MS News: Stem Cell Transplantation in Aggressive Disease; Drug Costs Soar

By bmartin on March 22, 2011 9:11 AM | Permalink

While Greek investigators report the favorable long-term results of hematopoietic (not embryonic) stem cells in "aggressive"* multiple sclerosis, the price of approved MS drugs goes up, up, up.

In an unblinded phase 1/2 trial of 35 patients who underwent HSCT (published in the latest issue of Neurology),

16 experienced improvements in their EDSS scores by a median of 1 point (range, 0.5-5.5), which lasted for a median of 2 years. (Of note, a "lifesaving response" was observed in 1 patient with "malignant" MS.)
The median progression-free survival (PFS) lasted 5.4 years in patients with secondary or relapsing MS and 1.5 years in patients with primary progressive disease.
Good prognostic factors were relative youth (age, <35 years) and a shorter time between the diagnosis of MS and transplantation.
PFS at 15 years was significantly greater in patients with active MR lesions (44% vs 10%).
The number and volume of enhancing MR lesions dropped significantly, starting with stem-cell mobilization and increasing after transplantation. Enhancing lesions were persistently suppressed for 12-14 years.

The rationale for using HSCT (in an effort to reconstitute the immune system) in MS is based on the suspected autoimmune nature of the disease and favorable animal-model data.

Meanwhile, Bloomberg reports that the price of injected disease-modifying MS drugs rose as much as 39% last year. The reason: To offset the expected erosion of market share due to Gilenya (fingolimod; Novartis), the first disease-modifying pill for MS. But Gilenya comes with its own considerable sticker shock.

A price survey at this blog in October provided the following monthly costs for the big 4 MS drugs (per destinationrx.com):

Avonex, $2941.92
Betaseron, $6196.61
Rebif, $2809.91
Copaxone, $3267.05

Bloomberg quotes the approval price of Gilenya at $4800 per month (although the company promises a little financial respite for non-Medicare patients).

Today's visit to destinationrx.com provides the following monthly prices, suggesting a smaller (but still sizeable) increase in medication costs during the last 5 months.

Gilenya: $4268.16

Avonex: $3119.04 (6% increase from October)

Betaseron: $6196.61 (no change)

Rebif: $3048.84 (8.5% increase)

Copaxone: $3753.83 (15% increase)

EDSS = Expanded Disability Status Scale; HSCT = hematopoietic stem cell transplantation.

* "[A]dvanced and refractory disease with increasing disability."

T2-weighted supraventricular horizontal MR image from Harvard's Whole Brain Atlas. Multiple subcortical MS lesions are evident, including a very prominent lesion in the frontal area. The web site also offers a very cool time-lapsed movie of developing MS lesions.

P.S. The monthly cost of Biogen Idec's Tysabri (natalizumab)—a highly effective, but potentially risky, second-line agent—is $3554.66.

The median cost of HSCT in 2004, according to an analysis at the Dana-Farber Cancer Institute, was $102,574 during the first 100 days and $128,800 for the first year of treatment. The bulk of these costs were for hospitalization (particularly in the early phase of treatment).