本帖最后由 凤凰涅盘 于 2011-11-9 20:26 编辑
Written by Stephen Rose, Ph.D., Chief Research Officer, Foundation Fighting Blindness
November 8, 2011 – Hope has never run higher in the fight against retinal degenerative diseases, thanks to clinical trials now underway for gene, stem cell and pharmaceutical therapies. As a result, people affected by these vision-robbing conditions are naturally eager to figure out what emerging treatment approach is going to work best for them.
While it is tempting for someone affected to focus on a “magic bullet” to stop or reverse their disease, I strongly encourage people to consider the Foundation’s comprehensive portfolio of emerging treatments when thinking about the future of their vision. Here are three important reasons why:
We can’t predict how future research will unfold
Science always has surprises. As an example, until a year or two ago, we believed that corrective gene therapy would be suitable only for treating early-stage disease when a person had a lot of photoreceptors left to save. But recently, Foundation-funded researchers from the University of California, Berkeley and other groups are using gene therapy to enable ganglion cells -- cells in the retina that survive long after photoreceptors are lost -- to provide vision. This research advance has now put gene therapy on the map for potentially reversing total blindness. Previously, we thought that only stem cells or artificial retinas could restore vision in advanced disease.
We need to have back-up plans
In the next couple of years, as we see a big increase in the number of clinical trials for inherited retinal diseases, some setbacks will be inevitable. We need to keep in mind that clinical trials are experiments and, invariably, will not always achieve optimal results. It is critical that we have multiple treatments available, or in the pipeline, for each disease, in case a promising course of action does not pan out. This is one reason pharmaceutical therapies are so important. Generally speaking, they can treat a broader range of conditions than, for example, a gene therapy directed at a specific genetic defect. I envision pharmaceuticals often serving as bridges, or as alternatives, to more targeted therapies.
Treatment decisions will be personal
Several factors will play a role in determining which treatments might work best for an individual, including his or her genetic profile, stage of disease, age and even tolerance for risk. Let’s say in the future that a middle-aged person with retinitis pigmentosa has been taking a drug for many years that’s done a good job preserving vision, and along comes a new gene or stem cell therapy. Does that person try a new treatment or stick with what is known to work? There is no right or wrong course of action. What’s important is that we fund a diversified portfolio of research, so patients have options. Multiple treatment alternatives may seem like a luxury now, but we are working hard to make that day a reality as soon as possible.
Find out more about Foundation research
The Foundation currently funds 130 grants at 73 institutions around the world. Grants are selected through a rigorous review process conducted by the Foundation’s Scientific Advisory Board, which is comprised of the world’s top retinal researchers. A complete list of the Foundation’s grants is at: www.FightBlindness.org/grants.
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新兴治疗什么工作最适合我？史蒂芬写的玫瑰，博士，首席研究官，战胜失明基金会2011年11月8日–希望从来没有高于对抗视网膜退行性疾病，由于目前正在进行临床试验的基因，干细胞和药物疗法。因此，人们受到这些vision-robbing条件自然是急于找出新的治疗方法是将最适合他们的工作。虽然这是很有诱惑力的人的影响集中在一个“魔术子弹”，以阻止或逆转疾病，我强烈鼓励人们考虑的基础上的综合投资组合的新兴疗法在思考未来的愿景。这里有三个重要原因：我们不能预测未来的研究将展开科学总是惊喜。作为一个例子，直到一年或两年前，我们相信，纠正基因治疗将只适用于治疗早期疾病，当一个人有很多光感受器去拯救。但最近，基金会资助的研究人员从加利福尼亚大学，伯克利和其他团体使用基因疗法使神经节细胞——细胞在视网膜光感受器丧失生存很久以后——提供视觉。本研究进展基因治疗已经把地图上的潜在逆转全色盲。以前，我们认为只有干细胞或人工视网膜可以恢复视力的先进的疾病。我们需要有后备计划在接下来的几年，我们看到一个大数量的增加临床试验遗传性视网膜疾病，一些挫折是不可避免的。我们需要记住，临床试验的实验，无一例外，并非总是达到最佳效果。至关重要的是，我们有多个治疗，或在管道，每一种疾病，如一个充满希望的作法不挑锅。这是一个原因，药物治疗是非常重要的。一般来说，他们可以治疗范围更广泛的条件，例如，一个基因治疗针对特定的基因缺陷。我设想***往往充当桥梁，或作为替代，以更有针对性的治疗。处理决定将个人几个因素将发挥作用，确定哪些治疗可能是一个人，包括他或她的遗传资料，疾病阶段，年龄和风险容忍度。让我们说，在未来，一个中年人视网膜色素变性一直采取药物多年，做好维护视力，并沿是一个新的基因和干细胞治疗。那人尝试新的治疗或坚持什么是众所周知的工作？是没有正确或错误的作法。最重要的是，我们基金多元化投资组合的研究，使病人有选择。多种替代治疗方法可能看起来像是一个奢侈，但是我们正在努力使这一天尽快实现。找出更多有关基础研究目前基金会赠款资金13073个世界各地的机构。补助金是通过严格的审查过程进行了基础的科学咨询委员会，这是由世界顶尖的视网膜的研究人员。一个完整的名单上的基金会赠款是：www.fightblindness.org/grants。 |
