(FDA)

During a two-week span in November, the Food and Drug Administration approved three new drugs to treat multiple myeloma, a rare form of blood cancer that kills an estimated 11,000 Americans a year. The approvals, along with others in recent years, are beginning to alter the prognosis of a disease that once amounted to a death sentence for many patients.

Until last month, seven drugs had been approved for multiple myeloma, which causes malignant plasma cells to build up in bone marrow, where they crowd out healthy cells, hinder the body's ability to fight infection, and lead to problems, such as tumors and kidney damage. Now there are 10 treatments available, some unlike any that have come before.

On Nov. 16, the FDA approved Darzalex, the first "monoclonal antibody" for multiple myeloma, which works by helping certain immune systems cells identify and attack cancer cells. Days later came an approval for Ninlaro, a "proteasome inhibitor" aimed at hindering the ability of cancer cells to grow and survive; it is taken as a once-a-week pill. Finally, on Nov. 30, came the approval of Empliciti, another drug intended to harness the body's immune system to attack and kill malignant cells. Each of the drugs were approved for patients who already have tried other treatments, only to see their cancer persist.

The FDA granted Darzalex and Emplicitie its "breakthrough" designation, a process designed to expedite the development and review of drugs that demonstrate substantial improvement over existing therapies for serious conditions. All three of the new drugs received "priority review," meaning the FDA evaluated them much faster in order to speed them to patients in need.

Despite the understandable excitement in the multiple myeloma community, the new drugs are not a panacea. As with many cancer treatments, some patients showed a dramatic response to the new therapies, but data showed many others did not. Even in those patients who did respond, the benefits were not necessarily indefinite. In addition, as with other promising treatments approved in recent years, the new myeloma drugs are expensive — in some cases, the cost exceeds $10,000 a month.

To better understand significance of the past month for the estimated 90,000 people living with multiple myeloma in the United States, as well as for the more than 25,000 patients expected to be diagnosed over the coming year, we spoke with Walter Capone, president of the Multiple Myeloma Research Foundation. The nonprofit foundation, established in 1998, is the largest private funder of multiple myeloma research. Its investments aided the development of all three drugs approved by FDA in November.

The following conversation has been edited for length and clarity.

Washington Post: You’ve said that this is a “watershed moment” for people with multiple myeloma. Why is that?

Walter Capone: It’s very reminiscent of some unbelievably transformative times that we’ve seen in other disease areas, where there were no treatments, or basically the diseases were a death sentence. I’m thinking, in particular, of my experience in the field of HIV drug development, going back to the late 1990s. And also the work in the area of hepatitis C drug development seven or eight years later.

First, it’s unprecedented in virtually any disease that you’d have three brand new drugs approved for a (rare disease) in the span of two weeks. To my knowledge, that’s just never happened before. For it to happen in myeloma, it’s just indicative of the community’s effort to …. help bring the critical mass necessary to make transformative change happen. That kind of result in that short a period of time is truly a watershed.

When we reflect back on HIV, it was the advent of multiple classes of drugs, with different mechanisms of action, coming together to be able to be used in combination to help a majority of patients break through the ceiling of response —and duration of response — to treatment. To really put their illness into more of a manageable state, rather than swinging from one therapy or single drug to another, and having it be a matter of time until those drugs failed.

Here, we finally have, like we had with HIV, an ability to really overcome the ability for these malignant cells to work around the ways in which these drugs attack and try to silence their activity. Now we have ways of combining these drugs in succession so we can extend the duration of response and deepen the degree of response of these medications in trying to control myeloma.

WP: Some of these drugs have been deemed breakthroughs by the FDA and, as you noted, are the first of their kind. At the same time, they are generally for patients who have already tried other lines of therapy. And when you look at the data, obviously only a portion of patients actually respond. Can you talk about the significance of the benefits we’ve seen in these new drugs, and how they still fall short of [the kind of broad response] you’d like to see?

Capone: The method by which new therapies generally get approved is based on the improvement and efficacy, or response, relative to drugs that are currently approved in the same patients — as well as an assessment of the risk or safety of those drugs. The most rapid route for many of these therapies to be approved is to demonstrate meaningful improvement in patients who have the poorest prospects with regard to their treatment. They have basically been exposed to everything else, and nothing seems to be working. So even a modest improvement in that group of patients is seen as a major advance.

