Author: David Altman

Neurologic clinical trials in a dish?

Neuron_in_tissue_culture
Neuron in tissue culture (Wikipedia creative commons)

Translational neuroscience research has seen a disappointing streak of failed clinical drug trials. While the need for therapeutics that target the nervous system is growing, recent results in diseases like Alzheimer’s and autism have disappointed, and many companies have begun to downsize their R&D investments. Prospects are glum for patients who need new therapies to help manage their disorders.

The frustration is that drug candidates that have shown promise in animal models have not demonstrated efficacy in humans. Mouse models are not proving to be sufficient surrogates for human neurologic disease. Human brains and brain cells are built and function differently, and many neurodevelopmental disorders—hard enough to diagnose in human children—don’t have identifiable behavioral counterparts in mice. As I hear over and over from scientists, there is no such thing as a mouse with autism.

A study, published in Cell Stem Cell this June and conducted by Clifford Woolf, MD, PhD, et al, is among the first to demonstrate the power of an alternative technique: modeling disease in neurons derived from induced pluripotent stem cells (iPS cells).

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From thalidomide to Pomalyst: Better living through chemistry

Thalidomide advertisement, c. 1961
Thalidomide advertisement, c. 1961

In times past, a pharmaceutical chemist’s main focus was to synthesize novel molecules to treat diseases. Today, an increasingly popular alternative is to re-engineer an existing drug—and continually improve it even after FDA approval. That’s how Robert D’Amato, MD, PhD, developed Pomalyst®, recently approved to treat multiple myeloma and the most potent analog to date of thalidomide.

Pomalyst_CelgeneCorpThalidomide has its own fascinating history. Originally developed by Chemie Grünenthal GmbH in the 1950s, it was the result of a search for an anti-anxiety drug to compete with Valium, and was approved for use in Europe as a sleep aid and depression treatment. Eventually, doctors found it useful for treating nausea, and started prescribing it off-label to pregnant women with morning sickness.

The results were disastrous. Thalidomide turned out to be a teratogen, causing severe birth defects. 

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A good deal: Pharma and academia team up to use stem cells to find autism treatments

In a four-way collaboration, skin cells from patients with autism will be used to make pluripotent stem cells. These will be made into neurons — for study of what goes awry at the cellular level in autism, and for testing of drugs. (Miserlou/Wikimedia Commons)

In recent years, creative new partnerships have demonstrated big pharma’s recognition that academic medical centers hold many important cards in clinical research: scientific expertise, animal models of disease, patient samples and phenotypic data.

Increasingly, these partnerships involve academic and company researchers developing joint grant proposals in targeted areas, selected (by joint agreement) for company sponsorship. Some, like the Immune Disease Institute’s $25M arrangement with GlaxoSmithKline, are specific to one academic institution; others, like Pfizer’s Centers for Therapeutic Innovation (CTI) program, provide the same resources under the same deal structure to multiple institutions. Each new deal advances the interaction and understanding between academia and pharma around the common goal of finding new compounds and bringing them to clinic.

Now, in an exciting twist on its track record of partnerships with academic institutions, Roche has brought together three Harvard-affiliated organizations to screen and identify new drugs for the treatment of autism spectrum disorders (ASDs).

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A little business help goes a long way for kids with developmental disorders

Even a small idea, given a small boost, can have a high impact. (Rick Kimpel/Flickr)

When I tell people I work at the Technology and Innovation Development Office at Children’s (TIDO), they usually think I work to commercialize patented blockbuster drug candidates. But many of the most satisfying projects I help promote are innovations that don’t involve as much risk, time and investment, yet make a big difference for patients. Commercializing these innovations can help the greater good, and is part of what propels me to work at a licensing office at a pediatric hospital.

And sometimes it doesn’t take much to help them along.

The Sonnewheel Body Mass Index Calculator and the Vidatak communications board for patients unable to speak or write are some products supported by TIDO without income being the primary goal. Another great example, which we blogged about recently, is helping make routine blood draws less stressful for kids with learning differences and their parents.

The Blood Draw Learning Kit grew out of a serendipitous meeting.

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Licensing: It’s not just the money

(Photo: iChaz/Flickr)

An article popped up in my Google alerts that gave me some excitement.  A survey from the Association of University Technology Managers, reported in Mass High Tech, placed Children’s Hospital Boston fifth in licensing income among all U.S. hospitals. We were ranked just below the Mayo Clinic, which has more than double the research funding of Children’s. Massachusetts General Hospital was second on the list and Brigham and Women’s was eighth.*

I don’t often get to see quick financial results from my work (I’m the marketing and communications specialist in Children’s Technology and Innovation Development Office (TIDO), which licenses the Hospital’s technologies). But what I do get to see regularly is just as important to our mission: small advances that barely impact the hospital’s bottom line but have a large significance to our patients.

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Affordable pneumococcal vaccines that work globally

Streptococcus pneumoniae kills over a million young children a year, most of them in the developing world. Yet currently available pneumococcal vaccines are only effective against strains circulating in Europe and the US.  How can we make affordable vaccines that work globally?

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