If not, they are talking going back to drugs I’ve been on in a combination, cyclo­phos­phamide, dexa­metha­sone, Pomalyst, and daratumamab. Pomalyst would be daily and daratumumab weekly.

So any information people might have about BFCR4350A for multiple myeloma would be appreciated.

[WholeNotherWorld]

Re: BFCR4350A for multiple myeloma

by WholeNotherWorld on Wed May 02, 2018 7:26 am

You are in a very difficult situation and my thoughts and prayers are for you.

I don't have information about BFCR4350A for you right now. But, if you are considering partici­pation in a Phase 1 trial, your doctor or research coordinator can help you understand the goal of the study. Read the consent form carefully. Here's some information about Phase 1 trials in general that you may want to know (via the American Cancer Society):

1. The first few people in the study often get a very low dose of the treatment and are watched very closely. If there are only minor side effects, the next few participants may get a higher dose. This process continues until doctors find a dose that’s most likely to work while having an acceptable level of side effects.
2. The focus in phase I is looking at what the drug does to the body and what the body does with the drug.
3. Safety is the main concern at this point. Doctors keep a close eye on the people and watch for any serious side effects. Because of the small numbers of people in phase I studies, rare side effects may not be seen until later.
4. Placebos (sham or inactive treatments) are not part of phase I trials.
5. These studies usually include a small number of people (typically up to a few dozen).
6. Often, people with different types of cancer can take part in the same phase I study.
7. These studies are usually done in major cancer centers.
8. These studies are not designed to find out if the new treatment works against cancer.

Phase 1 studies are very important first step in the development of a new drug, which would be further tested for effectiveness against a particular kind of cancer. Volunteers for Phase 1 studies are brave pioneers.

Good luck in your decision! Please let us know what you decide to do.

[Multibilly]

Re: BFCR4350A for multiple myeloma

by Multibilly on Wed May 02, 2018 8:57 am

Hi Ann,

BFCR4350A is a new bi-specific T-cell engager drug (also known as a BiTE). In a nut, a BiTE is like a monoclonal antibody drug that has been developed to go after two separate targets. But in this case, one antibody attaches itself to your T-cells (T-cells are one of the big dogs in your immune system that normally seek out and destroy cancer cells). The other antibody attaches itself to the myeloma cell. The BiTE tag on the T-cell is engineered to in turn recognize the BiTE tag on the myeloma cell. The tagged T-cell then attaches itself to the tagged myeloma cell and does it's dirty work to kill off the myeloma cell.

In a simplistic way, the approach used with BiTEs is much like that of the much-talked-about CAR-T cell therapy that's been in the news. But unlike CAR-T cell therapy, one doesn't have to extract a patient's T-cells and re-engineer those cells outside of the body in order for the T-cells to be able to recognize the tagged myeloma cells.

Note that BFCR4350A primarily targets the antigen (a protein) on the surface of the myeloma cell known as FCRH5 along with 4 other protein targets. There's also a Phase 1 BiTE trial for myeloma that has opened for PF-06863135, which targets the BCMA antigen on the surface of the myeloma cell. BCMA is an antigen target that is also being used in some of the CAR-T cell approaches.

BiTEs which use different protein targets are currently going through Phase 2 clinical trials for treating lymphoma, leukemia, etc.

If you are in a Phase 1 trial, you would be helping determine the safety and efficacy of the BFCR4350A drug. While I think you can look through some of the results of the Phase 1 trials for BiTEs such as blinatumomab (Blincyto), I wouldn't necessarily use the results and side effects from those trials as an indicator for what you might experience with BFCR4350A.

Best of luck with your decision. Please keep us updated on your journey.