FDA允许扩展干细胞治疗肌萎缩侧索硬化症临床试验

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FDA allows expansion of stem cell trial for ALS: D0 q7 `  W" O4 C4 a, u) g( ^
April 18, 20135 ?& M9 H8 p6 ?7 q7 y0 g+ }: ?
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U-M may recruit ALS patients for stem cell clinical trial& F  Z+ y4 u1 y7 E" g

' k( A: a/ G3 C5 UA clinical trial using human neural stem cells — injected into the spinal cord — to halt or even reverse the deadly effects of Lou Gehrig’s Disease may begin recruiting patients at the University of Michigan as early as this summer.7 r4 M: b. L2 u: M$ l0 h; i3 t6 U
Until now, the surgeries have taken place at Emory University in Atlanta, led in part by a former U-M neurosurgery resident, Dr. Nicholas Boulis, and overseen by U-M physician and neurology professor Dr. Eva Feldman. The trial is the only one if its kind because the neural stem cells are injected directly into the spinal cord.  ?9 o1 Y$ {  g  Z) A+ T! G
At Emory, 15 patients underwent surgery during Phase I, which was focused primarily on safety. At least one appeared to improve dramatically for a short time, regaining use of his legs. Feldman attended each surgery.9 k3 ?$ K; ^* r9 X3 Q( c
The go-ahead Monday by the U.S. Food and Drug Administration to expand the trial to Phase II means the surgeries can take place at U-M as well. The second phase will involve 15 patients split between U-M and Emory, according to U-M and the provider of the stem cells, Maryland-based Neuralstem.
, Q) e+ }, B3 I) p/ D: lParticipants must be ambulatory and live close to those universities.
: b* r4 h+ i3 D; g0 C1 A2 @  GCurrently, there is no cure for amyotrophic lateral sclerosis, often called ALS or Lou Gehrig’s disease. One drug extends life, but usually just by months.
2 _) H  q7 Z! zThe disease moves swiftly, with most people living two to five years after diagnosis. ALS deadens nerves, withers muscles and, in a final assault, cuts off a person’s ability to breathe even as their mind remains intact.& @. [! G9 Y# D* W2 E$ k7 L$ a* G
Dave Murray, 55, of Sterling Heights said Wednesday he was “thrilled” by the trial’s move to U-M, though it’s unclear whether he would be eligible.
$ ~  s+ d+ e1 K5 k0 RThe former security alarm installer already has been a participant in two other clinical trials.
, ~  ?# H* e# u8 w5 e“I might be past the point of eligibility, but I’m always happy with any news that we might be moving forward,” he said. “It’s such a horrible disease.”
# c& f7 R4 F2 _7 v9 h* y# r8 B) s3 rTwo years ago, he was sitting with his coat draped over his arms on an exam table when a doctor gave him the diagnosis, told him he had three, maybe five, years left. Only the sound of his doctor washing her hands at the tiny sink broke the suffocating silence that followed.
1 A2 j* i  C1 J“The doctor, she was very compassionate,” recalled his wife, Sheryl. “She left us room to cry. She said ‘Take whatever time you need.’ ”1 B6 z& f7 B' I' f
Feldman, the physician overseeing the trial, has spent her career stalking ALS and searching for a cure. She has watched helplessly as countless patients have died over the years — as many as five a week and as young as 16, she told the Free Press in 2012.9 Q% t0 u/ |  g% b+ M# e, N
The trial is still early and will move slowly as she and other researchers continually assess their results and report the findings to the FDA.
9 K' h7 I7 w: X3 k% N2 i* c0 zPhase II means researchers can begin assessing the effectiveness of the procedure, not just its safety. In a lengthy surgery, a specially designed apparatus is attached to the spine and inserts human stem cells into a person’s spinal cord.
, T% T5 Z% J/ y- ~% ~( n9 f" ^: E7 P$ aFeldman and others theorize that these new cells, once in the spinal cord, act as nursemaids to damaged nerve cells, sending out repair signals, and somehow halting the progression of the disease.
$ q" a% Q* \: W% G* ~The cells were derived from a cell line that dates to the spinal cord of an aborted fetus in 2000. The cells are different from the embryonic stem cells that were the subject of a controversial ballot proposal in Michigan in 2008, when voters approved lifting the ban on embryonic stem cell research.
+ d% g8 E4 f, W5 o9 a! LU-M’s Institutional Review Board, which oversees clinical trials to make sure they are scientifically and ethically sound, must sign off on the experimental surgeries before U-M begins recruiting.( N7 W# I, ?- o
Despite its limitations, the trial offers hope for those who see little of it once they are handed a diagnosis, said Sue Burstein-Kahn, executive director of ALS of Michigan. Her father died of ALS.
$ o  K0 ]; ~/ j9 B0 A, g+ K- rShe called the FDA approval “wonderful” in that it could provide insights to a treatment for future patients.
: u) c* j5 e6 x3 m& i“We need ALS research fast-tracked,” she said. “This isn’t even about a cure. People would be happy with the treatment.”7 H: T0 D9 V" _& Y8 }% b. ]
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Contact Robin Erb: 313-222-2708 or rerb@freepress.com.
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