When you look at the last drug approvals in myeloma — the last four, in fact — they’ve really focused on patients who have been exposed to and/or have failed everything else already and are looking for something else to help sustain them. Because of the nature of these patients and their disease having advanced as far as it has …. they generally are tougher types of cancer that we’re trying to treat. That’s why, when you see activity in these kinds of patients, it gets to be pretty exciting.

We absolutely have to continue our efforts to develop new therapies and advance new treatments and mechanisms of actions. Many of the patients being treated in the trials we just talked about for these recently approved drugs have failed those drugs, too. They need other solutions, so we’ve got to constantly keep that pipeline going.

Fortunately, there are many more patients that haven’t gotten to that stage yet. It’s by being able to use these very recently approved drugs — either in combination with each other, or in combinations that haven’t been tested yet with the other seven approved drugs [for multiple myeloma] — that we can really begin to see how we extend the duration of disease-free or progression-free time that patients have.

WP: How do you believe having these new treatments available might change the trajectory of this disease?

Capone: On one side, people who have never heard of a disease like multiple myeloma and might have just gotten the unfortunate news that this has come into their life and is something they have to deal with — their outlook on this disease is [going to be] an entire generation removed from where it was even two or three years ago. With 10 drugs now approved, we have a platform that can offer a much longer disease-free or disease-controlled trajectory for patients.

As recently as four years ago, median survival for myeloma patients was just over three years, somewhere between three and five years. With the advent of what we call “triple therapy,” that horizon expanded out to more like five to seven years. With these new drugs, we could be talking a decade or more. That is very much akin to the tipping point we had with HIV two decades ago.

For patients who are looking for options, who unfortunately lost response to their initial therapy or subsequent therapies, we now have basically an extension, a bridge for them to be able to at least manage their disease, to keep it in check and manage its progression … Having that bridge, and having the opportunity to reset the clock back to where it was two or three years ago, for many patients that’s what these drugs also help provide.

WP: These drugs, like so many new drugs these days, are quite expensive. Do you worry about that from a patient perspective? And how might we measure whether a new drug is actually worth its price?

Capone: It’s a really critical question. And it’s not one that’s confined to myeloma specifically. It’s the case across oncology. It was the case when I was working in HIV, and we introduced the first fusion inhibitor. That was the most expensive therapy because it was the most costly to make.

It’s absolutely the case that advances in science and innovative medicines carry with them high development costs that result in high therapeutic costs. On the other hand, these therapies are only effective if patients are able to access them. The same emphasis that we’re putting on innovating the approaches to overcoming disease, we have to put on ensuring we have equal access for these patients, regardless of where they reside, who’s treating them or what type of insurance they have. That’s absolutely an issue that’s most acute in our minds, as a research foundation that was founded by a patient and focused on patients, first and foremost.

In addition to these treatments, we have one of the most costly procedures in oncology today, which also happens to be one of the most effective: bone marrow transplant. Much like solid organ transplants or liver plant in hepatitis C, it’s a cost of a minimum of a quarter million dollars or more. Bone marrow transplant is one of the most important determinants of a [myeloma] patient’s ability to respond to and control their disease. So, access to that type of intervention, as well as the drugs, is absolutely critical.

WP: It’s obviously been a significant month for people with multiple myeloma. What does the pipeline ahead look like? How would you expect this disease to look different a decade from now?

Capone: I think it’s reasonable to expect, as we continue to elucidate the underlying biology and makeup of myeloma, that we will see this disease transition to one that is able to be more chronically managed.

Especially if we can get patients when they are just evidencing smoldering disease, when there’s a switch that’s gone a little bit off with their immune system. If we can use approaches to help reset their immune system or at least work much like a vaccine does, so that we turn on the immune system to recognize those plasma cells that go awry, then we’ll actually be able to probably control myeloma over time. The types of therapies that we’re talking about today might be less relevant or only applicable [to some patients]. So, getting the disease in its earliest stage and activating the immune system in the process probably has the best prospects to chronically manage it.

As we look between now and that time, the additional treatments and new mechanisms that are being targeted to help arrest the progression of these malignant cells does seem promising. We’re able to continue building on the understanding that we have today. These interventions are going to be stepping stones.

Over the next decade, there might not be this exact watershed moment, but there will be further transformation to helping us being able to control this disease in the vast majority of patients.

READ MORE:

How and $84,000 drug got its price: 'Let's hold our position ... whatever the headlines'

For one rare disease community, a rare moment of hope

Nearly 60 percent of Americans — the highest ever — are taking prescription drugs

Are risks worth the rewards when nonprofits act like venture capitalists?