When "Orphan" is a Good Word

Often mesothelioma treatment comes in cycles. A patient is diagnosed and receives first-line treatment, after which they may be stable for a period of time. (This is where I am now.) But then the cancer almost invariably comes back, triggering either a return to more aggressive treatment, such as chemo, or a switch to a new treatment. And then the cycle repeats, with stability for a period of time again if the patient is lucky. 

What this means is that we mesothelioma patients are always eager to hear about new treatments coming along. Even if our current treatments are working well (as my immunotherapy is), we expect to need other treatments in the future. We also continue to hold out hope that one of those new treatments will break the cycle and effect a cure.

The drug approval process is a lengthy one, taking years. One way that patients can receive new drugs faster is to participate in clinical trials. Another thing that can speed up the approval for drugs that treat rare cancers is "orphan drug status."

It sounds terrible, doesn't it? Actually, though, orphan drug status is designed to help bring to approval and production drugs that treat rare diseases, officially defined in the US as diseases affecting less than 200,000 people nationally. Developing these drugs is not financially profitable for drug companies, so the government steps in and gives the drug orphan status, which comes with various financial incentives. So when a drug that is being developed for mesothelioma receives orphan status, we meso patients rejoice.

That's what happened recently to a promising new drug presently designated as VT3989. It is the first of a new class of drugs called a TEAD inhibitor and is about to go to Phase III trials. This drug is expected to be particularly effective against tumors that have a certain mutation known as NF2 (which mine does), and so far it looks like the drug is well tolerated by patients. 

So I am cheering on this particular orphan, hoping that it will be approved by the time I need a new treatment drug. Or if it isn't, that it will still be in clinical trials and I'll be able to get it that way.

Another exciting development on the horizon is a generalized mRNA vaccine that works together with immunotherapy drugs like Keytruda to help the immune system target cancer. It has shown great promise in mouse trials for a broad range of cancers. Hopefully it will continue to show promise and move on to human trials soon, if not in this country then in others.

For now, though, the Keytruda (immunotherapy) is doing the trick for me. I am very easily fatigued but am otherwise feeling pretty well. It occurred to me the other day that I could describe my present situation as "chronic cancer." These two words are not usually found next to each other! It's just one more weird aspect of my life lately. But I'll take it over many of the alternatives as I hope and wait for a cure.

P.S. If you would like to join me in sponsoring mesothelioma research, visit my fundraising page at the Mesothelioma Applied Research Foundation: https://curemeso.givecloud.co/fundraisers/amalia-gnanadesikans-fund-for-research.