Right to try bill called both last chance, false hope

A rare neuromuscular disease is eating away at Rose Marie Steffenson’s digestive system.

Her muscles have atrophied and nerves are dying. She gets nourishment through one tube and goes to the bathroom through another.

Doctors have said there is no cure. The best they can do is makeSteffenson comfortable.

Unwilling to accept her death sentence, Steffenson’s family began researching ways to save her. A Colorado doctor recommended stem cell treatment, unavailable in the U.S.

“We checked around in Mexico, but they didn’t have the right kind of stem cell; China turned her down because she was too far into it,” said Steffenson’s sister, Rebecca Pizzuti.

A controversial clinic in Delhi, India, agreed to treat Steffenson, 55. After raising $45,000 and spending two months in India, Steffenson improved. She gained weight and can eat some on her own. But it was only the first of what could be multiple treatments. After being home two months, she hopes to return to India for another round of treatment.

Doctors there wanted her back in June, but it could take the family until September to raise the $25,000 it needs to cover the cost.

These are the people State Rep. JoannGinal of Fort Collins wants to help.

“These are the … people we’re looking at to help prevent them from having to go to all that expense and travel,” said Ginal, who co-sponsored a Right-to-Try bill that unanimously passed the Colorado House two weeks ago and awaits Senate action.

Ginal’s bill is sometimes equated to the “Dallas Buyers Club,” the Oscar-nominated movie about an AIDS patient who brought life-extending drugs from Mexico to the U.S. at a time when the disease was considered a death sentence.

House Bill 1281 would give terminally ill patients who have exhausted all other treatment options access to experimental drugs or treatments that have passed basic safety tests but that have not been proven effective. A patient’s physician and the pharmaceutical company would have to agree to the treatment, but the drug companies would not be required to pay for the treatment.

Drug companies already can provide compassionate use of their products, but that approval can take months, said Ginal, a Democrat.

“Everyone probably knows someone or had someone in the family” who the bill could benefit, she said. “This bill is trying to eliminate the procedural burden with the (U.S. Food and Drug Administration) when people apply for compassionate use. Sometimes it can take four to six months before approval. By that time, often the patient has died or they’re too far gone. This speeds up the (approval) process.”

It’s a bill that could potentially help Steffenson and her family.

Steffenson lived in Loveland and raised her two children there for 15 years. It’s the place she considers home even though she moved to Milliken two years ago.

“It’s very painful to watch her,” Pizzuti said. “There was a period of time where she just wanted to die because the pain was so bad. But she wants to live. She has so much to live for.”

The clinic and doctor that treated Steffenson — Dr. GeetaShroff at the Nu Tech Mediworld clinic — are controversial. Critics in the medical community say Shroff is preying on patients’ fears and hopes for a miracle. Proponents believe Shroff’s stem cell work is saving lives.

It was a risk Steffenson’sfamily readily took and are happy they did, given her improvement, Pizzuti said.

Pros and cons

Only a few other states — Arizona, Missouri and Louisiana — have similar Right-to-Try legislation pending. The Colorado proposal has seen little opposition.

The libertarian Goldwater Institute, which lobbied Ginal to carry a bill and helped craft it, believes terminally ill patients deserve the option.

“States should enact Right-to-Try measures to protect the fundamental right of people to try to save their own lives,” the Institute writes on its website. “Without state action, terminal patients are at the mercy of a federal bureaucracy that can literally cause death by delays, denials and unnecessary procedural requirements.”

The group Science Based Medicine, however, calls it a “stupendously bad idea.”

“It advocates allowing access to drugs that have only passed phase 1 testing,” Dr. David Gorski, SBM managing editor, said in a telephone interview. “My main concern is that it is likely to harm far more patients than it helps.”

“There are worse things than dying,” Gorski said. “What about something that makes your death worse or remaining life shorter … that is quite likely with some of these experimental drugs.”

Right-to-Try bills are not new but thus far no state has adopted such a bill. Gorski said the “Dallas Buyers Club” movie has led to renewed interest.

“These ideas were around before the movie, but it wouldn’t surprise me if the movie popularized the idea and resurrected them from the grave.”

Within the state, the Colorado Pharmacists Society has taken no position on the bill, according to a spokesman. The Colorado Hospital Association is “actively monitoring” it but also has not taken a position, said Kevin Downey, vice president of communications and media relations.

The Colorado Hospital Association testified about hospitals’ concerns during the bill’s first hearing in the House and helped draft an amendment that provides an exemption for hospital patients.

The FDA only approves investigational drugs for patients in a hospital that is part of a clinical trial or if the drugs have received FDA approval for “compassionate use,” Downey said. “The patients covered by this bill do not fit into those two categories; therefore, if this kind of drug were dispensed in a hospital against the current federal laws, a hospital could also lose its certification for Medicare and Medicaid.”

Holding on to hope

Steffenson’s family is trying to raise a second round of funding to get back to India. “We have to get her there one way or another,” Pizzuti said. “We have to save her.”

Long-term, Steffenson’s family hopes to start a nonprofit to raise the money to help Steffenson and others suffering from the same disease.

“Until we can get the progression slowed down and get ahead of it, she will get worse,” said Pizzuti, who’s optimistic the treatments will work. Steffenson is already up to 122 pounds — she was down to 94 pounds when she went to India — and may be able to have her colostomy reversed.

“I still believe in miracles and I still believe that I have a lot to live for,” Steffenson said. “I want more than anything else to be well; and if I don’t get the miracle that I believe in, I won’t be well and I think that sums it up.”

Right to Try at a glance

• • • Bill number: House Bill 1281
    • House sponsors: Rep. Joann Ginal, D-Fort Collins; Rep. Janak Joshi, R-Colorado Springs
    • Senate sponsors: Sen Irene Aguilar, D-Denver; Sen. George Rivera, R-Pueblo
    • Purpose: The bill would permit terminally ill patients to participate in clinical trials and use investigational drugs
    • Where it stands: Passed the Colorado House of Representatives, awaits Senate action.
    • Bill text:http://bit.ly/right2try

Want to help?

For more information about Rose Marie Steffenson’s journey, visit www.facebook.com/helprose.